January | February | March | April | May | June | July | August | September | October | November | December |
Company | Product | Description | Indication | Phase III status | Date | Therapeutic area |
Aravive Inc., of Houston | Batiraxcept | Ultra-high affinity decoy protein that binds to GAS6 and thereby prevents AXL signaling | Platinum-resistant ovarian cancer | Achieved full enrollment in study testing combination with paclitaxel; top-line data expected in mid-2023 | 1/4/23 | Cancer |
Astellas Pharma Inc., of Tokyo | Zolbetuximab | Monoclonal antibody targeting Claudin 18.2 | CLDN18.2-positive, HER2-negative, locally advanced unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma | In the 565-patient Spotlight study, zolbetuximab plus mFOLFOX6 reduced the risk of progression or death by 24.9% compared to mFOLFOX6 alone (p=0.0066); the combination reduced the risk of death by 25% (p=0.0053) compared to mFOLFOX6 alone | 1/19/23 | Cancer |
Beigene Ltd., of Beijing | Tislelizumab | Humanized IgG4 anti-PD-1 monoclonal antibody | Gastric or gastroesophageal junction cancer | Interim results showed study met 1 of the primary endpoints of overall survival; manageable safety profile | 1/18/23 | Cancer |
Daiichi Sankyo Co. Ltd., of Tokyo | Datopotamab deruxtecan (Dato-DXd) | TROP2-directed DXd antibody-drug conjugate | Untreated advanced or metastatic nonsquamous non-small-cell lung cancer | First patient dosed in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) with or without platinum chemotherapy | 1/30/23 | Cancer |
Deciphera Pharmaceuticals Inc., of Waltham Mass. | Qinlock | Switch-control tyrosine kinase inhibitor | Gastrointestinal stromal tumor previously treated with imatinib | In patients with tumors with mutations with KIT exon 11 and exon 17/18, median progression-free survival (PFS) was 14.2 months for Qinlock compared to 1.5 months for sunitinib (HR=0.22, p<0.0001); for patients with tumors with mutations in KIT exon 11 and 13/14, median PFS was 4 months for Qinlock compared to 15 months for sunitinib (HR=3.94, p=0.0005) | 1/24/23 | Cancer |
Deciphera Pharmaceuticals LLC, of Waltham, Mass. | Qinlock (ripretinib) | Switch-control tyrosine kinase inhibitor | Advanced gastrointestinal stromal tumor | Results from planned exploratory analysis of Intrigue study of Qinlock using circulating tumor DNA (ctDNA) from a subgroup of patients showed median progression-free survival of 14.2 months vs. 1.5 months for sunitinib (p<0.0001); objective response rate of 44.4% vs. 0% (p=0.0001); overall survival not estimated in ripretinib vs. 17.5 months for sunitinib (p=0.0061) | 1/3/23 | Cancer |
Geron Corp., of Foster City, Calif. | Imetelstat | Telomerase inhibitor | Lower risk myelodysplastic syndromes | Study met primary 8-week transfusion independence (TI) endpoint and key secondary 24-week TI endpoint with highly statistically significant and clinically meaningful improvements; TI of 47% and 9% for imetelstat and placebo, respectively, (p<0.001) at 8 weeks; TI of 33% and 2% for imetelstat and placebo, respectively, (p<0.001) at 24 weeks; increase in hemoglobin levels (p<0.001); mean reduction in RBC transfusion units compared to placebo (p=0.042); hematologic improvement-erythroid achieved for imetelstat vs. placebo (42.4% vs. 13.3%, p<0.001) | 1/4/23 | Cancer |
IO Biotech ApS, of Copenhagen | IO102-IO103 | Cancer immunotherapy targets indoleamine 2,3-dehydrogenase (IDO) and PD-L1. | Advanced melanoma | Independent data monitoring committee recommended to continue without any modifications in IOB-013/KN-D18 trial in combination with pembrolizumab; ongoing enrollment | 1/9/23 | Cancer |
Ipsen SA, of Paris | Onivyde | Liposomal injection of irinotecan | Metastatic pancreatic ductal adenocarcinoma | Data showed study met its primary and secondary endpoint; statistically significant improvement in overall survival of 11.1 months compared to 9.2 months (p=0.04); statistically significant improvement in median progression-free survival of 7.4 months vs. 5.6 months for nab-paclitaxel and gemcitabine (p=0.0001); 41.8% objective response rate | 1/20/23 | Cancer |
Janssen Research & Development LLC, part of Johnson & Johnson, of New Brunswick, N.J. | Carvykti (ciltacabtagene autoleucel) | BCMA-directed CAR T therapy | Relapsed or refractory multiple myeloma | Cartitude-4 study met its primary endpoint of significant improvement in progression-free survival at the first pre-specified interim analysis; independent data monitoring committee recommended the unblinding of the study | 1/27/23 | Cancer |
Junshi Biosciences Co. Ltd., of Shanghai | Toripalimab | Anti-PD-1 monoclonal antibody | Operable non-small-cell lung cancer | Results in combination with platinum-containing doublet chemotherapy as perioperative treatment achieved primary endpoint | 1/17/23 | Cancer |
Merck & Co. Inc., of Rahway, N.J. | Keytruda (pembrolizumab) | Monoclonal antibody targeting PD-1 | Metastatic hormone-sensitive prostate cancer | Keynote-991 study testing Keytruda plus enzalutamide and androgen deprivation therapy (ADT) will be stopped based on recommendation of the independent data monitoring committee after an interim review of the data showed a lack of improvement in overall survival or radiographic progression-free survival for the Keytruda-based combination compared to placebo plus enzalutamide and ADT | 1/25/23 | Cancer |
Merck & Co. Inc., of Rahway, N.J. | Keytruda (pembrolizumab) | Monoclonal antibody targeting PD-1 | First-line, advanced or unresectable biliary tract cancer | In the Keynote-996 study, Keytruda plus gemcitabine and cisplatin improved overall survival compared to gemcitabine and cisplatin alone; data will be presented at an upcoming medical meeting | 1/25/23 | Cancer |
Morphosys AG, of Munich, Germany | Pelabresib (CPI-0610) | BET inhibitor | Myelofibrosis | Top-line results of Manifest-2 study in combination with ruxolitinib expected in early 2024 | 1/9/23 | Cancer |
Point Biopharma Inc., of Indianapolis | 177Lu-PNT2002 | PSMA-targeted radioligand | PSMA-expressing metastatic castration resistant prostate cancer | Completed enrollment for the randomization phase; enrolled 390 patients | 1/12/23 | Cancer |
Roche Holding AG, of Basel, Switzerland | Tecentriq (atezolizumab) | Anti-PD-L1 | Early stage hepatocellular carcinoma | Study in combination with Avastin (bevacizumab, Roche Holding AG) met primary endpoint of recurrence-free survival; immature overall survival data; consistent safety profile | 1/19/23 | Cancer |
Servier SA, of Paris, and Taiho Oncology Inc., of Princeton, N.J. | Lonsurf (trifluridine/tipiracil) | Thymidine-based nucleoside analogue and thymidine phosphorylase inhibitor | Refractory metastatic colorectal cancer | Results in combination with bevacizumab met its primary endpoint of overall survival (OS); statistically significant and a clinically meaningful improvement in OS of 3.3 months compared to trifluridine/tipiracil alone (10.8 months vs. 7.5 months; p<0.001); 39% reduction in the risk of death in patients; statistically significant improvement for the trifluridine/tipiracil plus bevacizumab combination vs. trifluridine/tipiracil alone in progression-free survival (5.6 months vs. 2.4 months, 0.36-0.54, p<0.001) | 1/17/23 | Cancer |
Anthos Therapeutics Inc., of Cambridge, Mass. | Abelacimab | Dual-acting fully human monoclonal antibody targeting both factor XI and factor XIa | High-risk patients with atrial fibrillation (AF) deemed unsuitable for current anticoagulants | First patient enrolled; planning to enroll 1900 patients | 1/3/23 | Cardiovascular |
Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. | Orladeyo (berotralstat) | Plasma kallikrein inhibitor | Hereditary angioedema | Enrolled first patient in pivotal Apex-P trial testing once-daily oral drug in pediatric patients, ages 2 to <12 | 1/26/23 | Dermatologic |
Biofrontera Inc., of Woburn, Mass. | Ameluz | Aminolevulinic acid hydrochloride gel | Actinic keratosis on the extremities, neck and trunk | First patient dosed | 1/9/23 | Dermatologic |
Dong-A ST Co. Ltd., of Seoul, South Korea | DMB-3115 | Biosimilar of ustekinumab, a recombinant monoclonal antibody | Moderate to severe chronic plaque psoriasis | Results showed DMB-3115 and Stelara (ustekinumab, Janssen Pharmaceutical) demonstrated therapeutic equivalence at percent change from baseline in PASI at week 8 and week 12; no clinically significant difference in safety | 1/17/23 | Dermatologic |
Inflarx NV, of Jena, Germany | Vilobelimab | Monoclonal anti-human complement factor C5a antibody | Ulcerative pyoderma gangrenosum | Presented the design of randomized, controlled adaptive phase III design | 1/5/23 | Dermatologic |
Journey Medical Corp., of Scottsdale, Ariz. | DFD-29 (minocycline modified-release capsules) | Tetracycline antibiotic | Papulopustular rosacea | Fully enrolled and randomized all of the patients; top-line data expected in the first half of 2023 | 1/10/23 | Dermatologic |
Genethon, of Paris | GNT-003/GNT-0003 | Gene therapy combines normal copies of the UGT1A1 gene coding for the bilirubin metabolizing enzyme with an AAV vector | Crigler-Najjar syndrome | Launched pivotal study; planning to enroll patients aged 10 years and older | 1/10/23 | Endocrine/Metabolic |
Innovent Biologics Inc., of China, and Rockville, Md. | Mazdutide/IBI-362 | Glucagon-like peptide 1 receptor (GLP-1R) and glucagon receptor (GCGR) dual agonist | Type 2 diabetes | First patient dosed | 1/10/23 | Endocrine/Metabolic |
Maggie's Pearl LLC, of Sturgis, Mich. | Epalrestat | Oral and brain-penetrant drug | PMM2-CDG, or phosphomannomutase-2-congenital disorder of glycosylation | Initiated phase III study to evaluate safety, tolerability, and clinical and metabolic improvement in pediatric patients | 1/9/23 | Endocrine/Metabolic |
Oramed Pharmaceuticals Inc., of New York | ORMD-0801 | Oral insulin candidate | Type 2 diabetes | Trial did not meet its primary and secondary endpoint; no serious drug-related adverse events | 1/12/23 | Endocrine/Metabolic |
Sciwind Biosciences Co. Ltd., of China and San Francisco | Ecnoglutide (XW-003) | Long-acting glucagon-like peptide-1 (GLP-1) derivative | Type 2 diabetes | Initiated dosing in adult patients; to evaluate the efficacy and safety of once-weekly subcutaneous administration of ecnoglutide in approximately 210 treatment-naïve patients | 1/30/23 | Endocrine/Metabolic |
Visen Pharmaceuticals Co. Ltd., of Shanghai, China | Palopegteriparatide | Prodrug of parathyroid hormone | Hypoparathyroidism | Completed double blind study | 1/6/23 | Endocrine/Metabolic |
Galmed Pharmaceuticals Ltd., of Tel Aviv, Israel | Aramchol | Stearoyl CoA desaturase-1 inhibitor | Liver fibrosis and nonalcoholic steatohepatitis | Open-label part of the Armor study showed histological improvement in fibrosis (?1 stage) demonstrated in 39% of subjects according to NASH CRN, in 61% of subjects with statistically significant (p<0.0001) reduction in fibrosis score; statistically significant (p<0.0001) reduction in liver stiffness; statistically significant (p<0.0001) reduction in biochemical markers of liver injury ALT and AST; statistically significant reductions in major fibrosis biomarkers: FIB-4 (p<0.0001), Pro-C3 (p<0.0001) and ELF (p= 0.0038) at week 24; good safety profile | 1/4/23 | Gastrointestinal |
Index Pharmaceuticals Holding AB, of Stockholm | Cobitolimod | TLR9 agonist | Moderate to severe left-sided ulcerative colitis | Dose selection expected in the fourth quarter of 2023; patient recruitment underway | 1/27/23 | Gastrointestinal |
Madrigal Pharmaceuticals Inc., of Conshohocken, Pa. | Resmetirom | Liver-directed THR-beta agonist | Nonalcoholic steatohepatitis (NAS) with liver fibrosis | Study achieved both liver histological improvement endpoints; NASH resolution with ?2-point reduction in NAS and no worsening of fibrosis p-value of <0.0001 at 80-mg and 100-mg; ?1-stage improvement in fibrosis with no worsening; p=0.0002 and <0.0001 at 80-mg and 100-mg, respectively | 1/6/23 | Gastrointestinal |
Phathom Pharmaceuticals Inc., of Florham Park, N.J. | Vonoprazan | Potassium-competitive acid blocker | Non-erosive gastroesophageal reflux disease | Top-line results of PHALCON-NERD-301 study met the primary endpoint at 10-mg and 20-mg doses; highly statistically significant greater percentage of 24-hour heartburn-free days as compared to placebo (p<0.0001), with mean 46.4% for vonoprazan at 10 mg, 46% for vonoprazan at 20 mg compared to 27.5% for placebo; full results expected in 2023; well-tolerated | 1/9/23 | Gastrointestinal |
Mallinckrodt plc, of Dublin | Terlivaz (terlipressin) | Derivative of the vasoconstrictor vasopressin | Hepatorenal syndrome | Pooled analysis showed higher percentage of terlipressin-treated patients with ACLF grade 0-1 or grade 2 achieved HRS reversal compared with placebo (43% vs. 18% for ACLF grade 0-1; p<0.0001) and (28% vs. 15% for ACLF grade 2; p=0.02); no differences in the incidence of HRS reversal in ACLF grade 3 (p=0.46) | 1/23/23 | Genitourinary/Sexual Function |
Biomarin Pharmaceutical Inc., of San Rafael, Calif. | Valoctocogene roxaparvovec (Roctavian) | Adeno-associated virus vector encoding human coagulation factor VIII | Severe hemophilia A | Study achieved primary and secondary endpoints (p<0.001 for both) from 3 years of follow-up from its ongoing global phase III Gener8-1 study; 92% of patients remained off prophylaxis at the end of year 3; mean annualized bleed rate reduced by 80% from baseline and factor VIII usage reduced by 94% in year 3 compared to baseline | 1/8/23 | Hematologic |
Grifols SA, of Barcelona, Spain | Grifols Fibrin Sealant | Plasma-protein based agent | Control of surgical bleeding in pediatric patients | Top-line results in pediatric patients met all primary and secondary endpoints; 95% efficacy rate, achieving hemostasis within 4 minutes of application; good safety and tolerability profile; comparable adverse events between both arms | 1/11/23 | Hematologic |
GSK plc, of London | Momelotinib | ACVR1/ALK2, JAK1 and JAK2 inhibitor | Myelofibrosis patients with anemia | Momentum trial showed clinically significant improvement in disease-related symptoms after 24 weeks of treatment; 50% or more reduction in Myelofibrosis Symptom Assessment Form Total Symptom Score; significant reduction in spleen size; non-hematological side effects; ongoing patient follow-up; long-term survival continues to be monitored; results published in The Lancet | 1/27/23 | Hematologic |
Hutchmed Ltd., of Hong Kong | Sovleplenib | Syk inhibitor; small molecule | Primary immune thrombocytopenia | Completed patient enrollment; top-line results expected in the second half of 2023 | 1/3/23 | Hematologic |
Sanofi SA, of Paris | Efanesoctocog alfa | Recombinant factor VIII therapy | Hemophilia A | Data published in The New England Journal of Medicine from Xtend-1 trial showed drug delivered normal to near-normal factor activity levels (>40%) for majority of the week with once-weekly dosing; median and mean annualized bleeding rates (ABR) were 0 (IQR: 0.00-1.04) and 0.71 (95% CI: 0.52-0.97), respectively; statistically significant and clinically meaningful reduction in ABR (77%) vs. prior factor VIII prophylaxis (p<0.001); nearly all (97%) bleeding episodes resolved with a single injection; prophylaxis improved physical health (p<0.001), pain intensity (p=0.03) and joint health (p=0.01) when comparing 52-week and baseline measurements | 1/25/23 | Hematologic |
Takeda Pharmaceuticals Co. Ltd., of Osaka, Japan | TAK-755 | Recombinant ADAMTS13 protein | Congenital thrombotic thrombocytopenic purpura | Preplanned Interim analysis showed TAK-755 reduced the incidence of thrombocytopenia events by 60% | 1/5/23 | Hematologic |
Allovir Inc., of Waltham, Mass. | Viralym-M (posoleucel) | Allogeneic, off-the-shelf, multivirus-specific T-cell therapy | Preventing clinically significant infections or diseases from viruses in allogeneic hematopoietic cell transplant | Planning to complete enrollment by the end of 2023; data readouts in 2024 | 1/9/23 | Immune |
Zenas Biopharma LLC, of Waltham, Mass. | Obexelimab | High-affinity bifunctional antibody that inhibits B-cell lineages by simultaneously binding to CD19 and Fc?RIIB | IgG4-related disease (IgG4-RD) | First patient dosed | 1/11/23 | Immune |
Aridis Pharmaceuticals Inc., of Los Gatos, Calif. | AR-301 | Monoclonal antibody targeting S. aureus alpha-toxin | Ventilator-associated pneumonia caused by gram-positive bacteria Staphylococcus aureus | Top-line results from AR-301-002 study showed improvement in clinical cure rate of ?10% with adjunctive use of AR-301 along with standard-of-care (SOC) antibiotics; with limited sample size evaluated, statistical significance was not reached for the primary endpoint of clinical cure rate on day 21 compared to antibiotics alone (68.9% [42/61 patients] for AR-301 + SOC vs. 57.6% [34/59] for SOC alone, [p= 0.23]) | 1/25/23 | Infection |
Finch Therapeutics Inc., of Somerville, Mass. | CP-101 | Microbiome therapeutic | Clostridium difficile infection | Company reported its decision to discontinue the Prism4 phase III study; decision follows an assessment by management team and board of directors of several factors, including slower-than-anticipated enrollment | 1/24/23 | Infection |
Janssen Pharmaceutical Cos., of Johnson and Johnson, of New Brunswick, N.J. | Vaccine regimen containing a mosaic-based adenovirus serotype 26 vector (Ad26.Mos4.HIV) | Vaccine regimen containing a mosaic-based adenovirus serotype 26 vector (Ad26.Mos4.HIV) | HIV infection | Data and safety monitoring board reported Mosaico phase III study showed vaccine not effective compared to placebo among study participants; no safety issues; discontinued study | 1/18/23 | Infection |
Moderna Inc., of Cambridge, Mass. | mRNA-1345 | Vaccine consisting of mRNA encoding for a stabilized RSV prefusion F glycoprotein, encapsulated in lipid nanoparticles; single-dose vaccine | Respiratory syncytial virus | Study achieved primary endpoint; vaccine efficacy of 83.7% against RSV lower respiratory tract disease (p<0.0001); well-tolerated; no safety concerns | 1/17/23 | Infection |
Discgenics Inc., of Salt Lake City | IDCT (rebonuputemcel) | Allogeneic discogenic cell therapy | Lumbar disc degeneration | Results from 2-year clinical data achieved primary endpoint; clinically meaningful, statistically significant improvements in low back pain (VAS), function (ODI), and quality of life (EQ-5D) by 12 weeks; sustained at 6 months, 1 year, 1.5 years, and 2 years post-injection; statistically significant improvements in disc volume and reached statistical significance at week 52 (p=0.0284) and week 104 (p=0.028); overall patient follow-up was 85% | 1/23/23 | Musculoskeletal |
AC Immune SA, of Lausanne, Switzerland, and partner Life Molecular Imaging, of Berlin | PI-2620 | Tau PET tracer with high binding affinity and selectivity for aggregated tau | Alzheimer’s disease | Imaged first patient | 1/18/23 | Neurology/Psychiatric |
Aculys Pharma Inc., of Tokyo | Pitolisant | Histamine H3 receptor antagonist/inverse agonist | Excessive daytime sleepiness associated with obstructive sleep apnea syndrome | Started the 148-patient study in Japan comparing pitolisant to placebo | 1/24/23 | Neurology/Psychiatric |
Annovis Bio Inc., of Berwyn, Pa. | Buntanetap | Inhibitor of neurotoxic aggregating proteins | Parkinson's disease | Based on current enrollment, interim analysis of patient who received 2 months of therapy expected to occur in the second quarter of 2023; analysis will determine the total number of patients that will be enrolled | 1/25/23 | Neurology/Psychiatric |
Aribio Co. Ltd., of Seongnam, South Korea | AR-1001 | Inhibits neuron apoptosis, restores synaptic plasticity and activates autophagy removal of amyloid plaques and tau proteins | Alzheimer’s disease | First subject enrolled; planning to enroll 800 participants | 1/5/23 | Neurology/Psychiatric |
Cingulate Inc., of Kansas City, Kan. | CTx-1301 | Extended-release tablet formulation of dexmethylphenidate | Attention deficit hyperactivity disorder | Initiated adult dose-optimization study to assess the onset and duration of efficacy and safety; initial results expected in the first half of 2023 | 1/4/23 | Neurology/Psychiatric |
Multidisciplinary Association for Psychedelic Studies (MAPS), of San Jose, Calif. | MDMA-assisted therapy | 3,4-methylenedioxymethamphetamine-assisted therapy | Post-traumatic stress disorder | Study met primary and secondary endpoints; no serious adverse events | 1/5/23 | Neurology/Psychiatric |
Neuroderm Ltd., of Rehovot, Israel | ND-0612 | Continuous delivery of carbidopa/levodopa via subcutaneous infusion | Parkinson's disease | Study met its primary endpoint of "Good ON" time with a statistically significant difference (p<0.0001) of 1.72 hours; positive and clinically meaningful results for the key secondary endpoint of "OFF" time (p<0.0001); MDS-Unified Parkinson's Disease Rating Scale Part II score (MDS-UPDRS motor experiences of daily living subscore) (p<0.0001); Patient Global Impression of Change (p<0.0001); Clinical Global Impression of Improvement (p<0.0001); well-tolerated; 5.5% discontinued due to adverse events | 1/9/23 | Neurology/Psychiatric |
Nrx Pharmaceuticals Inc., of Radnor, Pa. | NRX-101 | Oral; Fixed-dose combination product of D-cycloserine and lurasidone | Bipolar depression and sub-acute suicidality | Dosing of the first patient expected in the early 2023 | 1/3/23 | Neurology/Psychiatric |
Teva Pharmaceutical Industries Ltd., of Tel Aviv, Israel | TEV-44749 | Long-acting formulation of the atypical antipsychotic olanzapine | Schizophrenia | Started 640-patient study comparing 3 dose levels to placebo; primary endpoint is change from baseline to week 8 in the Positive and Negative Syndrome Scale total score | 1/24/23 | Neurology/Psychiatric |
Bausch + Lomb, unit of Bausch Health Cos. Inc., of Laval, Quebec, and Novaliq GmbH, of Heidelberg, Germany | NOV-03 (perfluorohexyloctane) | Ophthalmic liquid formulation | Dry eye disease associated with Meibomian gland dysfunction | Results published in the Ophthalmology showed study met both co-primary endpoints and all key secondary endpoints with statistical significance; change in total corneal fluorescein staining (tCFS) statistically significantly greater in the NOV-03 arm compared to the control saline group (p<0.001); eye dryness VAS score was statistically significantly improved in the NOV-03 arm compared to control group (p<0.001) at week 8; tCFS and eye dryness VAS score were statistically significant compared to saline at week 2 (p<0.01 for both); well-tolerated | 1/4/23 | Ocular |
Glaukos Corp., of Aliso Viejo, Calif. | Epioxa (Epi-on) | Corneal cross-linking therapy | Keratoconus | Initiated subject enrollment; to complete by the end of 2023 | 1/10/23 | Ocular |
Glaukos Corp., of Aliso Viejo, Calif. | GLK-301 (Ilution) | Sterile ophthalmic topical cream | Dry eye disease | Top-line results showed GLK-301 demonstrated improvement in the quality of tear film; improvement in quality of vision; planning to start phase IIb study in early 2023 | 1/10/23 | Ocular |
Oculis SA, of Lausanne, Switzerland | OCS-01 | High concentration, topical formulation of dexamethasone | Diabetic macular edema | Completed enrollment | 1/5/23 | Ocular |
Ocuphire Pharma Inc., of Farmington Hills, Mich. | Nyxol | Preservative-free, ophthalmic formulation of phentolamine mesylate; non-selective alpha-1 and alpha-2 adrenergic antagonist | Presbyopia | Initiated VEGA-2 phase III pivotal study; first patient enrolled in late December; to evaluate efficacy and safety for 2 labels as Nyxol alone and in combination with low-dose pilocarpine | 1/9/23 | Ocular |
Regenerx Biopharmaceuticals Inc., of Rockville, Md., | RGN-259 | Sterile, preservative-free, eye drop | Neurotrophic keratopathy | Data showed significant improvements at multiple time points in ocular discomfort, foreign body sensation, and dryness; no adverse effects | 1/3/23 | Ocular |
Achieve Life Sciences Inc., of Seattle | Cytisinicline | Plant-based alkaloid with a high binding affinity to the nicotinic acetylcholine receptor | Smoking cessation | Last subject dosed in ORCA-3 trial; randomized 792 subjects | 1/9/23 | Other/Miscellaneous |
Infant Bacterial Therapeutics AB, of Stockholm | IBP-9414 | Pharmaceutical-grade probiotic L. reuteri | Very low birth weight infants | Data from the Connection study, published in the British Journal of Gastroenterology, showed time to sustained feeding tolerance strongly influenced the duration of NICU stay | 1/24/23 | Other/Miscellaneous |
Rhythm Pharmaceuticals Inc., of Boston | Imcivree (setmelanotide) | Melanocortin-4 receptor agonist | Bardet-Biedl syndrome and obesity | Results published in the Orphanet Journal of Rare Diseases showed significant and clinically meaningful change in quality of life, with a mean change from baseline of +11.2 points on the Pediatric Quality of Life Inventory (PedsQL) measure at 52-week analysis in 9 patients younger than 18; significant and clinically meaningful improvement in weight-related quality of life, with a mean change from baseline of +12 points in 11 adult patients who completed the Impact of Weight on Quality of Life Questionnaire-Lite (IWQOL-Lite); IWQOL-Lite score significantly correlated with changes in percent body weight (p=0.0037) and body mass index (p=0.0098) | 1/23/23 | Other/Miscellaneous |
Bellerophon Therapeutics Inc., of Warren, N.J. | Inopulse | Pulsatile nitric oxide delivery system | Fibrotic interstitial lung disease | Last patient enrolled; top-line results in mid- 2023; 145 patients enrolled | 1/18/23 | Respiratory |
Actinium Pharmaceuticals Inc., of New York | Iomab-B | Iodine-131 (131I)-labeled murine monoclonal IgG1 antibody (BC8) targeting CD45 | Active relapsed or refractory acute myeloid leukemia | Sierra trial met primary and secondary endpoints; significant and clinically meaningful improvement in the secondary endpoint of event-free survival; 78% reduction in the probability of an event (p<0.0001); doubled 1-year survival compared to the control arm excluding crossover patients (26.1% vs 13.1%) as well as median overall survival (6.4 months vs. 3.2 months); well-tolerated with 4 times lower rates of sepsis (6.1% vs. 28.6%); Iomab-B enabled unprecedented access to BMT with 100% engraftment in patients compared to 18% of patients in the control arm; Iomab-B produced a 75% post-BMT complete remission (CR) rate compared to 6.3% post-BMT CR in the control arm | 2/19/23 | Cancer |
Bristol Myers Squibb Co., of Princeton, N.J. | Abecma (idecabtagene vicleucel) | Anti-BCMA CAR T-cell therapy | Multiple myeloma | Results from Karmma-3 study at a median follow-up of 18.6 months showed clinically meaningful and statistically significant improvement in the primary endpoint of progression-free survival (PFS) compared with standard regimens; median PFS of 13.3 months vs. 4.4 months (p<0.0001); 51% reduction in risk of disease progression or death with Abecma; overall response rate also met statistical significance with the majority of patients (71%); 39% achieved a complete response or stringent complete response vs. 5% response (p<0.0001); no new safety signals; results published in The New England Journal of Medicine, EBMT and the European Hematology Association | 2/10/23 | Cancer |
Bristol Myers Squibb Co., of Princeton, N.J. | Opdivo (nivolumab) | PD-1 immune checkpoint inhibitor | Locally advanced unresectable or metastatic urothelial carcinoma | Results from 3 years of follow-up in the CheckMate-274 trial showed median disease-free survival (DFS) of 22 months compared to 10.9 months with placebo; risk reduction of 29%; median DFS of 52.6 months vs. 8.4 months with placebo; 48% reduction in the risk of disease recurrence or death in patients whose tumor cells express PD-L1 ?1%; non-urothelial tract recurrence-free survival (NUTRFS) at 25.9 months compared to 13.7 months for placebo; median NUTRFS of 52.6 months with Opdivo vs. 8.4 months with placebo in patients whose tumor cells express PD-L1 ?1%; distant metastasis-free survival of 47.1 months vs. 28.7 months; median DMFS not reached vs. 20.7 months with placebo in patients whose tumor cells express PD-L1 ?1%; progression-free survival was 61.2 months vs. 47.1 months with placebo; median PFS not reached vs. 9.4 months with placebo in patients whose tumor cells express PD-L1 ?1% | 2/17/23 | Cancer |
Bristol Myers Squibb Co., of Princeton, N.J.; Exelixis Inc., of Alameda, Calif.; and Ipsen SA, of Paris | Cabometyx (cabozantinib) | Kinase inhibitor | Advanced renal cell carcinoma | Results from 3-year Checkmate-9ER pivotal trial in combination with Opdivo (nivolumab, Bristol Myers Squibb Co.) showed sustained survival and response rate benefits; 30% reduction in the risk of death; improvement in median OS vs. sunitinib (49.5 months vs. 35.5 months, respectively); double median PFS vs. sunitinib (16.6 months vs. 8.4 months, respectively) (p<0.0001); ORR 55.7% vs. 28.4%; median DOR of 23.1 months compared to 15.2 months with sunitinib; post-hoc analysis with 44 months median follow-up showed improvements were observed in both median PFS and OS | 2/13/23 | Cancer |
Can-Fite Biopharma Ltd., of Petach Tikva, Israel | Namodenoson | Small orally bioavailable drug that binds to A3 adenosine receptor | Hepatocellular carcinoma | Results published in Purinergic Signalling showed survival rate of 44% vs. 18% for placebo for 12-month | 2/14/23 | Cancer |
Coherus Biosciences Inc., of Redwood City, Calif., and Junshi Biosciences Co. Ltd., of Shanghai | Toripalimab | Anti-PD-1 monoclonal antibody | Recurrent or metastatic nasopharyngeal carcinoma | Final results from Jupiter-02 study in combination with chemotherapy showed statistically significant and clinically meaningful improvement in overall survival | 2/15/23 | Cancer |
G1 Therapeutics Inc., of Research Triangle Park, N.C. | Trilaciclib | CDK4/6 inhibitor | Metastatic colorectal cancer | Top-line preserve-1 study in combination with FOLFOXIRI and bevacizumab achieved its co-primary endpoint; clinically meaningful and statistically significant reductions in both occurrence of severe neutropenia during induction (placebo=20% vs. trilaciclib=1%; p<0.001); mean duration of severe neutropenia in cycles 1 through 4 (placebo=1.3 days vs. trilaciclib=0.1 days; p<0.001).; clinically meaningful reduction in the rate of chemotherapy-induced diarrhea; reductions in febrile neutropenia (placebo=5% vs. trilaciclib=0%) and ESA administration (placebo=7% vs. trilaciclib=3%); termination of study due to inconsistent results | 2/13/23 | Cancer |
Glycomimetics Inc., of Rockville, Md. | Uproleselan | Targeted antagonist of E-selectin | Acute myeloid leukemia | Independent data monitoring committee reviewed the interim utility analysis; recommended the study to continue to the originally planned final overall survival event; no safety issues | 2/15/23 | Cancer |
Jaguar Health Inc., of San Francisco | Crofelemer | Oral plant-based non-opioid agent | Chemotherapy-induced diarrhea | Achieved 75% patient enrollment; target trial enrollment of 256 patients expected to complete in the second quarter of 2023 | 2/21/23 | Cancer |
Junshi Biosciences Co. Ltd., of Shanghai | Toripalimab | Anti-PD-1 monoclonal antibody | Advanced triple-negative breast cancer | Prespecified interim analysis of Torchlight study in combination with paclitaxel for injection (albumin-bound) achieved primary endpoint; prolonged progression-free survival and resulted in improvement in overall survival in PD-L1-positive patients; no new safety signals | 2/20/23 | Cancer |
Kite, of Santa Monica, Calif., a Gilead Co. | Tecartus (brexucabtagene autoleucel) | CAR T-cell therapy | Relapsed/refractory B-cell acute lymphoblastic leukemia | Data from 3-year follow up from Zuma-3 study showed median overall survival (OS) of 26 months; high rates of durable response CR+CRi 71%; consistent safety profile | 2/9/23 | Cancer |
Merck & Co. Inc., of Rahway, N.J. | Keytruda (pembrolizumab) | Monoclonal antibody targeting PD-1 | Stage III-IV or recurrent endometrial carcinoma | NRG-GY018 trial in combination with carboplatin and paclitaxel met its primary endpoint of progression-free survival | 2/3/23 | Cancer |
Merck & Co. Inc., of Rahway, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 | HER2-negative locally advanced unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma | Keynote-859 study in combination with chemotherapy showed significantly improved overall survival; reduced risk of death by 22% (p<0.0001) compared to chemotherapy alone; median overall survival was 12.9 months vs. 11.5 months; median PFS of 6.9 months vs. 5.6 months; ORR was 51.3% vs. 42%; complete response rate of 9.5% vs. 6.2% and a partial response rate of 41.8% vs. 35.7% chemotherapy alone (p=0.00009); median duration of response was 8 months vs. 5.7 months | 2/16/23 | Cancer |
Merck & Co. Inc., of Rahway, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 monoclonal antibody | Metastatic castration-resistant prostate cancer | Discontinued KEYNOTE-641 study in combination with enzalutamide and androgen deprivation therapy based on the recommendation of an independent data monitoring committee; no improvement in radiographic progression-free survival or overall survival (OS) compared to placebo plus enzalutamide and ADT; crossed a prespecified futility boundary for OS; no new safety signals | 2/28/23 | Cancer |
Merck & Co. Inc., of Rahway, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 monoclonal antibody | Metastatic nonsquamous non-small-cell lung cancer | KEYNOTE-789 in combination with pemetrexed plus platinum-based chemotherapy did not meet its dual primary endpoint of overall survival (OS); improvement in OS at the final analysis of the study compared to pemetrexed with platinum-based chemotherapy; results did not meet statistical significance per the prespecified statistical plan; improvement in progression-free survival compared to chemotherapy alone but results did not reach statistical significance; no new safety signals | 2/28/23 | Cancer |
Merck KGaA, of Darmstadt, Germany | Bavencio (avelumab) | Human anti-PD-L1 antibody | Advanced urothelial carcinoma | Long-term follow up data of Javelin study showed median OS of 29.7 months vs. 20.5 months best supportive care (BSC); median OS of 31 months for cisplatin plus gemcitabine+BSC vs. 23 months for BSC alone; median OS of 25.8 months for carboplatin plus gemcitabine+BSC vs. 17.6 months for BSC alone; no new safety concerns | 2/13/23 | Cancer |
Orion Corp., of Espoo, Finland | Nubeqa (darolutamide) | Androgen receptor inhibitor | Metastatic hormone-sensitive prostate cancer | Sub-group analysis from phase III Arasens trial plus androgen deprivation therapy (ADT) and docetaxel showed increased overall survival; reduced risk of death in patients (p<0.0001); results simultaneously published in the Journal of Clinical Oncology | 2/16/23 | Cancer |
Pfizer Inc., of New York | Talzenna (talazoparib) | Poly ADP-ribose polymerase inhibitor | Metastatic castration-resistant prostate cancer | Talapro-2 study in combination with Xtandi (enzalutamide) showed statistically significant and clinically meaningful improvement in radiographic progression-free survival (rPFS) compared to placebo plus Xtandi; reduced the risk of disease progression or death by 37% vs. placebo + Xtandi (p< 0.001); median rPFS not reached vs. 21.9 months for placebo + Xtandi; overall survival immature; clinically meaningful improvement in median rPFS in HRR-deficient (p<0.001); HRR-non-deficient or unknown (p=0.004); median time to definitive clinically meaningful deterioration in global health status/quality of life was also longer, 30.8 vs 25.0 months (p=0.04) | 2/16/23 | Cancer |
Telix Pharmaceuticals Ltd., of Melbourne, Australia | TLX250-CDx (89Zr-DFO-girentuximab) | Imaging agent | Clear cell renal cell carcinoma | Trial achieved primary and secondary endpoints; confirmed favorable safety and tolerability profile; sensitivity of ?84% and specificity of ?84% in all 3 readers (86%/87% overall); achieved ?85% sensitivity and ?89% specificity in small renal masses (cT1a ?4cm); no unexpected safety signals | 2/19/23 | Cancer |
Bayer AG, of Whippany, N.J. | Asundexian (BAY-2433334) | Oral inhibitor of factor Xia | Atrial fibrillation; non-cardioembolic ischemic stroke or high-risk transient ischemic attack | First patient enrolled; expected to enroll 27,000 patients in 40 countries | 2/8/23 | Cardiovascular |
Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. | Orladeyo (berotralstat) | Plasma kallikrein inhibitor | Hereditary angioedema | Long-term Apex-S study showed well-tolerated; mild and transient treatment-related adverse events; no new safety signals; clinically meaningful and sustained reductions in HAE attack rates; median attack rate of 0.0 attacks/month was observed in 20 of 24 months; improved quality of life (QoL); patient-reported improvements from baseline to week 96 in total AE-QoL score | 2/24/23 | Dermatologic |
Dermavant Sciences Ltd., of Long Beach, Calif. | Vtama (tapinarof) | Nonsteroidal topical cream/aryl hydrocarbon receptor agonist | Atopic dermatitis | Completed patient enrollment; top-line results expected in the first half of 2023 | 2/13/23 | Dermatologic |
Innovent Biologics Inc., of San Francisco | IBI-112 (picankibart) | Recombinant anti-interleukin 23p19 subunit antibody | Moderate to severe plaque psoriasis | First patient dosed | 2/16/23 | Dermatologic |
Kalvista Pharmaceuticals Inc., of Cambridge, Mass. | Sebetralstat | Orally disintegrating formulation of plasma kallikrein inhibitor | Hereditary angioedema | Enrolled more than 50% of the 114 targeted number of patients in Konfident study; top-line data in the second half of 2023 | 2/14/23 | Dermatologic |
Leo Pharma A/S, of Ballerup, Denmark | Delgocitinib cream | Pan-JAK inhibitor | Moderate to severe chronic hand eczema (CHE) | Top-line data showed study achieved primary and secondary endpoint; well-tolerated | 2/10/23 | Dermatologic |
Novartis AG, of Basel, Switzerland | Cosentyx (secukinumab) | Interleukin-17A inhibitor | Moderate to severe hidradenitis suppurativa | Long-term data published in The Lancet showed more than 50% experienced a meaningful reduction in pain; more than 60% of patients were free of flares at week 52; no new safety signals | 2/4/23 | Dermatologic |
Palvella Therapeutics Inc., of Wayne, Pa. | Qtorin | 3.9% rapamycin anhydrous gel | Pachyonychia congenita | Top-line results of Vapaus study expected in mid-2023; enrolled 80 patients | 2/21/23 | Dermatologic |
Amicus Therapeutics Inc., of Philadelphia | AT-GAA (cipaglucosidase alfa) | Alpha-glucosidase stimulator | Pompe disease | Long-term follow up data showed durable mean improvements in percent predicted six-minute walk distance (6MWD) from baseline in ERT-experienced and -naïve patients and sustained through 104 weeks of follow-up; pulmonary function remained stable; decline in FVC; durable reductions in key biomarkers of muscle damage and disease substrate; decline in serum CK levels to week 52; reduction in Hex4; mild to moderate in severity; no new safety signals | 2/22/23 | Endocrine/Metabolic |
Applied Therapeutics Inc., of New York | AT-007 (govorestat) | Penetrant aldose reductase inhibitor | Sorbitol dehydrogenase (SORD) deficiency | Prespecified interim data showed AT-007 reduced sorbitol levels by a mean of approximately 52% over 90 days of treatment (p<0.001 vs. placebo) in patients; safe and well-tolerated | 2/15/23 | Endocrine/Metabolic |
Chiesi Global Rare Diseases, a business unit of Parma, Italy-based Chiesi Farmaceutici SpA | PRX-102 (pegunigalsidase alfa) | Modified stabilized version of the recombinant human alpha-galactosidase A protein | Fabry disease | Long-term results from Balance study showed noninferiority to agalsidase beta based on the median estimated glomerular filtration rate (eGFR) annualized slope | 2/24/23 | Endocrine/Metabolic |
Junshi Biosciences Co. Ltd., of Shanghai | JS-002 (ongericimab) | Recombinant humanized anti-PCSK9 monoclonal antibody | Primary hypercholesterolemia and mixed dyslipidemia | Study met primary endpoints; significant lipid-lowering efficacy in both studies with good safety | 2/27/23 | Endocrine/Metabolic |
Sanofi SA, of Paris | Nexviazyme (avalglucosidase) | Enzyme replacement therapy | Late-onset Pompe disease | Long-term Comet extension study showed sustained treatment effect; sustained improvements in respiratory and motor function; patients who switched from alglucosidase alfa to Nexviazyme treatment during the extension period experienced stabilization of treatment effect showing a 1.18 point improvement in FVC percent-predicted and an average increase of 0.29 meters in walking distance (6MWT), compared to baseline; no new safety signals | 2/27/23 | Endocrine/Metabolic |
Sanofi SA, of Paris | Nexviazyme (avalglucosidase) | Enzyme replacement therapy | Late-onset Pompe disease | Subgroup analyses showed patients who responded to alglucosidase alfa treatment in the primary analysis period had increased respiratory function (p<0.001), which was maintained throughout the extension period with Nexviazyme treatment (p=0.88); patients who did not respond to alglucosidase alfa treatment in the primary analysis period had reduced respiratory function while taking alglucosidase alfa (p=0.016); switching to Nexviazyme halted this decline in the extension period (p=0.81) | 2/27/23 | Endocrine/Metabolic |
Theratechnologies Inc., of Montreal | Tesamorelin | GHRH receptor agonist | Metabolic syndrome in HIV infection | Data showed prevalence did not differ significantly between responders and non-responders (34.2% vs. 43.8%, respectively, by NCEP; p=0.077); prevalence of metabolic syndrome decreased in responders, significantly lower prevalence compared to non-responders (30.8% vs. 48.5%; p<0.001); triglycerides reduction (17.5% in responders vs. 8.0% in non-responders; p=0.026) and waist circumference decrease (13.3% vs. 5.8%; p=0.034) | 2/22/23 | Endocrine/Metabolic |
Galapagos NV, of Mechelen, Belgium | Jyseleca (filgotinib) | JAK inhibitor | Crohn's disease | Top-line results from Diversity study missed the co-primary endpoints of clinical remission and endoscopic response at week 10 in 2 induction cohorts; achieved the co-primary endpoints of clinical remission (43.8% vs. 26.4%; p=0.0382) and endoscopic response (30.4% vs. 9.4%; p=0.0038) at week 58 at 200-mg; consistent safety profile | 2/9/23 | Gastrointestinal |
Eloxx Pharmaceuticals Inc., of Watertown, Mass. | ELX-02 | Small-molecule drug candidate designed to restore production of full-length functional proteins | Alport syndrome in patients with nonsense mutations | First patient dosed; top-line results expected in the first half of 2023 | 2/27/23 | Genitourinary/Sexual Function |
Roche Group AG, of Basel, Switzerland | Crovalimab | Anti-C5 recycling monoclonal antibody | Paroxysmal nocturnal hemoglobinuria | Data from Commodore2 study met its co-primary efficacy endpoints of transfusion avoidance and control of hemolysis levels; achieved disease control; favorable benefit-risk profile | 2/7/23 | Hematologic |
Akari Therapeutics plc, of New York | Nomacopan | Bispecific recombinant inhibitor of complement C5 activation and leukotriene B4 | Pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy | Study moved to part B; enrollment expected in 2024 for adults; enrollment not completed for the youngest age group (ages 0.5 to <2 years) in part A | 2/13/23 | Immune |
Akari Therapeutics plc, of New York | Nomacopan | Bispecific recombinant inhibitor of complement C5 activation and leukotriene B4 | Pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy | Case study result of 6-year old male patient from phase III part A showed reduction in terminal complement activity by 95% from a high baseline CH50 of 299.6U Eq/ml; sC5b9 normalized; neurological symptoms following a period of hypertension; stopped for 3 days and restarted after the diagnosis not related to nomacopan; resolved gut pathology and thrombocytopenia; patient discharged and remains well and in remission; no adverse events reported during 72-day treatment period | 2/21/23 | Immune |
Sanofi SA, of Paris | Tolebrutinib | Brain-penetrant Bruton’s tyrosine kinase inhibitor | Myasthenia gravis | Discontinued phase III study, citing crowded therapeutic space | 2/3/23 | Immune |
Takeda Pharmaceutical Ltd., of Osaka, Japan | Vedolizumab | Humanized monoclonal antibody designed to specifically antagonize the alpha4beta7 integrin | Graft-vs-host disease | Graphite study met its primary endpoint; achieved a statistically significant and clinically meaningful improvement in lower gastrointestinal GvHD-free survival compared to placebo (85.5% vs. 70.9%) by day 180 (p<0.001); statistically significant and intestinal aGvHD-free and relapse-free survival by day 180 (p=0.0043); grade C-D aGvHD-free (with any organ involvement) survival (p=0.0204); no new safety signals | 2/21/23 | Immune |
Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. | Peginterferon lambda | Type III interferon | Mild-to-moderate COVID-19 | Results published in the New England Journal of Medicine showed 51% reduction in COVID-19-related hospitalizations or emergency room visits greater than 6 hours vs. placebo; reduced risk of hospitalization by 42% and COVID-19 related death by 50% | 2/9/23 | Infection |
Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | IONIS-HBVRx (bepirovirsen) | Antisense medicine | Chronic hepatitis B infection | Initiated 2 randomized, double-blind, placebo-controlled phase III studies to evaluate safety and efficacy | 2/1/23 | Infection |
Merck & Co. Inc., of Rahway, N.J. | Lagevrio (molnupiravir) | Antiviral | COVID-19 | Study did not met primary endpoint; did not demonstrate a statistically significant reduction in the risk of COVID-19 following household exposure to another individual with COVID-19; consistent safety profile | 2/21/23 | Infection |
Merck & Co. Inc., of Rahway, N.J. | Islatravir | Nucleoside reverse transcriptase translocation inhibitor; oral | HIV-1 infection | Initiated patient enrollment in phase III study in combination with doravirine 100 mg | 2/22/23 | Infection |
Moderna Inc., of Cambridge, Mass. | mRNA-1010 | mRNA-based seasonal influenza vaccine | Seasonal influenza infection | Interim results showed an acceptable safety and tolerability profile; non-inferiority was not met for seroconversion rates and geometric mean titer ratios for the influenza B/Victoria- and B/Yamagata-lineage strains; superiority on seroconversion rates for A/H3N2 and A/H1N1, superiority on geometric mean titer ratios for A/H3N2, and non-inferiority on geometric mean titer ratios for A/H1N1 | 2/16/23 | Infection |
Pfizer Inc., of New York, and Valneva SE, of Saint-Herblain, France | VLA-15 | Lyme disease vaccine | Lyme disease prophylaxis | Company decided to discontinue a significant percentage of participants in the U.S. enrolled for Valor phase III study; half of the total recruited participants in the trial being discontinued following violations of Good Clinical Practice at certain clinical trial sites run by a third-party clinical trial site operator | 2/17/23 | Infection |
Seres Therapeutics Inc., of Cambridge, Mass. | SER-109 | Oral microbiome therapeutic candidate consisting of a consortium of highly purified Firmicutes spores | Recurrent C. difficile infection | Results from Ecospor IV study published in the JAMA NETWORK OPEN showed 91.3% and 86.3% of subjects remained free of C. difficile infection recurrence at 8 and 24 weeks; well-tolerated; no treatment-related adverse events; rapid and steady improvement in HRQOL | 2/14/23 | Infection |
Shionogi & Co. Ltd., of Osaka, Japan | Ensitrelvir | 3CL protease inhibitor | COVID-19 | Study achieved primary and secondary endpoints; time to resolution of 5 symptoms of COVID-19 was 167.9 hours in patients (125 mg ensitrelvir) compared to 192.2 hours in the placebo group (p=0.0407); time to achieve a negative infectious viral titer was significantly shorter with ensitrelvir compared with placebo (a median time of 36.2 hours vs. 65.3 hours, p<0.0001); well-tolerated; no serious adverse drug reactions; no deaths | 2/21/23 | Infection |
Shionogi & Co. Ltd., of Osaka, Japan | Ensitrelvir | 3CL protease inhibitor | Long COVID | Exploratory data showed ensitrelvir significantly decreased overall risk of the presence of long COVID compared to placebo (14.5% and 26.3% reported consecutive symptoms in the 125-mg ensitrelvir and placebo groups, respectively, 45% relative risk reduction, p<0.05) significantly decreased overall risk of reporting 4 neurological symptoms at day 85 (29.4% and 44% reported symptoms at either day 85 or day 169 in the 125-mg ensitrelvir and placebo groups, respectively, 33% relative risk reduction, p<0.05); declined relative risk reduction 25% (p=0.1774) and 26% (p<0.05) vs. placebo for 14 COVID-19 symptoms and the 4 neurological symptoms, respectively, in overall population of patients | 2/21/23 | Infection |
Valneva SE, of Saint Herblain, France | VLA-1553 | Single-shot chikungunya vaccine | Chikungunya virus infection | Completed enrollment and vaccination; results expected in mid-2023 | 2/14/23 | Infection |
Viiv Healthcare Ltd., of London, and Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson | Cabenuva | Extended-release injectable suspensions of cabotegravir and rilpivirine hydrochloride | HIV infection | Study achieved primary endpoint of non-inferior virologic efficacy vs. daily oral Biktarvy (bictegravir/emtricitabine/tenofovir alafenamide [BIC/FTC/TAF]); mean treatment satisfaction scores (HIVTSQs) improved significantly in CAB+RPV LA compared to BIC/FTC/TAF from baseline (p<0.001); well-tolerated | 2/23/23 | Infection |
Westvac Biopharma Co. Ltd., of Chengdu, China, | Recombinant COVID-19 vaccine (Sf9 cells) | Recombinant COVID-19 vaccine (Sf9 cells) | COVID-19 | Vaccine induced high titers of neutralizing antibodies against omicron XBB.1.5, BQ.1, BF.7, BA.5, BA.2.75 and other subvariants | 2/19/23 | Infection |
Atyr Pharma Inc., of San Diego | Efzofitimod | Fusion protein comprising immunomodulatory domain of histidyl-tRNA synthetase fused to the FC region of a human antibody | Sarcoidosis | First patient dosed | 2/6/23 | Inflammatory |
Amylyx Pharmaceuticals Inc., of Cambridge, Mass. | AMX-0035 | Fixed-dose of sodium phenylbutyrate and ursodoxicoltaurine | Amyotrophic lateral sclerosis | Completed enrollment; top-line results in 2024; study enrolled 664 patients | 2/2/23 | Musculoskeletal |
Blueprint Medicines Corp., of Cambridge, Mass. | Ayvakit (avapritinib) | Kinase inhibitor | Indolent systemic mastocytosis | Pioneer trial showed Ayvakit achieved rapid, durable and statistically significant reductions on all measures of pathological mast cell burden compared to placebo at 24 weeks; proportion with ?50% reduction in serum tryptase, ?50% reduction in KIT D816V variant allele fraction and 50% reduction in bone marrow mast cell aggregates (p<0.0001 for all); statistically significant and clinically meaningful improvement in total symptom score TSS (p=0.003), ?30% reduction in TSS (p=0.009) and ?50% reduction in TSS (p=0.005); achieved a statistically significant and clinically meaningful improvement in the mean percent change in Mastocytosis Quality of Life Questionnaire (MC-QoL) total score compared to placebo at 24 weeks (p=0.001); well-tolerated; favorable safety profile | 2/26/23 | Musculoskeletal |
Mitsubishi Tanabe Pharma Canada Inc., of Toronto, a subsidiary of Mitsubishi Tanabe Pharma America Inc. | Radicava (edaravone, oral suspension) | Neuroprotectant; antioxidant agent; free radical scavenger; oral suspension | Amyotrophic lateral sclerosis | Results published in the Muscle & Nerve showed well-tolerated at week 48; no new safety signals; no serious treatment related adverse events; 25% of patients discontinued; no deaths due to drug | 2/27/23 | Musculoskeletal |
AB Science SA, of Paris | Masitinib | Targets microglia and mast cells | Mild to moderate Alzheimer’s disease | Results published in Alzheimer's Research & Therapy showed AB09004 study met its primary analysis endpoint; significantly slowed cognitive deterioration relative to placebo with a manageable safety profile at 4.5 mg/kg/day; significant treatment effect relative to placebo in the primary endpoint of ADAS-Cog; overall improvement in cognition (ADAS-cog from baseline of -1.46) vs. +0.69 increased cognitive deterioration; ADAS-cog between-group difference of -2.15 (p=0.0003); non-significant trend toward improved overall function relative to placebo as measured by the ADCS-ADL score p=0.038 | 2/28/23 | Neurology/Psychiatric |
Cassava Sciences Inc., of Austin, Texas | Simufilam | Small-molecule drug designed to restore normal shape and function of altered filamin A (FLNA) protein | Alzheimer’s disease | Total of 953 patients enrolled; planning to complete enrollment for both phase III studies by the end of 2023 | 2/8/23 | Neurology/Psychiatric |
Corium Inc., of Boston | Azstarys (serdexmethylphenidate and dexmethylphenidate) | Alpha 2 adrenoceptor agonist | Children with attention-deficit hyperactivity disorder | Results published in the Journal of Child and Adolescent Psychopharmacology showed sustained symptom improvement; children scored on average 16.1, decline of -25.3 (p<0.001) on ADHD rating scale-5 after 1 month treatment; significantly improved in the clinical severity of assessed by the Clinical Global Impressions–Severity (CGI-S) scale (p<0.0001 after 1 month of treatment); well-tolerated in children ages 6 to 12; safe; 8 serious treatment-emergent adverse events unrelated to study drug | 2/22/23 | Neurology/Psychiatric |
Horizon Therapeutics plc, of Dublin | Uplizna (inebilizumab) | B-cell depleting anti-CD19 monoclonal antibody | COVID-19 infection in neuromyelitis optica spectrum disorders | Data showed low incidence rate of infections (0.024 events per patient year) in Uplizna treated patients; vaccine titers showed no meaningful reduction after 3.5 years of treatment; favorable safety profile | 2/23/23 | Neurology/Psychiatric |
Idorsia Pharmaceuticals Ltd., of Allschwil, Switzerland | Clazosentan | Fast-acting, endothelin A (ETA) receptor antagonist | Delayed cerebral ischemia in patients following aneurysmal subarachnoid hemorrhage | Study did not meet the primary endpoint | 2/6/23 | Neurology/Psychiatric |
Pfizer Inc., of New York | Zavegepant | CGRP receptor antagonist | Migraine | Study met its co-primary endpoints; well-tolerated; no serious adverse events; co-primary efficacy endpoints of pain freedom (24% vs 15%, p<0·0001) and freedom from most bothersome symptom MBS (40% vs 31%, p=0·0012) at 2 hours post-dose; more effective than placebo on 13 of the 17 secondary endpoints | 2/16/23 | Neurology/Psychiatric |
Pharmazz Inc., of Willowbrook, Ill. | Sovateltide | Endothelin B receptor agonist | Acute ischemic stroke | Data showed statistically significant and clinically meaningful improvements in multiple neurological outcomes in patients; well-tolerated; mRS showed a statistically significantly greater number of patients (52.8% in control and 76.1% in sovateltide groups); improvement of ?2 points on mRS; mean score reduction (p=0.005) and greater number of patients (64.3% in control and 82.1% in sovateltide groups) with an improvement of ?6 points on NIHSS (p=0.019); improvement of ?40 points on BI (p=0.064); no drug related adverse events | 2/10/23 | Neurology/Psychiatric |
Alteogen Inc., of Daejeon, South Korea | ALT-L9 | Biosimilar of Eylea (aflibercept) | Neovascular age-related macular degeneration | Completed patient enrollment; enrolled 431 patients | 2/22/23 | Ocular |
Eluminex Biosciences Ltd., of Suzhou, China | EB-301 | Corneal implant from recombinant human type III collagen | Corneal blindness secondary to stromal lesions amenable to anterior lamellar keratoplasty (ALK) surgery | Completed clinical enrollment in part A of a 2-part pivotal study | 2/14/23 | Ocular |
Essex Biotechnology Ltd., of Hong Kong | HLX04-O | Recombinant anti-VEGF humanized monoclonal antibody | Wet age-related macular degeneration | First patient dosed | 2/10/23 | Ocular |
Neurophth Therapeutics Inc., of Wuhan, China | NR-082 | Gene therapy expressing a mitochondria codon-optimized NADH-dehydrogenase subunit 4 | Leber hereditary optic neuropathy with ND4 mutation | Completed patient enrollment | 2/21/23 | Ocular |
Roche Holding AG, of Basel, Switzerland, and its subsidiary Genentech Inc. | Vabysmo (faricimab) | Bispecific antibody for the eye targeting Ang-2 and VEGF-A | Macular edema due to branch and central retinal vein occlusion | Study achieved primary and secondary endpoint; non-inferior visual acuity gains compared to aflibercept; achieved rapid and robust drying of retinal fluid from baseline; vision gains were +16.9 eye chart letters in the Vabysmo arm and +17.5 letters in the aflibercept arm at 24 weeks in Balaton studies; vision gains were +16.9 letters in the Vabysmo arm and +17.3 letters in the aflibercept arm at 24 weeks in the Comino studies; absence of blood vessel leakage in the retina compared to aflibercept patientsin a pre-specified exploratory endpoint; consistent safety profile | 2/10/23 | Ocular |
Pharming Group NV, of Leiden, the Netherlands | Leniolisib | Oral, selective PI3K-delta inhibitor | Activated phosphoinositide 3-kinase delta syndrome | First pediatric patient enrolled | 2/21/23 | Other/Miscellaneous |
Akeso Inc., of Hong Kong | Cadonilimab (AK-104) | Bispecific antibody targeting PD-1/CTLA4 | Gastric cancer | Patient enrollment completed in phase III study in combination with chemotherapy; interim analysis expected by the end of 2023 | 3/14/23 | Cancer |
Ambrx Biopharma Inc., of San Diego | ARX-788 | Antibody-drug conjugate consisting of HER2-targeted monoclonal antibody incorporating non-native amino acid para-acetylphenylalanine linked to monomethyl auristatin F through PEG4-containing non-cleavable linker | HER2-positive metastatic breast cancer | Interim analysis of the ACE-Breast-02 study met its prespecified interim primary efficacy endpoint with statistical significance; greater progression-free survival benefit compared to the active control | 3/1/23 | Cancer |
Anaptysbio Inc., of San Diego, and GSK plc, of London | Jemperli (dostarlimab) | Anti-PD-1 inhibitor | Primary advanced or recurrent endometrial cancer | Interim results of Jemperli plus standard-of-care chemotherapy followed by dostarlimab alone compared to chemotherapy plus placebo showed 72% and 36% reduction in the risk of disease progression or death observed in the dMMR/MSI-H population and overall patient population, respectively; clinically meaningful overall survival trend observed; results published in The New England Journal of Medicine | 3/27/23 | Cancer |
Astellas Pharma Inc., of Tokyo | Xospata (gilteritinib) | FLT3 inhibitor | FMS-like tyrosine kinase 3 (FLT3)-internal tandem duplication (ITD) mutated acute myeloid leukemia | Top-line results of Morpho study did not meet its pre-defined primary endpoint of relapse-free survival compared to placebo; not statistically significant at the primary analysis; study including follow-up will be stopped as per the study protocol | 3/9/23 | Cancer |
Astrazeneca plc, of Cambridge, U.K. | Imfinzi (durvalumab) | Human monoclonal antibody that binds to the PD-L1 protein; blocked the interaction of PD-L1 with the PD-1 and CD80 proteins | Resectable early-stage (IIA-IIIB) non-small-cell- lung cancer | Interim analysis of Aegean study in combination with neoadjuvant chemotherapy before surgery and as adjuvant monotherapy after surgery demonstrated a statistically significant and clinically meaningful improvement in event-free survival vs. neoadjuvant chemotherapy alone; well-tolerated; no new safety signals | 3/9/23 | Cancer |
Astrazeneca plc, of Cambridge, U.K. | Tagrisso (osimertinib) | Irreversible EGFR-TKI | Early-stage (IB, II and IIIA) EGFRm non-small-cell-lung cancer | High-level results from the Adaura study demonstrated a statistically significant and clinically meaningful improvement in overall survival compared to placebo | 3/9/23 | Cancer |
Bayer AG, of Leverkusen, Germany | Nubeqa (darolutamide) | Androgen receptor inhibitor | Hormone-sensitive prostate cancer | Initiated phase III study in combination with androgen deprivation therapy | 3/23/23 | Cancer |
Bristol Myers Squibb Co., of New York | Opdivo (nivolumab) | PD-1 immune checkpoint inhibitor | Resectable non-small-cell lung cancer | Results from Checkmate-816 study in combination with platinum-based chemotherapy with median follow-up of 41.4 months reduced the risk of disease recurrence, progression or death by 32% in 3-year follow-up results; event-free survival (EFS) rate of 57% with Opdivo with chemotherapy compared to 43% with chemotherapy alone; time to distant metastasis or death showed as 71% vs. 50%; overall survival remained immature; 78% of patients treated with neoadjuvant Opdivo and chemotherapy were alive compared to 64% with chemotherapy; consistent safety profile; no new safety signals; grade 3-4 treatment-related and surgery-related adverse events as 36% and 11% of patients in the Opdivo with chemotherapy arm, respectively vs. 38% and 15% in the chemotherapy arm, respectively | 3/30/23 | Cancer |
Cel-Sci Corp., of Vienna, Va. | Multikine | Leukocyte interleukin injection | Locally advanced squamous cell carcinoma of the head and neck | Data showed 43% survival extension; progression-free survival was 8.4% higher at 5 years for patients treated with Multikine+ CIZ+SOC as compared to patients treated with SOC control alone; death rate was 15.6% vs. 48.7% death rate for the control patients who had an early tumor response; 43.8% death rate vs. 48.7% death rate for control in patients who did not have an early tumor response | 3/8/23 | Cancer |
Deciphera Pharmaceuticals Inc., of Waltham, Mass. | Vimseltinib | Selective switch-control kinase inhibitor of CSF1R | Tenosynovial giant cell tumor | Completed enrollment; expects to report top-line results in the fourth quarter of 2023 | 3/1/23 | Cancer |
Exelixis Inc., of Alameda, Calif. | Cabometyx (cabozantinib) | Kinase inhibitor | Locally advanced or metastatic clear cell or non-clear cell (papillary or unclassified only) renal cell carcinoma | Contact-3 study did not meet its primary endpoint of progression-free survival in combination with atezolizumab vs. cabozantinib alone; no new safety signals | 3/2/23 | Cancer |
Inovio Pharmaceuticals Inc., of Plymouth Meeting, Pa. | VGX-3100 | DNA vaccine | Cervical high-grade squamous intraepithelial lesions | Top-line results from Reveal2 study showed statistical significance was achieved in the all-participants population for the endpoint of lesion regression and viral clearance; biomarker-selected population meeting the primary endpoint was 28.6% vs. 0%, which was not statistically significant (p=0.115); clearance of virus in the all-participants population of 203 participants was statistically significant (27.6% of patient achieved endpoint vs. 8.7% for placebo, p=0.001); viral clearance in the all-participants population of Reveal2 was 37.3% in the 3-dose treatment group vs. 8.7% for placebo; well-tolerated; no treatment-related serious adverse events; mild to moderate adverse events | 3/1/23 | Cancer |
Inovio Pharmaceuticals Inc., of Plymouth Meeting, Pa. | VGX-3100 | DNA vaccine | Cervical high-grade squamous intraepithelial lesions | Ad-hoc integrated efficacy analysis of the results for both REVEAL1 and REVEAL2 study showed statistical significance in both the biomarker-selected and all-participants populations; combined biomarker-selected population of 92 participants achieved endpoint was 54.4% vs. 12.5% placebo (p<0.001); combined all-participants population of 404 participants achieved endpoint was 25% vs. 9.8% (p<0.001) | 3/1/23 | Cancer |
Kite, a unit of Foster City, Calif.-based Gilead Sciences Inc. | Yescarta (axicabtagene ciloleucel) | CAR T targeting CD19 | Relapsed/refractory large B-cell lymphoma | In the Zuma-7 study, Yescarta improved overall survival compared to standard of care; data to be presented at an upcoming scientific meeting | 3/21/23 | Cancer |
Merck & Co. Inc., of Rahway, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 therapy | Resectable stage II, IIIA or IIIB non-small-cell lung cancer | KEYNOTE-671 study met one of its dual primary endpoints; in combination with chemotherapy before surgery and continuing as a single agent after surgery showed a statistically significant improvement in event-free survival vs pre-operative chemotherapy; statistically significant improvements in key secondary endpoints of pathological complete response and major pathological response | 3/1/23 | Cancer |
Merck & Co. Inc., of Rahway, N.J. | Keytruda (pembrolizumab) | Monoclonal antibody targeting PD-1 | Stage III-IV or recurrent endometrial carcinoma | NRG?GY018 study in combination with standard-of-care chemotherapy (carboplatin and paclitaxel) then continued as a single agent every 6 weeks for up to 14 cycles showed statistically significant and clinically meaningful improvement in progression-free survival (PFS); after a median follow-up of 7.9 months, significantly reduced the risk of disease progression or death by 46% (p<0.00001) compared to chemotherapy alone in pMMR cohort of 591 evaluable patients; median PFS was 13.1 months vs. 8.7 months; at median follow-up of 12 months, treatment significantly reduced risk of disease progression or death by 70% ( p<0.00001) compared to chemotherapy alone in dMMR cohort of 225 evaluable patients; median PFS was not reached vs. 7.6 months; results published in The New England Journal of Medicine | 3/27/23 | Cancer |
Novartis AG, of Basel, Switzerland | Kisqali (ribociclib) | CDK4/6 inhibitor | HR+/HER2-negative early breast cancer | Independent data safety monitoring board recommended to stop the trial as primary endpoint of invasive disease-free survival (iDFS) has been met; Kisqali in combination with endocrine therapy significantly reduced the risk of disease recurrence, compared to standard adjuvant ET alone | 3/27/23 | Cancer |
PDS Biotechnology Corp., of North Brunswick, N.J. | PDS-0301 (NHS-IL12) | Fusion protein consisting of a tumor-targeting antibody conjugated to interleukin 12 | Unresectable, recurrent/metastatic human papillomavirus 16-positive head and neck squamous cell carcinoma | Initiated phase III study in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) | 3/30/23 | Cancer |
Pfizer Inc., of New York, and Astellas Pharma Inc., of Tokyo | Xtandi/enzalutamide | Androgen receptor signaling inhibitor | Non-metastatic hormone-sensitive prostate cancer | Top-line results from Embark trial met its primary endpoint with a statistically significant and clinically meaningful improvement in metastasis-free survival (MFS) for patients treated with Xtandi plus leuprolide vs. placebo plus leuprolide; overall survival data not yet mature; statistically significant and clinically meaningful improvement in MFS for patients treated with Xtandi monotherapy vs. placebo plus leuprolide; reached statistical significance in time to prostate-specific antigen (PSA) progression and time to first use of new antineoplastic therapy; no new safty signals; | 3/16/23 | Cancer |
Renovorx Inc., of Los Altos, Calif. | Renovogem | Drug-device combination product candidate | Locally advanced pancreatic cancer | Interim analysis showed median overall survival by 24 weeks with statistical significance was not reached to stop the study early (p=0.051) | 3/8/23 | Cancer |
Renovorx Inc., of Los Altos, Calif. | Renovogem | Drug-device combination product candidate | Locally advanced pancreatic cancer | Interim analysis showed 6-month median overall survival benefit for patients; 60% improvement vs. the study control arm; greater than 65% reduction in adverse events | 3/28/23 | Cancer |
Servier SAS, of Paris | Vorasidenib | Oral, brain-penetrant dual inhibitor of mutant IDH1 and IDH2 | Residual or recurrent IDH-mutant low-grade glioma | Indigo trial as monotherapy met its primary endpoint of progression-free survival and the key secondary endpoint of time to next intervention; prespecified interim analysis were both statistically significant and clinically meaningful | 3/14/23 | Cancer |
Springworks Therapeutics Inc., of Stamford, Conn. | Nirogacestat | Oral gamma secretase inhibitor | Desmoid tumors | Results published in the New England Journal of Medicine showed trial met its primary endpoint of improving progression-free survival, statistically significant improvement for nirogacestat over placebo; 71% reduction in the risk of disease progression (p< 0.001); median PFS was not reached in the nirogacestat arm and 15.1 months in the placebo arm; improved PFS across pre-specified subgroups; confirmed objective response rate based on RECIST v1.1 was 41% with nirogacestat versus 8% with placebo (p<0.001); complete response rate was 7% in the nirogacestat arm and 0% in the placebo arm; statistically significant and clinically meaningful improvements in patient-reported outcomes; significantly reduced pain (p<0.001) and other desmoid tumor-specific symptoms (p<0.001); significantly improved physical/role functioning (p<0.001); overall health-related quality of life (p?0.01) | 3/8/23 | Cancer |
Telix Pharmaceuticals Ltd., of Australia | TLX250-CDx (89Zr-DFO-girentuximab) | Imaging agents | Clear cell renal cell carcinoma | Additional data showed high degree of consistency between readers with a very high sensitivity (85.5%) and specificity (87%); 85.5%/89.5% for small masses (<4 cm) confirming optimal SUVmax thresholds for image interpretation; correlation of CAIX expression on histology with imaging results | 3/13/23 | Cancer |
TME Pharma NV, of Berlin | NOX-A12 | CXCL12 inhibitor | Glioblastoma | Gloria study in combination with standard of care radiotherapy and bevacizumab showed 83% of patients alive after 12 months; median overall survival not yet reached | 3/27/23 | Cancer |
Amarin Corp. plc, of Dublin | Vascepa (icosapent ethyl) | Oral ethyl-eicosapentaenoic acid | Acute coronary syndrome (recent ACS) | Post-hoc analysis from Reduce-IT study showed Vascepa substantially and significantly reduced the risk of first and total ischemic events by 37% and 36%, respectively in patients with recent ACS without increasing bleeding, supporting early initiation of Vascepa after ACS; absolute risk reduction for first events with Vascepa over 5 years for the primary composite endpoint was 9.3% with a number needed to treat (NNT) of 11; absolute risk reduction for first events in patients with ACS ?12 months was 4.7% with an NNT of 21; consistent tolerability and adverse event patterns with placebo; bleeding event rates were no more frequent compared to placebo with dual antiplatelet therapy | 3/5/23 | Cardiovascular |
Janssen Research & Development LLC, of Raritan, N.J, a unit of Johnson & Johnson | Opsynvi (macitentan 10 mg and tadalafil 40 mg) | Dual endothelin receptor A and B antagonist and PDE5 inhibitor | Pulmonary arterial hypertension | A DUE study met its co-primary endpoint; marked pulmonary hemodynamic improvement and highly statistically significant; consistent and robust PVR reduction; PVR change was significantly greater vs. macitentan (p<0.0001) and tadalafil (p<0.0001); not statistically significant in 6MWD; clinically relevant improvement in 6MWD vs. macitentan (p=0.380) and tadalafil (p=0.059); no new safety observations | 3/6/23 | Cardiovascular |
Lexicon Pharmaceuticals Inc., of The Woodlands, Texas | Sotagliflozin | Dual SGLT1 and SGLT2 inhibitor | Heart failure (HF) | Post-hoc analysis in patients with worsening HF showed it was statistically significant (p<0.05) and remained significant throughout follow-up; sub-group analysis of patients with left ventricular ejection fraction (LVEF) < 50% or ? 50% showed statistically significant reduction in the risk for the primary outcome on day 27 | 3/4/23 | Cardiovascular |
Merck & Co. Inc., of Rahway, N.J. | Sotatercept | Activin signaling inhibitor | Pulmonary arterial hypertension | Sotatercept significantly improved exercise capacity; increased 6MWD by 40.8 meters (p<0.001) from baseline at week 24; statistically significant and clinically meaningful improvements in 8 of 9 secondary outcome measures; reduced the risk of clinical worsening or death by 84% compared to placebo with a median follow-up of 32.7 weeks (p<0.001); results published in The New England Journal of Medicine | 3/6/23 | Cardiovascular |
Almirall SA, of Barcelona, and Eli Lilly and Co., of Indianapolis | Lebrikizumab | Monoclonal antibody that binds to IL-13 | Moderate to severe atopic dermatitis | Both Advocate 1 and Advocate 2 study met the co-primary endpoints; Advocate 1 results published in the New England Journal of Medicine showed proportions of patients achieved IGA 0/1 at week 16 were 43.1% of patients treated with lebrikizumab vs. 12.7% of those treated with placebo; EASI-75 responses were 58.8% vs. 16.2%, respectively; proportions for IGA 0/1 were 33.2% in patients treated with lebrikizumab vs. 10.8% in those treated with placebo in Advocate 2 study; EASI-75 responses were 52.1% vs. 18.1%, respectively; statistically significant (p<0.0001); improved measures of itch and itch interference on sleep; mild to moderate in severity treatment-related adverse events; week 52 results published in the British Journal of Dermatology; 74% of patients dosed every 4 weeks and 76% of patients dosed every 2 weeks maintained clear or almost clear skin (IGA 0 or 1) at 1 year of treatment; 79% of patients dosed every 4 weeks and every 2 weeks maintained 75% or greater skin improvement (EASI-75) at 1 year of treatment in Advocate 1 study; 81% of patients dosed every 4 weeks and 65% of patients dosed every 2 weeks maintained clear or almost clear skin (IGA 0 or 1) at 1 year of treatment and 85% of patients dosed every 4 weeks and 77% of patients dosed every 2 weeks maintained EASI-75 response at 1 year of treatment in Advocate 2 study; clinically meaningful reductions in itch at 1 year of treatment (Advocate 1: 80% of patients dosed every 4 weeks, and 81% of those dosed every 2 weeks; Advocate 2: 88% of patients dosed every 4 weeks and 90% of those dosed every 2 weeks) | 3/31/23 | Dermatologic |
CSL Behring, a unit of Melbourne, Australia-based CSL Ltd. | CSL-312 (garadacimab) | Monoclonal antibody targeting factor XIIa | Hereditary angioedema | Results published in The Lancet showed vanguard study met its primary and secondary endpoints; statistically lower monthly attack rate compared to placebo over the 6-month treatment period (p< 0.0001); mean monthly attack rate was 0.27 for patients treated with garadacimab vs. 2.01 for placebo; median monthly attack rate of 0 for patients treated with garadacimab vs. 1.35 for placebo; attack rate reduction in means of 86.5% vs. placebo and an attack rate reduction in medians of 100% vs. placebo; patients treated with garadacimab once a month experienced a reduction in the number of time-normalized moderate or severe attacks; reduction in the number of HAE attacks requiring on-demand rescue medication compared to placebo; improvements in patient-reported outcomes, with 81.6% as good or better in Subject's Global Assessment of Response to Therapy (SGART) response in the 6-month trial compared to 33.3% in placebo; favorable safety profile | 3/1/23 | Dermatologic |
Dermavant Sciences Ltd., of Long Beach, Calif. | Vtama (tapinarof) | Nonsteroidal topical cream/aryl hydrocarbon receptor agonist | Atopic dermatitis | Top-line results of Vtama achieved primary and secondary endpoints in pediatric subjects down to 2 years old and adult subjects; statistically significant improvement in the vIGA-AD score of clear (0) or almost clear (1) with at least a 2-grade improvement from baseline at week 8 (p<0.0001); statistically significant improvement in the proportion of subjects with ?75% improvement in EASI75 from baseline at week 8 (p<0.0001); highly statistically significant improvement ?4-point reduction in Peak Pruritis Numeric Rating Scale (PP-NRS) in itch (p=0.0015); no safety or tolerability signals in children as young as 2; mild to moderate adverse events; low study discontinuation rate due to adverse events (1.5% VTAMA vs. 3.0% vehicle); contact dermatitis (1.1% VTAMA vs. 1.5% vehicle) and follicular event (8.9% VTAMA vs. 1.5% vehicle) | 3/15/23 | Dermatologic |
Janssen Pharmaceutical Co., a unit of Johnson & Johnson, of New Brunswick, N.J. | Stelara (ustekinumab) | Human interleukin (IL)-12 and IL-23 antagonist | Moderate to severe ulcerative colitis | Results from Unifi Lte study (n=205) showed 58% (119/205) of patients were in clinical remission; 80% of patients were in clinical response; 79.5% of patients were in modified Mayo score response; 67% of patients showed endoscopic improvement | 3/4/23 | Dermatologic |
Janssen Pharmaceutical Co., part of New Brunswick, N.J.-based Johnson & Johnson | Tremfya (guselkumab) | Fully human monoclonal antibody that selectively binds to the p19 subunit of IL-23 and inhibits its interaction with the IL-23 receptor | Moderate to severe plaque psoriasis | Post-hoc analysis of Voyage-2 study showed durable clinical efficacy in adult patients; mean scalp-specific Investigator Global Assessment (ss-IGA) score rapidly improved from 2.9 at week 0 to 0.2 at week 24, and 0.3 at week 48; improvements in scalp psoriasis and quality-of-life measures at week 48 | 3/17/23 | Dermatologic |
Kintor Pharmaceutical Ltd., of China | KX-826 (pyrilutamide) | Androgen receptor antagonist | Androgenetic alopecia | Completed enrollment of 740 subjects in China | 3/28/23 | Dermatologic |
Leo Pharma Inc., of Madison, N.J. | Adbry (tralokinumab) | Human monoclonal antibody targeting IL-13 | Moderate to severe atopic dermatitis | Results from ECZTRA 6 trial showed Adbry significantly reduced the abundance of Staphylococcus aureus in the lesional and non-lesional skin of adolescents after 16 weeks; improvement in skin gene expression from a lesional to a non-lesional skin profile; improvement occurred in 29% and 41% of the differentially expressed genes in lesional skin in patients treated with Adbry (150-mg and 300-mg groups, respectively), compared to a 4% improvement in the placebo; Interim analysis of adolescent patients in the ECZTEND trial showed similar safety profile to the adult population for up to 3 years; 75% improvement in the extent and severity (EASI-75) at 2 years of treatment with Adtralza at 8/10 patients; | 3/17/23 | Dermatologic |
Hanmi Pharmaceuticals Co. Ltd., of Seoul, South Korea | Rosuzet | Single-pill combination of rosuvastatin and ezetimibe | Dyslipidemia | Racing study results published in the Lancet showed Rosuzet is non-inferior to high-intensity statin monotherapy; LDL cholesterol concentrations of less than 70 mg/dL at 1, 2, and 3 years were observed in 73%, 75%, and 72% of patients in Rosuzet group vs. 55%, 60%, and 58% of patients in the high-intensity statin monotherapy group, respectively (p<0.0001); post-hoc analysis showed LDL cholesterol concentrations of less than 55 mg/dL at 1, 2, and 3 years were observed in 42%, 45%, and 42% of patients in Rosuzet group and 25%, 29%, and 25% of patients in the high-intensity statin monotherapy group, respectively; intolerance-related discontinuation or dose reduction in 88 patients in Rosuzet group and 150 patients in the high-intensity statin monotherapy group (p<0.0001) | 3/17/23 | Endocrine/Metabolic |
Hanmi Pharmaceuticals Co. Ltd., of Seoul, South Korea | Rosuzet | Single-pill combination of rosuvastatin and ezetimibe | Dyslipidemia | Sub-analysis of Racing study results published in European Heart Journal showed incidence of the primary outcome was similar between the 2 groups; 10% in Rosuzet group and 11.3% in the high-intensity statin monotherapy group (P=0.460); LDL-C target achievement rate (less than 70 mg/dL) was significantly higher than that in the high-intensity statin monotherapy group after 3-year follow-up; reduced total cholesterol and triglyceride; intolerance-related discontinuation or dose reduction was significantly lower | 3/17/23 | Endocrine/Metabolic |
Novo Nordisk A/S, of Bagsvaerd, Denmark | Wegovy (semaglutide) | GLP-1 receptor agonist | Obesity and type 2 diabetes | Pioneer plus trial achieved its primary endpoint; statistically significant and superior reduction in HbA1c (1.9% and 2.2% at 25-mg and 50-mg doses, respectively) at week 52 vs. 14-mg (1.5%) dose of oral semaglutide from mean baseline HbA1c of 9%; statistically significant higher weight loss of 7 kg and 9.2 kg, respectively, compared with a reduction of 4.5 kg with oral semaglutide 14-mg from mean baseline body weight of 96.4 kg; safe and well-tolerated; mild to moderate adverse events | 3/24/23 | Endocrine/Metabolic |
Abbvie Inc., of North Chicago | Skyrizi (risankizumab) | IL-23 inhibitor | Ulcerative colitis | Top-line results met the primary endpoint of clinical remission and secondary endpoints in adult patients at 1200-mg intravenous; 20.3% of patients receiving risankizumab achieved clinical remission compared to 6.2% of patients receiving placebo (p<0.00001); significantly greater proportion of patients treated with risankizumab achieved endoscopic improvement at week 12 compared to placebo (36.5% vs 12.1%; p<0.00001); 24.5% of patients treated with risankizumab achieved histologic-endoscopic mucosal improvement at week 12 vs 7.7% of those receiving placebo (p<0.00001); consistent safety profile | 3/23/23 | Gastrointestinal |
Celltrion USA Inc., of Jersey City, N.J. | CT-P13 SC | Subcutaneous formulation of infliximab | Inflammatory bowel disease | Liberty-CD study (n=343) showed clinical remission rate was greater in the CT-P13 SC arm than placebo (62.3% and 32.1% respectively, P<0.0001) at week 54; rate of clinical remission at week 54 was significantly greater in CT- P13 SC (43.2%) compared to the placebo (20.8%) (P<0.0001) in LIBERTY-UC study (n=438) | 3/3/23 | Gastrointestinal |
Janssen Pharmaceutical Cos., a unit of Johnson & Johnson, of New Brunswick, N.J. | Stelara (ustekinumab) | Human interleukin (IL)-12 and IL-23 antagonist | Moderately to severely active Crohn’s disease (CD) and ulcerative colitis (UC) | Final pooled safety analysis of phase II/III study showed well-established safety experience in adult patients with UC through up to 4 years and CD through 5 years; frequently reported serious infections of anal abscess, pneumonia, cellulitis, and abdominal abscess were similar between Stelara and placebo, except gastroenteritis (0.25 Stelara vs. 0.11 placebo) | 3/4/23 | Gastrointestinal |
Astellas Pharma Inc., of Tokyo | Fezolinetant | Neurokinin-3 receptor antagonist | Moderate to severe vasomotor symptoms due to menopause | Skylight-1 study results published in The Lancet study met the 4 co-primary efficacy endpoints; statistically significant improvements from baseline in VMS frequency and severity at 4 and 12 weeks compared to placebo at 30 mg and 45 mg; improvements from week 1 and maintained throughout the remainder of the 52-week study period; treatment-emergent adverse events showed 37% and 43% at 30-mg and 45-mg doses, respectively vs. 45% of placebo participants; improvements in sleep disturbance were observed, statistical significance was not met for either fezolinetant dose at 12 weeks; reported improvements at 4 and 12 weeks compared with placebo for Patient Global Impression of Change in Sleep Disturbance (PGI-C SD) and Patient Global Impression of Severity in Sleep Disturbance (PGI-S SD) scales; significant and clinically meaningful improvement for quality of life at 4 and 12 weeks that was maintained through 52 weeks | 3/13/23 | Genitourinary/Sexual Function |
Calliditas Therapeutics AB., of Stockholm | Nefecon | Budesonide delayed-release capsules | Primary IgA nephropathy | Top-line results from NefIgard study met its primary endpoint; highly statistically significant benefit over placebo (p< 0.0001) in estimated glomerular filtration rate over the 2-year period of 9 months of treatment with Nefecon or placebo and 15-months of follow-up off drug; statistically significant and clinically meaningful with a sustained treatment benefit; durable urine protein-to-creatinine ratio reductions during the 15-month follow-up period off treatment | 3/12/23 | Genitourinary/Sexual Function |
AB2 Bio Ltd., of Lausanne, Switzerland | Tadekinig alfa (r-hIL-18BP) | Recombinant human interleukin-18 binding protein inhibiting IL-18 | Primary monogenic IL-18 driven hemophagocytic lymphohistiocytosis | Completed enrollment; top-line results expected in the second half of 2023 | 3/7/23 | Hematologic |
Incyte Corp., of Wilmington, Del. | Parsaclisib | Oral inhibitor of PI3K-delta | Myelofibrosis | Independent data monitoring committee mentioned LIMBER-304 study did not meet primary endpoint in combination with ruxolitinib; recommendation to stop the study was not due to safety | 3/3/23 | Hematologic |
Protagonist Therapeutics Inc., of Newark, Calif. | Rusfertide (PTG-300) | Injectable hepcidin mimetic | Polycythemia vera | Top-line results showed rusfertide achieved highly statistically significant improvements vs. placebo in the primary endpoint; statistically significant higher number of responders on rusfertide vs. placebo (p=0.0003, 69.2% vs. 18.5%); 2 of 26 subjects on rusfertide were phlebotomized, keeping 92.3% patients phlebotomy free in the rusfertide arm (p=0.0003) in 12 weeks of the blinded randomized withdrawal; well-tolerated; no new safety signals | 3/15/23 | Hematologic |
Swedish Orphan Biovitrum AB, of Stockholm, and Sanofi SA, of Paris | Altuviiio (efanesoctocog alfa, BIVV-001) | Recombinant factor VIII therapy | Hemophilia A | Top-line results from the study achieved primary endpoint in XTEND-Kids study; no factor VIII inhibitors detected; high sustained factor VIII levels throughout the weekly dosing interval with a median annualized bleeding rate (ABR) of 0.00; and an estimated mean ABR of 0.89 | 3/2/23 | Hematologic |
Abbvie Inc., of North Chicago | Rinvoq (upadacitinib) | Selective and reversible JAK inhibitor | Systemic lupus erythematosus (SLE) | Top-line results in combination with elsubrutinib met the primary endpoint of SLE Responder Index (SRI-4) and steroid dose less than or equal to 10 mg of prednisone equivalent once per day at week 24 ; no new safety signals | 3/23/23 | Immune |
DBV Technologies SA, of Montrouge, France | Viaskin Peanut | Epicutaneous patch; immunotherapy | Peanut allergy | First patient screened in peanut-allergic children ages 4-7 | 3/7/23 | Immune |
Immunovant Inc., of New York, and Harbour Biomed Co. Ltd., of Suzhou, China | HBM-9161 (batoclimab) | Anti-FcRn monoclonal antibody | Generalized myasthenia gravis | Top-line results of study met its primary and secondary endpoint; well-tolerated with no new safety signals | 3/6/23 | Immune |
Octapharma USA, of Hoboken, N.J. | Cutaquig | Immune globulin subcutaneous [human]-hipp, 16.5% solution infusions | Primary immunodeficiency disease | Results showed stable serum IgG levels; reduction in systemic side effects as compared to intravenous immune globulin (IVIG); improved compliance and reductions in hospitalizations; 33.3% of patients attained ?90% of the allowed maximum volume of 100 mL/site; mean number of infusion sites were reduced to 2.3 vs. 3.3 and 3.9 in cohorts 2 and 3, respectively; maximum realized infusion flow rate was 67.5 mL/hr/site in cohort 2; higher infusion flow rates were well-tolerated and reduced the duration of infusion; mean total trough levels for every other week dosing demonstrated equivalency to weekly dosing (p value = 0.0017) in cohort 3; no serious bacterial infections | 3/6/23 | Immune |
Organogenesis Holdings Inc., of Canton, Mass. | Renu | Cryopreserved amniotic suspension allograft | Knee osteoarthritis | Independent data monitoring committee provided directional guidance on the results of the interim analysis; recommended that the trial proceed without modification and without increase to sample size | 3/1/23 | Inflammatory |
Selecta Biosciences Inc., of Watertown, Mass., and Swedish Orphan Biovitrum AB, of Stockholm | SEL-212 | Pegadricase, a pegylated uricase, plus Immtor, which prevents antidrug antibodies | Chronic refractory gout | In the Dissolve I and II studies, 56% and 47% of patients treated monthly with the 0.15-mg/kg dose of SEL-212 achieved a response, defined as achievement and maintenance of reduction in serum urate <6mg/dL for at least 80% of the time during month 6, respectively, compared to 4% and 12% of patients who received placebo, respectively (p<0.0001 and p=0.0002, respectively) | 3/21/23 | Inflammatory |
Cytokinetics Inc., of South San Francisco | Reldesemtiv | Next-generation fast skeletal muscle troponin activator | Amyotrophic lateral sclerosis | Data monitoring committee reviewed unblinded data from COURAGE-ALS study; recommended the discontinuation of the clinical trial due to futility; no evidence of effect in patients treated with reldesemtiv relative to placebo on the primary endpoint of change from baseline to 24 weeks in ALSFRS-R or in key secondary endpoints; discontinued treatment in all patients including those in the open-label extension study | 3/31/23 | Musculoskeletal |
Mitsubishi Tanabe Pharma America Inc., of Jersey City, N.J. | Radicava (edaravone, oral suspension) | Neuroprotectant; antioxidant agent; free radical scavenger; oral suspension | Amyotrophic lateral sclerosis | Completed enrollment for phase IIIb study; top-line results expected in 2024 | 3/23/23 | Musculoskeletal |
Astrazeneca plc, of Cambridge, U.K., and Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | Eplontersen | Ligand-conjugated antisense medicine | Hereditary transthyretin-mediated amyloid polyneuropathy | Eplontersen continued to demonstrate a statistically significant and clinically meaningful change from baseline vs. an external placebo group on the co-primary endpoints of modified Neuropathy Impairment Score +7 (mNIS+7) at 66 weeks; met its third co-primary endpoint, statistically significant and sustained reduction in serum TTR concentration vs. an external placebo group; consistent safety and tolerability profile observed at 35 weeks | 3/27/23 | Neurology/Psychiatric |
Biovie Inc., of Santa Monica, Calif. | NE-3107 | Oral small-molecule, blood-brain permeable, anti-inflammatory, insulin sensitizing and ERK-binding properties | Alzheimer’s disease | Achieved its revised enrollment target of 400 patients; top-line results from the study due in October 2023 | 3/2/23 | Neurology/Psychiatric |
Bristol Myers Squibb Co., of New York, and Janssen Pharmaceuticals Inc., a unit of Johnson & Johnson, of New Brunswick, N.J. | Milvexian | Oral factor XIa inhibitor | Acute non-cardioembolic ischemic stroke or transient ischemic attack | Launched phase III Librexia study; initiated enrollment; first patient enrolled | 3/2/23 | Neurology/Psychiatric |
Eisai Co. Ltd., of Tokyo | Leqembi (lecanemab-irmb) | 100 mg/mL injection; humanized immunoglobulin gamma 1 monoclonal antibody directed against aggregated soluble and insoluble forms of amyloid beta (A?) | Early Alzheimer’s disease | Clarity studies showed amyloid-related imaging abnormalities (ARIA) were higher for patients receiving lecanemab compared to those on placebo; incidence of ARIA-E was 13.1% in the lecanemab group and 1.5% in the placebo group when no antiplatelet or anticoagulant medication was used; 10.4% in the lecanemab group and 0.84% in the placebo group when antiplatelet medication was used; 4.8% in the lecanemab group and 2.7% in the placebo group when anticoagulant medication was used; adjusted mean change from baseline in EQ-5D-5L and QOL-AD of subject showed 49% and 56% less decline, respectively at month 18; care partner burden measured Zarit Burden Interview and QOL-AD by partner resulted in 38% and 23% less decline, respectively; results published in the New England Journal of Medicine | 3/31/23 | Neurology/Psychiatric |
Eli Lilly and Co., of Indianapolis | Solanezumab | Monoclonal antibody targeting amyloid | Alzheimer's disease | Double-blind portion of the study showed solanezumab did not slow cognitive decline on the primary outcome measure PACC (p=0.26); higher baseline amyloid levels were strongly associated with a greater risk of progression to symptomatic Alzheimer's disease (p<0.001); consistent safety profile | 3/8/23 | Neurology/Psychiatric |
Emalex Biosciences Inc., of Chicago | Ecopipam (EBS-101) | Dopamine-1 receptor antagonist | Tourette syndrome | First patient dosed; enrolled patients receive medication for up to 24 weeks | 3/1/23 | Neurology/Psychiatric |
Harmony Biosciences Holdings Inc., of Plymouth Meeting, Pa. | Wakix (pitolisant) | Selective histamine 3 receptor antagonist/inverse agonist | Idiopathic hypersomnia | Reported an accelerated timeline for completing phase III Intune study; expected to complete enrollment in the second quarter of 2023; top-line data in the fourth quarter of 2023 | 3/29/23 | Neurology/Psychiatric |
Horizon Therapeutics plc, of Dublin | Uplizna (inebilizumab) | B-cell depleting anti-CD19 monoclonal antibody | Neuromyelitis optica spectrum disorder | Post-hoc analysis from MRI findings showed participants experienced subclinical optic nerve findings without new symptoms at the end of the randomized controlled period; shorter (median length 6 mm) than in those with an optic nerve attack (15 mm, p<0.001); number of clinical optic neuritis attacks was significantly reduced during this time period | 3/14/23 | Neurology/Psychiatric |
Intra-Cellular Therapies Inc., of New York | Caplyta (lumateperone) | Combination of antagonist activity at central serotonin 5-HT2A receptors and postsynaptic antagonist activity | Major depressive episodes in patients with major depressive disorder with mixed features and in patients with bipolar depression with mixed features | Top-line results of 403 study achieved primary and secondary endpoints at 42 mg; statistically significant and clinically meaningful reduction in the MADRS total score compared to placebo at week 6; combined patient population of MDD with mixed features and bipolar depression with mixed features (5.7-point reduction v. placebo; p<0.0001); MDD with mixed features (5.9-point reduction v. placebo; p<0.0001); bipolar depression with mixed features (5.7-point reduction v. placebo; p<0.0001); statistically significant on the key secondary endpoint of the clinician’s assessment of improvement in overall severity on the Global Impression of Severity Scale (CGI-S); combined MDD with mixed features and bipolar depression with mixed features (p<0.0001); MDD with mixed features (p=0.0003); bipolar depression with mixed features (p<0.0001); favorable safety and tolerability profile | 3/28/23 | Neurology/Psychiatric |
Janssen Pharmaceutical Cos., of Beerse, Belgium, unit of Johnson & Johnson | Spravato (esketamine nasal spray) | NMDA antagonist | Treatment-resistant major depressive disorder | Escape-TRD study achieved remission from week 6 and at every subsequent time-point through week 32 compared to the quetiapine XR arm; 55% of patients in the esketamine arm achieved remission compared to 37% in the quetiapine XR arm; significantly greater proportion of participants in the esketamine arm vs. quetiapine XR arm experienced response from day 15 and at every subsequent time-point through week 32; absolute response rate at week 8 (55.4% vs. 39.1%) and week 32 (75.5% vs.55.5%); significantly improved reduction of depressive symptoms; treatment discontinuation due to treatment-emergent adverse events were lower in the esketamine (4.2% vs. 11%) | 3/27/23 | Neurology/Psychiatric |
Karuna Therapeutics Inc., of Boston | Karxt | Combination of xanomeline and trospium chloride; muscarinic agonist and antagonist | Schizophrenia | Top-line results met its primary endpoint; statistically significant and clinically meaningful 8.4-point reduction in Positive and Negative Syndrome Scale (PANSS) total score compared to placebo (p<0.0001) at week 5; sustained statistically significant reduction of symptoms from week 2 (p<0.05) through the end of the trial; clinically meaningful and statistically significant 3.5-point reduction in PANSS positive subscale compared to placebo at week 5 (p<0.0001); statistically significant reduction in PANSS negative subscale and PANSS negative Marder factor subscale compared to placebo at week 4 (p<0.05); well-tolerated | 3/20/23 | Neurology/Psychiatric |
Neurocrine Biosciences Inc., of San Diego | Ingrezza (valbenazine) | VMAT2 inhibitor; capsule | Tardive dyskinesia | Long-term outcome results from older and elderly patients showed clinically meaningful reductions of TD symptoms; >80% of participants in the ?65 age group met the >50% improvement in the Abnormal Involuntary Movement Scale (AIMS) response threshold; minimal scores in psychiatric symptom scale; treatment-emergent adverse events as 76.8% and 72.7% for ?55 years and ?65 years, respectively; serious events as 20.5% and 18.2% in both groups | 3/3/23 | Neurology/Psychiatric |
Oculis Holding AG., of Lausanne, Switzerland | OCS-01 | High-concentration, preservative-free, topical Optireach formulation of dexamethasone | Inflammation and pain following cataract surgery | Completed enrollment of 240 patients | 3/16/23 | Neurology/Psychiatric |
Prilenia Therapeutics BV, of Naarden, the Netherlands | Pridopidine | Oral, small-molecule, highly selective and potent sigma-1 receptor (S1R) agonist | Huntington’s disease | Last patient completed last visit of the blinded treatment period in the Proof-HD study; results expected in the early second quarter of 2023 | 3/28/23 | Neurology/Psychiatric |
Relmada Therapeutics Inc., of Coral Gables, Fla. | REL-1017 | NMDA receptor channel blocker; chemical molecule | Major depressive disorder | Implemented critical changes to Reliance II (Study 302); 2 phase III two-arm trial ongoing; initiating new 304 study ; study 304 planning to initiate by mid-2023 | 3/23/23 | Neurology/Psychiatric |
Tonix Pharmaceuticals Holding Corp., of Chatham, N.J. | TNX-102 SL | Cyclobenzaprine HCl sublingual tablets | Fibromyalgia | Study met its prespecified primary endpoint; significantly reducing daily pain compared to placebo (p=0.01) in participants; higher rate of responders to TNX-102 SL (47%) than to placebo (35%; p=0.006) in primary endpoint was analyzed as a ?30% pain responder analysis; improvements in sleep quality, mitigation of fatigue, and fibromyalgia-specific global symptomatic and functional recovery at 5.6-mg dose; discontinuation rates were similar for TNX-102 SL and placebo (17.7% and 16.5%, respectively); well-tolerated | 3/30/23 | Neurology/Psychiatric |
Vistagen Therapeutics Inc., of South San Francisco | Fasedienol (PH-94B) | Rapid-onset pherine nasal spray | Social anxiety disorder | Long-term intranasal administration for 4 times a day in daily life found to be safe and well-tolerated (n=500); mean reduction on the Liebowitz Social Anxiety Scale (LSAS) was 16, 20 and 24 points with 36%, 44% and 55% experiencing a 20?point or greater reduction at 1, 2 and 3 months, respectively; 28.6% of the 385 patients assessed after 1 month were much or very much improved in Clinician Global Impression of Improvement (CGI-I); 26.8% of the 385 patients assessed after 1 month considered themselves much or very much improved in Patient Global Impression of Change (PGI-C) | 3/22/23 | Neurology/Psychiatric |
Bausch + Lomb, unit of Bausch Health Cos. Inc., of Laval, Quebec, and Novaliq GmbH, of Heidelberg, Germany | NOV-03 (perfluorohexyloctane) | Ophthalmic liquid formulation | Dry eye disease associated with Meibomian gland dysfunction | Mojave study published in the American Journal of Ophthalmology met both primary sign and symptom efficacy endpoints; well-tolerated; reduction from baseline in total corneal fluorescein staining (tCFS) was statistically greater in the NOV-03 arm compared to the control saline group (p<0.001); eye dryness Visual Analog Scale (VAS) score statistically significantly improved vs. control (p<0.001); tCFS and VAS dryness score were statistically significant compared to saline at week 2 (p=0.001 and p<0.001); well-tolerated; ocular adverse events (12.9% NOV-03 group, 12.3% control group); mild to moderate adverse events | 3/22/23 | Ocular |
Brim Biotechnology Inc., of Taipei, Taiwan | BRM-421 | Topical ophthalmic solution; synthetic peptide comprising 29 amino acids derived from pigment epithelium-derived factor (PEDF) with neurotrophic and anti-inflammation properties | Dry eye disease | First patient enrolled; top-line results expected at the end of 2023 | 3/2/23 | Ocular |
Gensight Biologics SA, of Paris | Lumevoq (GS-010, lenadogene nolparvovec) | Recombinant adeno-associated virus type 2 encoding the human mitochondrial NADH dehydrogenase subunit 4 gene | Leber hereditary optic atrophy | Pooled analysis of safety data from 5 clinical studies published in the American Journal of Ophthalmology showed good overall safety profile; mild in intensity systemic adverse events | 3/9/23 | Ocular |
Gensight Biologics SA, of Paris | Lumevoq (GS-010, lenadogene nolparvovec) | Recombinant adeno-associated virus type 2 encoding the human mitochondrial NADH dehydrogenase subunit 4 gene | Leber hereditary optic atrophy | Results from 3-year follow-up of Reflect study showed sustained efficacy; subjects bilaterally injected showed +20 ETDRS letters vs. Nadir (p<0.0001) in the first affected eye; +17 ETDRS letters vs. Nadir (p<0.0001) in the second affected eye; subjects unilaterally injected showed +18 ETDRS letters vs. Nadir (p<0.0001) in the first affected eye; +14 ETDRS letters vs. Nadir (p<0.0001) in the second affected eye; good safety profile; no study discontinuation related to systemic or ocular adverse events; no serious ocular adverse event | 3/13/23 | Ocular |
Iveric Bio Inc., of Parsippany, N.J. | Avacincaptad pegol | Complement C5 inhibitor | Geographic atrophy secondary to age-related macular degeneration | Exploratory time-to-event analysis from Gather1 and 2 study independently showed signals up to 59% risk reduction in rate of vision loss compared to sham treatment at 12 months at 2 mg; combined analysis of both studies experienced a 56% reduction in rate of vision loss compared to sham treatment at 12 months at 2 mg | 3/1/23 | Ocular |
Luye Pharma Group, of Shanghai, and Boan Biotechnology Co. Ltd., of Yantai, China | BA-9101 (OT-702) | Aflibercept intravitreous injection | Retinal diseases | Completed patient enrollment | 3/13/23 | Ocular |
Thea Pharma Inc., of Lexington, Mass. | Iyuzeh (latanoprost ophthalmic solution; preservative-free) | Topical prostaglandin F2? analogue | Primary open-angle glaucoma or ocular hypertension | Large pivotal study showed similar efficacy compared to Xalatan (latanoprost) in patients; similar clinically meaningful reductions in intraocular pressure from baseline (?18 mmHg) with fewer ocular adverse events (13.9% vs. 22.5%) | 3/3/23 | Ocular |
Visus Therapeutics Inc., of Seattle | Brimochol PF | Fixed-dose combination of carbachol and brimonidine tartrate | Presbyopia | Completed patient enrollment; last visit has been conducted in BRIO-I phase III study; top-line data in the second quarter of 2023 | 3/22/23 | Ocular |
Areteia Therapeutics Inc., of Chapel Hill, N.C. | Dexpramipexole | Eosinophil lowering small molecule | Severe eosinophilic asthma | First patient dosed in both Exhale-2 and Exhale-4 study | 3/9/23 | Respiratory |
Insmed Inc., of Bridgewater, N.J. | Brensocatib | Small-molecule, oral, reversible inhibitor of dipeptidyl peptidase 1 (DPP1) | Non-cystic fibrosis bronchiectasis | Completed enrollment of adult patients; top-line results expected in the second quarter of 2024 | 3/31/23 | Respiratory |
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and Sanofi SA, of Paris | Dupixent (dupilumab) | Fully human monoclonal antibody inhibiting IL-4 and IL-13 | Uncontrolled chronic obstructive pulmonary disease | Study met primary and all key secondary endpoints; 30% reduction in moderate or severe acute COPD exacerbations over 52 weeks (p=0.0005); improved lung function from baseline by 160 ml at 12 weeks compared to 77 ml for placebo (p<0.0001); benefit vs. placebo sustained through week 52 (p=0.0003); improvement in patient-reported health-related quality of life; consistent safety profile | 3/23/23 | Respiratory |
Achieve Life Sciences Inc., of Seattle | Cytisinicline | Plant-based alkaloid with a high binding affinity to the nicotinic acetylcholine receptor | Smoking cessation and nicotine addiction | Additional findings from the ORCA-2 trial showed successful abstinence was observed in subgroups of smokers who received cytisinicline; consistently higher rates of abstinence in subjects who received either 6 or 12 weeks of cytisinicline treatment; 82% of subjects completing the 12 weeks of treatment with study compliance; no treatment-related serious adverse events; mild adverse events | 3/3/23 | Toxicity and Intoxication |
Achieve Life Sciences Inc., of Seattle | Cytisinicline | Plant-based alkaloid with a high binding affinity to the nicotinic acetylcholine receptor | Smoking cessation | Last subject last study follow-up visit completed in the ORCA-3 trial; top-line data expected in the second quarter of 2023 | 3/29/23 | Toxicity and Intoxication |
Actinium Pharmaceuticals Inc., of New York | Iomab-B | Iodine-131 (131I)-labeled murine monoclonal IgG1 antibody (BC8) targeting CD45 | Active relapsed or refractory acute myeloid leukemia | Results from Sierra showed 100% bone marrow transplant (BMT) access and engraftment with Iomab-B vs. only 17% in the control arm; 75% of Iomab-B patients achieved complete remission (CR/CRp) within 30 days following BMT vs. only 6.3% of patients on the control arm; 22% of Iomab-B treated patients achieved a dCR vs. 0% of control patients (p<0.0001); 92% 1-year survival rate and 60% 2-year survival rate; median overall survival not reached; event-free survival was 26% vs. 0.2% in favor of Iomab-B (p<0.0001); median OS was doubled in Iomab-B patients - 6.4 months vs. 3.2 months in control arm in patients who did not cross over – as was 1-year survival – 26.1% vs. 13.1%; favorable safety profile with more than 4-times lower rates of sepsis (6.1% vs. 28.6%) | 4/27/23 | Cancer |
Astellas Pharma Inc., of Tokyo | Zolbetuximab | Monoclonal antibody targeting Claudin 18.2 | CLDN18.2-positive, HER2-negative, locally advanced unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma | Data from the Spotlight study published in The Lancet showed zolbetuximab plus the chemotherapy combination mFOLFOX6 reduced the risk of disease progression or death by 25% compared with mFOLOX6 alone (p=0.0066); the combination also reduced the risk of death by 25% compared to mFOLOX6 alone (p=0·0053) | 4/14/23 | Cancer |
Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Rahway, N.J. | Lynparza (olaparib) | PARP inhibitor | Advanced high-grade epithelial ovarian cancer without tumor BRCA mutations | Duo-O study in combination with durvalumab, chemotherapy and bevacizumab demonstrated a statistically significant and clinically meaningful improvement in progression-free survival vs. chemotherapy plus bevacizumab; overall survival and other secondary endpoints are immature | 4/5/23 | Cancer |
Beigene Ltd., of Beijing | Tislelizumab | Humanized IgG4 anti-PD-1 monoclonal antibody | Advanced unresectable or metastatic gastric or gastroesophageal junction (G/GEJ) adenocarcinoma | Rationale305 study met its primary endpoint of overall survival in combination with chemotherapy; superior overall survival (OS) compared with chemotherapy in patients | 4/20/23 | Cancer |
Biolinerx Ltd., of Tel Aviv, Israel | Motixafortide (Aphexda) | Stem cell mobilization agent | Multiple myeloma | Genesis trial achieved statistical significance (p<0.0001) across all primary and secondary endpoints; favorable safety profile; well-tolerated; grade 1/2 injection site reactions | 4/17/23 | Cancer |
Eisai Co. Ltd., of Tokyo, and Merck & Co. Inc., of Rahway, N.J. | Lenvima and Keytruda | Multiple receptor tyrosine kinase inhibitor and anti-PD1 | Unresectable or metastatic melanoma | Discontinued LEAP-003 trial; independent data monitoring committee reviewed data from a planned interim analysis; did not demonstrate an improvement in overall survival; statistically significant improvement in progression-free survival | 4/10/23 | Cancer |
Eisai Co. Ltd., of Tokyo, and Merck & Co. Inc., of Rahway, N.J. | Lenvima and Keytruda | Multiple receptor tyrosine kinase inhibitor and anti-PD1 | Unresectable and metastatic colorectal cancer | LEAP-017 study did not meet its primary endpoint of OS; did not meet statistical significance per the pre-specified statistical analysis plan; trend toward improvement was also observed in key secondary endpoints of PFS, ORR, DOR | 4/10/23 | Cancer |
Foghorn Therapeutics Inc., of Cambridge, Mass. | FHD-609 | Targets BRD9 | Synovial sarcoma and SMARCB1-deleted tumors | Paused enrollment due to a grade 4 QTc prolongation event in a synovial sarcoma patient at the second highest dose; enrollment completed for dose escalation portion of the study; maximum tolerated dose has been identified | 4/24/23 | Cancer |
I-Mab Biopharma Ltd., of Shanghai | Lemzoparlimab (TJC-4) | CD47 antibody | High-risk myelodysplastic syndrome | First patient treated in combination with azacitidine | 4/24/23 | Cancer |
Junshi Biosciences Co. Ltd., of Shanghai | Senaparib | PARP inhibitor | Advanced ovarian cancer | Completed prespecified interim analysis; Independent data monitoring committee said trial met the predefined efficacy endpoint; treatment significantly extended progression-free survival | 4/12/23 | Cancer |
Junshi Biosciences Co. Ltd., of Shanghai | Toripalimab | Anti-PD-1 monoclonal antibody | Resectable stage III non-small-cell lung cancer | Interim analysis of Neotorch study in combination with chemotherapy significantly improved event-free survival (p < 0.0001) and reduced risk of disease recurrence, progression events or death by 60%; median EFS was not reached in the toripalimab arm vs. 15.1 months in the placebo arm; major pathological response (MPR) and pathologic complete response (pCR) rates per blinded independent pathologic review vs. higher compared to the placebo arm, with rates of 48.5% vs. 8.4% (p<0.0001) and 24.8% vs. 1% (p<0.0001), respectively; median OS was not reached in the toripalimab arm vs. 30.4 months in the placebo arm; well-tolerated; no new safety signals; after neoadjuvant therapy , more patients from the toripalimab arm underwent surgery compared to the placebo arm (82.2% vs. 73.3%); 95.8% and 92.6% of the patients underwent surgery and achieved R0 resection, respectively | 4/20/23 | Cancer |
Junshi Biosciences Co. Ltd., of Shanghai | Toripalimab | Anti-PD-1 monoclonal antibody | Advanced renal cell carcinoma | Prespecified interim analysis from Renotorch study met the predefined efficacy boundary; significant improvement in progression-free survival, objective response rate and overall survival | 4/27/23 | Cancer |
Sellas Life Sciences Group Inc., of New York | Galinpepimut-S | WT1-targeting peptide immunotherapeutic | Acute myeloid leukemia | Independent data monitoring committee recommended to continue trial without modifications; next committee meeting scheduled in the third quarter 2023 | 4/18/23 | Cancer |
Urogen Pharma Ltd., of Princeton, N.J. | Jelmyto (mitomycin) | Pyelocalyceal solution is a mitomycin-containing reverse thermal gel containing 4 mg mitomycin per mL gel | Lower-grade upper tract urothelial cancer | Subanalysis showed investigators reported no difference in outcomes at first endoscopic evaluation based on UTUC tumor location (ureter vs. renal pelvis) (p=0.644); 37.8% patients with ureteral tumor had significant ureteral stenosis at first post-treatment evaluation | 4/30/23 | Cancer |
Lianbio Co. Ltd., of Shanghai | Mavacamten (Camzyos) | Cardiac myosin inhibitor | Symptomatic obstructive hypertrophic cardiomyopathy | Explore study in Chinese patients met its primary endpoint; statistically significant and clinically meaningful improvement in Valsalva left ventricular outflow tract (LVOT) gradient from baseline to week 30 compared to placebo (p<0.001); improvement for all secondary endpoints; consistent safety profile with previous studies; no new safety signals; no patients experienced decreases in left ventricular ejection fraction (LVEF) <50 % that required dose interruption | 4/26/23 | Cardiovascular |
Applied Therapeutics Inc., of New York | AT-007 (govorestat) | Penetrant aldose reductase inhibitor | Galactosemia | Govorestat demonstrated consistent and sustained clinical benefit on activities of daily living, behavioral symptoms, cognition, adaptive behavior and tremor; study not met primary endpoint; systematic improvement over time was demonstrated for the overall primary endpoint (p=0.1030) and for a pre-specified sensitivity analyses including cognition (p=0.0698); favorable trend towards improvement with govorestat vs. placebo in patients with severe speech deficits; post-hoc analysis showed highly statistically significant benefit of active treatment vs. placebo (p=0.0205), which strengthened over time in global statistical test including behavior and activities of daily living but excluding speech & language components; statistically significant benefit on tremor at 18 months (p=0.0428), as measured by the Archimedes Spiral Drawing Test, and adaptive skills as assessed by the BASC-3 Adaptive Skills Index (p=0.0265); improvement in galactitol levels; safe and well-tolerated ; no treatment-related serious adverse events | 4/24/23 | Endocrine/Metabolic |
Daewoong Pharmaceutical Ltd., of Seoul, South Korea | Enavogliflozin | SGLT-2 inhibitor | Type 2 diabetes | Data showed change of glycated hemoglobin (HbA1c) level at week 24 after administration of enavogliflozin showed -0.88%p for Envlo group and 0.11% p for placebo group; no significant differences were found in the incidence of adverse events, adverse drug reaction, and serious adverse events between the 2 groups; statistically significant (p<0.0001) the proportion of subjects achieving a therapeutic glycemic response (HbA1c < 7% or < 6.5%); results published in the international SCIE journal Diabetes, Obesity and Metabolism | 4/24/23 | Endocrine/Metabolic |
Newamsterdam Pharma BV, of Naarden, the Netherlands | Obicetrapib | Oral, once-daily CETP inhibitor | Heterozygous familial hypercholesterolemia | Completed patient enrollment; top-line results expected in the second half of 2024 | 4/24/23 | Endocrine/Metabolic |
Madrigal Pharmaceuticals Inc., of Conshohocken, Pa. | Resmetirom | Liver-directed THR-beta agonist | Nonalcoholic steatohepatitis with liver fibrosis | Completed enrollment in MAESTRO-NASH biopsy trial | 4/18/23 | Gastrointestinal |
Palisade Bio Inc., of Carlsbad, Calif. | LB-1148 | Oral formulation of broad-spectrum serine protease inhibitor | Return of bowel function following abdominal surgery | First patient enrolled | 4/5/23 | Gastrointestinal |
Akebia Therapeutics Inc., of Cambridge, Mass. | Vadadustat | Hypoxia-inducible factor prolyl hydroxylase inhibitor | Anemia due to chronic kidney disease | Least square mean difference in Hb compared to Mircera (methoxy polyethylene glycol-epoetin beta, CSL Ltd.) was -0.43 g/dL and -0.23 g/dL for patients treated with 600 mg and 900 mg of vadadustat, respectively, achieving the pre-specified non-inferiority margin of -0.75 g/dL | 4/3/23 | Genitourinary/Sexual Function |
Aurinia Pharmaceuticals Inc., of Victoria, British Columbia | Lupkynis (voclosporin) | Calcineurin inhibitor | Lupus nephritis | Top-line data showed significant and greater reductions in proteinuria; maintaining stable renal function as evidenced by a stable estimated glomerular filtration rate (eGFR) slope over time; histologic activity improvement with stable chronicity scores similar to the active control arm of MMF and low dose steroids alone over the 18 months | 4/5/23 | Genitourinary/Sexual Function |
Humacyte Inc., of Durham, N.C. | Human acellular vessel | Human acellular vessel | Hemodialysis patients with end-stage renal disease | Completed enrollment | 4/11/23 | Genitourinary/Sexual Function |
Travere Therapeutics Inc., of San Diego | Filspari (sparsentan) | Endothelin and angiotensin II receptor antagonist | IgA nephropathy | Data published in The Lancet and presented at the World Congress of Nephrology 2023 showed Filspari produced a mean 49.8% reduction in proteinuria from baseline, compared 15.1% for patients treated with irbesartan (p<0.0001); complete remission (urine protein excretion <0.3 g/day) rate was 20.8% for patients treated with Filspari and 7.9% for the irbesartan group (p=0.0005); partial remission (urine protein excretion <1.0 g/day) rate was 70.3% for patients receiving Filspari, compared to 44.1% for the irbesartan group (p<0.0001) | 4/1/23 | Genitourinary/Sexual Function |
Travere Therapeutics Inc., of San Diego | Filspari (sparsentan) | Endothelin and angiotensin II receptor antagonist | IgA nephropathy | Interim results from Protect study showed rapid and sustained proteinuria reduction; mean reduction in proteinuria from baseline of 49.8%, compared to a mean reduction in proteinuria from baseline of 15.1% for Irbesartan-treated patients (p<0.0001); achieved complete remission (urine protein excretion <0.3 g/day) and partial remission (urine protein excretion <1.0 g/day) of proteinuria compared to patients on Irbesartan; complete remission at any time over the course of the double-blind treatment period occurred in 20.8% of participants in the Filspari group and 7.9% of participants in the Irbesartan group (p=0.0005); 70.3% of participants in the Filspari group achieved partial remission, compared to 44.1% of participants in the Irbesartan group (p<0.0001); well-tolerated; results published in the Lancet | 4/13/23 | Genitourinary/Sexual Function |
Belief Biomed Group, of Shanghai | BBM-H901 | Gene therapy | Hemophila B | Completed patient dosing | 4/24/23 | Hematologic |
Genexine Inc., of Seoul, South Korea | GX-E4 (efepoetin alfa) | Long-acting EPO formulation | Chronic kidney disease-induced anemia | Interim results showed response rate of GX-E4 administered every 2 weeks was 69.6%; hemoglobin level was maintained at 91.2% vs. Mircera maintained the response rate at 63.2% and the hemoglobin level at 87.2% during the same period | 4/11/23 | Hematologic |
Morphosys AG, of Munich, Germany | Pelabresib (CPI-0610) | BET inhibitor | Myelofibrosis | Completed enrollment in Manifest-2 study in combination with ruxolitinib; top-line results expected by the end of 2023 | 4/4/23 | Hematologic |
Novartis AG, of Basel, Switzerland | Iptacopan | Oral targeted factor B inhibitor of alternative complement pathway | Paroxysmal nocturnal hemoglobinuria | Appoint-PNH study as monotherapy met its primary endpoint and demonstrated clinically meaningful benefits across secondary endpoints; 92.2% of patients achieved a 2 g/dL or more hemoglobin-level increase from baseline without the need for red blood cell transfusions after the 24-week core treatment period; 62.8% of patients achieved hemoglobin levels of 12 g/dL or more without the need for red blood cell transfusions; 97.6% of patients achieved red blood cell transfusion independence at 24 weeks; lactate dehydrogenase decreased by 83.55% from baseline at 24 weeks; clinically meaningful improvements in fatigue; tolerability and safety profile consistent with previously reported data | 4/26/23 | Hematologic |
Merck KGaA, of Darmstadt, Germany | Evobrutinib | BTK inhibitor | Relapsing multiple sclerosis | U.S. FDA placed a partial clinical hold on the initiation of new patients on evobrutinib and patients with fewer than 70 days exposure to study medication in the U.S.; readout expected in the fourth quarter of 2023 | 4/12/23 | Immune |
Atea Pharmaceuticals Inc., of Boston | Bemnifosbuvir | Purine nucleos(t)ide prodrug | COVID-19 | Morningsky study showed 71% relative reduction in risk of hospitalization in non-hospitalized adult and adolescent patients; 82% reduction for hospitalization was seen in a subset of patients older than 40 in exploratory analysis; did not meet its primary endpoint of improved time for alleviation of COVID-19 symptoms; safe and well-tolerated; no treatment-related serious adverse events | 4/12/23 | Infection |
Bavarian Nordic A/S, of Copenhagen | MVA-BN | Vaccines | Prevention of respiratory syncytial virus | Results from adults ?60 years of age now accrued the number of cases required to complete the primary efficacy analysis; top-line results expected in mid-2023 | 4/11/23 | Infection |
Citius Pharmaceuticals Inc., of Cranford, N.J. | Mino-Lok | Antibiotic lock solution | Catheter-related bloodstream infections | Trial enrolled 190 patients; achieved 85/92 events | 4/24/23 | Infection |
Gilead Sciences Inc., of Foster City, Calif. | Veklury (remdesivir) | Viral RNA polymerase | Hospitalized COVID-19 with moderately and severely reduced kidney function | No new safety signals were observed in the study of 242 patients who were randomized 2:1 to receive Veklury or placebo; no additional adverse reactions to Veklury identified in the 163 patients on hemodialysis receiving Veklury for up to 5 days | 4/16/23 | Infection |
GSK plc, of London | Gepotidacin | Triazaacenaphthylene antibiotic | Uncomplicated urinary tract infections | Gepotidacin demonstrated non-inferiority to nitrofurantoin; demonstrated statistically significant superiority vs. nitrofurantoin; therapeutic success in 50.6% of patients compared to 47% for nitrofurantoin in Eagle-2 study; therapeutic success in 58.5% of patients compared to 43.6% for nitrofurantoin in Eagle-3 study; both trials were stopped for non-inferiority based on pre-defined non-inferiority success boundaries; Eagle-3 phase III trial met the pre-defined boundary for superiority | 4/15/23 | Infection |
Moderna Inc., of Cambridge, Mass. | mRNA-1283 | Next-generation, refrigerator-stable mRNA vaccine | COVID-19 | First subject dosed | 4/11/23 | Infection |
SAB Biotherapeutics Inc., of Sioux Falls, S.D | SAB-185 | Human IgG1 (polyclonal) antibody therapeutic candidate | COVID-19 | Top-line results from Activ-2 study demonstrated benefit in sustained symptom resolution in study participants with COVID-19 caused by omicron as compared to participants who received REGEN-COV (Regeneron Pharmaceuticals Inc.); 66% of participants treated with SAB-185 reached full symptom resolution for at least 4 consecutive days by day 28, while only 50% of participants on REGEN-COV met this endpoint (p=0.010); median time to symptom resolution for at least 4 consecutive days was 7 days shorter for SAB-185; median time to symptom resolution for at least 2 consecutive days was 6 days shorter for SAB-185 as compared to those who were treated with REGEN-COV (p=0.021); median time to symptom resolution for at least 4 consecutive days was 7 days shorter for SAB-18 and the median time to symptom resolution for at least 2 consecutive days was 4 days shorter for SAB-185 than REGEN-COV in non-omicron patients, though neither of these analyses met statistical significance | 4/26/23 | Infection |
Shionogi & Co. Ltd., of Osaka, Japan | Fetcroja (cefiderocol) | Siderophore cephalosporin antibiotic | Aerobic gram-negative infections | Interim results showed cefiderocol achieved clinical cure in 65% of patients; 81% of patients were alive within 30 days | 4/17/23 | Infection |
Lyra Therapeutics Inc., of Watertown, Mass. | LYR-210 | Bioresorbable nasal implant | Chronic rhinosinusitis | Resumed screening and enrollment in Enlighten study | 4/25/23 | Inflammatory |
Amo Pharma Ltd., of London | AMO-02 (tideglusib) | Disrupting the pathogenic RNA repeat in CDM1 and inhibiting excess levels of the kinase GSK3? | Congenital-onset myotonic dystrophy | Completion of treatment for the final patient enrolled | 4/25/23 | Musculoskeletal |
Cingulate Inc., of Kansas City, Kan. | CTx-1301 | Extended-release tablet formulation of dexmethylphenidate | Attention deficit hyperactivity disorder | Completed the first cohort of its phase III adult onset and duration trial; enrolled 25 adults; results expected in the third quarter of 2023 | 4/4/23 | Musculoskeletal |
Galapagos NV, of Mechelen, Belgium | Filgotinib | Oral, once-daily JAK1 preferential inhibitor | Axial spondyloarthritis | First patient randomized; top-line results expected in the second half of 2025 | 4/26/23 | Musculoskeletal |
Tonix Pharmaceuticals Holding Corp., of Chatham, N.J. | TNX-102 SL | Cyclobenzaprine HCl sublingual tablets | Fibromyalgia | Eliminating interim analysis; top-line data expected in the fourth quarter of 2023 | 4/13/23 | Musculoskeletal |
Abbvie Inc., of North Chicago | Qulipta (atogepant) | CGRP antagonist | Episodic migraine | Elevate study met its primary and secondary endpoints; demonstrated adult patients who previously failed 2 to 4 classes of conventional oral medications in the atogepant 60-mg once daily arm experienced a decrease of 4.20 days in their mean monthly migraine days (MMDs) across the 12-week treatment period, which was statistically significantly greater than the 1.85 day reduction observed in the placebo arm (p<0.0001); well-tolerated; consistent safety results | 4/21/23 | Neurology/Psychiatric |
Alzheon Inc., of Framingham, Mass. | ALZ-801 (valiltramiprosate) | Prodrug of tramiprosate, which inhibits the formation of toxic amyloid beta 42 oligomers | Alzheimer’s disease | Ongoing APOLLOE4 study fully blocked the formation of neurotoxic soluble beta amyloid (A?) oligomers; robust clinical efficacy in the highest-risk Alzheimer’s population; favorable safety with no increased risk of brain vasogenic edema; fully enrolled and to be completed in mid-2024 | 4/18/23 | Neurology/Psychiatric |
Astrazeneca plc, of Cambridge, U.K., and Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | Eplontersen | Ligand-conjugated antisense medicine | Hereditary transthyretin-mediated amyloid polyneuropathy | Eplontersen met all co-primary endpoints and secondary endpoints at 66 weeks vs. an external placebo group; consistent and sustained benefit on the three co-primary endpoints of serum transthyretin (TTR) concentration, neuropathy impairment and quality of life (Qol); achieved a least squares (LS) mean reduction of 82% in TTR serum concentration from baseline compared to an 11% reduction from baseline in the external placebo group (p<0.0001); halted disease progression as measured by modified Neuropathy Impairment Score +7 (mNIS+7); 0.28 point LS mean increase compared to a 25.06 point increase for the external placebo group from baseline (24.8 point LS mean improvement; p<0.0001); overall 47% of treated patients showed improvements in neuropathy at 66 weeks compared to baseline vs. 17% in the external placebo group; 53% of treated patients showed improvements in neuropathy at 66 weeks compared to baseline vs. 19% in the external placebo group from study completers; LS mean decrease (improvement) on the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN), compared to a 14.2 point increase (worsening) in the external placebo group (19.7 point LS mean improvement; p<0.0001); overall 58% of treated patients showed improvements in QoL at 66 weeks compared to baseline vs. 20% in the external placebo group; 65% of treated patients showed improvements in QoL at 66 weeks compared to baseline vs. 23% in the external placebo group in study completers; statistically significant benefits on both mNIS+7 and Norfolk QoL-DN at 35 weeks vs. the external placebo group and improved at 66 weeks; favorable safety and tolerability profile; no adverse events | 4/24/23 | Neurology/Psychiatric |
Cali Biosciences Co Ltd., of San Diego | CPL-01 | Extended-release injectable version of Naropin (ropivacaine hydrochloride) | Postoperative surgical pain | Initiated phase III studies; began dosing in hernia patients | 4/19/23 | Neurology/Psychiatric |
Eisai Co. Ltd., of Tokyo, and Bioarctic AB, of Stockholm | Leqembi (lecanemab-irmb) | 100 mg/mL injection; humanized immunoglobulin gamma 1 monoclonal antibody directed against aggregated soluble and insoluble forms of amyloid beta | Mild cognitive impairment due to Alzheimer’s disease | Long-term data based on a disease simulation model from Clarity AD study showed estimated lifetime risk of disease progression to mild, moderate and severe AD dementia reduced by 7.5%, 13.7% and 8.8%, respectively, in patients who received lecanemab+standard of care (SoC), compared to SoC alone; resulted in a delay of 2 to 3 years in the mean time to progression to more severe stages of AD, compared with SoC alone; admission to institutional care was delayed by 0.6 years in the lecanemab-treated group compared to SoC alone (SoC vs. lecanemab: 6.25 years vs. 6.85 years); increase of 0.71 quality-adjusted life-years compared to SoC; subgroup analysis showed earlier initiation of treatment with lecanemab with greater impact on disease progression; incremental mean times for transition to mild and moderate AD dementia were 2.55 and 3.15 years, respectively, when treating MCI due to AD in a subgroup analysis compared to SoC | 4/3/23 | Neurology/Psychiatric |
Impel Neuropharma Inc., of Seattle | INP-104 (Trudhesa) | Dihydroergotamine mesylate; nasal spray (0.725 mg per spray) | Migraine | Analysis showed no drug-drug interactions of clinical concern in presence of orally administered gepant medications; additional analysis from STOP-301 trial showed well-tolerated; limited treatment-related adverse events; consistent efficacy; 16% of patients pain relief started as early as 15 minutes; pain free at 2 hours after their first migraine attack; 98% were still pain free at 24 hours; 95% were still pain free through 2 days respectively, during weeks 21-24 | 4/26/23 | Neurology/Psychiatric |
Otsuka Pharmaceutical Co. Ltd., of Tokyo, and H. Lundbeck A/S, of Valby, Denmark | Aripiprazole | Long-acting injectable form | Schizophrenia and bipolar I disorder | Results published in the CNS Drugs showed well-tolerated; maintained consistent therapeutic plasma levels for at least 2 months as compared to aripiprazole 1-month plasma levels following repeated monthly dosing | 4/4/23 | Neurology/Psychiatric |
Pharma Two B Ltd., of Rehovot, Israel | P2B-001 | Fixed-dose combination of rasagiline mesilate and pramipexole hydrochloride | Parkinson's disease | Result showed comparable changes from baseline to week 12 with P2B-001 and commercial Prami-ER in total UPDRS scores (p=0.7197); non-inferiority (post-hoc) of P2B-001 was confirmed (margin of 3 points, p=0.0052); no significant differences between P2B-001 and Prami-ER were seen in total UPDRS responders (?4-point improvement), UPDRS Part II (activities of daily living/ADL) and UPDRS Part III (motor) scores; side effects (44.7% vs. 66.2%); better outcomes in daytime sleepiness, with significantly less worsening (shown by a lower ESS change from baseline) than that with Prami-ER (p < 0.0001); rate of new-onset EDS was significantly lower in patients treated with P2B-001 (8.5%) as compared to commercial Prami-ER (p<0.0001) | 4/24/23 | Neurology/Psychiatric |
Prilenia Therapeutics BV, of Naarden, the Netherlands | Pridopidine | Oral, small-molecule sigma-1 receptor (S1R) agonist | Huntington’s disease | Top-line results of Proof-HD study did not meet its primary and secondary endpoint change from baseline compared to placebo at 65 weeks; prespecified analyses that excluded participants taking neuroleptics and chorea medications showed clinically meaningful and nominally significant benefits for participants on pridopidine across multiple measures; robust and nominally significant beneficial effects in secondary endpoints; well-tolerated with no serious treatment-related adverse events | 4/25/23 | Neurology/Psychiatric |
Aldeyra Therapeutics Inc., of Lexington, Mass. | Reproxalap | Modulator of reactive aldehyde species | Allergic conjunctivitis | Completed enrollment in the Invigorate-2 study; top-line data expected in the first half of 2023 | 4/13/23 | Ocular |
Apellis Pharmaceuticals Inc., of Waltham, Mass. | Syfovre (pegcetacoplan injection) | Intravitreal targeted C3 therapy | Geographic atrophy secondary to age-related macular degeneration | Syfovre treated patients showed visual function and quality-of-life benefits in patients with extrafoveal lesions in 24-month analysis; 4.1-point benefit in vision-related quality-of-life outcomes; meaningful reduction in the loss of both photoreceptor (46% (OAKS; p<0.0001) and 46% (DERBY; p<0.0001 in every other month) and RPE 20% (OAKS; p=0.0002) and 21% (DERBY; p=0.0005 in every other month) cells compared to sham | 4/23/23 | Ocular |
Genentech Inc., a subsidiary of Roche Holding AG, of Basel, Switzerland | Vabysmo (faricimab) | Bispecific antibody for the eye targeting Ang-2 and VEGF-A | Wet age-related macular degeneration and diabetic macular edema | Post-hoc data from pooled data from the head-to-head dosing period (weeks 0-12) of the phase III Tenaya and Lucerne studies showed Vabysmo reduced retinal fluid from baseline compared to aflibercept (Sanofi SA/Regeneron Pharmaceuticals Inc.), as measured by reduction in central subfield thickness (CST); CST reductions were 145 µm in the Vabysmo arm and 133 µm in the aflibercept arm at 12 weeks; larger proportion of Vabysmo patients (77%) had absence of retinal fluid at 12 weeks vs. aflibercept (67%), as measured by subretinal and intraretinal fluid; separate post-hoc analysis of pooled data from the head-to-head dosing period (weeks 0-16) of the Yosemite and Rhine studies showed Vabysmo reduced the macular leakage area to 3.6 mm2 from baseline compared to 7.6 mm2 with aflibercept; 28.4% had resolution of leakage vs. aflibercept (15.2%) at 16 weeks | 4/25/23 | Ocular |
Krystal Biotech Inc., of Pittsburgh | Beremagene geperpavec (B-VEC) | Non-invasive, topical, redosable gene therapy designed to deliver 2 copies of the COL7A1 gene | Dystrophic epidermolysis bullosa | B-VEC was well-tolerated and associated with full corneal healing by 3 months as well as significant visual acuity improvement from hand motion to 20/40 at 7 months; no drug-related adverse events | 4/24/23 | Ocular |
Novaliq GmbH, of Heidelberg, Germany | Cyclasol (cyclosporine ophthalmic solution) | Anti-inflammatory | Dry eye disease | Results published in the American Medical Association (JAMA) Ophthalmology showed Cyclasol improved total and central corneal staining within 2 weeks; 71.6% of Cyclasol-treated patients showed such a response after 4 weeks of treatment significantly more than in the vehicle group | 4/12/23 | Ocular |
Palatin Technologies Inc., of Cranbury, N.J. | PL-9643 | Melanocortin agonist | Dry eye disease | Data demonstrated effectiveness across multiple clinical signs and reduced symptomatic ocular pain; positive affect across multiple regions of the eye; Melody-1 phase III study continuing patient enrollment; final data in the second half of 2023; no ocular adverse events; no patients discontinued | 4/27/23 | Ocular |
Regenerx Biopharmaceuticals Inc., of Rockville, Md. | RGN-259 | Promotes cell migration, anti-inflammation and wound healing | Neurotrophic keratitis | First of approximately 70 patients treated in the Seer-2 study; primary endpoint is complete corneal healing | 4/13/23 | Ocular |
Samsung Bioepis Co. Ltd., of Incheon, Korea | SB-15 | Proposed biosimilar to Eylea (aflibercept) | Neovascular age-related macular degeneration | Results showed 425 patients completed up to week 56; demonstrates comparable efficacy, safety, immunogenicity and pharmacokinetics profiles to reference aflibercept up to week 56; switching group had similar efficacy and safety compared to the continuing groups without treatment-emergent issues | 4/24/23 | Ocular |
Visus Therapeutics Inc., of Seattle | Brimochol PF | Fixed-dose combination of carbachol and brimonidine tartrate | Presbyopia | Top-line results met its pre-specified primary study endpoints; achieved highly statistically significant near vision improvements over 8 hours; well-tolerated; no treatment-related serious adverse events; clinically and statistically significant reductions in pupil size were also observed out to 10 hours; no study subjects discontinuing due to adverse events | 4/20/23 | Ocular |
Arrowhead Pharmaceuticals Inc., of Pasadena, Calif., and Takeda Pharmaceutical Ltd., of Osaka, Japan | Fazirsiran | RNAi therapy designed to reduce the production of mutant alpha-1 antitrypsin protein | Alpha-1 antitrypsin deficiency associated liver disease | First patient treated | 4/4/23 | Other/Miscellaneous |
Eli Lilly and Co., of Indianapolis | Mounjaro (tirzepatide) | Peptide co-agonist of gastric inhibitory polypeptide and glucagon-like peptide 1 | Obesity | Surmount-2 study achieved both co-primary objectives and all key secondary objectives compared to placebo; tirzepatide achieved average weight reductions of 13.4% at 10-mg and 15.7% at 15-mg doses compared to placebo (3.3%); reduction in A1C and other cardiometabolic parameters; adverse events were gastrointestinal-related and generally mild to moderate in severity; overall treatment discontinuation rates were 9.3% (10 mg), 13.8% (15 mg) and 14.9% (placebo) | 4/27/23 | Other/Miscellaneous |
Fibrogen Inc., of San Francisco | Pamrevlumab | Monoclonal antibody targeting connective tissue growth factor | Idiopathic pulmonary fibrosis | Completed enrollment of 372 patients; top-line data expected in mid-2024 | 4/3/23 | Respiratory |
Inhibrx Inc., of San Diego | INBRX-101 | Optimized recombinant human AAT-Fc fusion protein | Emphysema due to alpha-1 antitrypsin deficiency | Initiated registration-enabling trial; initial readout from the Elevaate trial expected to occur in late 2024 | 4/26/23 | Respiratory |
Nuance Pharma Co. Ltd., of Shanghai | Ensifentrine | Dual inhibitor of the enzymes phosphodiesterase 3 and 4 | Chronic obstructive pulmonary disease | First patient dosed in Enhance study | 4/6/23 | Respiratory |
Renovion Inc., of Chapel Hill, N.C. | ARINA-1 | Nebulized therapy | Bronchiolitis obliterans syndrome progression in adults with a bilateral lung transplant | First patient enrolled | 4/18/23 | Respiratory |
Astrazeneca plc, of Cambridge, U.K. | Tagrisso (osimertinib) | Irreversible EGFR-TKI | Locally advanced (stage IIIB-IIIC) or metastatic (stage IV) EGFRm non-small-cell lung cancer | Tagrisso in combination with chemotherapy demonstrated a statistically significant and clinically meaningful improvement in progression-free survival compared to Tagrisso alone; overall survival immature; safety results and discontinuation rates due to adverse events were consistent | 5/17/23 | Cancer |
Delta-Fly Pharma Inc., of Tokushima, Japan | DFP-10917 | Deoxycytidine nucleoside analogue | Acute myeloid leukemia | Plans to do the first interim analysis of the study | 5/15/23 | Cancer |
Hansa Biopharma AB, of Lund, Sweden | Idefirix (imlifidase) | Enzyme derived from Streptococcus pyogenes e | Anti-glomerular basement membrane (anti-GBM) disease | First patient dosed | 5/30/23 | Cancer |
Immunogen Inc., of Waltham, Mass. | Elahere (mirvetuximab soravtansine-gynx) | Antibody-drug conjugate comprising folate receptor alpha-binding antibody, cleavable linker and the maytansinoid payload DM4 | Platinum-resistant epithelial ovarian cancer | Elahere demonstrated a statistically significant and clinically meaningful improvement in overall survival (OS) compared to single-agent chemotherapy; median OS was 16.46 months vs. 12.75 months on chemotherapy (p=0.0046); 33% reduction in the risk of death; statistically significant and clinically meaningful improvement in progression-free survival (PFS) compared to chemotherapy (p<0.0001); 35% reduction in the risk of tumor progression or death; median PFS was 5.62 months compared to 3.98 months; 42.3% of ORR compared to 15.9%; no new safety signals; serious adverse events 24% vs. 33%; treatment-related events leading to discontinuation of study drug (9% vs. 16%) | 5/3/23 | Cancer |
Immutep Ltd., of Sydney, Australia | IMP-321 (eftilagimod alpha) | LAG-3 immunotherapy | Non-small-cell lung cancer | Received positive feedback from the U.S. FDA for phase II study; toxicological package and general aspects of the trial design were discussed | 5/16/23 | Cancer |
Innovent Biologics Inc., of Rockville, Md. | Sintilimab | Monoclonal antibody targeting PD-1 | EGFR-mutated non-squamous non-small-cell lung-cancer | Second interim analysis showed median follow-up duration of progression-free survival (PFS) were 12.9 months, 15.1 months and 14.4 months in arm A (sintilimab plus bevacizumab plus chemotherapy), B (sintilimab plus chemotherapy) and C (chemotherapy), respectively; median PFS of 7.2, 5.5 and 4.3 months in respective arms; arm B showed statistically significant and clinically meaningful improvement in PFS compared to arm C (p=0.016); PFS benefit (p<0.0001); median overall survival for arm A and arm C were 21.1 months vs. 19.2 months; arm A showed longer median time-to-deterioration of the Global Health Status Dimension Score of EORTC Quality of Life Questionnaire Core 30 (QLQ-C30) compared with arm C; treatment-related adverse events of grade ?3 were 56% in arm A, 41% in arm B and 49% in arm C; results published in The Lancet Respiratory Medicine | 5/7/23 | Cancer |
Jaguar Health Inc., of San Francisco | Crofelemer | Oral plant-based non-opioid agent | Cancer therapy-related diarrhea | Completed enrollment; target number of 256 patients enrolled | 5/9/23 | Cancer |
Jaguar Health Inc., of San Francisco | Crofelemer | Oral plant-based non-opioid agent | Cancer therapy-related diarrhea | Completion of targeted enrollment of 256 patients; top-line results in October 2023 | 5/25/23 | Cancer |
Mirati Therapeutics Inc., of San Diego | Sitravatinib | Oral receptor tyrosine kinase inhibitor | Nonsquamous non-small-cell lung cancer | Sapphire study did not meet its primary endpoint of overall survival at the final analysis in combination with Opdivo (nivolumab, Bristol Myers Squibb Co.) | 5/24/23 | Cancer |
Rain Therapeutics Inc., of Newark, Calif. | Milademetan | Oral mouse double minute 2 (MDM2) inhibitor | Dedifferentiated liposarcoma | Top-line results showed Mantra study did not meet its primary endpoint of progression-free survival (PFS); median PFS for milademetan was 3.6 months vs. 2.2 months for trabectedin (p=0.53); dose reductions in the milademetan arm were 44.2% vs. 29.1% in the trabectedin arm; discontinuations in the milademetan arm due to adverse events were 11.6% vs. 19% for trabectedin; treatment emergent serious adverse events in the milademetan arm were 36% vs. 48.1% in the trabectedin arm | 5/22/23 | Cancer |
Rakuten Medical Inc., of San Mateo, Calif. | ASP-1929 (cetuximab sarotalocan) | Antibody-dye conjugate targeting EGFR | Head and neck squamous cell carcinoma | Indian Central Drugs Standard Control Organization (CDSCO) granted global phase III study evaluating Alluminox treatment (photoimmunotherapy) using ASP-1929 in patients; planning to enroll 275 patients globally | 5/22/23 | Cancer |
Rayzebio Inc., of San Diego | RYZ-101 | Radiopharmaceutical therapy that delivers Actinium-225 | SSTR+ gastroenteropancreatic neuroendocrine tumors | First patient dosed; expected to enroll 210 patients | 5/31/23 | Cancer |
Soligenix Inc., of Princeton, N.J. | Hybryte (SGX-301) | Synthetic hypericin sodium; photodynamic light therapy | Cutaneous T-cell lymphoma | Flash study showed all patients had improvements in their cumulative CAILS score (average improvement of 36.4%); individual lesions showed that 7 of the 27 index lesions (25.9%) had at least a 50% improvement in their CAILS score and 4 of the 27 index lesions (14.8%) were completely resolved after as little as 8 weeks of treatment; no observable impact to normal sinus rhythm | 5/4/23 | Cancer |
Summit Therapeutics Inc., of Menlo Park, Calif. | Ivonescimab | Dual PD-1 checkpoint/VEGF inhibitor | Non-small-cell lung cancer | First patient enrolled in Harmoni study to test combination with chemotherapy in EGFR-mutated, locally advanced or metastatic disease that has progressed after treatment with third-generation EGFR tyrosine kinase inhibitors | 5/9/23 | Cancer |
Alnylam Pharmaceuticals Inc., of Cambridge, Mass. | Onpattro (patisiran) | RNAi therapeutic | Transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy | Interim analysis of exploratory data from the open-label extension Apollo-B study achieved primary endpoint and secondary endpoint during the 12-month double-blind period; favorable effects on functional capacity (6-MWT) and health status and quality of life (KCCQ-OS) were sustained in patients at 18 months; maintained relative stability of NT-proBNP and troponin I levels; no statistically significant differences were achieved for treatment difference in death and hospitalizations between patisiran and placebo | 5/20/23 | Cardiovascular |
Amarin Corp. plc, of Dublin | Vascepa/Vazkepa (icosapent ethyl) | Eicosapentaenoic acid | Angiographic coronary artery disease | In the Evaporate study, the drug improved mean distal segment FFRCT at 9- and 18-months of follow-up compared with placebo (p=0.02 and p=0.03, respectively) | 5/12/23 | Cardiovascular |
Daiichi Sankyo Co. Ltd., of Tokyo | Nilemdo (bempedoic acid) | Inhibits ATP citrate lyase | Reduction of risk of serious cardiovascular events | Clear outcome study showed 3% relative risk reduction in the primary endpoint of a 4-component composite of major adverse cardiovascular events (MACE-4) | 5/23/23 | Cardiovascular |
Mineralys Therapeutics Inc., of Radnor, Pa. | Lorundrostat | Aldosterone synthase inhibitor | Uncontrolled and resistant hypertension | First patient dosed; top-line data expected in the first half of 2024 | 5/2/23 | Cardiovascular |
Novartis AG, of Basel, Switzerland | Entresto (sacubitril/valsartan) | Neprilysin inhibitor/angiotensin receptor blocker | Heart failure in patients with an ejection fraction | PARAGLIDE-HF study showed a greater reduction in NT-proBNP concentration than enalapril and valsartan (p=0.049); secondary composites favored treatment but were not significant (p=0.16); reduced worsening renal functions; results published in the Journal of the American College of Cardiology | 5/21/23 | Cardiovascular |
Abeona Therapeutics Inc., of New York | EB-101 | Autologous, engineered cell therapy | Recessive dystrophic epidermolysis bullosa | Viital study showed both co-primary endpoints met; 81.4% of randomized EB-101–treated wounds demonstrating ?50% healing compared with 16.3% of untreated control wounds; significantly greater reduction in pain severity observed in randomized EB-101-treated wounds (3.07 mean pain reduction from baseline) compared with untreated control wounds (0.9 mean pain reduction from baseline) at 6 months; patient-reported outcomes related to itch and blistering showed significantly greater improvement with EB-101 treatment; improvement in caregiver-reported outcomes related to wound care and overall impression of wound pain; well-tolerated with no serious treatment-related adverse events | 5/11/23 | Dermatologic |
Almirall SA, of Barcelona, and Eli Lilly and Co., of Indianapolis | Lebrikizumab | Monoclonal antibody that binds to IL-13 | Moderate to severe atopic dermatitis | Post-hoc analysis from the 16-week induction periods of the Advocate 1 and Advocate 2 studies and the Adhere study showed 58% to 73% of adult and adolescent patients treated with lebrikizumab experienced improvement or clearance of face or hand dermatitis; improved or clear skin at week 16 | 5/1/23 | Dermatologic |
Arcutis Biotherapeutics Inc., of Westlake Village, Calif. | Zoryve (roflumilast) cream 0.3% | PDE4 inhibitor | Mild to moderate atopic dermatitis | Enrollment of the last subject in the Integument-Ped study in children ages 2 to 5; total of 652 children enrolled; top-line data expected in the third quarter of 2023 | 5/2/23 | Dermatologic |
Dermavant Sciences Ltd., of Long Beach, Calif. | Vtama (tapinarof) | Aryl hydrocarbon receptor agonist | Atopic dermatitis | Top-line results from Adoring1 study in adult and pediatric subjects down to 2 years of age met primary and secondary endpoints; 45.4% of subjects achieved the primary endpoint of vIGA-ADTM response of clear (0) or almost clear (1) with at least a 2-grade improvement from baseline at week 8 vs. 13.9% on vehicle (p<0.0001); 55.8% of subjects achieved EASI75 at week 8 (p<0.0001); statistically significant improvement in itch with a ? 4-point reduction in the patient reported Peak Pruritus Numeric Rating Scale (PP-NRS) (p=0.0366); no new safety or tolerability signals; 55.8% of subjects ? 12 years old, with a baseline PP-NRS score ? 4, achieved a ? 4-point reduction in the PP-NRS at week 8 (p=0.0366) | 5/16/23 | Dermatologic |
89bio Inc., of San Francisco | Pegozafermin | Specifically engineered glycoPEGylated analog of fibroblast growth factor 21 (FGF21) | Severe hypertriglyceridemia | Initiated Entrust phase III study to evaluate efficacy, safety and tolerability in patients | 5/23/23 | Endocrine/Metabolic |
Arrowhead Pharmaceuticals Inc., of Pasadena, Calif. | ARO-APOC3 | RNAi therapeutic targeting apolipoprotein C-III (APOC3) | Familial chylomicronemia syndrome | Completed enrollment; primary study completion expected in the second quarter of 2024 | 5/16/23 | Endocrine/Metabolic |
Neurocrine Biosciences Inc., of San Diego | Efmody (hydrocortisone modified-release hard capsules/MRHC) | Glucocorticoid receptor agonist | Congenital adrenal hyperplasia | Post-hoc analysis data showed greatest reductions in 17-OHP standard deviation score 24-hour profile than standard therapy group; greater reduction in androgen levels at an equivalent dose of MRHC | 5/12/23 | Endocrine/Metabolic |
Oramed Pharmaceuticals Inc., of New York, and Hefei Tianhui Biotechnology Co. Ltd., of Hefei, China | ORMD-0801 | Oral insulin | Type 2 diabetes | In the ORA-D-013-1 study, a pool of patients with specific parameters, such as body mass index, baseline HbA1c, age, gender and body weight, had an over 1% placebo adjusted, statistically significant, reduction in HbA1c, which was similar to data from the study in China | 5/15/23 | Endocrine/Metabolic |
Pathalys Pharma Inc., of Research Triangle Park, N.C., and Launch Therapeutics Inc., of Boston | Upacicalcet | Calcimimetic | Secondary hyperparathyroidism in end-stage kidney disease patients on hemodialysis | First patient enrolled | 5/31/23 | Endocrine/Metabolic |
PTC Therapeutics Inc., of South Plainfield, N.J. | Sepiapterin (formerly PTC-923) | Oral formulation of synthetic sepiapterin | Phenylketonuria | Aphenity study achieved primary endpoint in adult and pediatric patients; mean percent Phe reduction in sepiapterin-treated patients was 63%; subset of classical patients showed mean percent Phe reduction was 69%; highly statistically significant sepiapterin treatment benefit (p<0.0001) vs. minimal reductions in Phe levels observed in placebo group; well-tolerated; no serious adverse events | 5/17/23 | Endocrine/Metabolic |
Eli Lilly and Co., of Indianapolis | Mirikizumab | Humanized IgG4 monoclonal antibody | Moderately to severely active ulcerative colitis | New analysis from Lucent-1 and Lucent-2 studies showed remission of key symptoms, including bowel urgency, associated with significant improvement in IBDQ total scores among adults treated with mirikizumab; of those treated at 12 and 52 weeks, bowel urgency remission directly accounted for 44.8% and 32.5% improvement in IBDQ total score, respectively; 22.7% and 39.1% improvement, respectively, was mediated by rectal bleeding remission, and 32.5% and 28.4%, respectively, mediated by stool frequency remission | 5/9/23 | Gastrointestinal |
Everest Medicines Ltd., of Shanghai | Etrasimod | Once-a-day, selective sphingosine 1-phosphate receptor modulator | Moderate to severe active ulcerative colitis | Completed patient enrollment in Asia | 5/16/23 | Gastrointestinal |
Ironwood Pharmaceuticals Inc., of Boston | Linzess (linaclotide) | Guanylate cyclase-C agonist | Functional constipation | Linaclotide at 72 mcg met primary and secondary endpoints for increased frequency of spontaneous bowel movements (SBM); statistically significant and clinically meaningful improvement compared to placebo in 12-week SBM frequency rate; greater than 2-fold least squares mean change from baseline in SBMs/week compared to placebo (p<0.0001); improvement in stool consistency at weeks 12 with linaclotide compared to placebo; well-tolerated | 5/8/23 | Gastrointestinal |
Janssen Pharmaceutical Cos., of Johnson & Johnson, of Spring House, Pa. | Tremfya (guselkumab) | Binds to p19 subunit of IL-23 and inhibits its interaction with IL-23 receptor | Moderately to severely active ulcerative colitis | New data from Quasar induction study in adults who had inadequate response or intolerance to conventional and/or advanced therapies showed statistically significant and clinically meaningful improvements across symptomatic and histo-endoscopic outcome measures; significantly greater proportion treated with Tremfya vs. placebo (22.6% vs. 7.9%, p<0.001) achieved clinical remission at week 12, the study's primary endpoint; at week 4 22.6% achieved symptomatic remission vs. 12.9% for placebo (p<0.001); at week 12, 49.9% achieved symptomatic remission vs. 20.7% for placebo (p<0.001) | 5/9/23 | Gastrointestinal |
Mallinckrodt plc, of Dublin | Terlivaz (terlipressin) | Derivative of the vasoconstrictor vasopressin | Hepatorenal syndrome | Pooled analysis showed incidence of episodes of bradycardia was higher in terlipressin-treated patients (6.3%) compared with placebo-treated patients (0.8%; p<0.001); none of these episodes were considered serious; most patients did not require a change in treatment dose (terlipressin: 72.7% vs. placebo: p=0.507); median Maddrey discriminant function score was similar across treatment groups (terlipressin: 96.9 vs. placebo: 97.7; p=0.681) in Confirm study with alcoholic hepatitis patients; verified HRS reversal was achieved in 30.9% of patients in the terlipressin group vs. 7.7% in the placebo group (p=0.005); admission to the ICU was similar for patients in the terlipressin and placebo groups (17.3% vs. 17.9%); mean length of stay in the ICU was shorter for terlipressin (6.9 days) vs. the placebo group (12.4 days); numerical decrease in renal replacement therapy by day 30 in the terlipressin group vs. the placebo group (21%vs. 25.6%) | 5/8/23 | Gastrointestinal |
Mirum Pharmaceuticals Inc., of Foster City, Calif. | Livmarli (maralixibat) | Ileal bile acid transporter inhibitor; oral solution | Progressive familial intrahepatic cholestasis | MARCH-PFIC study in children met primary and secondary endpoints; significant and rapid improvements in pruritus (p=0.0186) and serum bile acids (p=0.0305); significant improvements in bilirubin and weight z-scores as well as a trend in height z-score improvement; no new safety signals | 5/19/23 | Gastrointestinal |
Vanda Pharmaceuticals Inc., of Washington | Tradipitant | Neurokinin-1 receptor antagonist | Motion sickness | Tradipitant at 170 mg and 85 mg showed superior to placebo in preventing vomiting, with 18.3% and 19.5% of participants experiencing vomiting, respectively, compared to 44.3% of participants on placebo (p<0.0001 for both) | 5/25/23 | Gastrointestinal |
Mithra Pharmaceuticals SA, of Liege, Belgium | Estelle | Contraceptive pill containing estetrol/estetrol monohydrate 15 mg and drospirenone 3 mg | Contraception | Completed recruitment for pediatric study; results expected in the first half of 2024 | 5/11/23 | Genitourinary/Sexual Function |
Travere Therapeutics Inc., of San Diego | Filspari (sparsentan) | Endothelin and angiotensin II receptor antagonist | Focal segmental glomerulosclerosis | Duplex study did not achieve the primary efficacy eGFR slope endpoint over 108 weeks of treatment; favorable difference on eGFR total slope and eGFR chronic slope compared to the irbesartan, not statistically significant; mean reduction in proteinuria from baseline of 50% compared to 32% for irbesartan; well-tolerated; comparable safety profile to irbesartan | 5/1/23 | Genitourinary/Sexual Function |
Astrazeneca plc., of Cambridge, U.K. | Danicopan (ALXN-2040) | Oral factor D inhibitor | Paroxysmal nocturnal hemoglobinuria | Danicopan as an add-on to standard of care C5 inhibitor therapy in Alpha study showed danicopan significantly improved signs and symptoms of clinically significant extravascular hemolysis (EVH) in the small subset of patients | 5/16/23 | Hematologic |
Fibrogen Inc., of San Francisco | Evrenzo (roxadustat) | HIF-PH inhibitors; Oral medication | Anemia in patients with transfusion-dependent lower risk myelodysplastic syndrome | Matterhorn phase III study did not meet its primary efficacy endpoint; proportion of patients who achieved red blood cell transfusion independence in the first 28 weeks was 47.5% for roxadustat compared to 33.3% for placebo (p=0.217); consistent adverse event profile | 5/5/23 | Hematologic |
Fibrogen Inc., of San Francisco | Evrenzo (roxadustat) | HIF-PH inhibitors; oral medication | Anemia in patients receiving concurrent chemotherapy for non-myeloid malignancies | Top-line results showed roxadustat met primary endpoint; non-inferiority compared to recombinant erythropoietin alfa on the primary endpoint of change in hemoglobin (Hb) level from baseline to the average level during weeks 9-13; consistent safety analysis | 5/18/23 | Hematologic |
Pfizer Inc., of New York | Marstacimab (PF-06741086) | Anti-tissue factor pathway inhibitor | Severe hemophilia A or B, with or without inhibitors | Basis study met its primary endpoints; statistically significant and clinically relevant reduction in annualized bleeding rate (ABR); marstacimab demonstrated superiority (P< 0.0001) with a 92% reduction in bleeds in cohort of patients treated with on-demand factor replacement intravenous therapy in the lead-in period; superiority (p=0.0376) with marstacimab compared to prophylaxis, with a 35% reduction in ABR; well-tolerated; no deaths and no thromboembolic events or events of consumptive coagulopathy recorded in hemophilia patients | 5/30/23 | Hematologic |
Pharmaessentia Corp., of Burlington, Mass. | Besremi (ropeginterferon alfa-2b-njft) | Monopegylated, long-acting interferon | Polycythemia vera | First patient dosed; top-line data expected in 2024 | 5/3/23 | Hematologic |
Alvotech Holdings SA, of Reykjavik, Iceland | AVT-05 | Monoclonal antibody; biosimilar to Simponi/Simponi Aria (golimumab) | Moderate to severe rheumatoid arthritis | Initiated confirmatory patient study | 5/4/23 | Immune |
Aurinia Pharmaceuticals Inc., of Edmonton, Alberta | Lupkynis (voclosporin) | Calcineurin inhibitor | Lupus nephritis | Data from kidney biopsy substudy of Aurora program (Aurora 1 and Aurora 2 extension study) showed addition of Lupkynis to standard of care mycophenolate mofetil (MMF) and low-dose steroids led to significantly earlier and greater reductions in proteinuria while maintaining stable renal function, as measured by estimated glomerular filtration rate slope over time; higher rates of both complete renal response and partial renal response observed in treated patients at month 36, consistent with overall Aurora 2, Lupkynis-treated population; patients in treatment arm demonstrated histologic activity improvement with stable chronicity scores similar to active control arm of MMF and low-dose steroids alone over 18-months average treatment period at time of repeat biopsy | 5/9/23 | Immune |
Stallergenes Greer plc, of London | Staloral | Sublingual solution of allergen extracts; allergen immunotherapy | Respiratory allergies | Initiated phase III study to confirm the safety and efficacy in children and adolescents with birch pollen-induced allergic rhino-conjunctivitis with or without asthma | 5/25/23 | Immune |
X4 Pharmaceuticals Inc., of Boston | Mavorixafor | Small-molecule antagonist of chemokine receptor CXCR4 | Warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome | Top-line study demonstrated statistically significant and clinically meaningful improvements across a number of key infection metrics vs. placebo; mavorixafor treatment resulted in a statistically significant reduction (60%) in annualized infection rate vs. placebo (p<0.01); reduction was greater with time on treatment with participants on mavorixafor experiencing less than 1 infection per year vs. 4.5 for placebo; achieved statistical significance (p<0.005) in second 6 months of the trial; >75% reduction in the percentage of individuals experiencing severe infections (grade 3 or higher) in the mavorixafor group vs. placebo group; >70% reduction in mean total days with infections (2 weeks on mavorixafor treatment vs. 7 weeks on placebo); well-tolerated; no drug-related serious adverse events, no treatment limiting toxicities; no discontinuations due to safety; 90% of participants in the trial continued to receive mavorixafor treatment in ongoing open-label extension study | 5/17/23 | Immune |
Ferring Pharmaceuticals Inc., of Saint-Prex, Switzerland, and Parsippany, N.J. | Rebyota (fecal microbiota, live-jslm) | Microbiota-based live biotherapeutic | Prevention of recurrence of Clostridioides difficile infection | Pooled safety population showed 82.1% did not experience an inflammatory bowel disease (IBD)-related medication change after 8 weeks; 7 participants stopped or initiated concomitant medication within 8 weeks of treatment; incidence of treatment-related adverse events was comparable in patients with or without IBD (57.1% vs. 63.1%); separate ad hoc analysis of the PUNCH CD3-OLS trial showed treatment success was comparable in patients with and without immunocompromising conditions (79.5% and 73.5%, respectively); sustained clinical response through 6 months was maintained in 80% and 85% of treatment responders with and without immunocompromising conditions respectively; no significant clonal engraftment in placebo-treated participants | 5/8/23 | Infection |
Innoviva Inc., of Burlingame, Calif. | Sulbactam-durlobactam | ?-lactam antibiotics | Acinetobacter baumannii-calcoaceticus complex infections | Data showed sulbactam-durlobactam demonstrated statistical non-inferiority vs. colistin for the primary endpoint of 28-day all-cause mortality in patients with carbapenem-resistant Acinetobacter infections; significant difference in clinical cure rates; favorable safety profile with a statistically significant lower incidence of nephrotoxicity; results published in the Lancet Infectious Diseases | 5/11/23 | Infection |
Pfizer Inc., of New York | MenABCWY | Pentavalent meningococcal vaccine | Meningococcal disease | Preliminary results showed MenABCWY met all primary endpoints; statistical non-inferiority compared to Bexsero (meningococcal group B vaccine) and Menveo (meningococcal group A, C, W-135, and Y conjugate vaccine), in individuals 10-25 years old; immunological effectiveness against a panel of 110 diverse meningococcal serogroup B (MenB) invasive strains; acceptable safety profile | 5/12/23 | Infection |
Redhill Biopharma Ltd., of Tel Aviv, Israel | RHB-204 | RNA polymerase inhibitor | Nontuberculous mycobacteria | Terminated phase III study due to low accrual rate; plans to shift resources to RHB-107 late-stage development | 5/22/23 | Infection |
Revive Therapeutics Ltd., of Toronto | Bucillamine | Anti-inflammatory | Mild to moderate COVID-19 | Independent data and safety monitoring committee review meeting by May 17, 2023 | 5/2/23 | Infection |
Revive Therapeutics Ltd., of Toronto | Bucillamine | Anti-inflammatory | Mild to moderate COVID-19 | Independent data monitoring committee recommended to halt the study; halted early due to statistical significance of the primary endpoint likely not going to be met; company will now proceed to unblind and seek an evaluation of the study’s data, including the COVID-19 clinical symptoms data | 5/30/23 | Infection |
Sanofi SA, of Paris | Beyfortus (nirsevimab) | Long-acting monoclonal antibody | Prevention of respiratory syncytial virus (RSV) lower respiratory tract disease (LRTD) | New data from Harmonie study showed 83.21% reduction in hospitalizations due to RSV-related LRTD in infants under 12 months of age who received a single dose of nirsevimab, compared to infants who received no RSV intervention; favorable safety profile | 5/12/23 | Infection |
Seres Therapeutics Inc., of Cambridge, Mass., and Nestlé Health Science, of Lausanne, Switzerland | Vowst (SER-109) | Fecal microbiota spores, live | Prevent the recurrence of Clostridioides difficile infection | Open-label ECOSPOR IV phase III study showed 91.3% of participants achieved clinical response at week 8, and 94.6% of those participants maintained a response through week 24; in participants with a first recurrence, response rates to Vowst were similar to the overall population, with 93.5% of participants achieving a clinical response at week 8 and 94.4% of those participants maintaining the response through week 24; 2 or more recurrences had 90.3% achieved a clinical response at week 8 and 94.6% of those participants maintained that response through week 24; post-hoc subgroup analysis showed all participants (8.7%), participants with first recurrence (6.5%) vs. those with 2 or more recurrences (9.7%), participants <65 years old (4%) vs. ?65 years old (13.1%), participants who received vancomycin (8.9%) vs. those who received fidaxomicin (8.3%), males (10.8%) vs. females (7.8%) | 5/8/23 | Infection |
Orapharma Inc., of Bridgewater, N.J. | Arestin | Microspheres/minocycline HCl | Periodontal disease | Study showed Arestin decreased key pathogens; statistically significant decrease with scaling and root planning; improvements in probing depth, clinical attachment loss, bleeding on probing, and gingival index | 5/18/23 | Inflammatory |
Selecta Biosciences Inc., of Watertown, Mass. | SEL-212 | Pegadricase, a pegylated uricase with Immtor designed to mitigate the formation of anti-drug antibodies | Chronic refractory gout | Dissolve I and II studies met primary efficacy endpoints; statistically significant response rate; percentage of patients who achieved and maintained a serum urate (SU) < 6 mg/dL for at least 80% of the sixth 28-day treatment period; response rate in the high-dose group was 56% in dissolve I and 46% in dissolve II; response rate in the high-dose group for patients ?50 years old was 65% and 47% in the dissolve I & II studies, respectively; reduction in SU from baseline was 60% for both dose levels; favorable safety profile and well-tolerated across both doses levels | 5/31/23 | Inflammatory |
Amgen Inc., of Thousand Oaks, Calif. | Prolia (denosumab) | Targets RANK Ligand | Postmenopausal women with osteoporosis | Real-world study showed 36% reduced risk of hip fractures; 43% reduced risk of nonvertebral fractures; 30% reduced risk of hospitalized vertebral fractures and not statistically significant; 39% reduced risk of major osteoporotic; nonvertebral and hospitalized vertebral fractures; 50% reduced risk of non-hip, nonvertebral fractures; greater reduction in major osteoporotic (MOP) fracture risk in longer duration | 5/8/23 | Musculoskeletal |
Boan Biotechnology Co. Ltd., of Shanghai | BA-6101; BA-1102 | Denosumab biosimilar | Postmenopausal women with osteoporosis | First patient enrolled | 5/4/23 | Musculoskeletal |
Ferring Pharmaceuticals Inc., of Saint Prex, Switzerland? | SI-6603 | Chemonucleolytic drug | Lumbar disc herniation | Top-line results showed study met primary endpoint; statistically significant improvement in worst leg pain score vs. control at 13 weeks after injection; well-tolerated | 5/26/23 | Musculoskeletal |
Bioxcel Therapeutics Inc., of New Haven, Conn. | BXCL-501 (dexmedetomidine) sublingual film | Orally dissolving film formulation of alpha-2 adrenergic receptor agonist | Bipolar disorders- or schizophrenia-associated agitation | Top-line part 1 of pivotal serenity III study did not reach statistical significance (p=0.077) for primary endpoint at 2 hours post-dose for the change in PEC differed from that with placebo (p=0.049) and 52% were PEC responders by 2 hours post-dose (p=0.019 vs. placebo); proportion responding was greater than with placebo as early as 1 hour (p=0.035) and remained so through 4 hours; proportion responding by CGI-I assessment was greater than with placebo at 2 hours post dose (p=0.039); well-tolerated; no reported serious adverse events; mild to moderate adverse events | 5/25/23 | Neurology/Psychiatric |
Cassava Sciences Inc., of Austin, Texas | Simufilam | Binds to altered conformation of filamin A protein (FLNA) | Alzheimer’s disease | Completed patient dosing in 6-month, randomized controlled trial in 125 patients; top-line results expected in the third quarter of 2023 | 5/11/23 | Neurology/Psychiatric |
Corium Inc., of Boston | Azstarys (serdexmethylphenidate and dexmethylphenidate) | Alpha 2 adrenoceptor agonist | Children with attention-deficit hyperactivity disorder | New analysis from long-term study published in Journal of Child and Adolescent Psychopharmacology showed transient decrease in weight in first 3 months of treatment; no significant difference from the reference population; increased height but less than the reference population | 5/22/23 | Neurology/Psychiatric |
Eli Lilly and Co., of Indianapolis | Donanemab | Monoclonal antibody targeting N3pG beta amyloid plaques | Alzheimer’s disease | Study met primary and secondary endpoints; 47% of participants on donanemab showed no decline on CDR-SB compared to 29% of participants on placebo, (p<0.001); 52% of participants completed their course of treatment by 1 year and 72% completed by 18 months and achieved plaque clearance; 40% less decline in ability to perform activities of daily living at 18 months as measured by ADCS-iADL (p<0.0001); 39% lower risk of progressing to the next stage of disease compared to placebo (p<0.001); combined population of high tau participants and intermediate tau population showed meaningful positive results across all clinical endpoints (p<0.001), with CDR-SB and iADRS showing 29% and 22% slowing of decline, respectively | 5/3/23 | Neurology/Psychiatric |
Harmony Biosciences Holdings Inc., of Plymouth Meeting, Pa. | Wakix (pitolisant) | Selective histamine 3 receptor antagonist/inverse agonist | Idiopathic hypersomnia | Completed enrollment 9 months before its estimated enrollment completion date; top-line data expected in the fourth quarter of 2023 | 5/25/23 | Neurology/Psychiatric |
Lyndra Therapeutics Inc., of Watertown, Mass. | Risperidone (LYN-005) | Long-acting antipsychotics | Schizophrenia and schizoaffective disorder | First subject dosed | 5/9/23 | Neurology/Psychiatric |
Neurocrine Biosciences Inc., of San Diego | Ingrezza (valbenazine) | Vesicular monoamine transporter 2 inhibitor | Tardive dyskinesia | Post-hoc analysis of patients with co-occurring schizophrenia, schizoaffective disorder or a mood disorder in the Kinect 3 and 4 studies showed 94.3% of patients had a clinician-reported minimally improved or better score at week 48 on the CGI-TD scale; 91.9% of patients reported a minimally improved or better score at week 48 on the PGIC scale; mean change in CDSS total score was -0.5; mean change in PANSS total score was -3.2 | 5/15/23 | Neurology/Psychiatric |
Painreform Ltd., of Herzliya, Israel | PRF-110 (ropivacaine gel, extended-release) | Sodium channel inhibitor | Postoperative pain | Results showed Cmax value among all of the 15 patients was 10% of the safety window set by U.S. FDA; favorable pharmacokinetic profile | 5/18/23 | Neurology/Psychiatric |
Alimera Sciences Inc., of Atlanta | Iluvien (fluocinolone acetonide intravitreal implant) | Glucocorticoid receptor agonist; 0.19-mg sustained-release intravitreal implant | Diabetic macular edema | Completed enrollment in Newday study; 300 patients enrolled | 5/24/23 | Ocular |
Hanall Biopharma Co. Ltd., of Seoul, South Korea | Tanfanercept | Topical anti-inflammatory agent | Dry eye disease | VELOS-3 trial did not demonstrate statistical significance for either of the 2 primary efficacy endpoints: improvement from baseline in central corneal staining score or improvement from baseline in Eye Dryness Score via Visual Analogue Scale (VAS) assessed at week 8; statistically significant improvement (p<0.001) in the secondary efficacy endpoint of unanesthetized Schirmer testing to quantify change from baseline in tear volume at week 8; post hoc analysis subset of subjects that met VELOS-3 inclusion criteria met statistical significance (p<0.05) for improvement in subjects in the tanfanercept arm relative to subjects in vehicle control arm as assessed at week 8; well-tolerated, without any significant new adverse events observed | 5/19/23 | Ocular |
Innovent Biologics Inc., of Suzhou, China, and Rockville, Md. | IBI-311 | Monoclonal antibody targeting insulin-like growth factor-1 receptor | Thyroid eye disease | First patient dosed | 5/7/23 | Ocular |
Visus Therapeutics Inc., of Seattle | Brimochol PF | Fixed-dose combination of carbachol and brimonidine tartrate | Presbyopia | Study met primary and secondary endpoints; met its prespecified U.S. FDA primary endpoint for binocular near visual acuity across multiple time points, without a loss of ?5 letters at distance across all time points through hour 6 (carbachol p=0.006; brimonidine p=0.039); achieved prespecified EU and U.K. primary endpoints from 0.5 to 8 hours duration (carbachol p=0.003; brimonidine p=0.001) and out to 10 hours (carbachol p=0.004; brimonidine p=0.001); statistically significant gain in distance vision of 2 letters at 8 hours vs. active control, carbachol (p=0.047), compared to baseline at all time points (p<0.001); well-tolerated with headache rates of less than 10%; no treatment-related serious adverse events; no discontinuations due to adverse events | 5/4/23 | Ocular |
Horizon Therapeutics plc, of Dublin | Tepezza (teprotumumab) | Fully human monoclonal antibody that targets the insulin-like growth factor 1 (IGF-1) receptor (IGF-1R) | Thyroid eye disease | First patient enrolled in Japan | 5/1/23 | Other/Miscellaneous |
Novo Nordisk A/S, of Bagsvaerd, Denmark | Wegovy (semaglutide) | GLP-1 receptor agonist | Obesity | Oasis1 study at 50-mg dose achieved a statistically significant weight loss of 17.4% after 68 weeks compared to a 1.8% reduction with placebo; 89.2% of those who received oral semaglutide 50 mg reached a weight loss of 5% or more after 68 weeks compared to 24.5% with placebo; 50-mg dose achieved a superior weight loss of 15.1% compared to a reduction of 2.4% with placebo and 84.9% achieved a weight loss of 5% or more, compared to 25.8% with placebo | 5/22/23 | Other/Miscellaneous |
Soleno Therapeutics Inc., of Redwood City, Calif. | DCCR | Extended-release tablets of diazoxide choline | Prader-Willi syndrome | Completed enrollment in the randomized withdrawal period of study; top-line results expected in the third quarter of 2023 | 5/4/23 | Other/Miscellaneous |
Sound Pharmaceuticals Inc., of Seattle | SPI-1005 | Anti-inflammatory | Meniere's disease | Completed enrollment | 5/4/23 | Other/Miscellaneous |
Bellerophon Therapeutics Inc., of Warren, N.J. | Inopulse | Pulsatile nitric oxide delivery system | Fibrotic interstitial lung disease | Last patient completed blinded treatment in the ongoing phase III Rebuild study; top-line results in mid-2023 | 5/11/23 | Respiratory |
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and Sanofi SA, of Paris | Dupixent (dupilumab) | Fully human monoclonal antibody inhibiting IL-4 and IL-13 | Chronic obstructive pulmonary disease | Boreas trial met primary and all key secondary endpoints; 30% reduction in moderate or severe acute COPD exacerbations over 52 weeks (p<0.001); 160-mL improvement in lung function from baseline at 12 weeks vs. 77 mL (p<0.001) and sustained through 52 weeks (p<0.001); 9.7-point improvement in health-related quality of life at 52 weeks vs. a 6.4-point improvement (p=0.002); 2.7-point reduction in respiratory symptom severity from baseline at 52 weeks vs. a 1.6-point reduction (p=0.001); prespecified analysis from a subgroup of patients with elevated levels (?20 ppb) of fractional exhaled nitric oxide (FeNO) showed significant 38% reduction in exacerbations compared to placebo at 52 weeks (p=0.005); improvement in lung function of 232 mL vs. 108 mL for placebo at 12 weeks (p=0.002) and sustained at 52 weeks, with an improvement in lung function of 247 mL vs. 120 mL for placebo (p=0.003); consistent safety results; results published in The New England Journal of Medicine and JAMA | 5/21/23 | Respiratory |
Zambon SpA, of Milan | Liposomal cyclosporine A (L-CsA-i) | Inhaled therapy | Bronchiolitis obliterans syndrome | Completed enrollment in 2 phase III studies | 5/1/23 | Respiratory |
Achieve Life Sciences Inc., of Seattle | Cytisinicline | Plant-based alkaloid with a high binding affinity to the nicotinic acetylcholine receptor | Smoking cessation | Top-line results of ORCA-2 study showed a statistically significant benefit in helping people to quit smoking compared to placebo; low rates of adverse events; 4.4 times higher odds, or likelihood, to have quit smoking during the last 4 weeks of treatment compared to subjects who received placebo (p<0.0001) at 12 weeks; continuous smoking cessation rate from week 9 to week 24 was 20.5% for the 12-week cytisinicline arm compared to 4.2% for placebo, with an odds ratio of 5.79 (p<0.0001); 2.85 times higher odds, or likelihood, to have quit smoking during the last 4 weeks of treatment compared to subjects who received placebo (p=0.0008) at week 6; continuous smoking cessation rate from week 3 to week 24 was 6.8% for the 6-week cytisinicline arm compared to 1.1% for placebo, with an odds ratio of 6.25 (p=0.0006) at week 6; well-tolerated; no treatment-related serious adverse events reported | 5/23/23 | Toxicity and Intoxication |
Abbvie Inc., of North Chicago | Venclexta (venetoclax) | BCL-2 inhibitor | Chronic lymphocytic leukemia | Murano phase III study met its primary efficacy endpoint of investigator (INV)-assessed PFS; median PFS with venetoclax and rituximab was not reached compared with 17 months for bendamustine and rituximab (p<0.0001); median follow-up for PFS was 23.8 months in the primary efficacy analysis; pneumonia as serious adverse reaction | 6/9/23 | Cancer |
Actinium Pharmaceuticals Inc., of New York | Iomab-B | Iodine-131 (131I)-labeled murine monoclonal IgG1 antibody (BC8) targeting CD45 | Active relapsed or refractory acute myeloid leukemia | Sierra results showed statistically significant improvement in the prespecified primary endpoint of durable complete remission; rapid engraftment and high initial complete remission/complete remission with incomplete platelet recovery rates; favorable toxicity profile; well-tolerated | 6/12/23 | Cancer |
Astellas Pharma Inc., of Tokyo | Xospata (gilteritinib) | FLT3 inhibitor FMS-like tyrosine kinase 3 (FLT3)-internal tandem duplication (ITD) mutated acute myeloid leukemia | Mutated acute myeloid leukemia | Morpho trial as a maintenance therapy following allogeneic hematopoietic stem cell transplantation did not demonstrate statistically significant improvement of relapse-free survival (RFS) in the entire cohort (p=0.0518); clinical improvement of RFS in a subgroup of patients with detectable MRD (p=0.0065) compared to patients without detectable MRD (p=0.575); favorable RFS for 50% of patients with detectable MRD pre- or post-HSCT, compared to those without detectable MRD; RFS in the North American sub-population showed an HR of 0.397 (P=0.0022) for gilteritinib vs. placebo | 6/9/23 | Cancer |
Astrazeneca plc, of Cambridge, U.K. | Imfinzi (durvalumab) | Monoclonal antibody targeting PD-L1 | Gastric and gastroesophageal junction cancer | Interim analysis in combination with standard-of-care FLOT (fluorouracil, leucovorin, oxaliplatin and docetaxel) neoadjuvant chemotherapy showed statistically significant and clinically meaningful improvement in the key secondary endpoint of pathologic complete response vs. neoadjuvant chemotherapy alone | 6/3/23 | Cancer |
Astrazeneca plc, of Cambridge, U.K. | Imfinzi (durvalumab) plus Imjudo (tremelimumab) | Human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1; human monoclonal antibody that targets the activity of cytotoxic T-lymphocyte-associated protein 4 (CTLA-4) | Advanced liver cancer | Himalaya phase III studies showed sustained, clinically meaningful overall survival (OS) benefit at 4 years; reduced the risk of death by 22% compared to sorafenib; median OS was 16.4 months vs. 13.8 months (p=0.0037); OS rate was 30.7% vs. 19.8% and 25.2% vs. 15.1% at 36 and 48 months; consistent safety profile; no new safety signals | 6/29/23 | Cancer |
Aveo Oncology Inc., of Boston | Fotivda (tivozanib) | Oral, next-generation VEGF receptor tyrosine kinase inhibitor | Advanced renal cell carcinoma | Completed enrollment in Tinivo-2 study in combination with Opdivo; results expected in the second half of 2024 | 6/27/23 | Cancer |
Beigene Ltd., of Beijing | Brukinsa (zanubrutinib) | BTK inhibitor | B-cell malignancies | Post-hoc analysis of monotherapy data from Aspen and Alpine study showed well-tolerated compared wih Brukinsa and ibrutinib; mild-to-moderate in severity adverse events and lead to treatment discontinuation | 6/9/23 | Cancer |
Biontech SE, of Mainz, Germany, and Oncoc4 Inc., of Rockville, Md. | Gotistobart (BNT316/ONC-392) | Anti-CTLA-4 antibody candidate | Non-small-cell lung cancer | First patient treated; expected to enroll 600 patients | 6/29/23 | Cancer |
Checkpoint Therapeutics Inc., of Waltham, Mass. | Cosibelimab | PD-L1 inhibitor | Advanced cancers | Pharmacokinetic modeling data from the pivotal trial support the comparability of cosibelimab 800 mg every 2 weeks and 1200 mg every 3 weeks; the BLA is under review by U.S. FDA with a PDUFA goal date of Jan. 3, 2024 | 6/28/23 | Cancer |
Eli Lilly and Co., of Indianapolis | Verzenio (abemaciclib) | CDK4/6 inhibitor; non-chemotherapy oral tablet | HR+, HER2-, node-positive early breast cancer | New analysis from monarche study in combination with endocrine therapy (ET) demonstrated an absolute benefit in invasive disease-free survival (IDFS) rate of 5.9% in those age 65 and older (n=850) and 6.4% in patients under 65; similar rates of adverse events in combination and ET alones; overall patient-reported quality of life similar in both groups | 6/2/23 | Cancer |
Innocare Pharma Inc., of Beijing | Orelabrutinib | BTK inhibitor | Relapsed/refractory marginal zone lymphoma | Orelabrutinib showed high response rates with durable disease remission in Chinese patients after median follow-up of 22.3 months; IRC-assessed ORR was 57.8%, and median duration of response (DoR) and median progression-free survival (PFS) was 34.3 months and 36 months, respectively; 12-month PFS rate was 84.3% and the rate of overall survival (OS) was 91.5% at 12 months; well-tolerated with most treatment-related adverse events as grade 1 or 2 | 6/15/23 | Cancer |
IO Biotech ApS, of Copenhagen | IO102-IO103 | Cancer immunotherapy targeting indoleamine 2,3-dehydrogenase (IDO) and PD-L1. | Advanced melanoma | Trial achieved enrollment of 225 patients; open-label, randomized phase III study in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) | 6/14/23 | Cancer |
Iovance Biotherapeutics Inc., of San Carlos, Calif. | Lifileucel | Autologous tumor-infiltrating lymphocyte cell therapy | Advanced melanoma | First patient randomized in TILVANCE-301 study in combination with Keytruda (pembrolizumab, Merck & Co. Inc.) | 6/15/23 | Cancer |
Junshi Biosciences Co. Ltd., of Shanghai | TAB-004 (tifcemalimab; JS-004) | Anti-BTLA monoclonal antibody | Limited stage small-cell lung cancer | Initiation of phase III study in combination with toripalimab; planning to enroll 756 patients | 6/29/23 | Cancer |
Medexus Pharmaceuticals Inc., of Toronto | Trecondyv (treosulfan) | Preparative regimen for allogeneic hematopoietic stem cell transplantation, to be used in combination with fludarabine | Myelodysplastic syndromes | Study showed 30% improvement (83.2% vs. 53.2%) in one-year overall survival | 6/13/23 | Cancer |
Merck & Co. Inc., of Rahway, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 therapy | HER2-positive gastric or GEJ adenocarcinoma | KEYNOTE-811 study in combination with trastuzumab and chemotherapy met one of its dual primary endpoints of progression-free survival (PFS); statistically significant improvement in PFS compared to placebo in combination with trastuzumab and chemotherapy in the intention-to-treat (ITT) study population; consistent safety profile similar to previous studies | 6/16/23 | Cancer |
Merck & Co. Inc., of Rahway, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 therapy | Locally advanced resectable gastric and gastroesophageal junction (GEJ) adenocarcinoma | Top-line results from KEYNOTE-585 study at a pre-specified interim analysis conducted by an independent data monitoring committee showed study met one of its primary endpoints of pathological complete response (pCR) rate and demonstrated a statistically significant improvement in pCR rates compared with chemotherapy alone; improvement in primary endpoint of event-free survival (EFS) in monotherapy arm and results did not meet statistical significance per the pre-specified statistical analysis plan; overall survival not tested; consistent safety profile | 6/20/23 | Cancer |
Novartis AG, of Basel, Switzerland | Kisqali (ribociclib) | CDK4/6 inhibitor | Stage II and III hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) early breast cancer | Data showed lowered the risk of cancer recurrence by 25.2% in patients in combination with endocrine therapy (ET) compared to ET alone (p=0.0014); consistent, clinically meaningful invasive disease-free survival (iDFS) benefit across key pre-specified subgroups; distant disease-free survival (DDFS) (26% risk reduction) and recurrence-free survival (RFS) (28% risk reduction); trend for improvement in overall survival (OS); favorable safety profile at 40-mg; low rates of symptomatic adverse events; grade 3 or higher QT interval prolongation and diarrhea were low for Kisqali at 1 % and 0.6%, respectively | 6/2/23 | Cancer |
Panbela Therapeutics Inc., of Minneapolis | Flynpovi | CPP-1X (eflornithine) and sulindac | Prevention of adenomas and second primary colorectal cancers in patients previously treated for stages 0 through III colon or rectal cancer | The SWOG Cancer Research Network’s Paces S0820 phase III trial passed a single planned futility analysis and will continue | 6/28/23 | Cancer |
Renovorx Inc., of Los Altos, Calif. | Renovogem | Intra-arterial administration of gemcitabine | Pancreatic cancer | Interim data demonstrated an 8-month median PFS benefit, 15 vs. 7 months; 65% reduction in adverse events compared to the control arm | 6/29/23 | Cancer |
Roche Group AG, of Basel, Switzerland | Crovalimab | Anti-C5 recycling monoclonal antibody | Paroxysmal nocturnal hemoglobinuria | Study showed 65.7% of patients achieved transfusion avoidance (TA) from baseline to week 25 with crovalimab and 68.1% with eculizumab; clinically meaningful improvement in FACIT-Fatigue score from baseline to week 25 occurred in both arms, numerically greater improvement with crovalimab vs. eculizumab; adverse events as 78% of participants treated with crovalimab and 80% treated with eculizumab; maintained disease control in people switching from currently approved complement inhibitors | 6/9/23 | Cancer |
Seagen Inc., of Bothell, Wash. | Adcetris (brentuximab vedotin) | CD30-targeting antibody-drug conjugate | Classical Hodgkin lymphoma | Interim analysis at 40 months met its co-primary endpoints; showed an unprecedented 94.9% 3-year progression-free survival (PFS) in patients with combination of chemotherapy (etoposide, cyclophosphamide, doxorubicin [Adriamycin], dacarbazine, and dexamethasone) vs. 92.3% for eBEACOPP (bleomycin, etoposide, doxorubicin (Adriamycin), cyclophosphamide, vincristine, procarbazine, and prednisone); 12-month post-treatment safety data consistent with previous studies; less peripheral neuropathy (98.1% vs. 97.3% for eBEACOPP); 1-year PFS was 97.5% and 3-year overall survival was 98.5% in both treatment arms | 6/20/23 | Cancer |
Takeda Pharmaceutical Co. Ltd., of Tokyo, and Hutchmed Ltd., of Hong Kong | Fruquintinib | VEGFR-1, -2 and -3 inhibitor | Metastatic colorectal cancer | Results from FRESCO-2 study showed fruquintinib reduced the risk of death by 34% in previously treated patients; data published in the Lancet | 6/15/23 | Cancer |
Viracta Therapeutics Inc., of San Diego | Nana-val (nanatinostat and valganciclovir) | HDAC inhibitor; antiviral agent | Relapsed or refractory Epstein-Barr virus+ peripheral T-cell lymphoma (R/R EBV+ PTCL) | The R/R EBV+ PTCL cohort of its pivotal NAVAL-1 clinical trial met the pre-specified efficacy threshold, based upon a pre-specified minimum number of objective responses achieved within the first 10 patients enrolled, for expansion into stage 2 of the study | 6/28/23 | Cancer |
Cytokinetics Inc., of South San Francisco | CK-274 (aficamten) | Selective, small-molecule cardiac myosin inhibitor | Symptomatic obstructive hypertrophic cardiomyopathy | Initiated phase III MAPLE-HCM study as monotherapy compared to metoprolol as monotherapy in patients; open for enrollment; planning to enroll 170 patients | 6/20/23 | Cardiovascular |
Idorsia Ltd., of Allschwil, Switzerland | Aprocitentan | Dual endothelin receptor antagonist | Resistant hypertension | Aprocitentan induced reduction in night-time systolic BP compared with dippers for both 12.5 mg (p<0.01) and 25 mg (p<0.01); normalization of the dipping pattern was achieved in 44%, 40% and 31% of non-dippers for 12.5 mg, 25 mg of aprocitentan, and placebo, respectively after 4 weeks; change in BP load for both daytime and nighttime in both doses were significant compared to placebo (p<0.001 for all comparisons) | 6/26/23 | Cardiovascular |
Milestone Pharmaceuticals Inc., of Montreal | Etripamil | Calcium channel blocker | Paroxysmal supraventricular tachycardia | RAPID trial achieved its primary endpoint; statistically significant and clinically meaningful difference in time to supraventricular tachycardia conversion compared to placebo; statistically significant greater proportion of patients who took etripamil converted within 30 minutes compared to placebo (64.3% vs. 31.2%; p<0.001); significant reductions in time to conversion in patients who took etripamil were evident early and durable, persisting throughout the observation window of the study compared to placebo; median time to conversion of 17.2 minutes for patients treated with etripamil vs. 53.5 minutes for patients treated with placebo; statistically significant improvement in multiple defined symptoms; reduction in emergency-department visits; mild to moderate adverse events and no serious events; results published in the Lancet | 6/20/23 | Cardiovascular |
Cali Biosciences Co. Ltd., of San Diego | CPL-01 | Long-acting formulation of the anesthetic ropivacaine | Bunions | Started a second phase III study in addition to the one to treat pain associated with hernia repair | 6/5/23 | Dermatologic |
Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | Donidalorsen | Ligand-conjugated antisense targeting the prekallikrein mRNA | Hereditary angioedema prophylaxis | Completed enrollment in the Oasis-HAE study; top-line data expected in the first half of 2024 | 6/1/23 | Dermatologic |
Timber Pharmaceuticals Inc., of Basking Ridge, N.J. | TMB-001 | Topical isotretinoin formulated with the Ipeg delivery system | Moderate to severe congenital ichthyosis | Interim analysis (n=9) showed minimal systemic absorption of isotretinoin or its major metabolites in patients who completed 15 days of study when applied to 75-90% of the body surface area | 6/20/23 | Dermatologic |
Ascendis Pharma A/S, of Copenhagen, Denmark | Transcon PTH (palopegteriparatide) | Prodrug designed to restore parathyroid hormone (PTH [1-34]) | Hypoparathyroidism | Pathway trial showed 95% of patients in the open-label extension achieved independence from conventional therapy (defined as no active vitamin D and calcium supplements of <600mg/day) and none required active vitamin D; 81% of participants achieved both normal serum calcium and independence from conventional therapy at week 52; mean albumin-adjusted serum calcium levels were maintained within the normal range (8.3–10.6 mg/dL); sustained improvements in disease-related physical and cognitive symptoms, as well as physical functioning and daily life, starting at the first scheduled follow-up after randomization or switching from placebo and sustained through week 52; bone mineral density Z-scores continued to trend toward age and sex; normalized 24-hour urine calcium; well-tolerated; no new safety signal; mild or moderate adverse events | 6/17/23 | Endocrine/Metabolic |
Intrabio Inc., of Oxford, U.K. | IB-1001 | Orally administered therapy | Niemann-Pick disease Type C | Top-line results met the primary endpoint and key secondary endpoints with high statistical significance; statistically significant and clinically meaningful 1.37-point reduction of Scale for the Assessment and Rating of Ataxia (SARA) compared to placebo after 12 weeks (p<0.001); modified SARA (mSARA p<0.001); safe and well-tolerated; no drug-related serious adverse events | 6/29/23 | Endocrine/Metabolic |
Neurocrine Biosciences Inc., of San Diego | Efmody (hydrocortisone modified-release hard capsules) | Glucocorticoid receptor agonist | Congenital adrenal hyperplasia | Post-hoc analysis of extension study of 24 months treatment showed median daily hydrocortisone dose was reduced from 30 mg to 20 mg; significantly achieved lower androgen levels at ?25 mg of hydrocortisone per day; 48% of the subjects achieved 17-OHP levels <36 nmol/L compared with 31% at baseline | 6/15/23 | Endocrine/Metabolic |
Prothena Corp. plc, of Dublin | Birtamimab | Monoclonal antibody targeting amyloid | Mayo stage IV AL amyloidosis | Phase III Vital clinical trial data showed a statistically significant survival benefit of 74% was observed for those treated with birtamimab plus standard of care (SOC) vs. 49% in patients on placebo plus SOC at 9 months; for two secondary endpoints, quality of life and cardiac function, it demonstrated statistically significant improvements over placebo in a post hoc assessment; data were published in Blood | 6/28/23 | Endocrine/Metabolic |
Rezolute Inc., of Redwood City, Calif. | RZ-358 | Human monoclonal antibody that binds to an allosteric site on insulin receptors in the liver, fat, and muscle | Congenital hyperinsulinism | Plans to initiate Sunrize, a pivotal phase III clinical study, in Europe and other geographies outside the U.S. in the fourth quarter of 2023, with top-line results anticipated in the first half of 2025 | 6/28/23 | Endocrine/Metabolic |
Synlogic Inc., of Cambridge, Mass. | SYNB-1934 | Bacteria replacements; gastrointestinal microbiome modulators | Phenylketonuria | Initiated phase III study; to enroll about 150 patients | 6/5/23 | Endocrine/Metabolic |
Abbvie Inc., of North Chicago | Skyrizi (risankizumab) | IL-23 inhibitor | Ulcerative colitis | Top-line results from Command study achieved primary and secondary endpoints in adult patients at 180-mg or 360-mg subcutaneous [SC] doses; 51% of patients treated with risankizumab 180 mg and 48% of patients treated with risankizumab 360 mg achieved endoscopic improvement at week 52 vs. 32% of patients in the induction-only control group (p<0.001); significantly more patients treated with risankizumab 180 mg and 360 mg achieved histologic endoscopic mucosal improvement at week 52 compared to those treated with induction only (43% and 42%, respectively, vs. 23% [p<0.001]); significantly higher proportion of patients who received risankizumab 180 mg or 360 mg achieved steroid-free clinical remission compared to the induction-only control group at week 52 (40% and 37%, respectively, vs. 25%; p<0.01); consistent with the safety profile observed in previous studies; no new safety risks | 6/15/23 | Gastrointestinal |
Cymabay Therapeutics Inc., of Newark, Calif. | Seladelpar | PPAR-delta agonist | Primary biliary cholangitis | Post-hoc analysis of Enhance study showed significant decrease in mean IL-31 levels from baseline; seladelpar 5 mg (p<0.001), 10 mg (p<0.001) compared to placebo (4.3 to 3.9 pg/mL, not significant); clinically meaningful improvement in pruritus (? 2 decreases in NRS score) showed greater dose-dependent reductions in IL-31 from baseline than those without pruritus improvement; significant correlations were observed between changes in IL-31 vs. reported pruritus NRS scores (p<0.0001), alkaline phosphatase (ALP) levels (p<0.01), and total bile acids (p<0.0001) in the seladelpar 10 mg | 6/21/23 | Gastrointestinal |
Defender Pharmaceuticals Inc., of St. Louis | DPI-386 (intranasal scopolamine) | Anticholinergic | Prevention of nausea and vomiting induced by motion | In the DPI-386-MS-33 study, proportion of participants receiving DPI-386 who did not report vomiting and did not request rescue treatment was significantly greater than participants who received placebo (p<0.0001); full results to be published in the near future | 6/5/23 | Gastrointestinal |
Genfit SA, of Lille, France, and Ipsen SA, of Paris | Elafibranor | Dual ?,? PPAR agonist | Primary biliary cholangitis (PBC) | Top-line data from Elative study met primary and secondary endpoint; 51% of patients on elafibranor 80 mg achieved a cholestasis response compared with 4% on placebo (p<0.0001); reductions in ALP and bilirubin levels at week 52; pruritus improvement was observed with a greater decrease from baseline in the PBC Worst Itch NRS score for patients on elafibranor compared to placebo, did not reach statistical significance; well-tolerated; consistent safety profile | 6/30/23 | Gastrointestinal |
Infant Bacterial Therapeutics AB, of Stockholm | IBP-9414 | Pharmaceutical-grade probiotic Lactobacillus reuteri | Prevention of necrotizing enterocolitis in newborn infants | Independent data monitoring committee performed the final planned safety analysis; continue recruitment as planned; enrolled more than 1,650 of the planned 2,158 premature babies | 6/21/23 | Gastrointestinal |
Madrigal Pharmaceuticals Inc., of Conshohocken, Pa. | Resmetirom | Liver-directed THR-beta agonist | Nonalcoholic steatohepatitis with liver fibrosis | MAESTRO-NASH trial achieved primary and secondary endpoints; 2-stage fibrosis improvement in the modified intent-to-treat (mITT) population (resmetirom 80 mg, 8%; resmetirom 100 mg, 10%; placebo, 3%, p<0.0001); NASH resolution and ?1-stage improvement in fibrosis in the mITT population (resmetirom 80 mg, 14%; resmetirom 100 mg, 16%; placebo, 5%, p<0.0001); NASH resolution with ?2-point reduction in NAS and no worsening of fibrosis in patients with both a baseline and week 52 biopsy (resmetirom 80 mg, 32%; resmetirom 100 mg, 39%; placebo, 11%, p<0.0001); ?1-stage fibrosis improvement with no worsening of NAS in patients with both a baseline and week 52 biopsy (resmetirom 80 mg, 30%; resmetirom 100 mg, 34%; placebo, 16%, p<0.0001); NASH resolution or ?1-stage fibrosis improvement in patients with both a baseline and week 52 biopsy (resmetirom 80 mg, 42%; resmetirom 100 mg, 50%; placebo, 19%, p<0.0001); resmetirom achieved significant reductions relative to placebo in key noninvasive tests; magnetic resonance imaging-proton density fat fraction (MRI-PDFF) reductions at week 52; study discontinuations in the 100-mg arm were increased relative to placebo only during the first few weeks of treatment and were similar in all treatment groups for the remaining period of the first 52 weeks; adverse event-related discontinuations in the resmetirom 100-mg arm were GI-related; no adjudicated cases of drug-induced liver injury | 6/22/23 | Gastrointestinal |
Astellas Pharma Inc., of Tokyo | Fezolinetant | Neurokinin 3 receptor antagonist | Moderate to severe vasomotor symptoms due to menopause | In the Daylight study, drug produced a statistically significant reduction from baseline in the frequency of moderate to severe vasomotor symptoms at week 24 compared to placebo | 6/27/23 | Genitourinary/Sexual Function |
Endo International plc, of Dublin | Xiaflex | Collagenase clostridium histolyticum | Peyronie's disease | Post-hoc data of pooled data from 2 randomized, double-blind, placebo-controlled phase III trials showed incremental reductions in penile curvature were obtained from each of the 4 CCH treatment cycles administered to men; results published in Urology | 6/5/23 | Genitourinary/Sexual Function |
Vera Therapeutics Inc., of Brisbane, Calif. | Atacicept | Recombinant fusion protein that contains the soluble transmembrane activator and calcium-modulating cyclophilin ligand interactor receptor that binds to the cytokines B lymphocyte stimulator and a proliferation-inducing ligand | IgA nephropathy | Initiated phase III randomized, double-blind, placebo-controlled study; to evaluate efficacy and safety of atacicept 150 mg in patients | 6/7/23 | Genitourinary/Sexual Function |
Karyopharm Therapeutics Inc., of Newton, Mass. | Xpovio (selinexor) | XPO1 inhibitor | JAK inhibitor naïve myelofibrosis | Initiated a pivotal phase III clinical trial to assess the efficacy and safety of once-weekly dosing; patients will be randomized 2:1 to ruxolitinib plus selinexor 60 mg or ruxolitinib plus placebo | 6/28/23 | Hematologic |
Sanofi SA, of Paris | Altuviiio (efanesoctocog alfa, BIVV-001) | Recombinant factor VIII therapy | Hemophilia A | Xtend-kids study met primary and key secondary endpoints; clearance of administered factor concentrates in the blood greater in pediatric population than in adults; highly effective bleed protection in both children and adults in once-weekly 50-IU/kg dose; | 6/26/23 | Hematologic |
Takeda Pharmaceuticals Co. Ltd., of Osaka, Japan | TAK-755 | Recombinant ADAMTS13 protein | Congenital thrombotic thrombocytopenic purpura | Interim data showed TAK-755 reduced the incidence of thrombocytopenia by 60% as compared to plasma-based therapy; favorable safety and tolerability profile; no patient had an acute TTP event; TAK-755 achieved a 5-fold increase in ADAMTS13 activity levels compared to those receiving plasma-based therapy | 6/26/23 | Hematologic |
Vertex Pharmaceuticals Inc., of Boston, and Crispr Therapeutics, of Zug, Switzerland | Exa-cel (exagamglogene autotemcel) | An autologous, ex vivo CRISPR/Cas9 gene-edited therapy | Transfusion-dependent beta thalassemia | Trial met primary and secondary endpoints; 88.9% achieved the primary endpoint of transfusion-independence for at least 12 consecutive months (TI12) and the secondary endpoint of transfusion-independence for at least 6 consecutive months (TI6) with a mean weighted hemoglobin (p<0.0001); mean duration of transfusion-independence was 20.5 months with a maximum of 40.7 months; 3 patients who did not achieve TI12, 1 patient has since stopped transfusions and has been transfusion-free for 2.9 months; 2 patients had substantial reductions (80% and 96%) in transfusion volume from baseline; increased total hemoglobin; mean proportion of edited BCL11A alleles was stable over time in bone marrow and peripheral blood; clinically significant improvements in patient-reported outcomes | 6/9/23 | Hematologic |
Vertex Pharmaceuticals Inc., of Boston, and Crispr Therapeutics, of Zug, Switzerland | Exa-cel (exagamglogene autotemcel) | An autologous, ex vivo CRISPR/Cas9 gene-edited therapy | Sickle cell disease | Study met primary and secondary endpoints; 94.1% achieved the primary endpoint of freedom from vaso-occlusive crises (VOCs) for at least 12 consecutive months (VF12, p=0.0001); mean duration of VOC-free was 18.7 months with maximum of 36.5 months; being free from hospitalizations related to VOCs for at least 12 consecutive months (HF12, p<0.0001); 1 patient who did not achieve VF12 did achieve HF12 and has a complex set of comorbidities; 1 patient who achieved VF12 had a VOC 22.8 months following exa-cel infusion; increased in fetal hemoglobin and total hemoglobin; mean proportion of edited BCL11A alleles was stable over time in bone marrow and peripheral blood; clinically significant improvements in patient-reported outcomes | 6/9/23 | Hematologic |
Bavarian Nordic A/S, of Copenhagen | ABNCoV2 | VLP-based, non-adjuvanted COVID-19 booster vaccine | COVID-19 prophylaxis | Top-line results of booster study met its primary objective, ABNCoV2 was non-inferior to Comirnaty; results expected in the third quarter of 2023 | 6/27/23 | Infection |
Bavarian Nordic A/S, of Copenhagen, Denmark | PXVX-0317 | Virus-like particle (VLP)-based chikungunya virus (CHIKV) vaccine candidate | Chikungunya virus infection | Study met its primary endpoints; immunogenic in healthy adults ?65 years of age up to day 22 post vaccination; strong induction of CHIKV neutralizing antibodies in 87% of vaccinees with neutralizing antibody titers exceeding the threshold agreed with authorities; fast onset of protection by 82% of subject at day 15 post the single vaccination; well-tolerated in older adult population and with similar rates of adverse events observed between the active and the placebo group; trial will continue for a 6-month follow-up for both safety and immunogenicity | 6/20/23 | Infection |
Gilead Sciences Inc., of Foster City, Calif. | Hepcludex (bulevirtide) | Viral entry inhibitor | Chronic hepatitis delta (HDV) infection | Myr-301 study from week 96 at 2 mg or 10 mg achieved combined response compared with week 48 with no signs of treatment resistance; response rates of 55% and 56% with 2 mg and 10 mg, respectively; consistent safety profile; no serious adverse events; increase in bile acids without a correlation to pruritus or other symptoms; injection site reactions occurred in a higher proportion of study participants at 10-mg; results published in the New England Journal of Medicine | 6/23/23 | Infection |
GSK plc, of London | Arexvy (recombinant adjuvanted vaccine; RSVPreF3) | Vaccine, adjuvanted, contains recombinant glycoprotein F stabilized in the prefusion conformation | Lower respiratory tract disease caused by respiratory syncytial virus infection | Data from the AReSVi-006 study showed primary endpoint of vaccine efficacy was 82.6% against RSV-LRTD and 94.1% against severe LRTD after season 1 efficacy with 6.7 months median follow-up; 77.3% and 84.6% efficacy against RSV-LRTD and severe LRTD, respectively, with 14 months median follow-up; well-tolerated; adverse events were injection-site pain, fatigue, myalgia, headache and arthralgia; mild to moderate events | 6/21/23 | Infection |
Pfizer Inc., of New York | Aztreonam-avibactam (ATM-AVI) | Combination of antibiotics | Serious bacterial infections due to Gram-negative bacteria | Results showed well-tolerated; no new safety findings; similar safety profile to aztreonam alone; cure rate in the intention to treat (ITT) analysis set was 76.4% vs.74% for meropenem/colistin (MER ± COL) treatment arm in 4 patients with complicated intra-abdominal infections (cIAI); cure rate was 85.1% for ATM-AVI ± metronidazole (MTZ) vs. 79.5% for MER ± COL in clinically evaluable analysis; cure rate in the ITT analysis set was 45.9% vs. 41.7% in patients with hospital-acquired pneumonia and ventilator-associated pneumonia; cure rate was 46.7% vs. 54.5% for clinically evaluable analysis | 6/1/23 | Infection |
Polypid Ltd., of Petach Tikva, Israel | D-PLEX??? | Polymer-lipid encapsulation matrix comprising doxycycline | Abdominal colorectal surgical site infections | First patient recruited and scheduled for surgery in its revised Shield II phase III study; planning to enroll 550 additional patients beyond the 40 patients already recruited; top-line results expected in mid-2024 | 6/22/23 | Infection |
Seres Therapeutics Inc., of Cambridge, Mass., and Nestlé Health Science, of Lausanne, Switzerland | Vowst (SER-109) | Fecal microbiota spores, live | Prevent the recurrence of Clostridioides difficile infection | Post-hoc analysis from patients enrolled in the ECOSPOR III and ECOSPOR IV phase III studies showed that at baseline (pretreatment), individuals experiencing a first recurrence or multiple recurrences exhibited a similarly low degree of gut microbiome diversity; both first recurrence and multiple recurrence patient groups had significant increase in microbiome diversity as well as increased levels of secondary bile acids after Vowst treatment | 6/17/23 | Infection |
Sorrento Therapeutics Inc., of San Diego | Olgotrelvir (STI-1558) | Oral Mpro Inhibitor | COVID-19 | Enrolled and dosed 1,200 patients; top-line data in the third quarter of 2023; maintained an excellent safety profile with no grade 3 toxicities reported | 6/26/23 | Infection |
Valneva SE, of Saint Herblain, France | VLA-1553 | Single-shot chikungunya vaccine candidate | Chikungunya virus infection | Data showed very high seroresponse rate of 98.9% in participants 28 days after receiving the single administration; immunogenicity profile similar in both younger and older adults; 96% of participants maintained seroresponse six months after vaccination; safe and well-tolerated in younger and older adults; results published in the Lancet | 6/13/23 | Infection |
Cingulate Inc., of Kansas City, Kan. | CTx-1301 | Extended-release tablet formulation of dexmethylphenidate | Attention deficit hyperactivity disorder | Completed phase III adult dose-optimization trial; results expected in the third quarter 2023 | 6/8/23 | Musculoskeletal |
Ferrer Internacional SA, of Barcelona, Spain | FNP-122 | Oral formulation of edaravone | Amyotrophic lateral sclerosis | Extension of phase III Adore study; to evaluate the efficacy and safety of long-term treatment | 6/21/23 | Musculoskeletal |
Fibrogen Inc., of San Francisco | Pamrevlumab | Monoclonal antibody targeting connective tissue growth factor | Duchenne muscular dystrophy | Top-line data of LELANTOS-1 study in non-ambulatory patients did not meet the primary endpoint of Performance of the Upper Limb 2.0 (PUL 2.0) score at week 52 compared to baseline; safe and well-tolerated; mild or moderate adverse events; top-line results of ambulatory patients expected in the third quarter of 2023 | 6/7/23 | Musculoskeletal |
Acadia Pharmaceuticals Inc., of San Diego | Daybue (trofinetide) | Synthetic analogue of amino?terminal tripeptide of IGF-1 | Rett syndrome | Study demonstrated statistically significant improvement compared to placebo on both co-primary efficacy endpoints; mean change from baseline to week 12 in the Rett Syndrome Behavior Questionnaire (RSBQ) total score was ?5.1 and ?1.7 in the Daybue and placebo groups, respectively; statistically significant improvement in Clinical Global Impression–Improvement (CGI-I) compared to placebo (p-value of 0.0030) | 6/8/23 | Neurology/Psychiatric |
Annovis Bio Inc., of Berwyn, Pa. | Buntanetap (ANVS-401 or Posiphen) | Inhibitor of neurotoxic aggregating proteins | Alzheimer's and Parkinson's disease | Reached full enrollment in 9 months; randomized, double-blind, placebo-controlled trial investigating the efficacy, safety and tolerability of buntanetap | 6/8/23 | Neurology/Psychiatric |
Annovis Bio Inc., of Berwyn, Pa. | Buntanetap (ANVS-401 or Posiphen) | Inhibitor of neurotoxic aggregating proteins | Parkinson's disease | Data safety monitoring board recommended to continue trial as planned; safe and well-tolerated; no drug-related serious events; very low dropout rate (6%); 640 patients were screened, and 523 were enrolled in 9 months | 6/20/23 | Neurology/Psychiatric |
Bioxcel Therapeutics Inc., of New Haven, Conn. | BXCL-501 (dexmedetomidine) sublingual film | Orally dissolving film formulation of alpha-2 adrenergic receptor agonist | Alzheimer’s disease-related agitation | Top-line results from Tranquility II reported; statistically significant and clinically meaningful 7.5 point reduction from baseline in Positive and Negative Syndrome Scale-Excitatory Component (PEC) total score was observed at 2 hours vs. 5.4 with placebo (p=0.0112) with the 60 mcg dose; it did not hit the endpoint with the 40 mcg dose; at 60 mcg, it reduced agitation symptoms at 1 hour during the first episode of agitation (p=0.0185) but did not meet the other key secondary endpoint of change from baseline in PEC score at 30 minutes; reduction in PEC total score from pre-dose vs. placebo at 1 hour (p=0.011) and 2 hours (p=0.0044) for all episodes of agitation at 60-mcg; 443 episodes for 149 patients were treated over 12 weeks across all doses; well-tolerated; no syncope or falls related to trial drug | 6/29/23 | Neurology/Psychiatric |
Cassava Sciences Inc., of Austin, Texas | Simufilam | Binds to altered conformation of filamin A protein (FLNA) | Alzheimer’s disease | Results showed oral administration of simufilam 100 mg twice daily patients for 28 days, lymphocytes showed normalized mTOR activity and restored mTOR sensitivity to insulin; results published in the journal Frontiers in Aging | 6/27/23 | Neurology/Psychiatric |
Horizon Therapeutics plc, of Dublin | Uplizna (inebilizumab) | B-cell depleting anti-CD19 monoclonal antibody | Neuromyelitis optica spectrum disorder (NMOSD) | Open-label portion of trial (n=134) showed no new NMOSD symptoms at the end of the 28 weeks; 15% found to have asymptomatic MRI lesions on the spinal cord; lesions were shorter than attack-associated lesions; formation of these lesions decreased; urinary tract infection and arthralgia as common adverse events | 6/30/23 | Neurology/Psychiatric |
PTC Therapeutics Inc., of South Plainfield, N.J. | Vatiquinone (PTC-743) | 15-lipoxygenase inhibitor | Mitochondrial disease associated seizures | MIT-E trial failed to meet primary endpoint of reduction in observable motor seizures; reduced seizure frequency in the overall study population; benefit in largest subgroup of children with Leigh syndrome for key secondary endpoints | 6/29/23 | Neurology/Psychiatric |
Reviva Pharmaceuticals Holdings Inc., of Cupertino, Calif. | Brilaroxazine | Serotonin and dopamine receptor modulator | Schizophrenia | 80% of patients enrolled in phase III Recover study; to assess the safety and efficacy of brilaroxazine in approximately 400 patients; top-line results expected in the third quarter of 2023 | 6/22/23 | Neurology/Psychiatric |
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Hyqvia (human normal immunoglobulin 10% and recombinant human hyaluronidase) | Subcutaneous immunoglobulin replacement therapy | Chronic inflammatory demyelinating polyneuropathy | Maintenance therapy in adult patients showed clinically significant reduction in relapse rate vs. placebo (9.7% vs. 31.4%; p = 0.0045); lower probability of functional worsening rates vs. placebo (37.5% vs 54.4%); longer time to relapse compared to placebo; change in R-ODS centile scores were lower compared to placebo; consistent safety profile; published in the Journal of the Peripheral Nervous System | 6/20/23 | Neurology/Psychiatric |
Tonix Pharmaceuticals Holding Corp., of Chatham, N.J. | TNX-102 SL | Cyclobenzaprine HCl sublingual tablets | Fibromyalgia | Relief study met its prespecified primary endpoint; significantly reduced daily pain compared to placebo (p=0.01); higher rate of responders to TNX-102 SL (47%) than to placebo (35%; p=0.006) in an exploratory analysis; improvements in sleep quality, mitigation of fatigue and fibromyalgia-specific global symptomatic and functional recovery at 5.6 mg; early discontinuation rates were similar for TNX-102 SL and placebo (17.7% and 16.5%, respectively); well-tolerated; top-line results expected in the fourth quarter of 2023; results published in Arthritis Care & Research | 6/15/23 | Neurology/Psychiatric |
Aldeyra Therapeutics Inc., of Lexington, Mass. | Reproxalap | Modulator of reactive aldehyde species | Allergic conjunctivitis | Top-line results of 0.25% reproxalap ophthalmic solution achieved primary and secondary endpoints; statistically significant ocular itching score reduction across all 11 prespecified primary endpoint comparisons (p<0.0001 for each comparison) from 110 to 210 minutes in the allergen chamber; statistically significant reduction from baseline compared to vehicle (p=0.004) for the key secondary endpoint of investigator-assessed ocular redness over the duration of the allergen chamber; statistically significant improvement in patient-reported ocular tearing score on a 0?3 point scale over the duration of the allergen chamber (p<0.0001) and change from baseline in total ocular severity score (11?point composite of the itching, redness and tearing scores) over the duration of the allergen chamber (p<0.0001) | 6/15/23 | Ocular |
Glaukos Corp., of Aliso Viejo, Calif. | Epioxa | Corneal cross-linking therapy | Keratoconus | Completed enrollment in the study; data expected by the end of 2024 | 6/5/23 | Ocular |
Lianbio Co. Ltd., of Shanghai | TP-03 | Lotilaner ophthalmic solution, 0.25% | Demodex blepharitis | Completed enrollment in phase III Libra study in Chinese patients; top-line results expected in the fourth quarter of 2023 | 6/8/23 | Ocular |
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Eylea (aflibercept) | VEGF inhibitor | Diabetic macular edema | Top-line, 2-year (96 weeks) data from the pivotal Photon trial showed that 89% of all aflibercept 8 mg patients maintained ?12-week dosing intervals through 2 years; of those assigned to a 16-week dosing regimen at baseline, 83% maintained ?16-week dosing intervals through 2 years; many patients met the extension criteria for even longer dosing intervals, including 43% for ?20-week intervals and 27% for 24-week intervals; and visual gains and safety of aflibercept 8 mg remained consistent with the established profile of Eylea (aflibercept) 2 mg injection | 6/28/23 | Ocular |
Bayer Inc., of Whippany, N.J. | Gadoquatrane | Extracellular macrocyclic gadolinium-based contrast agent | Abnormalities within organs and tissues | Started the Quanti clinical development program encompassing two large multinational phase III studies, Quanti CNS for suspected disease of the central nervous system and Quanti OBR for suspected diseases in other body regions, as well as a pediatric study | 6/27/23 | Other/Miscellaneous |
Horizon Therapeutics plc, of Dublin | Tepezza (teprotumumab) | Fully human monoclonal antibody that targets IGF-1 receptor | Thyroid eye disease and Graves’ disease linked to hearing loss as adverse events | Data from phase II and III study (n=12 in tinnitus, n=109 in hearing impairment or dysfunction compared to those without hearing-related events) showed baseline overall Graves’ Ophthalmopathy Quality of Life (GO-QOL) scores similar between those with and without hearing-related adverse events; mean overall GO-QOL scores improved significantly for both groups from baseline throughout the trials; experienced improvements in GO-QOL score from baseline and none withdrew from the study by week 24 | 6/17/23 | Other/Miscellaneous |
Horizon Therapeutics plc, of Dublin | Tepezza (teprotumumab) | Fully human monoclonal antibody that targets IGF-1 receptor | Active thyroid eye disease | Top-line results from Japanese patients with higher levels of disease activity met primary endpoint; 89% of patients had clinically meaningful improvement in proptosis (?2 mm) compared with placebo (11%) (p<0.0001) at week 24; consistent safety profile | 6/22/23 | Other/Miscellaneous |
Mirum Pharmaceuticals Inc., of Foster City, Calif. | Livmarli (maralixibat) | Ileal bile acid transporter inhibitor; oral solution | Cholestatic pruritus in patients with Alagille syndrome | Retrospective analysis showed improvement in pruritus from baseline to week 48 with Livmarli; 6-year event-free survival (EFS) and transplant-free survival (TFS) compared to patients with minimal to no improvement in pruritus, at 88% vs. 57% (p=0.0046) and 93% vs. 57%, (p=0.0007), respectively; 6-year EFS and TFS compared to those with higher values were 85% vs. 49% (p=0.0010) and 90% vs. 49% (p=0.0001), respectively, for serum bile acids of <200 ?mol/L at week 48; 50% reduction in sBA demonstrated increased EFS and TFS at 86% vs. 39% (p<0.0001) and 90% vs. 39% (p<0.0001); 6-year EFS and TFS showed 90% vs. 43% (p<0.0001) and 94% vs. 42%; (p<0.0001) for bilirubin <6.5 mg/dL | 6/14/23 | Other/Miscellaneous |
Bellerophon Therapeutics Inc., of Warren, N.J. | Inopulse | Pulsatile nitric oxide delivery system | Fibrotic interstitial lung disease | Top-line data did not meet its primary endpoint related to the change in moderate to vigorous physical activity; safe and well-tolerated; performing worse than placebo by 5.49 minutes per day (p=0.2646) minimal difference between the 2 groups with none approaching statistical significance; well-tolerated with no safety concerns; overall activity showed 3.51 count/min benefit in favor of iNO45 (p=0.8572) over 16 weeks; 6-minute walk distance showed 0.19-meter benefit (p=0.9866); patient-reported outcomes and treatment-emergent adverse events favorable to placebo; deaths were balanced (4% vs. 4.3%) | 6/5/23 | Respiratory |
Fibrogen Inc., of San Francisco | Pamrevlumab | Monoclonal antibody targeting connective tissue growth factor | Idiopathic pulmonary fibrosis | Top-line data of ZEPHYRUS-1 study did not meet the primary and secondary endpoint; change from baseline in forced vital capacity (FVC) at week 48 (p=0.29) compared to placebo; mean decline in FVC from baseline to week 48 was 260 ml in the pamrevlumab arm compared to 330 ml in the placebo arm; time to disease progression (FVC percent predicted decline of ?10% or death) was also not met; safe and well-tolerated; treatment-emergent adverse events were mild or moderate; serious events for 28.2% of patients in the pamrevlumab group and 34.3% of patients in the placebo group; second phase III discontinued based on the ZEPHYRUS-1, ZEPHYRUS-2 study | 6/26/23 | Respiratory |
Savara Inc., of Austin, Texas | Molgramostim | Recombinant granulocyte macrophage colony-stimulating factor | Autoimmune pulmonary alveolar proteinosis | Completed enrollment of 164 patients; top-line data expected in the second quarter of 2024 | 6/26/23 | Respiratory |
Verona Pharma plc, of London | Ensifentrine | Inhibits PDE3/4 | Chronic obstructive pulmonary disease (COPD) | Results from its phase III ENHANCE trials showed it demonstrated improvements in lung function, symptoms and quality of life measures, as well as a substantial reduction in the rate and risk of COPD exacerbations and a favorable safety profile; results were published in the American Journal of Respiratory and Critical Care Medicine | 6/28/23 | Respiratory |
Astrazeneca plc, of Cambridge, U.K., and Daiichi Sankyo Co. Ltd., of Tokyo | Datopotamab deruxtecan (Dato-DXd) | TROP2-directed DXd antibody-drug conjugate | Locally advanced or metastatic non-small-cell lung cancer | TROPION-Lung01 study met dual primary endpoint of progression-free survival with statistically significant improvement compared to docetaxel; overall survival not matured; consistent safety profile and no new safety signals | 7/3/2023 | Cancer |
Bristol Myers Squibb Co., of New York | Opdivo (nivolumab) | PD-1 immune checkpoint inhibitor | Unresectable or metastatic urothelial carcinoma | Checkmate-901 trial met the dual primary endpoints of overall survival (OS) and progression-free survival (PFS) as assessed by Blinded Independent Central Review (BICR) at final analysis in combination with cisplatin based chemotherapy; no new safety concerns | 7/11/2023 | Cancer |
Cel-Sci Corp., of Vienna, Va. | Multikine | Leukocyte interleukin injection | Naïve locally advanced primary head & neck squamous cell carcinoma | Data showed significant log rank test (2-sided, p=0.034) favoring the Multikine plus standard of care (SOC) group vs. SOC alone in Kaplan-Meier lifetable for low tumor cell PD-L1 (defined as TPS <10); 20% survival advantage at 5-year favoring both Multikine plus SOC (~60% alive) vs. SOC alone (~40% alive) | 7/11/2023 | Cancer |
CG Oncology Inc., of Irvine, Calif. | CG-0070 (cretostimogene grenadenorepvec) | Intravesically delivered oncolytic immunotherapy | High-risk non-muscle invasive bladder cancer (NMIBC) unresponsive to Bacillus Calmette-Guerin (BCG) | Completed patient enrollment for Bond-003 study; 110 patients enrolled | 7/24/2023 | Cancer |
Checkpoint Therapeutics Inc., of Waltham, Mass. | Cosibelimab | Anti-PD-L1 antibody | Locally advanced and metastatic cutaneous squamous cell carcinoma | Objective response rate was 55%, including 23% complete responses, for 31 patients with locally advanced disease, and 50%, including 18% complete responses, for 78 patients with metastatic disease; median duration of response hasn’t been reached for either group | 7/27/2023 | Cancer |
Elevar Therapeutics Inc., of Fort Lee, N.J. | Rivoceranib and camrelizumab (SHR-1210) | Orally administered tyrosine kinase inhibitor and humanized monoclonal antibody targeting PD-1 | Unresectable hepatocellular carcinoma | Cares310 study demonstrated statistically significant and clinically meaningful prolonged overall survival, progression-free survival as well as improved overall response rate vs. sorafenib; results published in The Lancet | 7/24/2023 | Cancer |
Gilead Sciences Inc., of Foster City, Calif. | Magrolimab | Monoclonal antibody that binds to CD47 | Higher-risk myelodysplastic syndrome | Enhance study discontinued due to futility based on a planned analysis; consistent safety profile | 7/21/2023 | Cancer |
Janssen Pharmaceutical Cos., a unit of New Brunswick, N.J.-based Johnson & Johnson | Rybrevant (amivantamab-vmjw) | Bispecific antibody | Advanced or metastatic non-small-cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations | Top-line results of Papillon study in combination with chemotherapy (carboplatin-pemetrexed) met its primary endpoint with a statistically significant and clinically meaningful improvement in progression-free survival (PFS) vs. chemotherapy alone; combination treatment safety profile consistent with the safety profiles of the individual regimens | 7/17/2023 | Cancer |
Karyopharm Therapeutics Inc., of Newton, Mass. | Selinexor | XPO1 inhibitor | Advanced or recurrent TP53 wild-type endometrial cancer | Exploratory subgroup analyses from the Siendo study showed improvements in median progression-free survival (PFS) for the intent-to-treat (ITT) population, but not clinically meaningful in the primary analysis; at median duration of follow-up of 25.3 months in selinexor-treated patients with TP53 wild-type endometrial cancer, median PFS of 27.4 months compared to 5.2 months for patients with TP53 wild-type endometrial cancer receiving placebo; median PFS was not reached compared to 4.9 months in placebo; no new safety signals | 7/25/2023 | Cancer |
Merck & Co. Inc., of Rahway, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 therapy | High-risk locally advanced cervical cancer | Independent data monitoring committee reviewed prespecified interim analysis of KEYNOTE-A18 study in combination with concurrent chemoradiotherapy; trial met 1 of its primary endpoints of progression-free survival with statistically significant and clinically meaningful improvement vs. concurrent chemoradiotherapy alone; no new safety signals | 7/19/2023 | Cancer |
Merck & Co. Inc., of Rahway, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 therapy | High-risk, early-stage ER+/HER2- breast cancer | Independent data monitoring committee reviewed prespecified interim analysis of KEYNOTE-756 study in combination with chemotherapy; study met one of its dual primary endpoints of pathological complete response (pCR) rate following the neoadjuvant part of the neoadjuvant/adjuvant study regimen; statistically significant improvement in pCR rate compared to neoadjuvant placebo plus chemotherapy; recommended study to continue without changes to evaluate the other dual primary endpoint of event-free survival (EFS); no new safety signals | 7/28/2023 | Cancer |
Moderna Inc., of Cambridge, U.K., and Merck & Co. Inc., of Rahway, N.J. | mRNA-4157/V940 | Personalized mRNA cancer vaccine | High-risk melanoma | Initiated phase III study in combination with Keytruda (pembrolizumab); first patient planning to enroll in Australia; planning to enroll 1,089 patients; primary endpoint of the study is recurrence-free survival, and secondary endpoints include distant metastasis-free survival, overall survival and safety | 7/26/2023 | Cancer |
Natera Inc., of Austin, Texas | Signatera | Personalized molecular residual disease assay | Colorectal cancer | Completed enrollment in Altair study and met goal to enroll >240 patients; primary endpoint of the study is disease-free survival; secondary endpoint of ctDNA clearance; primary results expected in mid-2024 | 7/28/2023 | Cancer |
Panbela Therapeutics Inc., of Minneapolis | SBP-101 (ivospemin) | Polyamine analogue designed to induce polyamine metabolic inhibition | Metastatic pancreatic ductal adenocarcinoma | Independent data safety monitoring board completed its prespecified review of safety data for treated patients in the trial; recommended that the study continue without modification; to evaluate ivospemin in combination with gemcitabine and nab-paclitaxel in patients; interim analysis in early 2024 | 7/10/2023 | Cancer |
Photocure ASA, of Oslo, Norway, and Shanghai-based Asieris Pharmaceuticals Ltd. | Blue light cystoscopy (BLC) | Used with Hexvix/Cysview (hexaminolevulinate HCl) to detect tumors | Non-muscle invasive bladder cancer | Completed patient enrollment | 7/6/2023 | Cancer |
Urogen Pharma Ltd., of Princeton, N.J. | UGN-102 (mitomycin) | Sustained-release formulation of the chemotherapy mitomycin | Low-grade, intermediate-risk non-muscle invasive bladder cancer | In the Atlas study, UGN-102 reduced the risk of recurrence, progression or death by 55%; complete response rate (CRR) at 3 months was 64.8% for patients who received UGN-102 compared to 63.6% for those who received a TURBT; in the Envision study, CRR at 3 months was 79.2% | 7/27/2023 | Cancer |
Verastem Oncology Inc., of Boston | Avutometinib + defactinib | Inactive complexes of MEK with ARAF, BRAF and CRAF + FAK inhibitor | Low-grade serous ovarian cancer | Finalized U.S. FDA design of its confirmatory phase III study; study planning in the second half of 2023 | 7/5/2023 | Cancer |
Zai Lab Ltd., of Shanghai | Zejula (niraparib) | PARP inhibitor | Advanced ovarian cancer | Zejula significantly extended progression-free survival (PFS) vs. placebo and reduced the risk of disease progression or death by 55%; median PFS in intent-to-treat (ITT) population was 24.8 vs. 8.3 (p<.001); overall survival data not mature in ITT; grade ≥3 treatment-emergent adverse events (TEAEs) and serious adverse events were reported in 54.5% vs. 17.8% and 18.8% vs. 8.5% in Zejula and placebo patients, respectively; 7.6% Zejula patients and 5.4% placebo patients discontinued therapy due to TEAEs; PFS not reached vs. 10.8 months in patients with the gBRCA mutation in subanalyses and was 19.3 vs. 8.3 months in patients without the gBRCA mutation; PFS of 24.8 vs. 8.3 months in patients with optimal debulking, and 16.5 vs. 8.3 months in patients with suboptimal debulking; results published in JAMA Oncology | 7/19/2023 | Cancer |
Bridgebio Pharma Inc., of Palo Alto, Calif. | Acoramidis (AG-10) | Stabilizes tetrameric transthyretin | Transthyretin amyloid cardiomyopathy | Data showed statistically significant result observed on primary endpoint with a win ratio of 1.8 (p<0.0001); 58% of ties in Finkelstein-Schoenfeld (F-S) primary analysis broken by all-cause mortality and frequency of cardiovascular-related hospitalization; statistical significance also achieved on an F-S test with those 2 parameters alone (p=0.0182); clinically meaningful and consistent separation observed on all measures of mortality, morbidity, function and quality of life; on-treatment survival rate of 81% vs. placebo survival rate of 74% (absolute risk reduction of 6.43%; relative risk reduction of 25%); statistically significant relative risk reduction of 50% (p<0.0001) observed on frequency of cardiovascular-related hospitalization; statistically significant and clinically meaningful treatment benefit observed at 30 months on the secondary endpoints of NT-proBNP (p<0.0001), KCCQ (p<0.0001) and 6-minute walk distance (p<0.0001); exploratory post hoc analyses enabled by tafamidis drop-in, albeit at low patient numbers, showed 42% greater increase in serum TTR levels and a 92% improvement in median NT-proBNP relative to placebo + tafamidis; no safety signals | 7/17/2023 | Cardiovascular |
George Medicines Pty Ltd., of London | GMRx2 | Single-pill, triple-component combination of telmisartan, amlodipine and indapamide | Hypertension | Completed recruitment; more than 1,300 patients enrolled | 7/12/2023 | Cardiovascular |
Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | Eplontersen | Antisense medicine designed to inhibit the production of transthyretin | Transthyretin-mediated amyloid cardiomyopathy | Completed enrollment; study enrolled more than 1,400 patients; results expected in the first half of 2025 | 7/31/2023 | Cardiovascular |
Newamsterdam Pharma Co. NV, of Naarden, the Netherlands | Obicetrapib | CETP inhibitor | Heterozygous familial hypercholesterolemia and/or established atherosclerotic cardiovascular disease | Completed enrollment of over 2,500 patients in the Broadway study; top-line data expected in the second half of 2024 | 7/25/2023 | Cardiovascular |
Abbvie Inc., of North Chicago | Rinvoq (upadacitinib, 15 mg) | Oral, once-daily reversible JAK inhibitor | Moderate to severe hidradenitis suppurativa | First patient dosed; to evaluate the efficacy and safety of upadacitinib compared to placebo in adults and adolescents (12 to under 18) | 7/24/2023 | Dermatologic |
Boehringer Ingelheim GmbH, of Ingelheim, Germany | Spevigo (spesolimab) | Humanized, selective antibody that blocks the activation of the interleukin-36 receptor | Generalized pustular psoriasis | Effisayil trial showed spesolimab significantly reduced the risk of flares by 84% over 48 weeks compared to placebo; no flares were observed after week 4 of spesolimab treatment in the high-dose group (n=123) | 7/4/2023 | Dermatologic |
Galderma SA, of Lausanne, Switzerland | Nemolizumab | Humanized monoclonal antibody directed against IL-31 receptor | Prurigo nodularis | Data showed rapid onset of action of nemolizumab monotherapy, with significantly more patients receiving the treatment achieving an itch-free state by week 4 vs. placebo (19.7% vs. 2.2%, p<0.0001); achieved a clinically relevant improvement in itch intensity at week 4 (41% for nemolizumab vs. 7.7% for placebo, p<0.0001) | 7/4/2023 | Dermatologic |
Incyte Corp., of Wilmington, Del. | Opzelura (ruxolitinib cream) | JAK1/JAK2 inhibitor | Atopic dermatitis | Top-line results of TRuE-AD3 study met its primary endpoint; significantly more patients treated with ruxolitinib cream 0.75% and 1.5% achieved Investigator’s Global Assessment Treatment Success (IGA-TS) than patients treated with vehicle control; no new safety signals | 7/11/2023 | Dermatologic |
Journey Medical Corp., of Scottsdale, Ariz. | DFD-29 | Minocycline modified-release capsules, 40 mg | Papulopustular rosacea | MVOR-1 top-line study achieved the co-primary and all secondary endpoints and subjects completed the 16-week treatment ; IGA success as 65%, 46.1% and 31.2% in DFD-29, Oracea and placebo, respectively; DFD-29 and Oracea groups was statistically significant (p=0.007); DFD-29 and the placebo groups was statistically significant (p<0.001); mean reduction of 21.3 lesions vs. 15.9 lesions for Oracea and 12.2 lesions from baseline to week 16 in placebo, statistically significant (p<0.001); no significant safety issues | 7/11/2023 | Dermatologic |
Journey Medical Corp., of Scottsdale, Ariz. | DFD-29 | Minocycline modified-release capsules, 40 mg | Papulopustular rosacea | MVOR-2 top-line study achieved the co-primary and all secondary endpoints and subjects completed the 16-week treatment; IGA success as 60.1%, 31.4% and 26.8% in DFD-29, Oracea and placebo, respectively, statistically significant (p<0.001); mean reduction of lesions as 18.4, 14.9 and 11.1, in DFD-29, Oracea and placebo, respectively, statistically significant (p<0.001); no significant safety issues | 7/11/2023 | Dermatologic |
Kintor Pharmaceuticals Ltd., of Suzhou, China | KX-826 | Androgen receptor antagonist | Androgenetic alopecia | Completed first patient enrollment; planning to enroll 270 male and female patients | 7/19/2023 | Dermatologic |
Palvella Therapeutics Inc., of Wayne, Pa. | Qtorin | 3.9% rapamycin anhydrous gel | Pachyonychia congenita | Top-line results did not show a treatment effect on the Patient Global Assessment of Activities Difficulty primary endpoint; well-tolerated; no participants withdrew due to drug-related adverse events; no drug-related serious adverse events; mild or moderate adverse events; | 7/20/2023 | Dermatologic |
Camurus AB, of Lund, Sweden | CAM-2029 (octreotide hydrochloride) | Synthetic peptide analogue of somatostatin; somatostatin receptor agonist; subcutaneous (SC) depot | Acromegaly | Results from Acroinnova 2 study (n=135) showed favorable safety profile and robust long-term efficacy over 52 weeks of treatment; statistically significant improvements for multiple endpoints from baseline; increased IGF-1 response rate in full population from 49.7% to 58.4% with a difference of 8.7%; new patients from 12% to 30.3% with a difference of 18.3%; treatment naïve patients from 20.2% to 93.8% with a difference of 73.7%; stable response rate from 92.8% to 89.4%; reduced acromegaly symptom burden; increased patient and treatment satisfaction scores; improved quality of life; well-tolerated; no severe adverse events ; 1 serious event of cholelithiasis, resolved and patient continued in the trial; 2 patients discontinued treatment due to adverse reactions; 1 patient had an adverse reaction leading to dose reduction | 7/17/2023 | Endocrine/Metabolic |
Newamsterdam Pharma BV, of Naarden, the Netherlands | Obicetrapib | Oral, once-daily CETP inhibitor | Heterozygous familial hypercholesterolemia and/or established atherosclerotic cardiovascular disease | Completed enrollment in Broadway study; target enrollment of 2,400 subjects was exceeded; top-line data expected in the second half of 2024 | 7/25/2023 | Endocrine/Metabolic |
Grifols SA, of Barcelona, Spain | Albutein | 20% Injectable Solution; Human Albumin | Decompensated cirrhosis and ascites | Completed enrollment in Preciosa phase III study; to determine the potential of long-term treatment to increase survival time in patients | 7/18/2023 | Gastrointestinal |
Chinook Therapeutics Inc., of Seattle | Zigakibart (BION-1301) | Anti-APRIL monoclonal antibody | IgA nephropathy | First patient enrolled in Beyond study | 7/28/2023 | Genitourinary/Sexual Function |
Everest Medicines Ltd., of Shanghai | Nefecon | Oral, delayed release formulation of budesonide | IgA nephropathy | Completed patient enrollment for the China open-label extension in NefIgard study; additional 9 months of treatment with Nefecon to all qualifying patients who have completed the NefIgard study; to evaluate the efficacy and safety of extended and repeated Nefecon treatment in patients | 7/31/2023 | Genitourinary/Sexual Function |
Akari Therapeutics plc, of London | Nomacopan | Bispecific recombinant inhibitor of complement C5 activation and leukotriene B4 (LTB4) activity | Pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy | Initiated enrollment in the registrational phase III pediatric study; phase III double-blind placebo-controlled clinical trial of nomacopan in adult patients enrollment will be in 2024 | 7/13/2023 | Immune |
Gamida Cell Ltd., of Boston | Omisirge (omidubicel-onlv) | Nicotinamide modified allogeneic hematopoietic progenitor cell therapy derived from cord blood | Hematopoietic stem cell transplantation | Data showed Omisirge recipients demonstrated an up to 70-fold advantage over patients who received umbilical cord blood (UCB) in median cell counts across most cell populations; 3 times more likely to achieve clinically relevant Th and NK cell counts of 100 cells/µL or above by 3 weeks; 33-fold higher median dose of CD34+ stem cells than UCB recipients; faster immune reconstitution by day 7; superior reconstitution of B cells, dendritic cells and monocytes; rate of grade 2/3 infections in the first year were significantly lower with Omisirge than with UCB; 29% vs. 70% for bacterial infections and 6% vs. 45% for viral infections; no differences were reported in incidence of acute or chronic GVHD between the Omisirge transplanted patients and the UCB transplanted patients; results published in Transplantation and Cellular Therapy | 7/11/2023 | Immune |
Genentech Inc., of South San Francisco, a member of the Roche Group | Ocrevus (ocrelizumab) | Monoclonal antibody targeting CD20-positive B cells | Relapsing forms and primary progressive of multiple sclerosis | Ocarina II study met its primary and secondary endpoints in patients | 7/13/2023 | Immune |
Bavarian Nordic A/S, of Copenhagen | MVA-BN | Vaccines | Lower respiratory tract disease of respiratory syncytial virus infection | Study did not meet all the primary endpoints in adults ≥60 years; 59% efficacy in preventing at least 2 predefined LRTD symptoms; 42.9% efficacy and missed the co-primary endpoint of the study; study discontinued | 7/22/2023 | Infection |
Gilead Sciences Inc., of Foster, Calif. | Biktarvy (bictegravir + emtricitabine + tenofovir alafenamide) | HIV-1 integrase inhibitor; nucleoside reverse transcriptase inhibitor | HIV infection | Open-label study results from pregnant women with HIV showed well-tolerated; all participants maintained virologic suppression at delivery (HIV-1 RNA <50 copies/mL); no cases of perinatal HIV transmission were observed; no adverse events leading to premature discontinuation and no drug-related events in the pregnant women or neonates; proportion of participants who were underweight decreased from 20.4% to 14.3%, while the proportion of normal weight participants increased from 67.3% to 73.5%, respectively, at week 56 in children with HIV; proportion of overweight or obese participants remained stable at 12.2%; lipid metabolism parameters improved during 48 weeks | 7/23/2023 | Infection |
Gilead Sciences Inc., of Foster, Calif. | Biktarvy (bictegravir + emtricitabine + tenofovir alafenamide) | HIV-1 integrase inhibitor; nucleoside reverse transcriptase inhibitor | HIV/HBV co-infection | Alliance study at week 96 showed numerically higher levels of hepatitis B viral suppression with Biktarvy (75% vs. 70%, p=0.64); Biktarvy had numerically higher alanine aminotransferase (ALT) normalization (72% vs. 57%, p=0.13) and hepatitis B surface antigen loss (23% vs. 14%, p=0.066) and seroconversion (9% vs. 7%, p=0.44); Biktarvy or dolutegravir+emtricitabine/tenofovir alafenamide (DTG+F/TDF groups) also had high rates of HIV suppression (HIV-1 RNA <50 copies/mL) at week 96 (87% vs. 88%, p=0.94); mean CD4 cell count increases of 261 and 229 cells/μl from baseline, respectively; similar safety findings; upper respiratory tract infection (19.8% vs. 14.8%), COVID-19 (38% vs. 36.1%), pyrexia (12.4% vs. 13.1%), ALT increase (8.3% vs. 12.3%) and nasopharyngitis (12.4% vs. 6.6%) for Biktarvy and DTG+F/TDF groups, respectively | 7/23/2023 | Infection |
Merck & Co. Inc., of Rahway, N.J. | V-116 | 21-valent pneumococcal conjugate vaccine | Pneumococcal infection prophylaxis | In the Stride-3 study, V-116 produced statistically significant immune responses compared to PCV-920 in vaccine-naïve adults for serotypes common to both vaccines; immune responses were also observed for serotypes unique to V-116; in the Stride-6 study, V-116 was immunogenic for all 21 pneumococcal serotypes in the vaccine in adults who previously received a pneumococcal vaccine at least 1 year prior | 7/27/2023 | Infection |
Novavax Inc., of Gaithersburg, Md., and Pfizer Inc., of New York, and Biontech SE, of Mainz, Germany | NVX-CoV2373/Comirnaty (BNT-162b2; tozinameran) | Recombinant protein-based COVID-19 vaccine/mRNA vaccine | COVID-19 | Com-COV3 study with mixed 2 doses in adolescents, ages 12-16, showed mild to moderate reactions; well-tolerated; robust immune responses in adolescents for at least 8 months; NVXCoV-2373 following 30-µg BNT-162b2 as a first dose showed highest humoral and peak cellular immune responses; neutralizing antibodies against omicron BA.1 and BA.2 were higher after NVXCoV-2373 than a 2-dose 30-µg BNT162b2 schedule; results published in the Journal of Infection | 7/6/2023 | Infection |
Revive Therapeutics Ltd., of Toronto | Bucillamine | Anti-inflammatory | Mild to moderate COVID-19 | Study (n=713) did not achieve statistical significance on the clinical endpoints; no deaths and 4 hospitalizations, of which 3 were from the placebo arm and 1 from the bucillamine low-dose group (300 mg/day); no hospitalizations occurred in the bucillamine large-dose group (600 mg/day); 29.1% improvement over placebo in time to normal oxygen saturation (SpO2); bucillamine and placebo arms had a median of 11 days for time to PCR negative and stay negative; bucillamine demonstrated a slightly shorter 75% of 14 days vs. 15 days in placebo | 7/6/2023 | Infection |
Horizon Therapeutics plc, of Dublin | Krystexxa (pegloticase) | Pegylated uric acid specific enzyme | Uncontrolled gout | Long-term Mirror randomized controlled study showed improvement in the patient response rate remained nearly 30% points higher with methotrexate co-therapy during month 12 (p<0.001); 23%-point increase in the complete resolution of at least 1 tophus at month 12 (p=0.048); consistent pharmacokinetic and immunogenicity; HAQ Pain and Health scores also progressively and meaningfully improved during treatment; Krystexxa with methotrexate group had greater improvement in both HAQ Pain (p=0.0272) and HAQ Health (p=0.0222) compared to Krystexxa with placebo; results published in ACR Open Rheumatology | 7/6/2023 | Inflammatory |
Brainstorm Cell Therapeutics Inc., of New York | Nurown | Mesenchymal stem cell therapy | Amyotrophic lateral sclerosis | Biomarker data showed reduction in neurofilament light (NfL) values from baseline to week 20 compared to placebo (p<0.05); greater decline from baseline at week 28 as measured by ALSFRS-R total score; had higher baseline NfL values (r=-0.33, p=0.0064) | 7/7/2023 | Musculoskeletal |
Ultragenyx Pharmaceutical Inc., of Novato, Calif., and Mereo Biopharma Group plc, of London | Setrusumab (UX-143) | Monoclonal antibody targeting sclerostin | Osteogenesis imperfecta | First patient dosed; trial evaluating setrusumab in pediatric and young adult patients | 7/6/2023 | Musculoskeletal |
Alzheon Inc., of Framingham, Mass. | ALZ-801 (valiltramiprosate) | Prodrug of tramiprosate, which inhibits the formation of toxic amyloid beta 42 oligomers | Alzheimer’s disease | APOLLOE4 phase III trial showed a high rate of cerebral amyloid angiopathy (CAA)-related lesions at baseline; robust clinical efficacy in the highest-risk population; favorable safety with no increased risk of brain vasogenic edema | 7/11/2023 | Neurology/Psychiatric |
Axsome Therapeutics Inc., of New York | Solriamfetol | Dopamine and norepinephrine reuptake inhibitor; trace-amine-associated receptor 1 (TAAR1) and 5HT1a receptors agonist | Attention deficit hyperactivity disorder | First patient dosed | 7/7/2023 | Neurology/Psychiatric |
Biogen Inc., of Cambridge, Mass., and Eisai Co. Ltd., of Tokyo | Lecanemab | Anti-amyloid beta protofibril antibody | Early Alzheimer’s disease | Lecanemab reduced amyloid-beta (Aβ) pathology and downstream biomarker changes; increase in plasma Aβ42/40 ratio was observed with lecanemab compared to placebo (adjusted mean change from baseline of lecanemab: 0.008, placebo: 0.001, p<0.0001); reduction in plasma p-Tau181 was observed with lecanemab compared to placebo (adjusted mean change from baseline of lecanemab: -0.575 pg/mL, placebo: 0.201 pg/mL, p<0.0001) | 7/19/2023 | Neurology/Psychiatric |
Cingulate Inc., of Kansas City, Kan. | CTx-1301 | Extended-release tablet formulation of dexmethylphenidate | Attention deficit hyperactivity disorder | Top-line results of CTx-1301-022 showed a trend toward significance (p=0.089); improvements on the Permanent Product Measure of Performance; well-tolerated; 9% experienced treatment emergent adverse events vs. 30% in placebo | 7/11/2023 | Neurology/Psychiatric |
Neumora Therapeutics Inc., of Watertown, Mass. | Navacaprant (NMRA-140) | Once-daily kappa opioid receptor (KOR) antagonist | Major depressive disorder | Planning to initiate Koastal phase III study includes Koastal-1, 2 and 3; to evaluate the efficacy and safety of monotherapy | 7/18/2023 | Neurology/Psychiatric |
NLS Pharma Ltd., of Stans, Switzerland | Mazindol extended release | Mazindol extended release | Narcolepsy | U.S. FDA reviewed the full protocol for the NLS-1031 study; independent Institutional Review Board approved full phase III study | 7/3/2023 | Neurology/Psychiatric |
Sunovion Pharmaceuticals Inc., of Marlborough, Mass., and Otsuka Pharmaceuticals Co. Ltd., of Tokyo | Ulotaront (SEP-363856) | Trace amine-associated receptor 1 (TAAR1) agonist with 5-HT1A agonist activity | Schizophrenia | Top-line results of Diamond 1 and Diamond 2 study did not meet their primary endpoint in acutely psychotic adults; reduction in the Positive and Negative Syndrome Scale (PANSS) total score over time in Diamond 1 study; superior to placebo on the primary endpoint of change from baseline in PANSS total score at week 6; ulotaront 75 mg/day and 100 mg/day did not demonstrate statistically significant improvement compared to placebo on the primary endpoint; numerically larger mean reductions in PANSS total score from baseline compared to placebo at week 6; safe and well-tolerated in both studies | 7/31/2023 | Neurology/Psychiatric |
Paxmedica Inc., of Tarrytown, N.Y. | PAX-101 | Suramin intravenous infusion | Autism spectrum disorder | Top-line results from PAX-HAT-301 study (n=349 patients) achieved primary endpoint with statistically significant and clinically meaningful results; health outcomes in the suramin-treated cohort were statistically significantly better than those in the natural history cohort; proportion of patients in the suramin-treated group alive and not meeting any supportive descriptive criteria of death, any clinical worsening or moribund status was 92% vs. 50% in the natural history cohort (p<0.001) | 7/24/2023 | Neurology/Psychiatric |
Annexon Inc., of South San Francisco | ANX-007 | Complement C1q subcomponent inhibitor | Geographic atrophy | Results from additional analyses of Archer trial showed drug well-tolerated through month 12; no increase in CNV rates between the treated and sham arms; no events of retinal vasculitis; protection against ≥15 BCVA letter loss; BCVA ≥10 letter loss and BCVA ≥20 letter loss demonstrated consistent; dose-dependent protection from vision loss; primary endpoint of mean rate of change (slope) in GA lesion area compared to sham at 12 months did not reach statistical significance; trend toward greater effect in the second 6 months of study for both treatment groups in lesion size | 7/30/2023 | Ocular |
Apellis Pharmaceuticals Inc., of Waltham, Mass. | Syfovre (pegcetacoplan injection) | Intravitreal targeted C3 therapy | Geographic atrophy secondary to age-related macular degeneration | Data showed no indication of drug-related immunogenicity; zero events of retinal vasculitis; no vasculitis events | 7/29/2023 | Ocular |
Belite Bio Inc., of San Diego | Tinlarebant | Oral, once-daily retinol binding protein 4 (RBP4) antagonist | Stargardt disease | Completed enrollment of 90 adolescent subjects in Dragon study; efficacy data in mid-2024 | 7/24/2023 | Ocular |
Belite Bio Inc., of San Diego | Tinlarebant | Oral, once-daily retinol binding protein 4 (RBP4) antagonist | Geographic atrophy | First subject dosed; planning to enroll 430 subjects | 7/27/2023 | Ocular |
Kodiak Sciences Inc., of Palo Alto, Calif. | Tarcocimab tedromer (KSI-301) | Anti-VEGF antibody biopolymer conjugate | Wet age related macular degeneration | Daylight study met the primary endpoint of noninferior visual acuity gains for tarcocimab dosed monthly compared to aflibercept dosed every 8 weeks following 3 monthly loading doses; safe and well-tolerated; intraocular inflammation occurred in 3.3% of patients treated with monthly tarcocimab and 0.4% of patients treated with aflibercept with no vasculitis or occlusion | 7/24/2023 | Ocular |
Kodiak Sciences Inc., of Palo Alto, Calif. | Tarcocimab tedromer (KSI-301) | Anti-VEGF antibody biopolymer conjugate | Diabetic macular edema | Gleam and Glimmer studies from treatment-naïve subjects did not meet their primary efficacy endpoints of showing noninferior visual acuity gains for tarcocimab dosed every 8 to 24 weeks after 3 monthly loading doses compared to aflibercept given every 8 weeks after 5 monthly loading doses; gained an average of 6.4 eye chart letters compared with 10.3 letters for patients treated with aflibercept in Gleam study; tarcocimab gained an average of 7.4 eye chart letters at the primary endpoint compared with 12.2 letters for patients treated with aflibercept in Glimmer study; unexpected increase in cataracts in both studies; no new or unexpected safety signals; intraocular inflammation in 1.3% and 0.2% of tarcocimab and aflibercept treated patients, respectively; no cases of intraocular inflammation with vasculitis or vascular occlusion; company discontinuing development of the tarcocimab program | 7/24/2023 | Ocular |
Nicox SA, of Sophia Antipolis, France | NCX-470 | Nitric oxide-donating bimatoprost eye drop | Open-angle glaucoma or ocular hypertension | Study met the primary efficacy endpoint of non-inferiority to latanoprost at all 9 timepoints measured in the Mont Blanc trial; statistically greater proportion of subjects with 10 mmHg or more IOP reduction compared to latanoprost at week 2, week 6, and month 3 ranging from 69% to 46% in the NCX-470 group compared to 60% to 34% in the latanoprost group; safe and well-tolerated | 7/3/2023 | Ocular |
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Eylea (aflibercept) | Targeted therapy | Diabetic macular edema | Results from Photon trial at 2 years (96 weeks) in the extended dosing intervals (9.5 for the 12-week aflibercept 8-mg group, 7.8 for the 16-week aflibercept 8-mg group and 13.8 for the Eylea group) showed 89% of patients maintained ≥12-week dosing intervals compared to 93% through 1 year; 84% maintained ≥16-week dosing intervals compared to 89% maintaining a 16-week dosing interval through 1 year; 44% met the criteria for ≥20-week dosing intervals at week 96, including 17% and 27% who were eligible for 20- and 24-week dosing intervals, respectively; no cases of retinal vasculitis, occlusive retinitis or endophthalmitis | 7/29/2023 | Ocular |
Eli Lilly and Co., of Indianapolis | Tirzepatide | Glucose-dependent insulinotropic polypeptide receptor and glucagon-like peptide-1 receptor agonist | Obesity or overweight with weight-related co-morbidities, excluding type 2 diabetes | In the Surmount-3 study, after 12 weeks of intensive lifestyle intervention, patients achieved an additional 21.1% mean weight loss with tirzepatide for a total mean weight loss of 26.6% from study entry to week 84; in the Surmount-4 study, patients achieved 21.1% weight loss during a 36-week tirzepatide lead-in period and an additional 6.7% weight loss during a 52-week continued treatment period, for a total mean weight loss of 26% over 88 weeks | 7/27/2023 | Other/Miscellaneous |
Harmony Biosciences Holdings Inc., of Plymouth Meeting, Pa. | Wakix (pitolisant) | Histamine 3 receptor antagonist/inverse agonist | Excessive daytime sleepiness in Prader-Willi syndrome | Completed end-of-phase II meeting with the U.S. FDA; planning to initiate phase III study in the fourth quarter of 2023 | 7/20/2023 | Other/Miscellaneous |
Grifols SA, of Barcelona, Spain | Prolastin-C | Alpha1-proteinase inhibitor (human) (alpha1-PI) | Emphysema due to alpha-1-antitrypsin deficiency | Sparta study met its enrollment target of 339 patients; to evaluate the efficacy and safety of two separate dose regimens | 7/13/2023 | Respiratory |
Zambon SpA, of Milan | CMS I-neb | Adaptive aerosol delivery system of colistimethate sodium | Non-cystic fibrosis bronchiectasis | PROMIS-I trial met its primary and secondary endpoint; twice-daily dose resulted in a statistically significant reduction of pulmonary exacerbations over the course of the 12-month study; annual exacerbation rate (AER) was significantly lower in patients receiving CMS I-neb vs. placebo (p=0.00101); treatment effect was even larger in adherent subjects (43.5% reduction in exacerbations, p=0.00080); improvements compared to placebo with prolonged time to first exacerbation in the CMS I-neb group (p=0.00074); frequency of severe exacerbations was also reduced (p=0.00300); significantly improved quality of life (p=0.0055); P. aeruginosa density was significantly reduced in the treatment arm (p<0.00001) after 28 days | 7/18/2023 | Respiratory |
Zambon SpA, of Milan | CMS I-neb | Adaptive aerosol delivery system of colistimethate sodium | Non-cystic fibrosis bronchiectasis | PROMIS-II study prematurely terminated after consultation with the U.S. FDA; due to both the COVID-19 pandemic and the view of investigators, there was no longer equipoise after the strongly positive PROMIS-I results; overall PROMIS-II study did not meet its primary endpoint (p=0.979); reduction in P. aeruginosa sputum density observed in the CMS group was consistent with those obtained in PROMIS-I and the preceding phase II trial; statistically significant (p<0.00001 visit 3 and p<0.00001 for overall); 27% reduction in annualized exacerbation rate for CMS vs placebo treated patients; similar rate of adverse events in both Promis-I and Promis-II study | 7/18/2023 | Respiratory |
Achieve Life Sciences Inc., of Seattle | Cytisinicline | Plant-based alkaloid with a high binding affinity to the nicotinic acetylcholine receptor | Smoking cessation | ORCA-2 study showed primary outcome measure was significantly higher for cytisinicline compared with placebo for both the 6- and 12-week treatment durations; 25.3% vs. 4.4% of participants were abstinent during weeks 3 to 6 in 6-week cytisinicline treatment vs. placebo; cytisinicline treatment had 8 times higher odds, or likelihood, to have quit smoking (p<0.001); 32.6% vs. 7% of participants were abstinent during weeks 9 to 12 in 12-week treatment; cytisinicline treatment had 6 times higher odds, or likelihood, to have quit smoking (p<0.001); rapid and sustained decline in cravings and smoking urges compared with placebo during the first 6 weeks of treatment; continuous abstinence rates were also statistically significant through 6 months; 2.9% of subjects discontinued cytisinicline due to an adverse event; no drug-related serious adverse events occurred; results published in the Journal of the American Medical Association | 7/11/2023 | Toxicity and Intoxication |
ADC Therapeutics SA, of Lausanne, Switzerland | Zynlonta (loncastuximab tesirine) | B-lymphocyte antigen CD19 modulator | Diffuse large B-cell lymphoma | Data showed 7 patients completed treatment and 5 continue in follow-up; overall response rate by central review was 16/20 (80%); median duration of response was 8 months and the median progression-free survival was 8.3 months; grade ò3 treatment-emergent adverse events; no new safety signals | 8/30/23 | Cancer |
Akeso Inc., of Guangdong, China | Ivonescimab | PD-1/VEGF bispecific antibody | Locally advanced or metastatic squamous non-small-cell lung cancer | First patient dosed; to evaluate the clinical efficacy and assess the safety of ivonescimab compared with tislelizumab in combination with chemotherapy | 8/18/23 | Cancer |
Akeso Inc., of Guangdong, China | Ivonescimab | PD-1/VEGF bispecific antibody | PD-L1+ locally advanced or metastatic non-small-cell lung cancer | Completed patient enrollment in a head-to-head study compared with pembrolizumab | 8/29/23 | Cancer |
Aravive Inc., of Houston | Batiraxcept | Ultra-high affinity decoy protein that binds to GAS6 and thereby prevents AXL signaling | Platinum-resistant ovarian cancer | Top-line results of Axlerate-OC trial did not meet its primary endpoint of progression-free survival (PFS) in the prespecified subset of patients na‹ve to prior bevacizumab treatment; median PFS in the batiraxcept plus paclitaxel arm was 5.4 months compared to 5.4 months in the paclitaxel arm in the bevacizumab-na‹ve population; median PFS in the batiraxcept plus paclitaxel arm was 5.1 months compared to 5.5 months in the paclitaxel arm in the overall population; no new safety signals | 8/2/23 | Cancer |
Cullinan Oncology Inc., of Cambridge, Mass. | Zipalertinib (CLN-081/TAS-6417) | Oral EGFR inhibitor | Advanced or metastatic non-small-cell lung cancer | Launched Rezilient3 study to assess progression-free survival of zipalertinib plus chemotherapy vs. chemotherapy in adult patients | 8/3/23 | Cancer |
Eli Lilly and Co., of Indianapolis | Retevmo (selpercatinib) | RET kinase inhibitor | RET fusion-positive advanced or metastatic non-small-cell lung cancer | Top-line results of Libretto-431 study met its primary endpoint; statistically significant and clinically meaningful improvement in progression-free survival; consistent adverse events with previous studies | 8/4/23 | Cancer |
Eli Lilly and Co., of Indianapolis | Retevmo (selpercatinib) | RET kinase inhibitor | RET-mutant medullary thyroid cancer | Top-line results from the Libretto-531 study met its primary endpoint; statistically significant and clinically meaningful improvement in progression-free survival; consistent adverse event with previously reported studies | 8/22/23 | Cancer |
Exelixis Inc., of Alameda, Calif. | Cabometyx (cabozantinib) | Small-molecule inhibitor of the tyrosine kinases c-Met and VEGFR2, and also inhibits AXL and RET | Advanced pancreatic neuroendocrine tumors (pNET) or advanced extra-pancreatic neuroendocrine tumors | Independent data and safety monitoring board recommended to unblind and stop the phase III Cabinet pivotal trial early due to a dramatic improvement in efficacy observed at an interim analysis; no new safety signals | 8/24/23 | Cancer |
Genentech, a unit of Basel, Switzerland-based Roche Holding AG | Tiragolumab | Monoclonal antibody targeting TIGIT | PD-L1-high locally advanced or metastatic non-small-cell lung cancer | Second interim results of Skyscraper-01 study in combination with Tecentriq (atezolizumab) showed median overall survival estimates of 22.9 months vs. 16.7 months in the Tecentriq monotherapy arm; well-tolerated; no new safety signals | 8/23/23 | Cancer |
Ipsen SA, of Paris, and Exelixis Inc., of Alameda, Calif. | Cabometyx (cabozantinib) | Small-molecule inhibitor of the tyrosine kinases c-Met and VEGFR2, and also inhibits AXL and RET | Metastatic castration-resistant prostate cancer | Contact-02 study in combination with Tecentriq (atezolizumab, Roche Holding AG) met 1 of 2 primary endpoints; statistically significant improvement in progression-free survival (PFS) at the primary analysis; improvement of overall survival; data were immature and did not meet the threshold for statistical significance; no new safety signals | 8/21/23 | Cancer |
Merck & Co. Inc., of Kenilworth, N.J. | Welireg (belzutifan) | Small-molecule inhibitor of hypoxia-inducible factor (HIF)-2a | Advanced renal cell carcinoma | Welireg showed a statistically significant and clinically meaningful improvement in progression-free survival (PFS) compared to everolimus, based on a pre-specified interim analysis conducted by an independent data monitoring committee; statistically significant improvement in the key secondary endpoint of objective response rate; trend toward improvement in overall survival but did not reach statistical significance | 8/18/23 | Cancer |
Merck & Co. Inc., of Rahway, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 therapy | Recurrent or metastatic head and neck squamous cell carcinoma | LEAP-010 trial in combination with Lenvima showed a statistically significant improvement in PFS and ORR vs. Keytruda plus placebo in the first analysis; did not demonstrate an improvement in OS compared to Keytruda plus placebo at the second analysis; protocol-specified threshold for statistical significance for OS deemed to be low; study closed | 8/25/23 | Cancer |
Qilu Pharmaceutical Co. Ltd., of Jinan, China | Iruplinalkib | Orally active inhibitor of ALK and ROS1 tyrosine kinase | ALK-positive advanced non-small-cell lung cancer | Interim analysis from the independent data monitoring committee with median follow-up time for the iruplinalkib and crizotinib groups was 23.98 months and 24.54 months, respectively; primary endpoint PFS (per RECIST v1.1 by IRC) met the pre-defined criteria; median PFS of 27.70 months and for placebo 14.62 months; duration of response and control of intracranial disease were superior to those from the crizotinib group; consistent safety profile; good safety profile and no increased safety risk compared to crizotinib | 8/17/23 | Cancer |
Seagen Inc., of Bothell, Wash. | Tukysa (tucatinib) | HER2 tyrosine kinase inhibitor | Advanced or metastatic HER2-positive breast cancer | Study achieved primary endpoint of progression-free survival in combination with ado-trastuzumab emtansine (Kadcyla); overall survival data not mature; discontinuations due to adverse events in the combination arm of the trial and no new safety signals | 8/16/23 | Cancer |
Stellas Life Sciences Group Inc., of New York | Galinpepimut-S | WT1-targeting peptide immunotherapeutic | Acute myeloid leukemia | Independent data monitoring committee recommended that the trial continue without modifications; enrollment in the study will be completed during the fourth quarter of 2023; primary endpoint is overall survival; charter provides for periodic reviews for safety, efficacy and futility in addition to the interim and final analyses | 8/22/23 | Cancer |
Telix Pharmaceuticals Ltd., of Melbourne | TLX591-Cdx | Imaging agent | Imaging of prostate cancer | First patient dosed in the pivotal study in China | 8/10/23 | Cancer |
Urogen Pharma Ltd., of Princeton, N.J. | UGN-102 (mitomycin) | Sustained-release formulation of the chemotherapy mitomycin | Low-grade, intermediate-risk non-muscle invasive bladder cancer | Atlas study demonstrated superiority to transurethral resection of bladder tumor surgery (TURBT) with a 55% reduction of risk for recurrence, progression, or death in patients who received UGN-102; tumor-free complete response rate at 3 months was 65% for patients compared to 64% complete response rate at 3 months for patients who underwent TURBT; well-tolerated; results published in The Journal of Urology | 8/8/23 | Cancer |
Bridgebio Pharma Inc., of Palo Alto, Calif. | Acoramidis (AG-10) | Stabilizes tetrameric transthyretin | Transthyretin amyloid cardiomyopathy | Study met primary endpoints (win ratio of 1.8), with statistical significance (p<0.0001); 81% survival rate on acoramidis vs. 74% survival rate on placebo for an absolute risk reduction of 6.4% and a relative risk reduction of 25%; 50% higher rate of tafamidis use on the placebo arm relative to the treatment arm; treatment group experienced a 14.9% CV-related mortality rate vs. 21.3% CV-related mortality rate in the placebo group for relative risk reduction of 30% on CV-related mortality; highly statistically significant treatment effect (p<0.0001 in each case) at 30 months on change from baseline in each of NT-proBNP, KCCQ and 6MWD; 42% greater increase in serum TTR levels relative to placebo + tafamidis in exploratory post hoc analysis; well-tolerated with no safety findings | 8/27/23 | Cardiovascular |
Bristol Myers Squibb Co., of New York | Camzyos (mavacamten) | Myosin inhibitor | Symptomatic obstructive hypertrophic cardiomyopathy | Long-term-56-week analysis of VALOR-HCM LTE demonstrated sustained improvements across key study endpoints; 8.9% of patients in Camzyos and 19.2% in the placebo cross-over group at week 40 decided to proceed with septal reduction therapy (SRT) or were SRT-eligible; Camzyos demonstrated sustained reduction in peak resting LVOT gradient; proportion with NYHA class improvement of ò1 class was observed in 93% of patients at week 56 vs. 73% of patients from the placebo cross-over group at week 40; patient-reported 23-item Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-23 CSS) improved 14.1 point increase vs. 11.7 point increase for the placebo crossover group; sustained reduction across biomarkers of cardiac wall stress and myocardial injury including reduction in N-terminal pro brain natriuretic peptide (NT-proBNP); no new safety signals ; data published simultaneously in JAMA Cardiology | 8/28/23 | Cardiovascular |
Bristol Myers Squibb Co., of New York | Camzyos (mavacamten) | Myosin inhibitor | Symptomatic obstructive hypertrophic cardiomyopathy | Results from 120-week analysis of EXPLORER-LTE showed no new safety signals; 75.9% of patients improved by ò1 NYHA class from baseline at the start of the LTE study to week 120; sustained improvements from baseline at the start of the LTE study in echocardiographic parameters, including E/e? average and NT-proBNP; mean LVEF remained within the normal range at all study visits | 8/28/23 | Cardiovascular |
Bristol Myers Squibb Co., of New York, and Lianbio, of Shanghai, and Princeton, N.J. | Camzyos (mavacamten) | Myosin inhibitor | Symptomatic obstructive hypertrophic cardiomyopathy | EXPLORER-CN trial in Chinese patients showed improvement in Valsalva LVOT peak gradient, LVOT obstruction, clinical symptoms, health status, cardiac biomarkers, and cardiac structure; no new safety signals; resting LVEF remained stable in both groups throughout treatment; no participants in the study experienced decreases in LVEF <50 % that required dose interruption; data published simultaneously in JAMA Cardiology | 8/28/23 | Cardiovascular |
Daiichi Sankyo Co. Ltd., of Tokyo | Nilemdo (bempedoic acid) | Inhibits ATP citrate lyase | High-risk of cardiovascular events | Prespecified outcome of Clear study showed 20% ( p=0.0001) relative risk reduction (RRR) in the primary endpoint of a 4-component composite of major CV adverse events (MACE-4); total events analysis includes 17% RRR for the 3-component composite of major adverse cardiovascular events (MACE-3); subanalysis showed risk of new-onset diabetes (NOD) when treated with bempedoic acid over a median 3.4 years follow-up (25% for MACE-4 and 58% for MACE-3); bempedoic acid both lowered LDL-C and reduced the risk of CV events with greater absolute benefit for those with diabetes; bempedoic acid did not increase HbA1c levels or the incidence of NOD (11.1% with bempedoic acid vs. 11.5% with placebo) | 8/27/23 | Cardiovascular |
Lexicon Pharmaceuticals Inc., of The Woodlands, Texas | Sotagliflozin | Dual SGLT1 and SGLT2 inhibitor | Heart failure | Post-hoc analysis of SOLOIST-WHF study of sotagliflozin resulted in significant relative risk reductions of 50% for readmission for non-fatal heart failure events, composite of cardiovascular death and readmission for heart failure at 30 or 90 days following hospital discharge vs. placebo; results published in the Journal of the American College of Cardiology (JACC): Heart Failure | 8/8/23 | Cardiovascular |
Novartis AG, of Basel, Switzerland, and Alnylam Pharmaceutical Inc., of Cambridge, Mass. | Leqvio (inclisiran) | siRNA therapy | Atherosclerotic cardiovascular disease | Long-term Orion-8 open-label extension trial showed consistent low-density lipoprotein cholesterol (LDL-C) reduction beyond 6 years of treatment; 8/10 patients achieved target LDL-C threshold; favorable safety profile | 8/28/23 | Cardiovascular |
Novo Nordisk A/S, of Copenhagen, Denmark | Wegovy (semaglutide) | GLP-1 receptor agonist | Obesity | Trial achieved primary endpoint; statistically significant and superior reduction in major adverse cardiovascular events (MACE) of 20% with semaglutide 2.4 mg compared to placebo; safe and well-tolerated | 8/8/23 | Cardiovascular |
Novo Nordisk A/S, of Copenhagen, Denmark | Wegovy (semaglutide) | GLP-1 receptor agonist | Heart failure with preserved ejection fraction (HFpEF) and obesity | STEP HFpEF trial showed large reductions in heart failure-related symptoms, physical limitations and improvements in exercise function, and resulted in greater weight loss in adults compared with placebo at once-weekly semaglutide 2.4-mg; mean change in the KCCQ-CSS was a 16.6-point increase at 52 weeks with semaglutide 2.4-mg vs 8.7 points with placebo (p<0.001); change in body weight was 13.3% reduction with semaglutide 2.4-mg vs. 2.6% reduction with placebo (p<0.001); mean increase in 6-Minute Walking Distance (6MWD) of 21.5 metres at 52 weeks with semaglutide vs. 1.2 metres with placebo (p<0.001); reduced inflammation; fewer serious adverse events; published simultaneously in the New England Journal of Medicine | 8/25/23 | Cardiovascular/Other |
Minghui Pharmaceutical Inc., of Shanghai | MH-004 | Topical cream containing a JAK inhibitor | Mild to moderate atopic dermatitis | First patient enrolled; to evaluate the safety and efficacy of the topical cream in a larger patient population | 8/17/23 | Dermatologic |
Novartis AG, of Basel, Switzerland | Remibrutinib (LOU-064) | BTK inhibitor | Chronic spontaneous urticaria | Remix-1 and 2 studies met all primary and secondary endpoints; rapid, clinically meaningful improvements across urticaria disease activity scores (UAS7) at week 12; clinically meaningful and statistically significant improvements in disease activity; rapid improvement as early as 2 weeks after treatment initiation; final results and submission in 2024 for week 52 | 8/9/23 | Dermatologic |
Genexine Inc., of Seoul, South Korea | Eftansomatropin alfa (GX-H9/TJ-101) | Long-acting growth hormone | Pediatric growth hormone deficiency | Top-line results met its primary endpoint of annualized height velocity (AHV) at week 52 and non-inferior to Norditropin; mean AHV was 10.76 (cm/year) for eftansomatropin alfa vs. 10.28 (cm/year) for Norditropin (p-value <0.0001); well-tolerated; no drug discontinuation | 8/30/23 | Endocrine/metabolic |
Lib Therapeutics Inc., of Cincinnati | Lerodalcibep | Next-generation, small binding protein alternative to a monoclonal antibody, PCSK9 inhibitor | Heterozygous familial hypercholesterolemia | Monthly lerodalcibep achieved both co-primary endpoints; achieved a mean reduction in LDL-C of 58.6% at week 24 and 65% at the co-primary endpoint of weeks 22 and 24 in intent-to-treat (ITT) patients compared to placebo; lerodalcibep achieved a placebo adjusted mean (SE) reduction in LDL-C of 63.6% at week 24 and at the week 22/24 mean of 70.2% (p value <0.0001 for both) in per-protocol analysis patients; mean reduction in apolipoprotein B of 45.6% and median reduction in Lp(a) of 24% compared to patients treated with placebo were statistically significant (p<0.0001); well-tolerated; mild injection site adverse event; results simultaneously published in the European Heart Journal | 8/28/23 | Endocrine/metabolic |
Merck & Co. Inc., of Rahway, N.J. | MK-0616 | Once-daily oral PCSK9 inhibitor | Hypercholesterolemia | Initiated phase III study; primary objective of this study is to evaluate the efficacy of MK-0616 compared with placebo on the mean percent change from baseline in LDL cholesterol at week 24; planning to enroll 17,000 participants; results expected by the end of 2023 | 8/25/23 | Endocrine/Metabolic |
Theracosbio Inc., of Marlborough, Mass. | Brenzavvy (bexagliflozin) | Oral SGLT2 inhibitor | Type 2 diabetes | Brenzavvy improved glycemic control and decreased systolic blood pressure levels and fasting plasma glucose levels; clinically meaningful and statistically significant 1.09% reduction of HbA1c from baseline with a placebo-adjusted treatment effect of -0.53%, as well as a mean change of -2.82%; treatment with rescue medication (6 patients Brenzavvy and 32 placebo)reduction of 1.07% from baseline HbA1c and a placebo-corrected treatment effect of -0.67%; decrease in systolic blood pressure levels by 5.03 mm Hg with a placebo-adjusted reduction of 7.07 mm Hg; mean change reduction of 8.19 mm Hg in the high glycemic group; decrease in fasting plasma glucose levels by 45.2 mg dL-1 with a placebo-adjusted reduction of 24.3 mg dL-1; results published in Diabetes, Obesity and Metabolism | 8/4/23 | Endocrine/Metabolic |
Cymabay Therapeutics Inc., of Newark, Calif. | Seladelpar | PPAR-delta agonist | Primary biliary cholangitis | Initiated phase III study; planning to enroll 75 patients | 8/10/23 | Gastrointestinal |
Calliditas Therapeutics AB, of Stockholm | Nefecon (targeted-release budesonide) | Corticosteroids | Primary IgA nephropathy | Data published from the Neflgard study in The Lancet showed there was a 6.11 mL/min/1.73 m2 decline in estimated glomerular filtration rate after 2 years for patients taking Nefecon compared with a 12 mL/min/1.73 m2 decline in patients taking placebo (p<0.0001) | 8/15/23 | Genitourinary/Sexual Function |
Palatin Technologies Inc., of Cranbury, N.J. | Vyleesi (bremelanotide) | Melanocortin 4 receptor agonist | Premenopausal women with hypoactive sexual desire disorder | Completed enrollment of 193 patients to evaluate the efficacy and safety of subcutaneously injected bremelanotide | 8/8/23 | Genitourinary/Sexual Function |
Hutchmed Ltd., of Hong Kong | Sovleplenib | Syk inhibitor; small molecule | Primary immune thrombocytopenia | ESLIM-01 study met its primary endpoint of durable response rate and all secondary endpoints in adult patients; consistent safety profile | 8/20/23 | Hematologic |
Pharming Group NV, of Leiden, the Netherlands | Leniolisib | Oral, selective PI3K-delta inhibitor | Activated phosphoinositide 3-kinase delta syndrome | First patient enrolled; to evaluate safety, tolerability and efficacy of leniolisib in 3 patients 12 and older | 8/9/23 | Immune |
Atea Pharmaceuticals Inc., of Boston | Bemnifosbuvir | Nucleotide polymerase inhibitor targets the SARS-CoV-2 RNA polymerase (nsp12) | COVID-19 | Enrollment continued in phase III study; protocol amended to broaden eligibility criteria for high-risk patients and adapt to current COVID-19 environment; top-line results expected in mid-2024 | 8/8/23 | Infection |
Bavarian Nordic A/S, of Copenhagen | ABNCoV2 | VLP-based, non-adjuvanted COVID-19 booster vaccine | COVID-19 prophylaxis | Study met primary objective; post-vaccination showed well-tolerated; no serious adverse events | 8/31/23 | Infection |
Bavarian Nordic A/S, of Copenhagen, Denmark | PXVX-0317 | Virus-like particle-based chikungunya virus vaccine candidateÿ | Chikungunya virus infection | Top-line results met all the co-primary endpoints; highly immunogenic in healthy adolescents and adults up to day 22 post vaccination; strong induction of chikungunya neutralizing antibodies in 98% of vaccinees in the active group; rapid onset of protective levels of immunity; robust and durable responses; 86% of the subjects had seroprotective levels of neutralizing antibodies 6 months post vaccination; well-tolerated in adolescent and adult population; mild or moderate adverse events | 8/6/23 | Infection |
Bioxytran Inc., of Boston | Prolectin-M | Gal-3 antagonist | Mild to moderate COVID-19 | Initiated phase III study; planning to enroll 408 patients | 8/8/23 | Infection |
Valneva SE, of Saint Herblain, France | VLA-1553 | Single-shot chikungunya vaccine candidate | Chikungunya virus infection | Independent safety committee found no safety concerns; adverse events were mild or moderate and resolved within 3 days; favorable safety profile; immunogenicity data expected in November 2023 | 8/28/23 | Infection |
Lyra Therapeutics Inc., of Watertown, Mass. | LYR-210 | Bioresorbable nasal implant | Chronic rhinosinusitis | Enlighten study fully enrolled; top-line results expected in the first half of 2024 | 8/29/23 | Inflammatory |
Fibrogen Inc., of San Francisco | Pamrevlumab | Monoclonal antibody targeting connective tissue growth factor | Duchenne muscular dystrophy | Top-line results of Lelantos-2 study did not meet the primary endpoint of change in the North Star Ambulatory Assessment (NSAA) total score from baseline to week 52 (p=0.5553); secondary endpoint measured by change from baseline at week 52 in 4-stair climb velocity, 10-meter walk/run test, time to stand, time to loss of ambulation, and proportion of patients with greater than 10 seconds in the 10-meter walk/run test also not met; safe and well-tolerated; mild or moderate adverse events; treatment-emergent serious adverse events were observed in 8.3% of patients in the pamrevlumab group and 2.8% of patients in the placebo | 8/29/23 | Musculoskeletal |
Stempeutics Research Pvt Ltd., of Bengaluru, India | Stempeucel-OA | Bone marrow-derived, cultured pooled, allogeneic mesenchymal stromal cells | Knee osteoarthritis | Results showed single injection significantly improved WOMAC total score compared with the placebo group at 6 and 12 months; significantly improved WOMAC pain, stiffness, and physical function sub-scores as well as VAS scores at 6 and 12 months; MRI T2 mapping showed no worsening of deep cartilage in the medial femorotibial compartment of the knee at 12-month follow-up; published in the American Journal of Sports Medicine | 8/23/23 | Musculoskeletal |
Tonix Pharmaceuticals Holding Corp., of Chatham, N.J. | TNX-102 SL | Cyclobenzaprine HCl sublingual tablets | Fibromyalgia | Completed enrollment of Resilent trial; total of 457 participants were randomized; top-line data expected in the fourth quarter of 2023 | 8/1/23 | Musculoskeletal |
Mitsubishi Tanabe Pharma America Inc., of Jersey City, N.J. | Radicava (edaravone, oral suspension) | Neuroprotectant; antioxidant agent; free radical scavenger; oral suspension | Amyotrophic lateral sclerosis | Post-hoc analysis showed 53% relative risk reduction of the cumulative occurrence of milestone events vs. placebo group (p=0.02); survival composite endpoint analyses at week 24 (p<0.01) and week 48 (p=0.02); no deaths during the double-blind period | 8/17/23 | Musculoskeletal |
Amneal Pharmaceuticals Inc., of Bridgewater, N.J. | IPX-203 | Extended-release oral formulation of carbidopa/levodopa | Parkinson's disease | Study met its primary and secondary endpoints; statistically significant improvement in daily ?Good On? time compared to optimized immediate-release carbidopa/levodopa; good-on time per dose increased by 1.55 hours with IPX-203 compared with immediate-release carbidopa-levodopa; well-tolerated with treatment related adverse events; results published in JAMA Neurology | 8/24/23 | Neurology/Psychiatric |
Medincell SA, of Jacou, France | F-14 (mdc-CWM) | Sustained-release formulation of celecoxib | Pain and inflammation after total knee replacement surgery | Completed patient enrollment; 151 patients enrolled; top-line data expected in the first quarter of 2024 | 8/30/23 | Neurology/Psychiatric |
Neurocrine Biosciences Inc., of San Diego | Ingrezza (valbenazine) | VMAT2 inhibitor | Chorea associated with Huntington disease | Exploratory analysis of Kinect-HD study showed greater placebo-adjusted improvement in chorea with Ingrezza at 40 mg at week 2, and greater efficacy compared to placebo was sustained through week 12; percentage of participants rated much improved or very much improved by CGI-C and PGI-C than placebo | 8/28/23 | Neurology/Psychiatric |
Neurocrine Biosciences Inc., of San Diego | Ingrezza (valbenazine) | VMAT2 inhibitor | Tardive dyskinesia (TD) | Data analysis of Kinect-4 study showed mean Abnormal Involuntary Movement Scale (AIMS) total score at baseline was 14.6 and decreased over 48 weeks and as aware/no distress at 48 weeks; patient-reported TD impact as measured by the Trajectories of Tardive Dyskinesia Impact Scale scores decreased from 16.5 at baseline to 6 at week 48 by the end of the Ingrezza treatment period | 8/29/23 | Neurology/Psychiatric |
Neuroderm Ltd., of Rehovot, Israel, and Mitsubishi Tanabe Pharma America Inc., of Jersey City, N.J. | ND-0612 | Continuous delivery of carbidopa/levodopa (CD/LD) via subcutaneous infusion | Parkinson's disease experiencing motor fluctuations | Study met its primary endpoint and the first 4 secondary endpoints; favorable efficacy over IR-LD/CD with a statistically significant increase (p<0.0001) of 1.72 hours in ON time without troublesome dyskinesia; reduction of OFF time (p<0.0001); reached statistical significance were the MDS-Unified Parkinson's Disease Rating Scale Part II score (MDS-UPDRS motor experiences of daily living sub-score) (p<0.0001), Patient Global Impression of Change (PGIC) (p<0.0001) and the Clinical Global Impression of Improvement (CGI-I) (p<0.0001); consistent with the well-established safety profile of oral standard of care; infusion site reactions (57% for ND0612 vs. 43% for oral immediate-release-LD/CD; 5.5% discontinued due to adverse events compared to 6.1% and 3.1%, respectively, of study participants in the oral LD/CD groups | 8/29/23 | Neurology/Psychiatric |
Pharma Two B Ltd., of Rehovot, Israel | P2B-001 | Fixed-dose combination of rasagiline mesilate and pramipexole hydrochloride | Parkinson's disease | Subgroup analysis showed greater symptomatic benefit assessed by Total-UPDRS; efficacy and safety profile of P2B-001 was not significantly different in younger vs. older patients; statistically significant greater benefit in Activities of Daily Living (UPDRS part II) vs. components; superior to extended-release pramipexole across all subgroups on ESS scores; significantly less daytime sleepiness compared to extended-release pramipexole at 12 weeks (pó0.001) in full-study cohort | 8/28/23 | Neurology/Psychiatric |
Relmada Therapeutics Inc., of Coral Gables, Fla. | REL-1017 | NMDA receptor channel blocker; chemical molecule | Major depressive disorder | First patient dosed in Relight study; planned enrollment of 300 patients; primary endpoint is the change in the MADRS total score from baseline to day 28 for REL-1017 compared to placebo | 8/23/23 | Neurology/Psychiatric |
Reviva Pharmaceuticals Holdings Inc., of Cupertino, Calif. | Brilaroxazine | Serotonin and dopamine receptor modulator | Schizophrenia | Completed enrollment of 402 patients in Recover study; top-line efficacy and safety data expected in October 2023; completion of 1-year open-label extension study expected in the third quarter of 2024 | 8/17/23 | Neurology/Psychiatric |
Vistagen Therapeutics Inc., of South San Francisco | Fasedienol (PH-94B) | Rapid-onset pherine nasal spray | Social anxiety disorder | Top-line results from Palisade-2 study met its primary and secondary endpoints; statistically significant rapid-onset reduction in patient-reported subjective units of distress scale (SUDS) score compared to placebo in a public speaking challenge (p=0.015); statistically significant reduction in proportion of responders compared to placebo as measured by the CGI-I scale (p=0.033); trial met an exploratory endpoint of the difference in the proportion of patient-assessed responders between fasedienol and placebo as measured by the Patient?s Global Impression of Change (PGI-C) scale; responders were self-rated very much less anxious or much less anxious, with 40.6% of fasedienol-treated patients rated as responders as compared to 18.6% of those treated with placebo (p=0.003); 20-point improvement in patient-assessed SUDS score from baseline (visit 2) to treatment (visit 3); 35.7% of fasedienol-treated patients statistically significant and clinically meaningful improvement in SUDS score as compared to 18.6% (n=71) in the placebo-treated group (p=0.020); well-tolerated; no severe or serious adverse events; mild or moderate adverse events |
8/7/23 | Neurology/Psychiatric |
Oculis SA, of Lausanne, Switzerland | OCS-01 | High-concentration, topical formulation of dexamethasone | Inflammation and pain following ocular surgery | Study met both hierarchical primary efficacy endpoints of the absence of inflammation at day 15 and the absence of pain at day 4, with robust statistical significance; the percentage of eyes with zero inflammation (absence of anterior chamber cells, score = 0) was statistically significantly greater with OCS-01 QD compared with vehicle at day 15 (57.2% vs. 24% p<0.0001); percentage of eyes with zero pain (absence of pain, score = 0) was statistically significantly greater with OCS-01 QD compared with vehicle at day 4 (OCS-01, 75.5% vs. vehicle, 52%, p<0.0001); no meaningful difference in intraocular pressure (IOP) between treatment groups with a mean change from baseline to day 15 of -0.90 mmHg in both the OCS-01 group and the vehicle group; well-tolerated with a favorable safety profile | 8/8/23 | Ocular |
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Aflibercept | Fusion protein with greater affinity towards VEGF-A | Wet age-related macular degeneration | Top-line data showed 78% maintained ò12-week dosing intervals throughout the 2-year study period, compared to 83% throughout the first year of study (48 weeks); 71% met the extension criteria for even longer dosing intervals, including 47% for ò20-week intervals and 28% for 24-week intervals; 70% maintained ò16-week dosing intervals throughout the two-year study period; 78% were eligible for ò16-week dosing, with 53% eligible for ò20-dosing week intervals at the end of 2 year dosing | 8/10/23 | Ocular |
Sandoz, a unit of Basel, Switzerland-based Novartis AG | SOK-583A1 | Biosimilar of aflibercept | Neovascular age-related macular degeneration | In the Mylight study, mean change of best corrected visual acuity from baseline to week 8 was similar between SOK-583A1 and the reference product | 8/15/23 | Ocular |
Tarsier Pharma Ltd., of Tel Aviv, Israel | TRS-01 | Eye drop formulation of dazdotuftide | Noninfectious anterior uveitis glaucoma | Study did not meet primary endpoint of ACC=0 at week 4 compared to steroids active control; steroid active control showed 2-fold higher rate of rebound (relapse of inflammation within 2 weeks) compared to the TRS-01 treatment arm and up to 6-fold higher rate of rebound compared to TRS-01 treatment arm in a pre-specified subgroup; 2.6-fold higher rate in the steroid active control arm presented a clinically meaningful IOP elevation after only 4 weeks of treatment compared to the TRS-01 arm; favorable safety and tolerability profile | 8/23/23 | Ocular |
4SC AG, of Planegg-Martinsried, Germany | Resminostat (Kinselby) | Orally administered class?I, IIb and IV histone deacetylase (HDAC) inhibitor | Cutaneous T-cell lymphoma | RESMAIN study showed statistically significant improvement in progression-free survival (PFS) of 97.6% compared to placebo, with a risk reduction of 38% in recently announced headline trial results (median PFS: 8.3 months vs. 4.2 months; p=0.015); median time to next treatment (median TTNT) vs. placebo showed a significant improvement of 8.8 months compared to 4.2 months (p= 0.002); mild to moderate side effects; manageable and reversible safety profile; clinically meaningful improvement in median total PFS of 24.3 months, compared to 14.9 months in additional analysis; significantly delayed the development of new, or worsening of existing, skin tumors | 9/25/2023 | Cancer |
Abbvie Inc., of North Chicago | Venclexta/Venclyxto (venetoclax) | B-cell lymphoma-2 inhibtor | T(11;14)-positive relapsed or refractory multiple myeloma after 2 or more treatments | In the Canova study, venetoclax plus dexamethasone (Vendex) produced a progression-free survival of 9.9 months compared to 5.8 months for pomalidomide plus dexamethasone (Pomdex) (HR=0.823, p=0.237); overall response rate was 62% for Vendex compared to 35% for Pomdex (p<0.001); median overall survival was 32.4 months in Vendex compared to 24.5 months for Pomdex (HR=0.697, p=0.067) | 9/29/2023 | Cancer |
Ascletis Pharma Inc., of Hangzhou, China | ASC-40/Denifanstat | Oral FASN inhibitor | Recurrent glioblastoma | Completed enrollment of 120 patients | 9/26/2023 | Cancer |
Asieris Pharmaceuticals Co. Ltd., of Shanghai, and Photocure ASA, of Oslo, Norway | APL-1702 (Cevira) | Photodynamic drug-device combination | High-grade squamous intraepithelial lesions | Study completed and met its primary endpoint | 9/20/2023 | Cancer |
Astrazeneca plc, of Cambridge, U.K., and Daiichi Sankyo Co. Ltd., of Tokyo | Datopotamab deruxtecan (Dato-DXd) | TROP2-directed DXd antibody-drug conjugate | Inoperable or metastatic hormone receptor (HR)-positive, HER2-low or negative (IHC 0, IHC 1+ or IHC 2+/ISH-) breast cancer | Tropion-Breast01 phase III study showed statistically significant and clinically meaningful improvement for the primary endpoint of progression-free survival compared to investigator’s choice of chemotherapy in patients; overall survival improved vs. chemotherapy; consistent safety profile with previous studies | 9/22/2023 | Cancer |
Bristol Myers Squibb Co., of New York | Opdivo (nivolumab) | PD-1 immune checkpoint inhibitor | Resectable non-small-cell lung cancer | Checkmate-77T study met its primary endpoint of improved event-free survival (EFS) as assessed by Blinded Independent Central Review (BICR); statistically significant and clinically meaningful improvement in EFS compared to neoadjuvant chemotherapy and placebo followed by surgery and adjuvant placebo; consistent safety profile | 9/22/2023 | Cancer |
Genmab A/S., of Copenhagen, Denmark and Seagen Inc., of Bothell, Wash. | Tivdak (tisotumab vedotin-tftv) | ADC composed of human monoclonal antibody targeted towards tissue factor (TF) with a protease-cleavable linker covalently attached to microtubule-disrupting agent monomethyl auristatin E (MMAE) | Recurrent or metastatic cervical cancer | Study met its primary endpoint of overall survival compared with chemotherapy alone; statistical significance in secondary endpoints of investigator-assessed progression-free survival and objective response rate; no new safety signals | 9/4/2023 | Cancer |
Hutchmed Ltd., of Hong Kong | Savolitinib (Orpathys) | Selective MET tyrosine kinase inhibitor | Mesenchymal epithelial transition factor (MET) exon 14 skipping alteration non-small-cell lung cancer | Data showed objective response rate was 60.7%; disease control rate was 95.2%; median follow-up of 11.1 months and median progression free survival was 13.8 months; median duration of response and overall survival not reached; no new safety signals | 9/12/2023 | Cancer |
Janssen Pharmaceutical Co., a unit of New Brunswick, N.J.-based Johnson & Johnson | Rybrevant (amivantamab-vmjw) | Bispecific antibody targeting EGFR and MET | First-line locally advanced or metastatic EGFR-mutated non-small-cell lung cancer | The phase III Mariposa study comparing Rybrevant plus lazertinib to osimertinib met its primary endpoint of improvement in progression-free survival; interim overall survival data trended to favoring Rybrevant plus lazertinib over osimertinib; data to be presented at an upcoming scientific congress | 9/28/2023 | Cancer |
Janssen Pharmaceutical Cos., a unit of New Brunswick, N.J.-based Johnson & Johnson | Rybrevant (amivantamab-vmjw) | Fully human bispecific antibody targeting EGFR and MET | Locally advanced or metastatic EGFR exon 19 deletions or L858R substitution non-small-cell lung cancer | MARIPOSA-2 study of Rybrevant and mesenchymal-epithelial transition, with and without lazertinib, and combined with chemotherapy met dual primary endpoint; statistically significant and clinically meaningful improvement in progression-free survival vs. chemotherapy alone in both experimental treatment arms in patients after disease progression on or after osimertinib; no new safety signals | 9/6/2023 | Cancer |
Merck & Co. Inc., of Rahway, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 therapy | Metastatic, nonsquamous non-small-cell lung cancer | Leap-006 trial in combination with Lenvima did not meet its dual primary endpoints of overall survival (OS) and progression-free survival (PFS) compared to Keytruda with chemotherapy; interim analyses did not demonstrate a statistically significant improvement in PFS or objective response rate; consistent safety profile with previous studies | 9/22/2023 | Cancer |
Merck & Co. Inc., of Rahway, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 therapy | Metastatic, nonsquamous non-small-cell lung cancer | Leap-008 study in combination with Lenvima did not meet its dual primary endpoints of OS and PFS vs. docetaxel; consistent safety profile with previous studies | 9/22/2023 | Cancer |
Merck & Co. Inc., of Rahway, N.J., and Seagen Inc., of Bothell, Wash. | Padcev (enfortumab vedotin) and Keytruda (pembrolizumab) | Antibody-drug conjugate targeting nectin-4 and anti-PD-1 therapy | Locally advanced or metastatic urothelial cancer | Top-line results of Keynote-A39 study met its dual primary endpoints of overall survival (OS) and progression-free survival (PFS); statistically significant and clinically meaningful improvement vs. chemotherapy in patients; statistically significant improvement in overall response rate; consistent safety profile with previous studies | 9/22/2023 | Cancer |
Novartis AG, of Basel, Switzerland | Lutathera ((177Lu) oxodotreotide) | Radioligand therapy | Somatostatin receptor (SSTR)-positive, grade 2 and 3, advanced gastroenteropancreatic neuroendocrine tumors | Study met its primary and secondary endpoints in combination with long-acting octreotide; significant improvement in progression-free survival and objective response rate; no new or unexpected safety findings | 9/25/2023 | Cancer |
Ose Immunotherapeutics SA, of Nantes, France | Tedopi | Cancer vaccine | Advanced or metastatic non-small-cell lung cancer | Tedopi showed statistically significant overall survival (reduced risk of death by 41%); 44.4% overall survival rate at 1 year with Tedopi vs. 27.5% with chemotherapy; clinically meaningful gain in median overall survival of 3.6 months in Tedopi arm with Tedopi OS at 11.1 months vs. 7.5 months for SoC (p=0.017); post progression survival was also significantly longer in the Tedopi arm (7.7 months vs. 4.6 months; p=0.004); significantly longer ECOG performance status (maintained general health condition with time to ECOG deterioration, 9 months vs. 3.3 months; p=0.006); better quality of life was observed with Tedopi (p= 0.04); (Global health status: p=0.045; Role Functioning: p=0.025); good tolerance profile; grade 3-5 severe adverse events (Tedopi 38% vs SoC 68%, p<0.001); no treatment-related adverse events; results published in the Annals of Oncology | 9/11/2023 | Cancer |
Roche Group, of Basel, Switzerland | Alecensa (alectinib) | Highly selective, central nervous system-active, oral medicine; ALK inhibitor | ALK-positive early-stage lung cancer | Study met its primary endpoint of disease-free survival (DFS) at a prespecified interim analysis; statistically significant and clinically meaningful improvement in DFS; overall survival data immature; no unexpected safety findings | 9/1/2023 | Cancer |
Sagimet Biosciences Inc., of San Mateo, Calif., and Ascletis Bioscience Co. Ltd., of Hangzhou, China | Denifanstat | Fatty acid synthase inhibitor | Recurrent glioblastoma | Completed enrollment in the 120-patient study testing denifanstat plus bevacizumab | 9/27/2023 | Cancer |
Veru Inc., of Miami | Enobosarm | Selective androgen receptor targeting agonist | Metastatic AR+ER+HER2- breast cancer (3rd-line or greater) | Discontinued ARTEST study to focus the clinical development of enobosarm therapy earlier in the treatment sequence, the second-line metastatic setting, for AR+ER+HER2- metastatic breast cancer in the phase III ENABLAR-2; well-tolerated without masculinizing adverse events or increases in hematocrit; overall response rates of 12.5% in the enobosarm group in a heavily pretreated population vs. no responses in the standard of care active control arm; overall response rate was 20% for enobosarm monotherapy vs. 0% for standard-of-care active control in patients who had ?3 lines of prior endocrine therapy in the metastatic setting | 9/11/2023 | Cancer |
Cytokinetics Inc., of South San Francisco | CK-274 (aficamten) | Selective, small-molecule cardiac myosin inhibitor | Symptomatic non-obstructive hypertrophic cardiomyopathy | Initiated enrollment for ACACIA-HCM phase III study | 9/6/2023 | Cardiovascular |
Dalcor Pharmaceuticals Inc., of Montreal | Dalcetrapib | Cholesterol ester transfer protein inhibitor | Reduce the occurrence of fatal and non-fatal myocardial infarction | Planning to conduct Dal-GenE-2 confirmatory trial in North America under a special protocol assessment agreement with the U.S. FDA; trial is expected to start in the third quarter of 2023 and interim efficacy analysis in 2026 | 9/13/2023 | Cardiovascular |
Janssen Pharmaceutical Cos. of Johnson & Johnson, of Beerse, Belgium | Macitentan (75 mg) | Dual endothelin receptor A and B antagonist | Chronic thromboembolic pulmonary hypertension | Decided to stop phase III Maciteph study due to futility after reviewing pre-planned interim analysis by independent data monitoring committee; no new safety signals | 9/6/2023 | Cardiovascular |
Merck & Co. Inc., of Rahway, N.J. | Sotatercept | Activin signaling inhibitor | Pulmonary arterial hypertension | Exploratory post-hoc analysis of Stellar study showed improvements from baseline in mean pulmonary arterial pressure, pulmonary vascular resistance, mean right atrial pressure and mixed venous oxygen saturation as well as cardiac efficiency; improvements in the ratio of tricuspid annular plane systolic excursion to systolic pulmonary artery pressure; simultaneous publication in the European Respiratory Journal | 9/11/2023 | Cardiovascular |
Merck & Co. Inc., of Rahway, N.J. | Sotatercept | Activin signaling inhibitor | Pulmonary arterial hypertension | Sotatercept was well-tolerated and the safety profile was similar to previous studies in SOTERIA open-label extension study; improvements in clinical efficacy measures measured at week 24 in SOTERIA were maintained at 1 year during the open-label period; mean changes from baseline at week 24 in 6MWD and NT-proBNP were largely maintained at 1 year; prostacyclin dose decreased in participants on infusion prostacyclin; participants had discontinued prostacyclins entirely; simultaneous publication in the European Respiratory Journal | 9/11/2023 | Cardiovascular |
Arcutis Biotherapeutics Inc., of Westlake Village, Calif. | Roflumilast | Topical PDE4 inhibitor | Atopic dermatitis | Integument-OLE long-term open-label study was well-tolerated, with no new safety signals up to 56 weeks in duration; efficacy maintained and improved; 61.5% and 66.2% of participants who rolled over from the roflumilast cream arm in Integument-1 or -2 demonstrated a 75% improvement from baseline in Eczema Area and Severity Index (EASI-75) after 28 weeks and 56 weeks, respectively; 46.1% and 51% of patients who rolled over from the roflumilast cream treatment arm in Integument-1 or -2 achieved IGA success at Week 28 and Week 56, respectively | 9/7/2023 | Dermatologic |
Arcutis Biotherapeutics Inc., of Westlake Village, Calif. | Roflumilast | Topical PDE4 inhibitor | Mild to moderate atopic dermatitis in children ages 2 to 5 years | Integument-ped study met its primary endpoint and all secondary endpoints; 25.4% of children treated with roflumilast cream 0.05% achieved IGA success, validated Investigator Global Assessment – Atopic Dermatitis (vIGA-AD) score of clear or almost clear plus a 2-grade improvement from baseline at week 4 compared to 10.7% treated with vehicle (p<0.0001); 39.4% of children treated with roflumilast cream 0.05% achieved a 75% improvement in EASI-75 at week 4 compared to 20.6% treated with vehicle (p<0.0001); 35.3% of children treated with roflumilast cream achieved a 4-point reduction in Worst Itch Numeric Scale (WI-NRS) at week 4 (vs. 18% for vehicle-treated subjects [nominal P=0.0002]); well-tolerated; low adverse events; ?3% of subjects in either arm was upper respiratory tract infection | 9/19/2023 | Dermatologic |
Vericel Corp., of Cambridge, Mass. | Nexobrid (anacaulase-bcdb) | Botanical drug product containing proteolytic enzymes | Deep thermal burns | Data from the Detect study published in the Journal of Burn Care & Research showed more than 93% of the patients treated with Nexobrid achieved complete eschar removal following one application compared with 4% in the gel vehicle arm (p<0.0001) | 9/28/2023 | Dermatologic |
Ascendis Pharma A/S, of Copenhagen, Denmark | Transcon PTH (palopegteriparatide) | Parathyroid hormone replacement therapy; administered once daily | Growth hormone deficiency | Enlighten trial concluded with 81 participants completing treatment based on their physician’s determination that treatment for pediatric was no longer required; 59% met or exceeded their average parental height standard deviation score (SDS), with mean Transcon hGH treatment duration of 3.2 years; safe and well-tolerated; no adverse events led to discontinuation of the study treatment | 9/23/2023 | Endocrine/Metabolic |
Crinetics Pharmaceuticals Inc., of San Diego | Paltusotine | Nonpeptide SST2 receptor agonist | Acromegaly | Study met primary and all secondary endpoints; statistical significance (p<0.0001) on the primary endpoint; proportion of participants taking paltusotine (83%) who maintained an insulin-like growth factor 1 (IGF-1) level ? 1 times the upper limit of normal (xULN) compared to those taking placebo (4%); change from baseline in IGF-1 level (p<0.0001); change from baseline in Acromegaly Symptoms Diary total score (p=0.02); proportion of participants who maintained a growth hormone level of <1 ng/mL (p=0.0003); well-tolerated; no serious or severe adverse events | 9/10/2023 | Endocrine/metabolic |
Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | Olezarsen (IONIS-APOCIII-LRx) | Antisense drug designed to inhibit production of apoC-III | Familial chylomicronemia syndrome | Top-line results of Balance study met its primary efficacy endpoint with a statistically significant reduction in triglyceride (TG) levels with the olezarsen 80-mg monthly dose at 6 months compared to placebo (p=0.0009); triglyceride lowering continued to improve at 12 months; 80-mg showed a 100% reduction in acute pancreatitis events compared to placebo and >75% reduction in apoC-III; substantial reduction in pancreatitis; 50-mg dose did not reach statistical significance at 6 months on the primary endpoint of triglyceride lowering (p=0.0775); favorable safety and tolerability profile; mild in severity adverse events; low incidence of injection site reactions; no hepatic or renal toxicity events; no clinically meaningful platelet reductions; 1 death not related to study drug | 9/26/2023 | Endocrine/Metabolic |
Neurocrine Biosciences Inc., of San Diego | Crinecerfont | Nonsteroidal corticotropin-releasing factor type 1 receptor antagonist | Classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency | Top-line data met its primary and secondary endpoints; statistically significant percent reduction in daily glucocorticoid (GC) dose vs. placebo while maintaining androgen control (p-value <0.0001); statistically significant decrease in androstenedione at week 4 vs. placebo (p-value <0.0001); 63% of patients on crinecerfont achieved a reduction to a physiologic GC dose vs. approximately 18% on placebo (p-value <0.0001) at week 24; well-tolerated; few serious adverse events, with none assessed as related to crinecerfont | 9/12/2023 | Endocrine/Metabolic |
Stealth Biotherapeutics Corp., of Boston | Elamipretide | Small mitochondrially targeted tetrapeptide | Primary mitochondrial myopathy | Achieved target enrollment and study will continue to enroll through month-end to allow for participation of additional interested patients; to assess efficacy and safety of single daily subcutaneous administration | 9/11/2023 | Endocrine/Metabolic |
Abbvie Inc., of North Chicago | Skyrizi (risankizumab) | Interleukin-23 inhibitor that selectively blocks IL-23 by binding to its p19 subunit | Moderately to severely active Crohn's disease | Top-line results of risankizumab met both primary and secondary endpoints; non-inferiority for clinical remission (Crohn's Disease Activity Index [CDAI]) at week 24 and superiority of endoscopic remission at week 48 vs. ustekinumab (remission rates of 59% vs. 40%); endoscopic remission (SES-CD ?4 and at least a 2-point reduction vs. baseline and no sub-score greater than 1 in any individual component) at week 48 met superiority of risankizumab vs. ustekinumab; remission rates were 32% in risankizumab group and 16% in ustekinumab group (p <0.0001); consistent safety profile; no new safety risks | 9/12/2023 | Gastrointestinal |
Cymabay Therapeutics Inc., of Newark, Calif. | Seladelpar | PPAR-delta agonist | Primary biliary cholangitis | Top-line results showed Seladelpar achieved primary and all key secondary endpoints; total of 61.7% of patients on seladelpar 10-mg met the primary composite endpoint related to serum alkaline phosphatase and bilirubin at 12 months vs. 20% on placebo (n=65; p<0.0001); anti-cholestatic effect of seladelpar was supported by the normalization of alkaline phosphatase at 12 months (key secondary endpoint) in 25% of patients on seladelpar vs. 0 on placebo (p<0.0001); least-squares mean percent reduction in alkaline phosphatase at 12 months was 42.4% in the seladelpar group vs. 4.3% in the placebo group (p<0.0001); statistically significant reduction in pruritus, or itch, (key secondary endpoint) after 6 months of treatment; Seladelpar-treated patients with a baseline Numerical Rating Scale (NRS) ?4 (moderate to severe pruritus) had a least-square mean reduction of 3.2 points in pruritus NRS compared to 1.7 points for patients in the placebo group (n=23; p<0.005); treatment-emergent adverse events, serious adverse events, and patient discontinuations were generally balanced across the treatment and placebo arms; favorable tolerability | 9/7/2023 | Gastrointestinal |
Cymabay Therapeutics Inc., of Newark, Calif. | Seladelpar | PPAR-delta agonist | Cirrhosis due to primary biliary cholangitis | Initiated Affirm study; planning to enroll 192 patients | 9/21/2023 | Gastrointestinal |
Eiger Biopharmaceuticals Inc., of Palo Alto, Calif. | Peginterferon lambda | Type III interferon | Chronic hepatitis delta | Decided to discontinue LIMT-2 study based on the recommendation of data safety monitoring board; observations of 4 patients with hepatobiliary events that resulted in liver decompensation | 9/12/2023 | Gastrointestinal |
Calliditas Therapeutics AB, of Stockholm | Kinpeygo | Oral version of the corticosteroid budesonide | Primary IgA nephropathy | Biomarker and subgroup analyses of the Neflgard study showed patients treated with Nefecon had a statistically significant decrease in IgA-IC levels compared to patients who received placebo at 3-, 6- and 9-months post randomization; subgroup analysis of 83 Asian patients and 275 white patients showed Nefecon preserved kidney function in both subgroups compared to placebo subgroups | 9/29/2023 | Genitourinary/Sexual Function |
Travere Therapeutics Inc., of San Diego | Filspari (sparsentan) | Endothelin and angiotensin II receptor antagonist | IgA nephropathy | Filspari achieved a clinically meaningful difference vs. irbesartan in eGFR total slope (p= 0.058); eGFR chronic slope (p=0.037); well-tolerated; overall safety profile in the study has been consistent between treatment groups; largest sustained reduction in proteinuria; slowest rates of eGFR decline in a controlled study of IgAN patients | 9/21/2023 | Genitourinary/Sexual Function |
Urovant Sciences Inc., a unit of Sumitomo Dainippon Pharma Co. Ltd., of Osaka, Japan | Gemtesa (vibegron) | Beta-3 adrenergic agonist | Overactive bladder symptoms receiving pharmacological therapy for benign prostatic hyperplasia | Study met its co-primary endpoints and all secondary endpoints at week 12 compared to placebo; statistically significant reduction in the key secondary endpoint of average number of nocturia episodes per night compared to placebo (p=0.0015); statistically significant reductions from baseline compared to placebo in the average number of urge urinary incontinence episodes per day (p=0.0034) and International Prostate Symptom Storage score (p<0.0001); statistically significant increase was seen in the average volume voided per micturition (p<0.0001); well-tolerated; consistent safety profile with no new safety signals compared to prior OAB studies | 9/11/2023 | Genitourinary/Sexual Function |
Remegen Co. Ltd., of Yantai, China | Telitacicept (RC-18) | B-cell lymphocyte stimulator/proliferation inducing ligand dual-target fusion protein | Rheumatoid arthritis | At week 24 of the 479-patient study, the 160-mg dose of telitacicept plus methotrexate had a significantly higher ACR20 response rate compared to patients who received methotrexate monotherapy | 9/26/2023 | Immune |
Cantex Pharmaceuticals Inc., of Weston, Fla. | Azeliragon | Receptor for advanced glycation end products inhibitor | Acute kidney injury and other life-threatening complications in patients hospitalized for COVID-19 | Started a study comparing azeliragon to placebo | 9/27/2023 | Infection |
Insmed Inc., of Bridgewater, N.J. | Arikayce | Amikacin liposome inhalation suspension | Newly diagnosed or recurrent nontuberculous mycobacterial (NTM) lung infection | Top-line results showed Arikayce achieved primary and secondary endpoints; statistically significantly higher culture conversion rates at month 7 vs patients in the comparator arm (78.8% vs. 47.1%, p=0.0010); 43.8% of patients achieved an improvement in Quality of Life – Bronchiectasis (QOL-B) respiratory score above the estimated meaningful within-subject score difference of 14.8 compared with 33.3% of patients in the comparator arm; study was not powered to show a statistically significant difference between treatment arms; strong trend toward significance was observed for improvement from baseline at month 7 (12.24 vs. 7.76, p=0.1073); no new safety signals | 9/5/2023 | Infection |
Moderna Inc., of Cambridge, Mass. | mRNA-1273.815 | Vaccine containing spike proteins for the XBB.1.5 sublineage of SARS-CoV-2 | COVID-19 | Data generated 8.7-fold to 11-fold increase in neutralizing antibodies against circulating variants, including BA.2.86, EG.5 and FL.1.5.1 variants | 9/6/2023 | Infection |
Moderna Inc., of Cambridge, Mass. | mRNA-1010 | mRNA-based seasonal influenza vaccine | Seasonal influenza infection | Study met all co-primary endpoints; similar adverse reactions to previous studies; higher HAI titers against A/H1N1, A/H3N2, B/Victoria compared Fluzone HD in phase I/II head-to-head study | 9/13/2023 | Infection |
Sorrento Therapeutics Inc., of San Diego | STI-1558 (Ovydso) | Protease inhibitor | COVID-19 | Top-line results showed study met primary and secondary endpoints; median time to sustained recovery of 11 COVID symptoms was 8.6 days in comparison with 11 days in the placebo group (p=0.0001); profound antiviral activity in COVID-19 patients at day 4 (3 days after treatment), reducing the viral RNA copy load by -0.8 log10 (p< 0.0001) compared with placebo group; improved recovery time by 2.3 days (7.6 days vs 9.9 days, p= 0.026) in patients with risk factors for severe COVID-19; reduced viral RNA copy load by -1.10 log10 (p<0.0001) at day 4 compared with the placebo group; mild adverse events; few serious events (4 in placebo and 3 in Ovydso) | 9/12/2023 | Infection |
YS Biopharma Co. Ltd., of Gaithersburg, Md. | PIKA rabies vaccine | Vaccine using the PIKA immunomodulating technology | Rabies post-exposure prophylaxis | First subject enrolled; planning to enroll 4,500 subjects | 9/26/2023 | Infection |
Astrazeneca SA, of Cambridge, U.K. | Fasenra (benralizumab) | Monoclonal antibody targeting IL-5 receptor alpha | Eosinophilic granulomatosis with polyangiitis | Study met primary endpoint; non-inferior rates of remission compared to mepolizumab in patients; consistent with safety and tolerability profile | 9/11/2023 | Inflammatory |
Amo Pharma Ltd., of London | AMO-02 (tideglusib) | Disrupting the pathogenic RNA repeat in CDM1 and inhibiting excess levels of the kinase GSK3? | Congenital-onset myotonic dystrophy | Top-line results of study did not meet the primary endpoint of a statistically significant benefit over placebo based on the U.S. FDA-authorized physician-completed rating scale; clinically and statistically significant benefit was achieved during analysis of a range of functional and objective assessments in the treatment group compared to placebo; clinically significant improvements in cognitive performance (p<0.05); reduction in a widely used biomarker of skeletal and cardiac muscle integrity (creatine phosphokinase, p<0.05); improvement in the 10-meter walk/run test (p=0.054); statistically significant benefit of treatment with AMO-02 compared to placebo (p<0.05); no reported treatment-related serious adverse events; safe and well-tolerated | 9/6/2023 | Musculoskeletal |
Biohaven Ltd., of New Haven, Conn. | Taldefgrobep alfa | Anti-myostatin adnectin | Spinal muscular atrophy | Completed enrollment; to evaluate the efficacy and safety of taldefgrobep at 48 weeks as an adjunctive therapy | 9/14/2023 | Musculoskeletal |
Fulcrum Therapeutics Inc., of Cambridge, Mass. | Losmapimod | selective p38?/? mitogen activated protein kinase (MAPK) inhibitor | Facioscapulohumeral muscular dystrophy | Completed enrollment with 260 patients; to evaluate the efficacy and safety | 9/7/2023 | Musculoskeletal |
Apnimed Inc., of Cambridge, Mass. | AD-109 | Fixed dose combination of aroxybutynin/atomoxetine | Obstructive sleep apnea | First patient dosed | 9/21/2023 | Neurology/Psychiatric |
Astrazeneca plc, of Cambridge, U.K., and Ionis Pharmaceuticals Inc., of Carlsbad, Calif. | Eplontersen | Antisense medicine targeting production of transthyretin | Hereditary transthyretin-mediated amyloid polyneuropathy | Data from the Neuro-TTRansform study published in The Journal of the American Medical Association showed treatment with eplontersen produced a least squares mean reduction of 82% in TTR serum concentration from baseline at 65 weeks, compared to an 11% reduction from baseline in the external placebo group (p<0.001) | 9/28/2023 | Neurology/Psychiatric |
Biovie Inc., of Santa Monica, Calif. | NE-3107 | Oral small molecule; blood-brain permeable; anti-inflammatory; insulin-sensitizing and ERK-binding properties | Mild to moderate Alzheimer’s disease | Last patient completed the last visit at week 30 | 9/26/2023 | Neurology/Psychiatric |
Cassava Sciences Inc., of Austin, Texas | Simufilam | Binds to altered conformation of filamin A protein (FLNA) | Alzheimer’s disease | Data and safety monitoring board reviewed interim safety report; recommended 2 phase III study continue as planned, without modification | 9/18/2023 | Neurology/Psychiatric |
Cingulate Inc., of Kansas City, Kan. | CTx-1301 | Extended-release tablet formulation of dexmethylphenidate | Attention deficit hyperactivity disorder | Study did not achieve statistical significance on the primary efficacy endpoint; trend toward significance in improving Permanent Product Measure of Performance (PERMP) scores with CTx-1301 compared to placebo; CGI-S scores showed significant improvements in severity of illness in patients taking CTx-1301 compared to placebo (p<0.001); reduction in Adult ADHD Investigator Symptom Rating Scale scores; favorable safety profile | 9/11/2023 | Neurology/Psychiatric |
Relmada Therapeutics Inc., of Coral Gables, Fla. | REL-1017 | NMDA receptor channel blocker; chemical molecule | Major depressive disorder | REL-1017 was well-tolerated; 418 patients had reached at least 6 months of treatment and 118 patients had reached at least 12 months of treatment at the time of study conclusion; rapid and sustained substantial improvement in Montgomery-Åsberg Depression Rating Scale (MADRS) total score over time for de novo patients only; 26.6% of de novo patients achieved clinical response by day 7, 51% by month 1, 60.7% by month 3, 63.4% by month 6, and 77.2% by month 12; clinical remission was achieved by 12.1% of de novo patients at day 7, 30.1% at month 1, 44% at month 3, 47.8% at month 6, and 54.4% at month 12; meaningful improvement in CGI-I Scale; significant reduction in Sheehan Disability Scale (50% at month 6); continual decline in anxiety symptoms over time as measured by the Hamilton Anxiety Rating Scale (HAM-A), 7.1 points at day 7, 9.6 points at month 1, 11.1 points at month 3, 11.5 points at month 6, and 13.5 points at month 12; no significant safety signal for weight gain, sexual dysfunction, cardiovascular issues, dissociative effects, withdrawal phenomena or abuse liability; low rates of adverse events and discontinuations due to adverse events; no new safety signals | 9/20/2023 | Neurology/Psychiatric |
Reviva Pharmaceuticals Holdings Inc., of Cupertino, Calif. | Brilaroxazine | Serotonin-dopamine stabilizer | Schizophrenia | Last patient completed Recover study; top-line results in October 2023 | 9/25/2023 | Neurology/Psychiatric |
Scisparc Ltd., of Tel Aviv, Israel | SCI-210 | Cannabidiol and palmitoylethanolamide combination | Autism spectrum disorder | Started the 60-patient crossover study comparing SCI-210 to cannabidiol monotherapy; primary endpoints include the Aberrant Behavior Checklist-Community parent questionnaire, the Clinical Global Impressions-Improvement scale and determining the effective therapeutic dosage | 9/29/2023 | Neurology/Psychiatric |
Affamed Therapeutics Ltd., of Shanghai | Dextenza | 0.4-mg dexamethasone ophthalmic insert | Ocular inflammation and pain following ophthalmic surgery | First patient treated; top-line data expected in the fourth quarter of 2023 | 9/20/2023 | Ocular |
Iveric Bio Inc., of Parsippany, N.J., and Astellas Pharma Inc., of Tokyo | Izervay (avacincaptad pegol intravitreal solution) | Complement C5 inhibitor | Geographic atrophy (GA) secondary to age-related macular degeneration | Top-line results from 24-month study met primary endpoint; significantly slow GA growth compared to sham at 24 months in monthly dosing regimen; similar reduction in the rate of GA growth vs. sham in the the every other month dosing; consistent safety profile with 12-month data; no new safety signals; 1 case of culture-positive endophthalmitis and 1 case of non-serious intraocular inflammation; no cases of occlusive or non-occlusive retinal vasculitis or ischemic neuropathy; rate of choroidal neovascularization was 12% in patients treated with Izervay and 9% in those treated with sham | 9/18/2023 | Ocular |
Palatin Technologies Inc., of Cranbury, N.J. | PL-9643 | Melanocortin agonist | Dry eye disease | Completed enrollment with 570 patients; top-line results expected in the fourth quarter of 2023; interim data from 120 patients showed excellent safety and tolerability | 9/7/2023 | Ocular |
Soleno Therapeutics Inc., of Redwood City, Calif. | DCCR | Extended-release tablets of diazoxide choline | Prader-Willi syndrome | Top-line results from the randomized withdrawal period showed hyperphagia-related behaviors markedly worsened in the placebo group compared to DCCR; statistically significant, clinically meaningful difference in mean change from baseline in the HQ-CT total score of 5 at week 16 (p=0.0022); strong trends toward worsening in the placebo group compared to DCCR over the course of the randomized withdrawal period (p=0.08 and 0.09) in CGI-S and CGI-I, respectively; well-tolerated; no new or unexpected safety signals; no serious adverse events or discontinuations due to adverse events | 9/26/2023 | Other/Miscellaneous |
Ascentage Pharma, of Suzhou, China, and Rockville, Md. | Olverembatinib | Tyrosine kinase inhibitor | Philadelphia chromosome-positive acute lymphoblastic leukemia | Registrational study of the drug in combination with chemotherapy in treatment-naïve patients has dosed the first patient | 10/17/23 | Cancer |
Asieris Pharmaceuticals Co. Ltd., of Shanghai | APL-1706 (Hexvix) | Imaging drug | Diagnosis or surgery of bladder cancer | Data from 97 patients diagnosed with Ta, T1 and CIS (mFAS), compared to standard white light cystoscopy, showed total of 42 patients (43.3%) detected 1 or more additional bladder cancer lesions through the combination of APL-1706 and blue light cystoscopy (p<0.0001); no serious adverse events; good tolerability | 10/12/23 | Cancer |
Astrazeneca plc, of Cambridge, U.K. | Imfinzi (durvalumab) | Monoclonal antibody targeting PD-L1 | Early stage and locally advanced gastric and gastroesophageal junction cancers | Pathologic complete response rate was 19% for patients treated with Imfinzi plus neoadjuvant FLOT chemotherapy compared to 7% for patients treated with neoadjuvant chemotherapy alone (p<0.00001); complete or near-complete responses rate was 27% with the Imfinzi combination and 14% with neoadjuvant chemotherapy alone | 10/20/23 | Cancer |
Beigene Ltd., of Basel, Switzerland, Beijing, and Cambridge, Mass. | Tevimbra (tislelizumab) | Humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to Fc-gamma (Fc?) receptors on macrophages | Advanced gastric or gastroesophageal junction adenocarcinoma | Phase III Rationale 305 trial showed the drug plus chemotherapy significantly improved overall survival (OS) in the intent-to-treat population as a first-line treatment; OS was 15 months compared with 12.9 months for chemotherapy alone (p=0.0011) | 10/17/23 | Cancer |
Beigene Ltd., of Basel, Switzerland, Beijing, and Cambridge, Mass. | Tevimbra (tislelizumab) | Humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to Fc-gamma (Fc?) receptors on macrophages | Resectable stage II or IIIA non-small-cell lung cancer | Rationale 315 data show it met its dual primary endpoints of major pathological response and event-free survival; 56.2% of patients receiving tislelizumab plus chemotherapy before surgery achieved a major pathological response vs. 15% receiving neoadjuvant chemotherapy alone; 40.7% of treated patients achieved the key secondary endpoint of pathological complete response vs. 5.7%; generally well-tolerated | 10/17/23 | Cancer |
Blue Earth Diagnostics Inc., of Monroe Township, N.J. | Posluma (flotufolastat F 18) PET | Imaging agent | Newly diagnosed prostate cancer | Results from subgroup analysis of Lighthouse trial focused on newly diagnosed, high-risk patients who had negative results with conventional imaging, showed sensitivity specificity for detection of pelvic lymph nodes in 174 patients who underwent surgery ranged from 24%-33% and 92%-96%, respectively, across 3 blinded, independent readers; detection rate for M1 lesions in 197 patients was 14%-25% across the 3 readers; of identified lesions, 16-25 were successfully verified as True Positive, providing a M1 Verified Detection Rate of 8.1%–13%, across the 3 readers | 10/4/23 | Cancer |
Briacell Therapeutics Corp., of Berkeley, Calif. | Bria-IMT | Cell-based immunotherapy | Advanced metastatic breast cancer | Institutional Review Board approved registration-enabling pivotal phase III study in combination with an immune checkpoint inhibitor | 10/3/23 | Cancer |
Bristol Myers Squibb Co., of New York | Opdivo (nivolumab) co-formulated with human hyaluronidase | Subcutaneous anti-PD-1 antibody | Advanced or metastatic clear cell renal cell carcinoma | In the Checkmate-67T study, subcutanous Opdivo was noninferior to intravenous Opdivo on both Cavgd28 (time-averaged Opdivo serum concentration over 28 days) and Cminss (trough serum concentration at steady state); subcutaneous treatment was also noninferior for objective response rate | 10/19/23 | Cancer |
Bristol Myers Squibb Co., of Princeton, N.J. | Opdivo (nivolumab) | PD-1 immune checkpoint inhibitor | Resectable non-small-cell lung cancer | Phase III Checkmate -77T trial showed neoadjuvant Opdivo with chemotherapy followed by surgery and adjuvant Opdivo reduced the risk of disease recurrence, progression or death by 42% (p=0.00025) | 10/17/23 | Cancer |
Bristol Myers Squibb Co., of Princeton, N.J. | Opdivo (nivolumab) | PD-1 immune checkpoint inhibitor | Unresectable or metastatic urothelial carcinoma | Phase III Checkmate -901 trial showed Opdivo in combination with cisplatin-based chemotherapy followed by Opdivo monotherapy reduced the risk of death by 22%, demonstrating a median overall survival of 21.7 months vs. 18.9 months with chemotherapy alone (p=0.0171) | 10/17/23 | Cancer |
Bristol Myers Squibb Co., of Princeton, N.J. | Opdivo (nivolumab) | PD-1 immune checkpoint inhibitor | Resectable non-small-cell lung cancer | Phase III Checkmate -816 trial of neoadjuvant Opdivo with chemotherapy showed a greater reduction in the risk of disease recurrence, progression or death than chemotherapy alone, demonstrating sustained event-free survival and promising overall survival trends, regardless of PD-L1 expression levels; overall survival data are immature but for those with PD-L1 expression ?1% Opdivo plus chemo reduced the risk of death by 63%; for those with PD-L1 <1%, it reduced it by 19% | 10/17/23 | Cancer |
Deciphera Pharmaceuticals Inc., of Waltham, Mass | Vimseltinib | CSF1R inhibitor | Tenosynovial giant cell tumor | In the Motion study, objective response rate (ORR) at week 25 was 40% for vimseltinib and 0% for placebo (p<0.0001); ORR per tumor volume score at week 25 was 67% for vimseltinib and 0% for placebo (p<0.0001); mean improvement from baseline in active range of motion at week 25 was 18.4% for vimseltinib compared to 3.8% for placebo (p=0.0077) | 10/30/23 | Cancer |
Eli Lilly and Co., of Indianapolis | Verzenio (abemaciclib) | CDK4/6 inhibitor | HR+, HER2-, node-positive early breast cancer | After a median follow-up of 4.5 years in the Monarche study, treatment with Verzenio reduced the risk of developing invasive disease by 32% (p<0.001) compared to placebo | 10/20/23 | Cancer |
Genentech, of South San Francisco, a member of the Roche Group | Alecensa (alectinib) | Highly selective, central nervous system-active, oral medicine; ALK inhibitor | Resected stage Ib to IIIa anaplastic lymphoma kinase-positive non-small-cell lung cancer | Alina study demonstrated a statistically significant and clinically meaningful improvement in disease-free survival, the primary endpoint, showing the drug reduces the risk of disease recurrence or death by 76% (p<0.0001) vs. platinum-based chemotherapy; a clinically meaningful improvement of central nervous system-DFS was also observed; overall survival data were immature | 10/18/23 | Cancer |
GSK plc, of London | Jemperli (dostarlimab) | Monoclonal antibody targeting PD-1 | Primary advanced or recurrent endometrial cancer | Ruby study met its primary endpoint of improvement of overall survival for Jemperli plus standard-of-care chemotherapy (carboplatin and paclitaxel), followed by Jemperli compared to placebo plus chemotherapy followed by placebo; data to be presented at an upcoming scientific meeting | 10/30/23 | Cancer |
Hansa Biopharma AB, of Lund, Sweden | Idefirix (imlifidase) | Enzyme derived from Streptococcus pyogenes | Desensitization therapy to enable kidney transplants in highly sensitized patients | Randomization of Confides trial; expected to conclude in mid-2024 | 10/11/23 | Cancer |
Henlius Biotech Inc., of Shanghai | Hansizhuang (serplulimab) | Anti-PD-1 monoclonal antibody | Limited-stage small-cell lung cancer | First patient dosed in combination with chemotherapy and concurrent radiotherapy | 10/25/23 | Cancer |
Lisata Therapeutics Inc., of Basking Ridge, N.J. | LSTA-1 | Integrin alpha-5/beta-3 modulator; neuropilin 1 modulator; integrin alpha-5/beta-5 modulator | Advanced solid tumors | Dosed first patient in the cholangiocarcinoma cohort of the Bolster Trial | 10/24/23 | Cancer |
Merarini Group, of Florence, Italy | Orserdu (elacestrant) | Selective estrogen receptor degrader | Early stage ER+/HER2- breast cancer and molecular relapse | Launched the EORTC 2129-BCG Treat ctDNA evaluating whether elacestrant can delay occurrence of metastasis or death compared to standard adjuvant endocrine therapy | 10/19/23 | Cancer |
Merck & Co. Inc., of Rahway, N.J. | Keytruda (pembrolizumab) | Monoclonal antibody targeting PD-1 | Localized muscle-invasive urothelial carcinoma and locally advanced urothelial carcinoma | In the Ambassador Keynote-123 study, treatment with Keytruda as an adjuvant reduced disease-free survival compared to the observation group; trial will continue to evaluate the other dual primary endpoint of overall survival | 10/5/23 | Cancer |
Merck & Co. Inc., of Rahway, N.J. | Keytruda (pembrolizumab) | Monoclonal antibody targeting PD-1 | Resectable stage II, IIIA or IIIB non-small-cell lung cancer | In the Keynote-671 study Keytruda plus chemotherapy before surgery plus Keytruda after surgery improved overall survival compared to placebo plus chemotherapy before surgery and placebo after surgery | 10/10/23 | Cancer |
Merck & Co. Inc., of Rahway, N.J. | Keytruda (pembrolizumab) | Monoclonal antibody targeting PD-1 | Resectable stage II, IIIA or IIIB non-small-cell lung cancer | In the Keynote-671 study, neoadjuvant Keytruda plus chemotherapy followed by Keytruda as a single agent after surgical resection reduced the risk of death by 28% compared to neoadjuvant placebo plus chemotherapy followed by adjuvant placebo, regardless of PD-L1 expression (p=0.00517); median overall survival not reached for patients treated with Keytruda compared to 52.4 months for the chemotherapy-placebo regimen | 10/20/23 | Cancer |
Merck & Co. Inc., of Rahway, N.J. | Keytruda (pembrolizumab) | Monoclonal antibody targeting PD-1 | HER2-positive locally advanced unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma | In the Keynote-811 study, Keytruda plus trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy reduced the risk of disease progression or death by 28% (p=0.0002) compared to trastuzumab and chemotherapy alone; for patients with PD-L1 combined positive score of ?1, Keytruda regimen reduced the risk of disease progression or death by 30% compared to trastuzumab and chemotherapy alone | 10/20/23 | Cancer |
Merck & Co. Inc., of Rahway, N.J. | Keytruda (pembrolizumab) | Monoclonal antibody targeting PD-1 | High-risk, early-stage estrogen receptor-positive, human epidermal growth factor receptor 2-negative breast cancer | In the Keynote-756 study, pathological complete response rate was 24.3% for Keytruda plus chemotherapy as neoadjuvant treatment, followed by Keytruda plus endocrine therapy as adjuvant treatment, compared to 15.6% in patients treated with neoadjuvant chemotherapy alone (p=0.00005); study is continuing to evaluate the other dual primary endpoint of event-free survival | 10/20/23 | Cancer |
Novartis AG, of Basel, Switzerland | Kisqali (ribociclib) | CDK4/6 inhibitor | Stage II and III hormone receptor-positive/human epidermal growth factor receptor 2-negative early breast cancer | Subgroup analysis of patients in the Natalee study showed Kisqali plus endocrine therapy improved the invasive disease-free survival rate compared to endocrine therapy alone for 10 different subgroups | 10/20/23 | Cancer |
Philogen SpA, of Sienna, Italy, and Sun Pharmaceutical Industries Ltd., of Mumbai, India | Nidlegy | Immunocytokines L19IL2 and L19TNF | Locally advanced fully resectable melanoma | Study compared neoadjuvant intratumoral Nidlegy followed by surgery (treatment arm) vs. surgery alone (control arm); at median follow-up of 27.6 months in both groups, the study met its primary endpoint with a statistically significant and clinically meaningful improvement in recurrence-free survival of the treatment arm compared to the control arm | 10/16/23 | Cancer |
Shanghai Junshi Biosciences Co. Ltd., of Shanghai | Toripalimab | Monoclonal antibody targeting PD-1 | First-line intermediate to high-risk unresectable or metastatic renal cell carcinoma | In the Renotorch study, toripalimab plus axitinib produced a median progression-free survival of 18 months compared to 9.8 months for patients treated with sunitinib (HR=0.65, p=0.0028); objective response rate was 56.7% for toripalimab-axitinib compared to 30.8% for sunitinib (p<0.001) | 10/27/23 | Cancer |
Stellas Life Sciences Group Inc., of New York | Galinpepimut-S | WT1-targeting peptide immunotherapeutic | Acute myeloid leukemia | Regal study showed patients achieved complete remission following second-line salvage therapy; expected to complete enrollment in November 2023; next meeting of the independent data monitoring committee for the Regal study is scheduled for the end of November 2023 | 10/13/23 | Cancer |
Biocardia Inc., of Sunnyvale, Calif. | CardiAMP cell therapy | CardiAMP cell therapy | Heart failure | Completed enrollment | 10/11/23 | Cardiovascular |
Renibus Therapeutics Inc., of Southlake, Texas | RBT-1 | Preconditioning agent; fixed-dose combination product of stannic protoporphyrin/iron sucrose; inducer of anti-inflammatory, antioxidant and iron-scavenging pathway | Reduce postoperative complications following cardiothoracic surgery | First patient dosed | 10/25/23 | Cardiovascular |
Allergan Aesthetics, part of Abbvie Inc., of Chicago | TrenibotulinumtoxinE (BoNT/E) | Short-acting neurotoxin | Moderate to severe glabellar lines | Top-line results showed all primary and secondary endpoints were met in M21-500 and M21-508 studies; statistical significance for improvement with BoNT/E vs. placebo (p<0.0001) in glabellar line severity on the Facial Wrinkle Scale (FWS) from baseline at day 7 (based on both subject and investigator assessments); statistical significance in patient-reported outcomes on overall treatment satisfaction | 10/24/23 | Dermatologic |
Abbvie Inc., of North Chicago | Rinvoq (upadacitinib, 15 mg) | Oral, once-daily reversible JAK inhibitor | Moderate to severe atopic dermatitis | Study achieved the co-primary endpoints of improvement in skin clearance, measured by an EASI score of 75 (EASI-75) and vIGA-AD 0/1 at week 16, compared to those who received placebo; achieved the secondary endpoint of improvement in skin clearance, measured by an EASI score of 90 (EASI-90), and an additional endpoint of itch reduction (WP-NRS 0/1) at week 16 compared to placebo-treated patients; well-tolerated; no new safety signals | 10/11/23 | Dermatologic |
Almirall SA, of Barcelona | Lebrikizumab | Monoclonal antibody targeting IL-13 | Moderate to severe atopic dermatitis | Post-hoc analysis of the Advocate 1 and 2 studies showed 84% of patients who had responded to lebrikizumab at week 16 achieved a clinically meaningful response in at least 1 domain of the disease (mild signs, symptoms or quality of life impact) after 52 weeks, and more than 57 % achieved response across the 3 domains; significantly higher proportion of patients treated with lebrikizumab achieved EASI ?7 (mild) and EASI ?1 (clear/almost clear) at week 16 compared to placebo | 10/13/23 | Dermatologic |
Almirall SA, of Barcelona, Spain | Ilumetri (tildrakizumab) | IL-23p19 inhibitor | Moderate to severe chronic plaque psoriasis | Interim data significantly improved patients wellbeing after 16 weeks and maintained up to week 28; improvements in patients skin symptoms and health-related quality of life after 28 weeks andshowed sustained efficacy and safety over 2 years in patients; improved other important patient-reported outcomes such as sleep quality, which is highly correlated with itch, pain, quality of life and work productivity | 10/11/23 | Dermatologic |
Almirall SA, of Barcelona, Spain and Eli Lilly and Co., of Indianapolis | Lebrikizumab | Monoclonal antibody targeting interleukin-13 | Moderate-to-severe atopic dermatitis | After 2 years in the Adjoin long-term extension study, 95%-96% of patients achieved at least a 75% improvement in Eczema Area and Severity Index (EASI-75), 72%-85% achieved an EASI-90 and 76%-86% achieved an Investigator’s Global Assessment of clear or almost clear | 10/20/23 | Dermatologic |
Anaptysbio Inc., of San Diego | Imsidolimab (IL-36R) | Anti-interleukin-36 receptor IgG4 antibody | Generalized pustular psoriasis | Study met its primary endpoint in the study population; 53.3% of patients who received a single dose of 750 mg intravenous imsidolimab achieved GPPPGA 0/1 (clear or almost clear) at week 4 compared to 13.3% of patients on placebo (p=0.0131); favorable safety and tolerability; low incidence and no increase of infections vs. placebo; 3.3% imsidolimab-treated patients had detectable ADA | 10/9/23 | Dermatologic |
Arcutis Biotherapeutics Inc., of Westlake Village, Calif. | Zoryve (roflumilast) cream 0.3% | PDE4 inhibitor | Plaque psoriasis | New data demonstrated improvements in both scalp and body itch; 65.3% of roflumilast-treated patients achieved a clinically significant reduction in itch compared to 30.3% of vehicle-treated patients at week 8 (p<0.0001) as measured by a four-point (4-pt) change from baseline in SI-NRS; rapid and significant improvement in scalp itch was observed 24 hours following first application as measured by SI-NRS (p=0.0164); improvement in body itch as measured by the Worst Itch Numeric Rating Scale (WI-NRS) was also observed at week 8 with 63.1% of those treated with roflumilast foam achieving a WI-NRS 4-pt response compared to 30.1% of those treated with vehicle (p<0.0001); 19.6% of patients treated with roflumilast foam reporting no scaling, itch, or pain compared to 7.1% of those treated with vehicle (P=0.0012) at week 8; 41.5% of roflumilast-treated patients achieved a PSD score of 0 vs. 13.6% of vehicle-treated patients (p<0.0001); 31.7% of roflumilast-treated patients achieved a PSD score of 0 vs. 10% of vehicle-treated patients (p<0.0001) in patients with severity of rich; 64.9% of individuals treated with roflumilast foam achieved a PSD score of 0 compared to 40.3% of individuals treated with vehicle (p<0.0001) in severity of pain patients; 66.4% of individuals treated with roflumilast foam achieved S-IGA Success compared to 27.8% of individuals treated with a vehicle foam at week 8 (p<0.0001), and 45.5% of individuals treated with roflumilast foam achieved B-IGA success compared to 20.1% of individuals treated with a matching vehicle foam at week 8 (p<0.0001); well-tolerated | 10/13/23 | Dermatologic |
Arcutis Biotherapeutics Inc., of Westlake Village, Calif. | Zoryve (roflumilast) | PDE4 inhibitor | Plaque psoriasis | At week 8, 72.1% of patients treated with Zoryve achieved a 50% reduction in Psoriasis Area and Severity Index (PASI-50) compared to 25.5% of those treated with vehicle (p<0.0001); 40.3% of patients treated with Zoryve achieved PASI-75 compared to 6.5% of vehicle-treated patients (p<0.0001); PASI-90 and PASI-100 was seen in 19.7% and 12.3% of patients treated with Zoryve and 2.3% and 0.8% of patients treated with vehicle, respectively (p<0.0001 and p<0.001, respectively) | 10/20/23 | Dermatologic |
Bristol Myers Squibb Co., of Princeton, N.J. | Sotyktu (deucravacitinib) | Oral, selective, allosteric TYK2 inhibitor | Moderate to severe plaque psoriasis | POETYK PSO long-term extension results showed clinical response was maintained at 73.2% for Psoriasis Area and Severity Index (PASI) 75 with 3 years of continuous Sotyktu treatment; consistent safety profile with no increases in adverse events or serious adverse events and no emergence of any new safety signals | 10/11/23 | Dermatologic |
Dermata Therapeutics Inc., of San Diego | DMT-310 | Once weekly topical product candidate derived from a naturally sourced freshwater sponge | Moderate to severe acne | Planning to initiate phase III Star-1 study by the end of 2023 | 10/26/23 | Dermatologic |
Dermavant Sciences Ltd., of Long Beach, Calif. | Vtama (tapinarof) | Aryl hydrocarbon receptor agonist | Atopic dermatitis | Data showed greater reductions in mean daily PP-NRS scores for tapinarof vs. vehicle observed as early as day 1, 24 hours after initial application in ADORING 1 (-1.2 vs. -0.9) and day 2 in ADORING 2 (-1.6 vs. -1.4); improvements in daily PP-NRS scores with tapinarof vs. vehicle continued through the first 2 weeks and through week 8 of both trials; achieved highly statistically significant and clinically meaningful reductions in mean weekly PP-NRS scores as early as week 1, the first assessment, for patients treated with tapinarof compared with vehicle (-2 vs.-1.2 [p<0.0001]) and (-2 vs. -1.3 [p=0.0010]), in ADORING 1 and ADORING 2, respectively; greater reductions in mean PP-NRS scores with tapinarof vs. vehicle were seen for all visits through week 8 (-4.1 vs. -2.6 and -4.1 vs. -2.4 [both p<0.0001]), in both ADORING 1 and ADORING 2 | 10/12/23 | Dermatologic |
Galderma SA, of Lausanne, Switzerland | Nemolizumab | Humanized monoclonal antibody directed against IL-31 receptor | Moderate to severe atopic dermatitis | ARCADIA 1 and 2 trials met their co-primary and all key secondary endpoints; 35.6% and 37.7% of nemolizumab-treated patients in ARCADIA 1 and 2, respectively, reached clearance or almost-clearance of skin lesions when assessed using the Investigator’ Global Assessment (IGA) score, compared to 24.6% and 26% in the placebo group (p<0.0006, p=0.001); 43.5 % and 42.1% of nemolizumab-treated patients in ARCADIA 1 and 2, respectively, achieved a 75% reduction in the Eczema Area and Severity Index (EASI), compared to 29% and 30.2% in the placebo group (p<0.0001, p=0.0011); 48.6% and 48.1% of nemolizumab-treated patients achieved at least 4-point reduction in itch in ARCADIA 1 and 2, respectively, as measured by the peak-pruritus numerical rating scale (PP-NRS) score, compared to 20.5% and 20.6% in the placebo group (p<0.0001), within 16 weeks of treatment; well-tolerated | 10/11/23 | Dermatologic |
Galderma SA, of Lausanne, Switzerland | Nemolizumab | Humanized monoclonal antibody directed against IL-31 receptor | Moderate to severe prurigo nodularis | Monotherapy significantly improved itch and skin lesions in adult patients; 58.4% of nemolizumab-treated patients achieved an at least 4-point reduction in itch, as measured by the peak-pruritus numerical rating scale (PP-NRS) score, compared to 16.7% in the placebo group (p<0.0001); 26.3% of nemolizumab-treated patients reached clearance or almost-clearance of skin lesions, when assessed using the Investigator’ Global Assessment (IGA) score, compared to 7.3% in the placebo group (p<0.0001); trial also met all key secondary endpoints, confirming rapid onset of action on itch as early as week 4 (41.1% compared to 6.3% in the placebo group; p<0.0001); well-tolerated | 10/11/23 | Dermatologic |
Incyte Corp., of Wilmington, Del. | Opzelura (ruxolitinib cream) | JAK1/JAK2 inhibitor | Atopic dermatitis | True-AD3 study showed 56.5% of patients treated with ruxolitinib cream 1.5% and 36.6% treated with ruxolitinib cream 0.75% achieved IGA-TS at Week 8 (p<=0.0001 for both) compared to 10.8% of patients treated with vehicle; more than half (67.2%/51.5%) of patients treated with ruxolitinib cream (1.5% and 0.75% respectively) achieved EASI75 compared to those treated with vehicle at week 8 (15.4%; P<0.0001 for both); NRS4 was achieved by 43.4% (1.5% treatment arm) and 37.5% (0.75% treatment arm) of patients at week 8 compared to those treated with vehicle (29.7%) in patients 6 to <12 years old; median time to NRS4 was 13.0/11.0 days (1.5% and 0.75% respectively) compared to 23 days for vehicle (hazard ratio, 1.74/1.77; P<0.05 for both); mean steady-state plasma concentrations (Css) of ruxolitinib at week 8 were 23.2 nM (1.5% ruxolitinib cream) and 11.3 nM (0.75% ruxolitinib cream); well-tolerated; no serious infections | 10/13/23 | Dermatologic |
Incyte Corp., of Wilmington, Del. | Opzelura (ruxolitinib cream) | JAK1/JAK2 inhibitor | Atopic dermatitis | Must study showed no serious side effects or led to treatment interruption or discontinuation; mean steady-state plasma concentrations (Css) of ruxolitinib cream through week 4 was 98.2 (148) nM; 54% of patients achieved an IGA of 0 or 1 and approximately 73% achieved NRS4 by week 4 | 10/13/23 | Dermatologic |
Incyte Corp., of Wilmington, Del. | Opzelura (ruxolitinib cream) | JAK1/JAK2 inhibitor | Atopic dermatitis | TRuE-AD3study met its primary endpoint with significantly more patients treated with ruxolitinib cream (0.75% and 1.5%) achieving Investigator’s Global Assessment Treatment Success (IGA-TS) than patients treated with vehicle control (non-medicated cream); secondary endpoints such as time to NRS4 (?4-point improvement in itch Numerical Rating Scale [NRS] score) and patients demonstrating at least a 75% improvement in the Eczema Area and Severity Index at week 8 were also achieved | 10/13/23 | Dermatologic |
Janssen Biotech Inc., part of New Brunswick, N.J.-based Johnson & Johnson | JNJ-2113 (formerly PN-235) | Oral interleukin-23 receptor antagonist | Moderate to severe plaque psoriasis | Advanced to phase III studies (ICONIC-LEAD and ICONIC-Total) | 10/9/23 | Dermatologic |
Journey Medical Corp., of Scottsdale, Ariz., and Dr. Reddy's Laboratories Ltd., of Hyderabad, India | DFD-29 (minocycline hydrochloride modified release capsules) | Targets bacterial 30S ribosomal subunit, inhibiting protein synthesis | Rosacea | In a comparative bridging study, bioavailability of minocycline was significantly lower after a single dose of DFD-29 (40 mg) under fasting and fed conditions compared to a single dose of Solodyn (105 mg) under fasting conditions | 10/20/23 | Dermatologic |
Kashiv BioSciences LLC., of Piscataway, N.J. | ADL-018 | Biosimilar candidate to Xolair (omalizumab) | Chronic idiopathic/spontaneous urticaria | Enrolled first patient | 10/2/23 | Dermatologic |
Leo Pharma A/S, of Ballerup, Denmark | Delgocitinib cream | Pan-JAK inhibitor | Chronic hand eczema | Trial achieved its key secondary endpoints at week 16; significantly greater proportion of delgocitinib-treated patients achieving a ?75% improvement in the Hand Eczema Severity Index (HECSI-75) compared to cream vehicle (49.5% vs. 18.2%; p<0.001); favourable safety profile | 10/13/23 | Dermatologic |
Leo Pharma A/S, of Ballerup, Denmark | Adtralza (tralokinumab) | Monoclonal antibody targeting IL-13 cytokine | Moderate to severe atopic dermatitis | Post-hoc analysis showed investigator's Global Assessment score of 0 (clear) or 1 (almost clear) (IGA 0/1) was observed in 52.6% of patients; 75% reduction in the Eczema Area and Severity Index (EASI-75) was observed in 84.5% of patients, and an EASI-90 in 64.4%; no new safety signals | 10/13/23 | Dermatologic |
Leo Pharma A/S, of Ballerup, Denmark | Delgocitinib | Pan-JAK inhibitor | Moderate to severe chronic hand eczema | In the Delta 3 open-label extension study, patients who were on delgocitinib in the primary Delta 1 and 2 studies maintained similar levels of symptom relief and treatment success over an additional 36 weeks of treatment; patients who were on cream vehicle in the primary studies reached similar levels of treatment success as the patients previously treated with delgocitinib cream | 10/30/23 | Dermatologic |
Leo Pharma A/S, of Madison N.J. | Adbry (tralokinumab-ldrm) | Monoclonal antibody targeting interleukin-13 | Atopic dermatitis | Post hoc analyses and real-world studies demonstrated the positive efficacy and safety of Adbry | 10/20/23 | Dermatologic |
Quoin Pharmaceuticals Ltd., of Ashburn, Va. | QRX-003 | Serine protease inhibitor | Netherton syndrome | Data showed well-defined positive improvement in pruritus, or itch in 5 subjects and reported absent or negligible pruritus on completion of dosing; 6th subject showed pruritus was effectively unchanged on completion of dosing; improvement in skin appearance in 6 subjects | 10/24/23 | Dermatologic |
UCB SA, of Brussels | Bimzelx (bimekizumab) | Humanized monoclonal IgG1 antibody targeting IL-17A and IL-17F | Moderate-to-severe hidradenitis suppurativa | Pooled data from Be Heard 1 and Be heard 2 studies showed higher response rates in the primary endpoint (HiSCR50) at week 16 vs. placebo (58 % Q2W/Q2W, 55.9 % Q2W/Q4W, 56.1 % Q4W/Q4W vs. 33.4 % for placebo); response in patients who switched from placebo to bimekizumab at week 16 approached that reached by patients on bimekizumab from baseline (70.5%) at week 48; bimekizumab-treated patients had improved responses at week 48 with approximately 6 in 10 patients achieving HiSCR75; numerically higher HiSCR100 responses in bimekizumab patients vs. placebo at week 16 and and improved responses at week 48; post hoc analysis showed that at week 16 higher proportions of bimekizumab-treated patients had mild HS vs. placebo (24.6¬–27.2% vs. 15.3%); improvements in International Hidradenitis Suppurativa Severity Score System (IHS4) categories were sustained over time across bimekizumab groups; no new safety signals | 10/13/23 | Dermatologic |
Ascendis Pharma A/S, of Copenhagen, Denmark | Transcon PTH (palopegteriparatide) | Sustained release of active parathyroid hormone | Chronic postsurgical hypoparathyroidism | In the Pathway study, 81% of patients achieved independence from conventional therapy while maintaining normocalcemia following treatment with Transcon PTH | 10/2/23 | Endocrine/Metabolic |
Ascendis Pharma A/S, of Copenhagen, Denmark | Transcon PTH (palopegteriparatide) | Sustained release of active parathyroid hormone | Chronic hypoparathyroidism | In the open-label portion of the Pathway study, skeletal dynamics trended toward a new skeletal steady state closer to age-appropriate norms | 10/16/23 | Endocrine/Metabolic |
Foresee Pharmaceuticals Co. Ltd., of Taipei, Taiwan | Leuprolide (FP-001) | 42 mg-controlled release | Central (gonadotropin-dependent) precocious puberty; gonadotropin-releasing hormone (GnRH) agonists | First patient dosed | 10/13/23 | Endocrine/Metabolic |
Mirum Pharmaceuticals Inc., of Foster City, Calif | Chenodal (chenodiol) | Bile acid | Cerebrotendinous xanthomatosis | In the Restore study, Chenodal reduced urine bile alcohols at the end of the withdrawal period by 20-fold compared to placebo (p<0.0001) | 10/2/23 | Endocrine/Metabolic |
Neurocrine Biosciences Inc., of San Diego | Crinecerfont | Corticotropin-releasing factor type 1 receptor antagonist | Classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency | In the Cahtalyst pediatric study, crinecerfont decreased serum androstenedione from baseline at week 4 more than placebo following a glucocorticoid stable period (p=0.0002) | 10/5/23 | Endocrine/Metabolic |
Sanofi SA, of Paris | Tzield (teplizumab-mzwv) | CD3-directed monoclonal antibody | Stage 3 autoimmune type 1 diabetes | Protect trial showed it met the primary endpoint, demonstrating superior beta cell preservation assessed by significantly slowing the decrease in mean C-peptide levels (area under the curve after a 4-hour mixed meal tolerance test) at trial completion, compared to placebo; the key secondary endpoints did not meet statistical significance, but numerical trends favoring Tzield were seen in relevant clinical parameters; full data were published in The New England Journal of Medicine | 10/18/23 | Endocrine/metabolic |
Abbvie Inc., of North Chicago | Skyrizi (risankizumab) | Monoclonal antibody targeting IL-23 | Crohn's disease after failure of 1 or more anti-TNF drugs | In the Sequence study, Skyrizi was non-inferior to Stelara (ustekinumab, J&J) with a clinical remission rate, defined as Crohn's Disease Activity Index < 150, at week 24 of 59% for Skyrizi and 40% for Stelara; endoscopic remission rates were 32% for Skyrizi and 16% for Stelara (p<0.0001) | 10/15/23 | Gastrointestinal |
Ardelyx Inc., of Waltham, Mass. | Ibsrela (tenapanor) | Locally acting inhibitor of NHE3 | Irritable bowel syndrome with constipation | In study testing drug in patients, ages 12-17, preliminary blinded safety data showed no serious treatment-emergent events; no unexpected safety concerns observed | 10/4/23 | Gastrointestinal |
Celltrion Inc., of Incheon, South Korea | Remsima (CT-P13) | Biosimilar reverencing infliximab | Inflammatory bowel disease | Post-hoc analyses of the Liberty-CD and Liberty-UC showed there were no statistical differences in efficacy outcomes between monotherapy and combination therapy with immunosuppressants after 54 weeks of treatment; in patients who lost response to the drug, dose escalation to 240 mg every 2 weeks improved clinical remission rate to 24.7% in UC and 53.8% in CD and endoscopic response to 28.2% in CD | 10/16/23 | Gastrointestinal |
Cymabay Therapeutics Inc., of Newark, Calif. | Seladelpar | PPAR-delta agonist | Primary biliary cholangitis | Post-hoc analysis showed a correlation between serum IL-31 levels and self-reported pruritus improvements in patients; seladelpar 5 mg (3.8 to 1.7 pg/mL, p < 0.001), 10 mg (4.2 to 1.7 pg/mL, p< 0.001) compared to placebo (4.3 to 3.9 pg/mL, not significant); clinically meaningful improvement in pruritus (? 2 decreases in numerical rating scale, or NRS, score) showed greater dose-dependent reductions in IL-31 from baseline than those without pruritus improvement; sgnificant correlations were also observed between changes in IL-31 vs. reported pruritus NRS scores (p< 0.0001), alkaline phosphatase levels (p<0.01), and total bile acids (p < 0.0001) at 10 mg | 10/24/23 | Gastrointestinal |
Eli Lilly and Co., of Indianapolis | Mirikizumab | Interleukin-23p19 antagonist | Moderately to severely active Crohn's disease | Study met the co-primary and all major secondary endpoints compared to placebo; statistically higher proportion achieved clinical response at week 12 and clinical remission at week 52 compared to placebo (45.4% vs. 19.6%, p<0.000001); statistically higher proportion achieved clinical response at week 12 and endoscopic response at week 52 compared to placebo (38% vs. 9%, p<0.000001); consistent safety profile; frequency of serious adverse events was greater in placebo than mirikizumab group | 10/12/23 | Gastrointestinal |
Ferring Pharmaceuticals A/S, of Saint-Prex, Switzerland, and Parsippany, N.J. | Rebyota (fecal microbiota, live–jslm) | Microbiome-based treatment | Recurrent Clostridioides difficile infection | A post-hoc analysis of the Punch CD3 study found the quartile with the highest C. difficile Quality of Life Survey (Cdiff32) score showed a significantly different microbiome composition from most other groups with lower scores; a subgroup analysis of patients with different comorbidities showed treatment success for 63% for those with cardiac disorders, 63% for those with renal disorders and 70% for those with gastrointestinal disorders | 10/23/23 | Gastrointestinal |
Index Pharmaceuticals Holding AB, of Stockholm | Cobitolimod | TLR9 agonist | Moderate to severe left-sided ulcerative colitis | Around 30% of patients have completed the final visit in Induction study | 10/11/23 | Gastrointestinal |
Ironwood Pharmaceuticals Inc., of Boston | Linzess (linaclotide) | Guanylate cyclase-C agonist | Functional constipation | A post-hoc analysis of the 12-week study found the use of rescue medications was 8.7% in children receiving linaclotide compared to 18.8% of children receiving placebo | 10/5/23 | Gastrointestinal |
Janssen Pharmaceuticals Inc., a unit of New Brunswick, N.J.-based Johnson & Johnson | Tremfya (guselkumab) | Monoclonal antibody targeting IL-23 p19 | Moderately to severely active ulcerative colitis | In the Quasar study, 61.5% of patients taking Tremfya achieved a clinical response at week 12 after IV induction therapy compared to 27.9% of patients taking placebo; for patients not in clinical response at week 12, 55% achieved clinical response at week 24 after Tremfya subcutaneous treatment | 10/23/23 | Gastrointestinal |
Janssen Pharmaceuticals Inc., a unit of New Brunswick, N.J.-based Johnson & Johnson | Tremfya (guselkumab) | Monoclonal antibody targeting IL-23 p19 | Moderately to severely active ulcerative colitis | Data showed clinical response was significantly achieved vs. placebo (61.5% vs. 27.9%); Tremfya-treated patients who were not in clinical response to IV induction therapy at week 12 and who then received subcutaneous treatment for an additional 12 weeks (55% achieved clinical response at week 24); cumulative clinical response at week 12 or 24 was achieved by 77.2%; no new safety signals; greater symptomatic improvements at week 12 (p<0.01); improvements in patient-reported outcomes of rectal bleeding (p<0.001) and absolute stool number(p<0.001) were observed as early after a single intravenous (IV) induction dose at week 12 | 10/23/23 | Gastrointestinal |
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and Sanofi SA, of Paris | Dupixent (dupilumab) | Fully human monoclonal antibody that inhibits the signaling of the IL-4 and IL-13 pathways | Eosinophilic esophagitis | Part B of phase III trial, following 16 weeks of Dupixent treatment or placebo in part A showed consistent efficacy and safety for up to one year (52 weeks) in children aged 1 to 11 years; 63% and 53% achieved histological disease remission; 0.97 and 0.89 reduction from baseline in disease severity and 0.89 and 0.86 reduction from baseline in extent, respectively, as measured at the microscopic level in biopsy specimens; 4.8 and 3.6-point reduction in abnormal endoscopic findings from baseline; 0.30 and 0.47-point numerical improvement in caregiver reported pediatric signs and symptoms, as measured by PESQ-C; 5.96 and 5.48 percentile increase in body weight for age percentile from baseline | 10/23/23 | Gastrointestinal |
Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Alofisel (darvadstrocel) | A dispersion of expanded allogeneic, adipose-derived mesenchymal stem cells | Complex Crohn's perianal fistulas | Admire-CD II study did not meet its primary endpoint of combined remission at 24 weeks, based on top-line data; the safety profile was consistent with prior studies | 10/18/23 | Gastrointestinal |
Novartis AG, of Basel, Switzerland | Iptacopan | Factor B inhibitor | IgA nephropathy | The Applause-IgAN study met its primary endpoint of reduction in proteinuria; data for estimated glomerular filtration rate slope over 24 months expected in 2025 | 10/2/23 | Genitourinary/Sexual Function |
Novartis AG, of Basel, Switzerland | Atrasentan | Endothelin A receptor antagonist | IgA nephropathy | At the 36-week interim analysis of the Align study, atrasentan produced a statistically significant reduction in proteinuria compared to placebo; study will continue to measure estimated glomerular filtration rate over 136 weeks with top-line results for the confirmatory endpoint expected in the first quarter of 2026 | 10/30/23 | Genitourinary/Sexual Function |
Novo Nordisk A/S, of Copenhagen, Denmark | Wegovy (semaglutide) | GLP-1 receptor agonist | Progression of renal impairment in people with type 2 diabetes and chronic kidney disease | Decided to stop the kidney outcomes trial based on a recommendation from the independent data monitoring committee; interim analysis met certain prespecified criteria for stopping the trial early for efficacy | 10/10/23 | Genitourinary/Sexual Function |
Omeros Corp., of Seattle | Narsoplimab | MASP-2 inhibitor | Immunoglobulin A nephropathy | An interim analysis showed the ARTEMIS-IGAN trial did not reach statistical significance on the primary endpoint of reduction in proteinuria from baseline compared to placebo | 10/16/23 | Genitourinary/Sexual Function |
Innocare Pharma Inc., of Beijing | Orelabrutinib | BTK inhibitor | Primary immune thrombocytopenia | First patient dosed | 10/27/23 | Hematologic |
Annexon Inc., of Brisbane, Calif. | ANX-005 | C1q inhibitor | Guillain-Barré Syndrome | Completed enrollment in the 225-patient, pivotal study; data expected in the first half of 2024 | 10/10/23 | Immune |
ARS Pharmaceuticals Inc., of San Diego | Neffy (epinephrine) | Adrenergic receptor agonist | Severe allergic reaction (type 1), including anaphylaxis | Neffy was well-tolerated with mild adverse events; robust responses on pharmacodynamic (PD) surrogates of efficacy observed even at 1 minute after dosing; repeated doses resulted in statistically greater mean maximum responses on PD surrogates for efficacy compared to Epipen; results published in Journal of Allergy and Clinical Immunology and American Academy of Allergy, Asthma and Immunology | 10/3/23 | Immune |
Bristol Myers Squibb Co., of New York | Zeposia (ozanimod) | S1P receptor modulator | Relapsing-remitting multiple sclerosis | New data after 8 years of follow-up showed 76% of patients treated with Zeposia were free of 6-month confirmed disability progression; low rates of progression independent relapse activity (PIRA) and relapse-associated worsening (RAW); PIRA and RAW were observed in 13.2% and 10.7% of participants treated with continuous Zeposia, respectively, after 8 years in the open-label extension study | 10/11/23 | Immune |
Genentech Inc., of South San Francisco, a member of the Roche Group | Ocrevus (ocrelizumab) | Monoclonal antibody targeting CD20-positive B cells | Relapsing forms and primary progressive of multiple sclerosis | Data showed subcutaneous injection was noninferior to intravenous infusion based on Ocrevus levels in the blood over 12 weeks; rapid, sustained and near-complete B-cell depletion that was similar to Ocrevus I.V. infusion (97% and 98% of patients, respectively, had B cell levels of 5 cells/µL or less when first measured at 14 days) and sustained over 24 weeks; rapid and near-complete suppression of MRI lesion activity by 24 weeks, with most patients having no T1 gadolinium-enhancing (T1 Gd+) lesions; no new/enlarging T2 lesions | 10/11/23 | Immune |
Genentech Inc., of South San Francisco, a member of the Roche Group | Ocrevus (ocrelizumab) | Monoclonal antibody targeting CD20-positive B cells | Relapsing forms and primary progressive of multiple sclerosis | 77% of patients with RMS were free from disability progression based on 48-week confirmed disability progression (CDP) events; 92% of patients were still walking unassisted; 36% were free from disability progression based on 48-week CDP events and 80% of those patients with PPMS treated continuously with Ocrevus over 10 years were still able to walk; favorable benefit-risk profile; no new or unexpected safety signals | 10/12/23 | Immune |
Genentech, a unit of Roche Holding AG, Basel, Switzerland | Fenebrutinib | BTK inhibitor | Multiple sclerosis | Data showed 12 weeks of continuous treatment, the mean fenebrutinib concentration was 43.1 ng/mL; significantly reduced the total number of new T1 gadolinium-enhancing (T1 Gd+) brain lesions; rapid onset of lesion reduction was observed by 4 weeks, with relative reductions of 92% and 90% in T1 Gd+ lesions and relative reductions of 90% and 95% in T2 lesions observed at 8 and 12 weeks, respectively; fenebrutinib were 4 times more likely to be free from any new T1 Gd+ brain lesions and new or enlarging T2 brain lesions at weeks 4, 8, and 12 combined, compared to patients who received placebo (odds ratio 4.005, p=0.0117); no new safety concerns | 10/13/23 | Immune |
Maat Pharma SA, of Lyon, France | MaaT-013 | Standardized, pooled-donor, enema microbiome ecosystem therapy | Acute graft-vs.-host disease | Independent Data Safety Monitoring Board unanimously recommended that the trial continues without modification; positive benefit/risk ratio based on a good safety profile and positive preliminary efficacy results with an overall response rate higher than pre-defined protocol assumptions; well-tolerated | 10/26/23 | Immune |
Basilea Pharmaceutica Ltd., Allschwil, Switzerland | Zevtera (ceftobiprole) | Cephalosporin antibiotic | Staphylococcus aureus infection | In the Eradicate study, exposure achieved with the applied dosing regimens was efficacious | 10/17/23 | Infection |
Ferring Pharmaceuticals A/S, of Saint-Prex, Switzerland, and Parsippany, N.J. | Rebyota (fecal microbiota, live-jslm) | Microbiome-based treatment | Recurrent Clostridioides difficile infection | An ad-hoc subgroup study of 98 patients with renal co-morbidity in the Punch CD3-OLS study, including 29 with chronic kidney disease and 5 with end-stage renal failure, showed 66% of patients remained free of C. diff infection recurrence for 8 weeks following Rebyota treatment, compared to 77% of 304 patients without renal co-morbidities; in a separate analysis of the Punch CD3 study, a Microbiome Health Index score of at least 7.2 was associated with a 14.2-18.4-point increase in patients’ C. difficile Quality of Life Survey score | 10/11/23 | Infection |
GSK plc, of London | Arexvy (recombinant adjuvanted vaccine; RSVPreF3) | Vaccine, adjuvanted, contains recombinant glycoprotein F stabilized in the prefusion conformation | Lower respiratory tract disease caused by respiratory syncytial virus infection | Preliminary results met primary endpoint; immune response in adults ages 50-59; co-primary endpoint was also met for the broader group of adults ages 50-59; common local adverse event was pain; common systematic adverse events were fatigue and headache | 10/25/23 | Infection |
Lianbio Co. Ltd., of Shanghai | TP-03 | Lotilaner; inhibits GABA-Cl channels | Demodex blepharitis | Top-line results from Libra study in Chinese patients showed statistically significant mite eradication vs. vehicle (p<0.001), meeting 1 co-primary endpoint; on the second co-primary endpoint, results showed a positive but not statistically significant trend (p=0.15) for complete collarette cure; well-tolerated with not treatment-related discontinuations | 10/30/23 | Infection |
Moberg Pharma AB., of Stockholm | MOB-015 | Topical formulation of terbinafine | Onychomycosis | Completed the recruitment of 384 patients; top-line results expected in Jan 2025 | 10/6/23 | Infection |
Moderna Inc., of Cambridge, Mass. | mRNA-1083 | Combination vaccine against influenza and COVID-19 | Influenza/COVID-19 | First subject dosed | 10/24/23 | Infection |
Theratechnologies Inc., of Montreal | Trogarzo (ibalizumab) | CD4-directed, post-attachment HIV-1 inhibitor | HIV infection with multidrug-resistant virus | The primary endpoint measuring a 90% confidence interval of the ratio of intramuscular injection to intravenous infusion (0.69, 1.08) did not meet the equivalence limits (0.8, 1.25); viral suppression was maintained in all HIV-positive subjects; well-tolerated | 10/13/23 | Infection |
University of Oxford and the European Clinical Research Alliance on Infectious Diseases | Various | Various | Influenza | Recovery study will expand to include patients with influenza, initially starting with testing Tamiflu (oseltamivir), Xofluza (baloxavir marboxil) and low dose corticosteroids | 10/30/23 | Infection |
YS Biopharma Co. Ltd., of Gaithersburg, Md. | Pika rabies vaccine | Prophylactic vaccine | Rabies infection | Completed subject enrollment; study includes 4,500 subjects | 10/31/23 | Infection |
Amylyx Pharmaceuticals Inc., of Cambridge, Mass. | AMX-0035 (taurursodiol) | Combination of sodium phenylbutyrate and ursodoxicoltaurine | Amyotrophic lateral sclerosis | Post-hoc analysis showed median overall survival was 10.4 months longer in AMX-0035 group than in the historical clinical trial control group; 52% lower risk of death over the duration of the follow-up period; results published in Annals of Clinical and Translational Neurology | 10/10/23 | Musculoskeletal |
Sarepta Therapeutics Inc., of Cambridge, Mass. | Elevidys (delandistrogene moxeparvovec) | Single-dose gene transfer therapy | Duchenne muscular dystrophy | Top-line results from Embark trial, in patients between ages of 4-7, missed the primary North Star Ambulatory Assessment endpoint but showed strong trends and nominal statistical significance on secondary endpoints, including time-to-rise assessment (p=0.0025) and 10-meter walk test (p=0.0048); timed functional endpoints, including stride velocity 95th centile, a digital endpoint that measures speed of walking via a wearable device (p=0.0402), and time to ascend four steps (p=0.0412), showed consistent treatment benefit in favor of Elevidys | 10/30/23 | Musculoskeletal |
Aculys Pharma Inc., of Tokyo | NRL-1 (diazepam) | Nasal spray; antiepileptic drug | Status epilepticus or epileptic seizures that may lead to status epilepticus | Achieved its primary efficacy endpoint, which was the proportion of patients who achieved resolution of clinically relevant seizures within 10 minutes after a single dose and who remained free from seizures or convulsions for 30 minutes, in a prespecified interim phase III analysis in Japanese patients ages 6 to 17; no adverse events leading to discontinuation or serious adverse events related to the drug were observed; Aculys intends to submit an NDA in Japan | 10/18/23 | Neurology/Psychiatric |
Alector Inc., of South San Francisco, Calif. | AL-001 (latozinemab) | Human monoclonal antibody designed to modulate progranulin (PGRN) | Frontotemporal dementia due to a progranulin gene mutation | Achieved target enrollment in Infront-3 study; 101 patients enrolled | 10/27/23 | Neurology/Psychiatric |
Athersys Inc., of Cleveland | Multistem (invimestrocel) | Bone marrow-derived regenerative stem cell therapy | Acute moderate-to-severe ischemic stroke | Interim analysis of 300 patients in the Masters-2 study determined that the study was insufficiently powered to achieve the primary endpoint at day 365; company plans to conduct additional analysis and has paused enrollment in the study | 10/10/23 | Neurology/Psychiatric |
Avadel Pharmaceuticals plc, of Dublin | Lumryz (sodium oxybate) | GABA-B receptor agonist | Cataplexy | Post-hoc analyses of the 3 Rest-on trial, showed treatment with Lumryz resulted in statistically significant and clinically meaningful improvement in excessive daytime sleepiness as measured by the Epworth Sleepiness Scale with the median score in the range considered normal at the end of the study | 10/20/23 | Neurology/Psychiatric |
Axsome Therapeutics Inc., of New York | AXS-05 (dextromethorphan-bupropion) | Oral NMDA receptor antagonist and sigma-1 receptor agonist | Alzheimer’s disease | Results showed rapid and sustained clinical response in patients; statistically significantly delayed the time to relapse of agitation symptoms as compared to placebo in placebo-controlled randomized withdrawal phase; well-tolerated; no new safety signals | 10/24/23 | Neurology/Psychiatric |
Biogen Inc., of Cambridge, Mass., and Eisai Co. Ltd., of Tokyo | Leqembi | Humanized anti- soluble aggregated amyloid-beta (A?) monoclonal antibody; intravenous infusion (200-mg, 500-mg lecanemab) | Mild cognitive impairment and mild dementia due to Alzheimer’s disease | Interim data showed weekly subcutaneous administration showed 14% greater amyloid plaque removal than biweekly intravenous administration; 76% of patients showed no decline and 60% showed clinical improvement at 18 months in low-tau / earlier stage AD population | 10/26/23 | Neurology/Psychiatric |
Biovie Inc., of Santa Monica, Calif. | NE-3107 | Oral small molecule; blood-brain permeable; anti-inflammatory; insulin-sensitizing and ERK-binding properties | Mild to moderate Alzheimer’s disease | Data showed statistically significant population changes from baseline were observed for all primary and secondary cognitive and functional assessments measured: ADAS-Cog12, ADCS-CGIC, MMSE, CDR, CDR-SB, ADCOMS, and ADL (p<0.0001 for all); significant reduction in amyloid burden from baseline; statistically significant increase in insulin and beta cell function (p<0.0001); decreased insulin sensitivity (p<0.0001); no events of hypoglycemia; no drug-related adverse events | 10/26/23 | Neurology/Psychiatric |
Cassava Sciences Inc., of Austin, Texas | Simufilam | Restores shape and function of altered filamin A | Alzheimer’s disease | Completed enrollment of 804 patients in the study designed to follow patients for 12 months | 10/2/23 | Neurology/Psychiatric |
Cassava Sciences Inc., of Austin, Texas | Simufilam | Restores shape and function of altered filamin A | Alzheimer’s disease | MRI data showed simufilam is not associated with treatment-emergent amyloid-related imaging abnormalities or anti-amyloid antibody drug | 10/25/23 | Neurology/Psychiatric |
Harmony Biosciences Holdings Inc., of Plymouth Meeting, Pa. | Wakix (pitolisant) | Histamine 3 receptor antagonist/inverse agonist | Idiopathic hypersomnia | Top-line results of Intune study showed 83% of patients who completed the 8-week open-label treatment period with pitolisant were responders and experienced a robust clinical response with an average Epworth Sleepiness Scale (ESS) change from baseline of – 9.4 points.; no statistically significant difference was observed between pitolisant and placebo groups on ESS; favorable effect in Idiopathic Hypersomnia Severity Scale (IHSS) and Sleep Inertia Questionnaire (SIQ); no new safety signals | 10/13/23 | Neurology/Psychiatric |
Johnson & Johnson, of New Brunswick, N.J. | Spravato (esketamine) | NMDA antagonist | Treatment-resistant depression | Data published in the New England Journal of Medicine from the phase IIIb Escape-TRD study showed treatment with Spravato was 1.54 times as likely to produce remission after 8 weeks compared to 8 weeks of treatment with quetiapine extended-release; relapse-free remission without treatment discontinuation at 32 weeks was 21.7% for Spravato compared to 14.1% for quetiapine XR | 10/4/23 | Neurology/Psychiatric |
Johnson & Johnson, of New Brunswick, N.J. | Spravato (esketamine) | NMDA antagonist | Treatment-resistant depression | Results from Escape-TRD study achieved 9-item patient health questionnaire (PHQ-9) scale-defined remission (56.8% vs. 43.3%; p<0.001) at week 8, with numbers increasing by week 32 (68.9% vs. 57.2%; p<0.01, respectively) compared to patients in the quetiapine arm | 10/9/23 | Neurology/Psychiatric |
Otsuka Pharmaceutical Co. Ltd., of Tokyo | Centanafadine | Oral serotonin-norepinephrine-dopamine triple-reuptake inhibitor | Attention deficit hyperactivity disorder | Top-line results in adolescents aged 13-17 met its primary endpoint by demonstrating improvements from baseline on the ADHD-RS-5 scale; statistically significant improvements compared to placebo for both the average effect of the high and low dose (p=0.0099) and for the high dose (p=0.0006) centanafadine-treated groups; low-dose centanafadine-treated group did not reach statistical significance | 10/27/23 | Neurology/Psychiatric |
Otsuka Pharmaceutical Co. Ltd., of Tokyo | Centanafadine | Oral serotonin-norepinephrine-dopamine triple-reuptake inhibitor | Attention deficit hyperactivity disorder | Top-line results in children aged 6-12 met its primary endpoint by demonstrating improvements from baseline on the ADHD-RS-5 scale; statistically significant improvements compared to placebo for both the average effect of the high and low dose (p=0.0039) and for the high dose (p=0.0008) centanafadine-treated group; low-dose centanafadine-treated group did not reach statistical significance; safety and tolerability results were consistent with the profile of centanafadine | 10/27/23 | Neurology/Psychiatric |
Reviva Pharmaceuticals Holdings Inc., of Cupertino, Calif. | Brilaroxazine | Serotonin-dopamine signaling modulator | Schizophrenia | In the Recover study, the 50-mg dose of brilaroxazine produced a -23.9-point improvement in the Positive and Negative Syndrome Scale total score compared to -13.8 points for placebo at week 4 (p<0.001); 15-mg dose was numerically superior to placebo | 10/30/23 | Neurology/Psychiatric |
UCB SA, of Brussels | Nayzilam (midazolam nasal spray) | Benzodiazepine receptor agonist | Epilepsy | Post-hoc analysis published in Epilepsy & Behavior showed median time to return to full baseline function (RTFBF), regardless of treatment with 1 or 2 doses, was 90 minutes; median time to RTFBF was stable over course of repeated, intermittent use | 10/31/23 | Neurology/Psychiatric |
Apellis Pharmaceuticals Inc., of Waltham, Mass. | Syfovre (pegcetacoplan injection) | Targets complement factor C3 | Geographic atrophy secondary to age-related macular degeneration | Data for 24 months of treatment published in The Lancet showed pegcetacoplan monthly and pegcetacoplan every other month slowed geographic atrophy lesion growth by 22% (p<0·0001) and 18% (p=0·0002) in the Oaks study and by 19% (p=0·0004) and 16% (p=0·0030) in the Derby study, respectively, compared with sham | 10/20/23 | Ocular |
Clearside Biomedical Inc., of Alpharetta, Ga | Arcatus (ARVN-001) | Triamcinolone acetonide injectable suspension for suprachoroidal use | Uveitic macular edema | Completed enrollment | 10/4/23 | Ocular |
Genentech Inc., a subsidiary of Roche Holding AG, of Basel, Switzerland | Vabysmo (faricimab) | Bispecific antibody for the eye targeting Ang-2 and VEGF-A | Retinal vein occlusion | In the Balaton and Comino studies, from week 24 to week 73, patients receiving Vabysmo extended their treatment intervals up to every 4 months while maintaining vision gains achieved in the first 24 weeks; data to be presented at an upcoming meeting | 10/9/23 | Ocular |
Innovent Biologics Inc., of Suzhou, China | IBI-302 | Ophthalmic recombinant human anti-VEGF and anti-complement bispecific fusion protein | Neovascular age-related macular degeneration | First patient dosed | 10/8/23 | Ocular |
Ocular Therapeutix Inc., of Bedford, Mass. | OTX-TKI | Axitinib intravitreal implant; VEGF inhibitor | Wet age-related macular degeneration | Initiated first pivotal trial | 10/3/23 | Ocular |
Oculis Holding AG, of Zug, Switzerland | OCS-01 | High-concentration, topical formulation of dexamethasone | Diabetic macular edema | In stage 1 of the Diamond study, Best Corrected Visual Acuity (BCVA) at week 6 was 7.2 letters for patients receiving OCS-01 compared to 3.1 for patients receiving vehicle (p=0.007); BCVA improvement at 12 weeks was 7.6 letters for OCS-01 and 3.7 letters for vehicle (p=0.016) | 10/10/23 | Ocular |
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | Eylea HD (aflibercept) | Targets vascular endothelial growth factor | Wet age-related macular degeneration | In the Pulsar study, 78% of patients were eligible for ?16-week dosing at the end of 2 years, including 53% eligible for ?20-week dosing intervals | 10/5/23 | Ocular |
Samsung Bioepis Co. Ltd., of Incheon, Korea | SB-15 | Proposed biosimilar to Eylea (aflibercept) | Neovascular age-related macular degeneration | Data showed safe and effective as Eylea; mean BVCA between the switching and the reference product groups was 65.3 at week 32 (prior to switching) and 65.8 at week 56 (post switching) vs. 65.2 at week 32 and 65.8 at week 56, respectively | 10/9/23 | Ocular |
Vyluma Inc., of Bridgewater, N.J. | NVK-002 | Low-dose atropine (0.01%) | Myopia | Doses of 0.01% and 0.02% concentrations continued to exhibit a strong safety profile with a low level (8% incidence) of treatment-emergent adverse events; no evidence of meaningful rebound in subjects who had discontinued active treatment and were washed out over 1 year; no evidence of tachyphylaxis in the treatment effect after 4 years | 10/11/23 | Ocular |
Boehringer Ingelheim GmbH, of Ingelheim, Germany, and Zealand Pharma A/S, of Copenhagen, Denmark | Survodutide (BI-456906) | Glucagon receptor/glucagon-like peptide-1 receptor agonist | Overweight or obesity | Phase III program consists of Synchronize-1 and Synchronize-2 in patients with comorbidities, including dyslipidemia, hypertension and obstructive sleep apnea; Synchronize-1 will enroll patients without type 2 diabetes, while Synchronize-2 will enroll patients with type 2 diabetes; primary endpoints of both studies are percent change in body weight at week 76 and the proportion of people who achieve body weight loss of 5% or more at week 76; a third study, Synchronize-CVOT, will enroll patients with cardiovascular disease, chronic kidney disease or risk factors for cardiovascular disease with a primary endpoint of time to first occurrence of 1 of 5 major adverse cardiac events | 10/5/23 | Other/Miscellaneous |
Novo Nordisk A/S, of Bagsvaerd, Denmark | Saxenda (liraglutide) | GLP-1 receptor agonist | Obesity | Started study in children aged 6 to 11 years of age | 10/20/23 | Other/Miscellaneous |
United Therapeutics Corp., of Silver Spring, Md. | Tyvaso (treprostinil) inhalation solution | PGI2 agonist | Progressive pulmonary fibrosis | First patient enrolled in the registration-phase Teton PPF study | 10/31/23 | Respiratory |
Abbisko Therapeutics Co. Ltd., of Shanghai | Pimicotinib (ABSK-021) | Oral small-molecule inhibitor of CSF-1R | Tenosynovial giant cell tumor | Enrollment ongoing in phase III study | 11/6/2023 | Cancer |
Adlai Nortye Biopharma Co. Ltd., of Hangzhou, China | Buparlisib (AN-2025) | Pan-PI3K inhibitor | Recurrent or metastatic head and neck squamous cell carcinoma | Completed patient enrollment | 11/17/2023 | Cancer |
Akeso Inc., of Hong Kong | Cadonilimab | PD-1/CTLA4 bispecific antibody | Unresectable locally advanced or metastatic gastric cancer or gastroesophageal junction adenocarcinoma | Interim analysis of AK104-302 study achieved its primary endpoint; statistically significant overall survival (OS) improvement in combination with chemotherapy; consistent safety profile | 11/7/2023 | Cancer |
Akeso Inc., of Hong Kong | Cadonilimab | PD-1/CTLA4 bispecific antibody | Recurrent/metastatic cervical cancer | Study in combination with platinum-based chemotherapy +/- bevacizumab met primary endpoints of progression-free survival (p<0.0001); overall survival not matured; no new safety signals | 11/27/2023 | Cancer |
Astrazeneca plc, of Cambridge, U.K. | Imfinzi (durvalumab) | Anti-PD-L1 antibody | Hepatocellular carcinoma | Emerald-1 study in combination with transarterial chemoembolization (TACE) and bevacizumab demonstrated a statistically significant and clinically meaningful improvement in the primary endpoint of progression-free survival (PFS) vs. TACE alone in patients; no new safety findings | 11/9/2023 | Cancer |
Astrazeneca plc, of Cambridge, U.K. | Imfinzi (durvalumab) | Anti-PD-L1 monoclonal antibody | Non-small-cell lung cancer | Pacific-2 trial in combination with chemoradiotherapy (CRT) did not achieve statistical significance for the primary endpoint of progression-free survival (PFS) vs. CRT alone for the treatment of patients with unresectable, stage III disease | 11/14/2023 | Cancer |
Clarity Pharmaceuticals Ltd., of Sydney | 64Cu-SAR-bisPSMA | PSMA imaging agent | Prostate cancer | Started registrational trial, Clarify, to assess diagnostic performance in detection of regional nodal metastasis in participants with high-risk prostate cancer prior to radical prostatectomy | 11/30/2023 | Cancer |
Coherus Biosciences Inc., of Redwood City, Calif., and Junshi Biosciences Co. Ltd., of Shanghai | Loqtorzi (toripalimab-tpzi) | Next-generation PD-1 monoclonal antibody that blocks PD-L1 and PD-L2 | Metastatic or recurrent locally advanced nasopharyngeal carcinoma | Final overall survival (OS) analysis of the Jupiter-02 trial showed first-line treatment with Loqtorzi plus chemotherapy significantly prolonged survival irrespective of PDL-1 status in results published in the Journal of the American Medical Association; the 2-year and 3-year OS rates were 78% vs. 65.1%, and 64.5% vs. 49.2%, respectively; consistent effect on OS observed in the majority of subgroups; superior progression-free survival (PFS) compared to chemotherapy alone, with a median PFS of 21.4 months vs. 8.2 months [HR=0.52 (95% CI: 0.37, 0.73)]; safety profile was consistent | 11/29/2023 | Cancer |
Curium Pharma Ltd., of London | 177Lu-PSMA L and T | Targeted therapy | Metastatic castration-resistant prostate cancer | Completed patient enrollment in Eclipse trial; enrolled 400 patients | 11/23/2023 | Cancer |
Ferring Pharmaceuticals A/S, of Saint-Prex, Switzerland | Adstiladrin (nadofaragene firadenovec) | Interferon alpha 2b gene stimulator | Non-muscle invasive bladder cancer | New 36-month follow-up data showed durable response; 25.5% (14/55 patients) remained free of high-grade recurrence; 90.4% 3-year overall survival rate, with more than half of cystectomy-free patients; follow-up analysis of study to continue with 5-year treatment and monitoring | 11/29/2023 | Cancer |
Gilead Sciences Inc., of Foster City, Calif. | Magrolimab | Monoclonal antibody that binds to CD47 | Acute myeloid leukemia | Discontinued phase III Enhance-2 study based on an ad hoc analysis and following review by an independent data monitoring committee | 11/7/2023 | Cancer |
GSK plc, of London | Blenrep (belantamab mafodotin) | Antibody-drug conjugate comprising a humanized BCMA monoclonal antibody covalently linked to the cytotoxic agent auristatin F via non-cleavable linker | Relapsed or refractory multiple myeloma | Trial met its primary endpoint of progression-free survival in combination with bortezomib plus dexamethasone (BorDex), significantly extended the time to disease progression or death vs. daratumumab plus BorDex; clinically meaningful overall survival (OS) trend (nominal p<0.0005) was also observed at the time of analysis | 11/27/2023 | Cancer |
IO Biotech ApS, of Copenhagen, Denmark | IO102-IO103 | Therapeutic peptide vaccine targeting indoleamine 2,3-dioxygenase and anti-programmed death ligand 1 | Advanced melanoma | Completed enrollment of 380 patients in the study testing O102-IO103 plus Keyturda (pembrolizumab, Merck & Co. Inc.); interim analysis expected in mid-2024; data for primary endpoint of progression-free survival expected in the second half of 2025 | 11/10/2023 | Cancer |
Karyopharm Therapeutics Inc., of Newton, Mass. | Selinexor | Oral exportin 1 (XPO1) inhibitor | Advanced or recurrent TP53 wild-type endometrial cancer | Exploratory subgroup analysis in TP53 wild-type patients as maintenance therapy had immature survival data; median overall survival not reached in either arm after a median follow-up of 28.9 months; improvements in median progression-free survival for the intent-to-treat population but were not clinically meaningful; no new safety signals | 11/6/2023 | Cancer |
Panbela Therapeutics Inc., of Minneapolis | SBP-101 (ivospemin) | Polyamine analogue designed to induce polyamine metabolic inhibition | Metastatic pancreatic ductal adenocarcinoma | Independent data safety monitoring board completed its prespecified review of Aspire trial; recommended study to continue without modification | 11/29/2023 | Cancer |
Sellas Life Sciences Group Inc., of New York | Galinpepimut-S | WT1-targeting peptide immunotherapeutic | Acute myeloid leukemia | Reached target patient enrollment outside of mainland China in Regal trial | 11/29/2023 | Cancer |
Servier SAS, of Paris | Vorasidenib | Oral, brain-penetrant dual inhibitor of mutant IDH1 and IDH2 | IDH1/2-mutant diffuse glioma | Indigo study of vorasidenib reduced tumor growth as measured by a blinded independent radiology committee; improvement in progression-free survival with a median of 27.7 months for vorasidenib vs. 11.1 months for placebo; tumor volume decreased by a mean of 2.5% every 6 months, while tumor volume increased by a mean of 13.9% every 6 months for patients randomized to the placebo arm | 11/18/2023 | Cancer |
Sol-Gel Technologies Ltd., of Ness Ziona, Israel | SGT-610 (patidegib gel, 2%) | Topical hedgehog inhibitor | Gorlin syndrome | Started trial evaluating drug in preventing new basal cell carcinoma lesions in Gorlin syndrome patients | 11/30/2023 | Cancer |
Springworks Therapeutics Inc., of Stamford, Conn. | Nirogacestat | Oral gamma secretase inhibitor | Desmoid tumors | Additional data from the phase III DeFi trial showed significantly improved mean physical functioning score from baseline per the GODDESS DTIS PF domain compared with placebo at the prespecified time point; significantly improved mean physical functioning score from baseline per the EORTC QLQ-C30 PF subscale compared with placebo; improved mean role functioning score from baseline per the EORTC QLQ-C30 RF subscale compared with placebo; clinically meaningful improvement in physical functioning | 11/1/2023 | Cancer |
Xspray Pharma AB., of Stockholm | XS-003 | Amorphous non-crystalline nilotinib | Chronic myeloid leukemia | Data showed bioavailability within the 80-125% range to Tasigna following oral administration with significantly lower dose | 11/21/2023 | Cancer |
Aerovate Therapeutics Inc., of Waltham, Mass. | AV-101 | Dry powder inhaled formulation of imatinib | Pulmonary arterial hypertension | First patient enrolled in phase III study | 11/20/2023 | Cardiovascular |
Bayer AG, of Berlin | Asundexian (BAY-2433334) | Oral direct inhibitor of activated coagulation factor XI | Atrial fibrillation | Initiated a third study, Oceanic-Afina, in patients 65 and older with atrial fibrillation at high risk for stroke or systemic embolism who are deemed ineligible for oral anticoagulation treatment due to an increased risk of bleeding | 11/6/2023 | Cardiovascular |
Bayer AG, of Berlin | Asundexian (BAY-2433334) | Oral direct inhibitor of activated coagulation factor XI | Atrial fibrillation | Independent data monitoring committee recommended to stop Oceanic-AF study due to an inferior efficacy of asundexian vs. control arm; recommended to continue Oceanic-Stroke phase III study as planned | 11/19/2023 | Cardiovascular |
Bridgebio Pharma Inc., of Palo Alto, Calif. | Acoramidis | Next-generation, oral, small-molecule stabilizer of transthyretin | Transthyretin amyloid cardiomyopathy | Attribute-CM data, at month 30, showed a highly statistically significant result demonstrated by a win ratio of 1.8 (p<0.0001) on the primary endpoint, a hierarchical analysis using the Finkelstein-Schoenfeld test prioritizing all-cause mortality (ACM), then cardiovascular-related hospitalization (CVH), then change from baseline in NT-proBNP, then change from baseline in 6MWD; the Kaplan-Meier composite ACM and CVH time-to-first event curves for treatment and placebo arms separated beginning at month 3 and continued to diverge through month 30 with a hazard ratio of 0.645 (p=0.0008) | 11/12/2023 | Cardiovascular |
Lexicon Pharmaceuticals Inc., of The Woodlands, Texas | Inpefa (sotagliflozin) | Oral inhibitor of SGLT2 and SGLT1 | Heart failure and major adverse cardiovascular events | A post-hoc analysis of data from the Scored trial demonstrated clinical benefit in HF and MACE related outcomes observed as early as 3 months in patients at high risk for cardiovascular events; for total cardiovascular deaths, hospitalizations for HF, or urgent visits for HF, the primary endpoint of the trial, the relative risk reduction was 26% (p<0.001); a statistically significant sustained reduction in the primary endpoint was seen at 95 days after randomization; for MACE, a secondary endpoint, relative risk reduction was 23% (p<0.001); a statistically significant sustained reduction in MACE was seen at 94 days after randomization | 11/12/2023 | Cardiovascular |
Lib Therapeutics Inc., of Cincinnati | Lerodalcibep | Third-generation PCSK9 inhibitor | Cardiovascular disease | Completed Liberate-CVD and Liberate-HR registrational-enabling studies for patients with CVD for at very high risk of CVD | 11/14/2023 | Cardiovascular |
Novo Nordisk A/S, of Copenhagen, Denmark | Wegovy (semaglutide, 2.4 mg) | GLP-1 receptor agonist | Obesity and overweight | Full Select cardiovascular outcomes results in overweight or obese patients without diabetes who have established cardiovascular disease showed Wegovy reduced the risk of major adverse cardiovascular events (MACE) by a statistically significant 20% consistently across age, gender, ethnicity and starting body mass index subgroups; significance was seen in reducing the risk of nonfatal heart attack by 28% compared to placebo; 2 other components of MACE did not show statistical significance, with Wegovy reducing cardiovascular death by 15% and nonfatal stroke by 7%; confirmatory secondary endpoints missed significance, showing the risk of composite heart failure events, including cardiovascular death, urgent heart failure visits and hospitalizations, were reduced by 18% vs. placebo; risk of death from any cause was reduced by 19% | 11/11/2023 | Cardiovascular |
Bio-Thera Solutions Ltd., of Guangzhou, China | BAT-8006 | Biosimilar of anti-IL-12 and anti-IL-23 human monoclonal antibody Stelara (ustekinumab, Johnson & Johnson( | Moderate to severe plaque psoriasis | Study met its primary endpoint, improvement from baseline in Psoriasis Area and Severity Index (PASI) score to week 12; also showed similar efficacy and comparable safety profile | 11/29/2023 | Dermatologic |
Dermata Therapeutics Inc., of San Diego | DMT-310 | Moderate-to-severe facial acne | Moderate to severe facial acne | Plans to start enrolling patients in the 550-patient STAR-1 study in December 2023; primary endpoints are the mean change from baseline in inflammatory and noninflammatory lesion counts and the Investigator Global Assessment response rate | 11/16/2023 | Dermatologic |
Inflarx NV, of Jena, Germany | Gohibic (vilobelimab) | Monoclonal anti-human complement factor C5a antibody | Ulcerative pyoderma gangrenosum | First patient dosed | 11/6/2023 | Dermatologic |
Novartis AG, of Basel, Switzerland | Remibrutinib | Bruton’s tyrosine kinase inhibitor | Chronic spontaneous urticaria | In the Remix-1 and -2 studies, remibrutinib was superior to placebo in weekly urticaria activity, itch and hives at week 12 | 11/9/2023 | Dermatologic |
Amarin Corp. plc, of Dublin, and Bridgewater, N.J. | Vascepa/Vazkepa (icosapent ethyl; IPE) | Eicosapentaenoic acid | Metabolic syndrome | Reduce-IT results showed that, among statin-treated patients in a subgroup with a history of metabolic syndrome but without diabetes at baseline (n=2,866), treatment with IPE with a median follow-up of 4.9 years led to a 29% relative risk reduction for the primary composite endpoint - cardiovascular death, nonfatal myocardial infarction (MI), nonfatal stroke, coronary revascularization (RV) or unstable angina resulting in hospitalization (H) (p<0.0001); it also showed a 41% reduction in total events (p<0.0001) vs. placebo; risk for cardiovascular death, nonfatal myocardial infarction or nonfatal stroke was reduced by 20% (p=0.05); 27% reduction in fatal/nonfatal MI (p=0.03); 47% reduction in urgent/emergent RV (p<0.0001); 58% reduction in H for unstable angina (p<0.0001); non-statistically significant reductions of 44% for cardiac arrest and 34% for sudden cardiac death | 11/12/2023 | Endocrine/Metabolic |
Minoryx Therapeutics SL, of Barcelona, Spain | Leriglitazone | PPAR? agonist | Cerebral adrenoleukodystrophy | Enrolled first of 40 patients in the study; primary endpoint is survival; secondary endpoints include the Loes score, major functional disabilities, activities of daily living and major neurocognitive impairment; data expected by early 2026 | 11/16/2023 | Endocrine/Metabolic |
Pharming Group NV, of Leiden, the Netherlands | Leniolisib | Oral small-molecule PI3K-delta inhibitor | Activated PI3K delta syndrome in children ages 1 to 6 years | First patient dosed | 11/21/2023 | Endocrine/Metabolic |
Everest Medicines Ltd., of Shanghai | Etrasimod | Once-daily, oral, sphingosine 1-phosphate (S1P) receptor modulator | Moderate to severe active ulcerative colitis | Top-line data of etrasimod resulted in a clinically meaningful and statistically significant improvement in the primary endpoint and all key secondary and other secondary endpoints (including mucosal healing, symptomatic remission and endoscopic normalization) after the 12-week induction treatment period; well-tolerated at 2 mg; no new safety findings | 11/30/2023 | Gastrointestinal |
Index Pharmaceuticals Holding AB, of Stockholm | Cobitolimod | TLR9 agonist | Moderate to severe left-sided ulcerative colitis | Independent data monitoring committee completed the planned dose selection analysis including safety review and assessment for futility; did not meet the primary endpoint; discontinued phase III study | 11/21/2023 | Gastrointestinal |
Index Pharmaceuticals Holding AB, of Stockholm | Cobitolimod | TLR9 agonist | Moderate to severe left-sided ulcerative colitis | Discontinuing phase III program after the independent data monitoring committee advised the company that the Induction Study I was unlikely to meet its primary endpoint | 11/21/2023 | Gastrointestinal |
Intercept Pharmaceuticals Inc., a unit of Alfasigma SpA, of Bologna, Italy | Obeticholic acid | Semi-synthetic bile acid derivative | Primary biliary cholangitis | Sub-analysis of the the Poise study showed patients achieved gamma-glutamyl transferase levels of <3.2× the upper limit of normal (ULN) and alkaline phosphatase levels of <1.5× the ULN | 11/10/2023 | Gastrointestinal |
Ipsen SA, of Paris, and Genfit SA, of Lille, France | Elafibranor | Oral, dual PPAR ?,? agonist | Primary biliary cholangitis | Full results from pivotal Elative trial show statistically significant improvements in biomarkers of disease progression across key endpoints with a significant treatment benefit achieved in the primary composite endpoint, demonstrating 47% placebo-adjusted difference (p<0.001) between patients on elafibranor 80 mg (51%) vs. patients on placebo (4%) achieving a biochemical response; data also published in The New England Journal of Medicine | 11/13/2023 | Gastrointestinal |
Madrigal Pharmaceuticals Inc., of Conshohocken, Pa. | Resmetirom | THR-? agonist | Nonalcoholic steatohepatitis | In the Maestro-NASH study, >70% of patients treated with resmetirom 100 mg experienced a ?30% reduction in magnetic resonance imaging-proton density fat fraction (MRI-PDFF); median reduction in MRI-PDFF was 52% | 11/10/2023 | Gastrointestinal |
Mirum Pharmaceuticals Inc., of Foster City, Calif. | Livmarli (maralixibat) | Ileal bile acid transporter inhibitor | Progressive familial intrahepatic cholestasis | Data from March and March-on extension trials showed significant and sustained improvements in pruritus severity, serum bile acid levels, total bilirubin and growth, with up to 2 years of treatment across broad range of genetic PFIC types; no new safety signals observed | 11/14/2023 | Gastrointestinal |
Astellas Pharma Inc., of Tokyo | Fezolinetant | Oral, nonhormonal treatment; blocks neurokinin B binding on kisspeptin/neurokinin/dynorphin neuron | Moderate to severe vasomotor symptoms associated with menopause | Data from 24-week phase IIIb Daylight trial in women considered unsuitable for hormone therapy showed statistically significant reduction of -1.93 (p<0.001) compared to placebo on primary endpoint of mean change from baseline in frequency of moderate to severe VMS; for key secondary endpoint of mean change from baseline in severity of moderate to severe VMS, data showed statistically significant reduction of -0.39 (p<0.001) vs. placebo; statistically significant reduction of -2.5 (p<0.001) vs. on mean change from baseline in patient-reported sleep disturbance | 11/30/2023 | Genitourinary/Sexual Function |
Bayer AG, of Leverkusen, Germany | Kerendia (finerenone) | Nonsteroidal mineralocorticoid receptor antagonist | Chronic kidney disease in patients with type 2 diabetes | Interim results showed 14% of patients treated with Kerendia reported hyperkalemia vs. 6.9% treated with placebo; treatment was continued in 92.3% of patients; interrupted or discontinued in 5.4% and 1% of patients, respectively | 11/4/2023 | Genitourinary/Sexual Function |
Idorsia Ltd., of Allschwil, Switzerland | Aprocitentan | Dual endothelin receptor antagonist | Stage 3 or 4 chronic kidney disease | Data showed both the 12.5-mg and 25-mg doses of aprocitentan resulted in blood pressure reduction from baseline to week 4 compared to placebo; urinary albumin-to-creatinine ratio reduced by 28% at 12.5 mg, 44% for aprocitentan 25 mg, and remained stable (reduction of 4%) in the placebo group; well-tolerated; adverse events as edema/fluid retention (18% and 24% of patients receiving aprocitentan 12.5 mg and 25 mg, respectively, vs. 2% with placebo, at week 4) | 11/3/2023 | Genitourinary/Sexual Function |
Travere Therapeutics Inc., of San Diego | Filspari (sparsentan) | Endothelin and angiotensin II receptor antagonist | IgA nephropathy | Filspari was well-tolerated in Protect study; achieved complete remission of proteinuria of less than 0.3 grams compared to those treated with irbesartan (31% vs. 11%); slowest rates of kidney function decline; lower rates of the composite endpoint of 40% decline in eGFR, kidney failure or death compared to irbesartan; mean reduction in proteinuria from baseline of 43% compared to 4% for irbesartan-treated patients; no drug-induced liver injury and no fluid overload; results published in the The Lancet | 11/3/2023 | Genitourinary/Sexual Function |
Travere Therapeutics Inc., of San Diego | Filspari (sparsentan) | Endothelin and angiotensin II receptor antagonist | Focal segmental glomerulosclerosis | Duplex study demonstrated a clinically meaningful and durable reduction in proteinuria; 50% reduction from baseline compared to a 32% reduction with the active control irbesartan; consistent and sustained achievement of complete remission of proteinuria in 18.5% of patients on sparsentan vs. 7.5% for irbesartan; confirmed 50% reduction in eGFR; well-tolerated; consistent safety profile; no drug-induced liver injury and no fluid overload; results published in The New England Journal of Medicine | 11/3/2023 | Genitourinary/Sexual Function |
Argenx SE, of Amsterdam | Vygart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) | Subcutaneous combination of IgG1 antibody fragment targeting the neonatal Fc receptor and hyaluronidase PH20 | Primary immune thrombocytopenia | The Advance-SC study didn’t meet primary endpoint with 13.7% of 124 patients treated with Vygart Hytrulo having a sustained platelet count response compared to 16.2%of 68 patients who took placebo (p=0.5081); secondary endpoints also weren’t met | 11/28/2023 | Hematologic |
Morphosys AG, of Munich | Pelabresib (CPI-0610) | BET inhibitor | Myelofibrosis | Manifest-2 study achieved primary endpoint in combination with ruxolitinib compared with placebo plus ruxolitinib in JAK inhibitor-naïve patients; statistically significant and clinically meaningful improvement in the proportion of patients achieving at least a 35% reduction in spleen volume (SVR35) at week 24; 50% reduction in total symptom score (TSS50 p=0.216) and absolute change in total symptom score (TSS) from baseline at week 24 (p=0.0545); TSS50 was achieved by 55% of patients in the pelabresib and ruxolitinib treatment arm at week 24 compared with 45% in the placebo plus ruxolitinib arm (p<0.05) in intermediate-risk patients; greater proportion of patients achieved hemoglobin response (? 1.5 g/dL from baseline) with the pelabresib and ruxolitinib combination than with placebo and ruxolitinib; well-tolerated; no new safety signals | 11/20/2023 | Hematologic |
Astrazeneca plc, of Cambridge, U.K. | Saphnelo (anifrolumab) | Monoclonal antibody targeting subunit 1 of the type I interferon receptor | Systemic lupus erythematosus | Post-hoc analysis of Saphnelo achieved remission vs. standard therapy alone; 33% of patients treated with Saphnelo were in remission compared with 21.4%; increase from 18.7% of patients treated with Saphnelo who achieved remission at the end of the first year compared with 8.6%; greater percentage of time in remission (17.2% vs. 8.3%, p=0.0022) and were more likely to sustain remission for 3 or more visits compared with standard therapy alone (30.2% vs. 17.2% p=0.0127) | 11/7/2023 | Immune |
DBV Technologies SA, of Montrouge, France | Viaskin Peanut 250-?g patch (DBV-712) | Epicutaneous patch | Peanut allergy | In the Epopex open-label extension of the Epitope study in toddlers, 81.3% of patients who completed the oral food challenge reached an eliciting dose of ?1,000 mg after 24 months of treatment; 55.9% of patients completed the oral food challenge at a cumulative dose of 3,444 mg without meeting stopping criteria; patients in the treatment arm of Epitope reported no treatment-related anaphylactic or serious treatment-related adverse events in the second year of treatment | 11/9/2023 | Immune |
Janssen Pharmaceuticals Inc., a unit of New Brunswick, N.J.-based Johnson & Johnson | Tremfya (guselkumab) | Monoclonal antibody targeting IL-23 p19 | Psoriatic arthritis | Analysis of Discover-1, Discover-2 and Cosmos study changes in PsA-5T-Ds score through week 24 correlated strongly with variations in PASDAS (p<0.0001) and moderately with variations in Disease Activity Index for PsA (DAPSA), clinical DAPSA and 36-Item short form survey (SF-36) physical component summary scores (p<0.0001) | 11/9/2023 | Immune |
Medeor Therapeutics Inc., of South San Francisco | MDR-101 | Allogeneic HLA-matched living donor hematopoietic stem cell therapy | Kidney transplant rejection | Study achieved primary endpoint; 63% of the study participants completed the trial and have been off immunosuppression therapy for 2 years; 4 additional patients have less than 6 months to complete the trial and currently remain free of immunosuppression therapy; 3 patients resumed immunosuppression therapy during the trial, and 1 patient withdrew from the study at 6 months | 11/2/2023 | Immune |
Stallergenes Greer plc, of London | Palforzia | Defatted powder of Arachis hypogaea L., semen (peanuts) | Peanut allergy in children ages 1 to 3 years | Results showed 73.5% of patients treated with Palforzia met the primary endpoint of tolerating the 600-mg single dose of peanut protein at 12 months compared with 6.3% of patients receiving placebo; favorable safety profile; 84.7% of patients on Palforzia and 93.8% of patients on placebo completed the study; no serious events; treatment-related systemic allergic reactions occurred in 2% vs. 0% in placebo | 11/20/2023 | Immune |
Gilead Sciences Inc., of Foster City, Calif. | Obeldesivir | Viral RNA polymerase | Non-hospitalized participants who are at high risk for developing severe COVID-19 | Discontinued phase III Birch study; decision was based on lower-than-expected COVID-19 incidence rates and related hospitalizations or all-cause death by day 29 and does not reflect any safety or efficacy concerns | 11/7/2023 | Infection |
Innoviva Inc., of Burlingame, Calif. | Zoliflodacin | Antibiotics | Gonorrhoeae infection | Top-line data showed study met its primary endpoint; statistical noninferiority of microbiological cure at the urogenital site when compared to treatment with intramuscular injection of ceftriaxone and oral azithromycin; favorable safety profile and well-tolerated; mild to moderate adverse events; no discontinuations reported due to adverse events, serious adverse events or deaths | 11/2/2023 | Infection |
Junshi Biosciences Co. Ltd., of Shanghai | VV-116 /mindeudesivir hydrobromide/JT-001 | Oral nucleoside analogue; anti-SARS-CoV-2 drug | Mild to moderate COVID-19 | VV-116 significantly reduced the time to sustained clinical symptom resolution compared to placebo; no observed safety concerns; superior to placebo in reducing the time to sustained clinical symptom resolution among 1,229 patients (p=0.0023); substantial reduction in the time to sustained clinical symptom resolution was observed with VV-116 compared to placebo (p=0.0009); time to sustained clinical symptom resolution was also shorter in the VV-116 group compared to the placebo group in elderly patient subgroup (?60 years old); results published in Lancet Infectious Diseases | 11/23/2023 | Infection |
Merck & Co. Inc., of Rahway, N.J. | V-116 | 21-valent pneumococcal conjugate vaccine | Pneumococcal disease prophylaxis | In the Stride-3 study, V-116 was non-inferior to PCV-20 for the 10 serotypes common to both vaccines as assessed by serotype-specific opsonophagocytic activity (OPA) geometric mean titers (GMTs) 30 days post-vaccination; V-116 was superior to PCV-20 for 10 of 11 serotypes included in V-116 but not in PCV-20 as assessed by serotype-specific OPA GMTs 30 days post-vaccination and the proportions of patients with a greater than or equal to 4-fold increase in OPA from day 1 to day 30 | 11/28/2023 | Infection |
Valneva SE, of Saint-Herblain, France | VLA-1553 | Vaccine | Chikungunya virus | Data in adolescents showed a single-dose induced levels of protective antibody titers in 98.8% of participants, ages 12-17, 28 days after vaccination; it was generally well-tolerated and showed a similar safety profile as was seen in adults | 11/13/2023 | Infection |
Biosplice Therapeutics Inc., of San Diego | Lorecivivint | Structure-modifying drug | Osteoarthritis of the knee | Patients who received 3 annual injections and completed the OA-07 extension trial showed an absolute decline in medial joint space width of 0.06 mm vs. baseline over 36 months; those on placebo after 2 years saw a decline of 0.21 mm; a significant 0.15-mm difference is seen (p=0.045); structural benefits were more pronounced in patients with less severe disease | 11/13/2023 | Inflammatory |
Ingeneron Inc., of Houston | Transpose RT system | Cell therapy platform consists of a processing unit, a set of disposables, and Matrase | Knee osteoarthritis | Study showed no serious treatment-related adverse events from 109 patients; results published in the journal Nature Medicine | 11/21/2023 | Inflammatory |
Ingeneron Inc., of Houston | ADRCs | Adipose-derived regenerative cells; cell therapy | Partial-thickness rotator cuff tears | Results from randomized, controlled study, which followed patients for 3.4 years after treatment, showed ADRCs safe and more effective than corticosteroid injections for improving shoulder function and reducing pain | 11/14/2023 | Musculoskeletal |
Tonix Pharmaceuticals Holding Corp., of Chatham, N.J. | TNX-102 SL (cyclobenzaprine) | Sublingual formulation of the drug that activates the 5-HT2A-serotonergic, ?1-adrenergic, H1-histaminergic, and M1-muscarinic receptors | Fibromyalgia | Completed the clinical phase of the 457-patient Resilient study; top-line data expected in late December 2023 | 11/15/2023 | Musculoskeletal |
Abbvie Inc., of North Chicago, Ill | Ubrelvy (ubrogepant 100 mg) | Orally administered calcitonin gene-related peptide (CGRP) receptor antagonist (gepant) | Acute treatment of migraine when administered during the prodrome of a migraine attack | Ubrelvy significantly reduced the likelihood of development of moderate or severe headache and reduced functional disability compared to placebo within 24 hours post-dose; absence of moderate or severe intensity headache within 24 hours was achieved following 46% of qualifying prodrome events when treated with Ubrelvy vs. 29% of placebo-treated events (p<0.0001); absence of moderate or severe intensity headache within 48 hours was achieved following 41% of qualifying prodrome events when treated with Ubrelvy vs. 25% of placebo-treated events (p<0.0001); more patients had normal function within the 24 hours after treatment of qualifying prodrome events (p<0.0001); absence of headache of any intensity within 24 hours was achieved following 24% of qualifying prodrome events when treated with Ubrelvy vs. 14% of placebo-treated events (p<0.0001) | 11/17/2023 | Neurology/Psychiatric |
Acadia Pharmaceuticals Inc., of San Diego | ACP-101 (carbetocin nasal spray) | Oxytocin receptor agonist | Hyperphagia in Prader-Willi syndrome | Initiated Compass PWS study to enroll about 170 children and adults, ages 5-30; primary endpoint is change from baseline to week 12 on the hyperphagia questionnaire for clinical trials score | 11/30/2023 | Neurology/Psychiatric |
Aribio Co. Ltd., of Seongnam-Si, South Korea | AR-1001 (mirodenafil dihydrochloride) | PDE5 inhibitor | Early Alzheimer’s disease | Expanding the AR1001-ADP3-US01 study to EU, U.K., Korea and China | 11/14/2023 | Neurology/Psychiatric |
Biovie Inc., of Santa Monica, Calif. | NE-3107 | Oral small-molecule, blood-brain permeable, anti-inflammatory, insulin sensitizing and ERK-binding properties | Alzheimer’s disease | Efficacy data showed improved performance on all cognitive and functional assessments, although the data missed statistical significance due to site exclusions; sponsor identified issues relating to GCP violations and protocol deviations (only a subset of enrolled patients to be included); patients experienced a treatment advantage after 6 months; decreased neuroinflammatory processes; average of -5.66 years of age deceleration of the DNA methylation advantage; company intends to work with U.S. FDA to employ the adaptive trial feature of the protocol to continue enrolling patients to achieve statistical significance | 11/29/2023 | Neurology/Psychiatric |
Cassava Sciences Inc., of Austin, Texas | Simufilam | Restores shape and function of altered filamin A | Alzheimer’s disease | Completed patient enrollment; total of 1,929 patients were randomized in 2 ongoing phase III studies | 11/6/2023 | Neurology/Psychiatric |
Compass Pathways plc, of London | COMP-360 | Psilocybin | Treatment-resistant depression | Expanding the COMP 005 and COMP 006 studies into the U.K. | 11/15/2023 | Neurology/Psychiatric |
Merck & Co. Inc., of Kenilworth, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 antibody | Renal cell carcinoma | Prespecified interim analysis from pivotal phase III Keynote-564 study met its key secondary endpoint of overall survival (OS); statistically significant and clinically meaningful improvement in OS compared to placebo; no new safety signals | 11/1/2023 | Neurology/Psychiatric |
Neurocrine Biosciences Inc., of San Diego | Ingrezza (valbenazine) | VMAT2 inhibitor; oral granules; sprinkle formulation | Chorea associated with Huntington's disease | Interim results from the ongoing open-label KINECT-HD2 study showed meaningful and sustained improvements in subjects; improved chorea at the first evaluation at week 2 at 40-mg dose, and efficacy sustained through week 50 at ? 80 mg; consistent adverse events in KINECT-HD study | 11/2/2023 | Neurology/Psychiatric |
Otsuka Pharmaceutical Co. Ltd., of Tokyo, and H. Lundbeck A/S, of Valby, Denmark | Rexulti (brexpiprazole) | Partial agonist of serotonin 5-HT1A and dopamine D2 receptors | Agitation associated with Alzheimer’s dementia | Rexulti showed a significant decrease in agitation symptoms; efficacy as measured by a 22.6-point reduction in participants mean CMAI total scores (p=0.003) vs.placebo; improvement in secondary endpoints; aggressive behavior (p= 0.004), physically nonaggressive behavior (restlessness, p=0.03), and verbally agitated behavior (p=0.01); well-tolerated; 86.8% of the brexpiprazole group and 88.9% of the placebo group completing trial; results published in JAMA Neurology | 11/6/2023 | Neurology/Psychiatric |
Pharma Two B Ltd., of Rehovot, Israel | P2B-001 | Fixed dose, extended-release combination of pramipexole 0.6 mg and rasagiline 0.75 mg | Parkinson’s disease | Data published in Movement Disorders showed least squares mean in the Unified Parkinson’s Disease Rating Scale at week 12 was –2.66 points for P2B-001 compared to pramipexole ER (p=0.0018) and –3.30 points for P2B-001 compared to rasagiline ER (p=0.0001); change for P2B-001 compared to PramiER was not significantly different (–8.35 and 7.98, respectively, p=0.7197) | 11/9/2023 | Neurology/Psychiatric |
Teva Pharmaceuticals Ltd., of Tel Aviv-Yafo, Israel, and Medincell SA, of Jacou, France | Uzedy | Extended-release injectable suspension of risperidone | Schizophrenia | Uzedy significantly prolonged time to impending relapse by 5 times with once-monthly dosing (p<0.0001) and 2.7 times with once-every-2-months dosing (p<0.0001) vs. placebo, with decreased risk of relapse by 80% and 62.5%, respectively; consistent safety profile; results published in The Lancet Psychiatry | 11/1/2023 | Neurology/Psychiatric |
Adverum Biotechnologies Inc., of Redwood City, Calif. | ADVM-022 (ixoberogene soroparvovec/ixo-vec) | AAV.7m8, carrying an aflibercept coding sequence | Wet age-related macular degeneration | Optic extension study results through 3 years of follow-up showed 84% reduction in annualized anti-VEGF injections at 2E11 dose; 53% of the participants at the 2E11 dose receiving no supplemental injections through 3 years; aflibercept protein levels have been sustained through follow-up, which is up to 4.5 years post-treatment; BCVA was maintained and CST was improved through 3 years; well-tolerated | 11/4/2023 | Ocular |
Apellis Pharmaceuticals Inc., of Waltham, Mass. | Syfovre (pegcetacoplan injection) | Targets complement factor C3 | Geographic atrophy secondary to age-related macular degeneration | Results from 3 years of continuous treatment showed Syfovre reduced GA lesion growth with both monthly (35%; p<0.0001) and every-other-month (EOM) (24%; p<0.0001) treatment compared to the projected sham arm; reduced nonsubfoveal GA lesion growth with both monthly (42%; p<0.0001) and EOM (28%; p=0.0015) treatment compared to the projected sham arm; reduced GA lesion growth by 19% (p<0.0001) after 1 year of treatment (combined monthly and EOM), compared to the sham treatment period, in patients who crossed over from the sham group; 1 serious adverse event of ischemic optic neuropathy | 11/4/2023 | Ocular |
Astellas Pharma Inc., of Tokyo, and Iveric Bio Inc., of Parsippany, N.J. | Izervay (avacincaptad pegol intravitreal solution) | Complement C5 inhibitor | Geographic atrophy secondary to age-related macular degeneration | Izervay every month demonstrated a statistically significant year-over-year reduction of 14% in the mean rate of GA growth at 2 years from baseline vs. sham (p=0.0165) through 2 years; reduction of 19% in the mean GA growth rate at 2 years vs. sham (nominal p=0.0015) in every other month; reduced the rate of ?15-letter persistent vision loss compared to sham over 2 years was not statistically significant; well-tolerated over 2 years; 1 case each of non-serious intraocular inflammation and culture-positive endophthalmitis; no cases of ischemic neuropathy or retinal vasculitis | 11/4/2023 | Ocular |
Kodiak Sciences Inc., of Palo Alto, Calif. | Tarcocimab tedromer (KSI-301) | Anti-VEGF antibody biopolymer conjugate | Non-proliferative diabetic retinopathy | Glow study met primary and secondary endpoints with high statistical significance; 2-step improvement on the Diabetic Retinopathy Severity Scale (DRSS) score; achieved a 29-fold increased response rate ratio; 41.1% of evaluable patients on tarcocimab with 2-step improvement vs. 1.4% of evaluable patients in the sham group (p<0.0001); improved and stable visual acuity and retinal anatomy; greater reductions in the proportion of patients developing sight-threatening complication, with 89% decreased risk, achieving 21% vs. 2.3% (p<0.0001); 95% risk reduction in the development of macular edema vs. sham, from 13.7% on sham vs. 0.7% on tarcocimab; rates of serious ocular adverse events and intraocular inflammation in patients treated with tarcocimab and sham were similar in both groups | 11/6/2023 | Ocular |
Galderma SA, of Zug, Switzerland | RelabotulinumtoxinA | Liquid botulinum toxin A | Glabellar lines | Top-line results from 2 phase IIIb trials showed both met primary endpoints, demonstrating significant improvement in both frown lines and crow’s feet, with rapid onset of action as early as day 1 and long duration | 11/30/2023 | Other/Miscellaneous |
Soleno Therapeutics Inc., of Redwood City, Calif. | DCCR | Extended-release tablets of diazoxide choline | Prader-Willi syndrome | Destiny study showed statistically significant, clinically meaningful decreases in HQ-CT total score from baseline in overall population after receiving DCCR for 52 weeks (p<0.0001); significant, clinically meaningful decrease in HQ-CT total score from baseline also observed after receiving DCCR for 13, 26 and 39 weeks (all p<0.0001); significantly greater reduction in HQ-CT score compared to the overall population (p<0.0001) in participants with severe hyperphagia at baseline; statistically significant improvements in all behavioral domains (p<0.0001); significant improvements in lean body mass-to-fat mass ratio were seen (p=0.0005); consistent with increases in lean body mass (p<0.0001) and no significant changes in body fat mass; significant reductions in serum leptin (p<0.0001) and insulin (p=0.0004), and improvement in Homeostatic Model Assessment for Insulin Resistance (HOMA-IR) (p=0.0033); significant increase in adiponectin was observed (p<0.0001); significant reduction in disease severity (p<0.0001 in both clinicians and caregivers using Clinical Global Impression of Severity and Caregiver Global Impression of Severity scores); well-tolerated; grade 1 adverse events; results published in Obesity | 11/6/2023 | Other/Miscellaneous |
GSK plc, of London | Ventolin (salbutamol) | low carbon version of its metered dose inhaler | Respiratory disorders | Planning to start phase III study; dosing of first patient planned in the first half of 2024 | 11/21/2023 | Respiratory |
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and Sanofi SA, of Paris | Dupixent (dupilumab) | Fully human monoclonal antibody that inhibits the signaling of the IL-4 and IL-13 pathways | Chronic obstructive pulmonary disease | Trial met its primary endpoint; 34% reduction in moderate or severe acute COPD exacerbations over 52 weeks (p=0.0002); improved lung function from baseline by 139 mL at 12 weeks compared to 57 mL for placebo (p=0.0001), with the benefit vs. placebo sustained at week 52 (115 mL for Dupixent vs. 54 mL for placebo, p=0.0182); consistent safety profile | 11/27/2023 | Respiratory |
Aura Biosciences Inc., of Boston | Belzupacap sarotalocan (bel-sar; AU-011) | Virus-like particle conjugated with an anticancer agent | Early-stage choroidal melanoma | First patient dosed | 12/7/2023 | Cancer |
Bristol Myers Squibb Co., of New York | Relatlimab and Opdivo (nivolumab) | LAG-3-blocking antibody and PD-1-blocking antibody | Microsatellite stable metastatic colorectal cancer | Discontinued Relativity-123 trial due to futility based on a planned analysis conducted by an independent data monitoring committee; unlikely to meet its primary endpoints upon completion; consistent safety profile with previously reported studies of the fixed-dose combination | 12/15/2023 | Cancer |
Bristol Myers Squibb Co., of Princeton, N.J. | Opdivo (nivolumab) plus Yervoy (ipilimumab) | PD-1 immune checkpoint inhibitor | Microsatellite instability-high or mismatch repair deficient metastatic colorectal cancer | Combination met the dual primary endpoint of progression-free survival | 12/7/2023 | Cancer |
CG Oncology Inc., of Irvine, Calif. | CG-0070 (cretostimogene grenadenorepvec) | Intravesically delivered oncolytic immunotherapy | High-risk non-muscle invasive bladder cancer (NMIBC) unresponsive to Bacillus Calmette-Guerin (BCG) | Interim data for BOND-003 showed 75.7% of patients evaluable for efficacy have achieved complete response at any time; 3- and 6-month landmark complete response rates were 68.2% and 63.6%, respectively; grade 1-2 local genitourinary symptoms; no grade 3 events; no treatment discontinuations due to adverse events | 12/1/2023 | Cancer |
Clarity Pharmaceuticals Ltd., of Sydney | 64Cu SAR-bisPSMA | Radio-diagnostic targeting prostate-specific membrane antigen | Prostate cancer | First of 383 patients dosed in the registrational Clarify study designed to measure whether 64Cu SAR-bisPSMA can detect regional nodal metastases | 12/22/2023 | Cancer |
Daiichi Sankyo Co. Ltd., of Tokyo, and Astrazeneca plc, of London | Dato-Dxd (datopotamab deruxtecan) | Engineered TROP2-directed DXd antibody-drug conjugate | Early stage triple-negative and HR-low, HER2-low or negative breast cancer | First patient dosed in Tropion-Breast04 study in combination with durvalumab; planning to enroll approximately 1,700 patients | 12/18/2023 | Cancer |
Daiichi Sankyo Co. Ltd., of Tokyo, and Astrazeneca plc, of London | Dato-Dxd (datopotamab deruxtecan) | Engineered TROP2-directed DXd antibody-drug conjugate | Advanced or metastatic triple-negative breast cancer whose tumors express PD-L1 | First patient dosed in Tropion-Breast05 study in combination with durvalumab and alone; planning to enroll 625 patients | 12/18/2023 | Cancer |
Eli Lilly and Co., of Indianapolis | Verzenio (abemaciclib) | CDK4/6 inhibitor | Post-menopausal patients with hormone receptor positive (HR+), human epidermal growth factor receptor 2 negative (HER2-) advanced or metastatic breast cancer | Monarch3 study had a median overall survival (OS) of more than 5.5 years – an increase of 13.1 months compared to the control arm in the intent-to-treat (ITT) population (66.8 vs. 53.7 months), although statistical significance for OS not reached (p=0.0664) in combination with an aromatase inhibitor (AI) compared to an AI alone as initial endocrine-based therapy for post-menopausal patients; OS for subpopulation were also not statistically significant (p=0.0757); median progression-free survival maintained (29 vs. 14.8 months nominal p<0.0001), with substantial difference in 6-year PFS rates (23.3% in the Verzenio arm vs 4.3% in the control arm); no new safety signals | 12/5/2023 | Cancer |
Genentech Inc., a unit of Basel, Switzerland-based Roche Holding AG | Inavolisib | Oral therapy; PI3K? inhibition | PIK3CA-mutated, hormone receptor (HR)-positive, HER2-negative, endocrine-resistant, locally advanced or metastatic breast cancer | Inavo120 study in combination with palbociclib and fulvestrant met its primary endpoint of progression-free survival (PFS) with statistically significant and clinically meaningful improvement compared to palbociclib and fulvestrant alone; well-tolerated; no new safety signals | 12/4/2023 | Cancer |
GSK plc, of London | Jemperli (dostarlimab) | Monoclonal antibody targeting PD-1 | Primary advanced or recurrent endometrial cancer | Top-line results of study met its primary endpoint of progression-free survival in combination with standard-of-care chemotherapy (carboplatin and paclitaxel) followed by dostarlimab plus Zejula (niraparib) as maintenance therapy; statistically significant and clinically meaningful benefit observed in both the overall patient population and in a subpopulation of patients with mismatch repair proficient/microsatellite stable (MMRp/MSS) tumors; secondary endpoint of overall survival immature; consistent safety profile | 12/18/2023 | Cancer |
Innovent Biologics Inc., of Rockville, Md., and Suzhou, China | Sintilimab | PD-1 immunoglobulin G4 monoclonal antibody | Advanced or metastatic gastric or gastroesophageal junction adenocarcinoma | Inteirm analysis of Orient-16 of sintilimab in combination with chemotherapy was published in JAMA for first-line treatment, demonstrating it significantly prolongs overall survival; significantly reduced risk of death (HR 0.77, 95%CI 0.63-0.94, p=0.009), and in patients with CPS ?5 (HR 0.66, 95%CI 0.50-0.86, p=0.002) successfully reached the prespecified superiority criteria; median overall survival prolonged by 2.9 months in all randomized patients, and 5.5 months in patients with CPS ?5; no additional safety signals were identified | 12/5/2023 | Cancer |
Lantheus Holdings Inc., of Bedford, Mass., and Point Biopharma Inc., of Indianapolis | ¹??Lu-PNT2002 | PSMA-targeted radioligand | Metastatic castration-resistant prostate cancer | Top-line results of Splash study met its primary endpoint; median radiographic progression-free survival (rPFS) per blinded independent central review of 9.5 months for patients compared to 6 months for patients treated with androgen receptor pathway inhibitor (ARPI); statistically significant 29% reduction in the risk of radiographic progression or death (p=0.0088); immature overall survival; favorable safety profile with grade ?3 treatment-emergent adverse events (30.1% vs.36.9%); serious events (17.1% vs. 23.1%); events leading to discontinuation (1.9% vs. 6.2%) | 12/18/2023 | Cancer |
Merarini Group, of Florence, Italy | Orserdu (elacestrant) | Selective estrogen receptor degrader | Early stage ER+/HER2- breast cancer and molecular relapse | Post-hoc analysis of Emerald study showed clinically meaningful improvement in progression-free survival; median PFS of 8.6 months vs. 1.9 months on SOC, with a 59% reduction in the risk of progression or death | 12/8/2023 | Cancer |
Merck & Co. Inc., of Rahway, N.J. | Keytruda (pembrolizumab) | Anti-PD-1 therapy | Metastatic squamous non-small-cell lung cancer | Stopped its phase III study on the recommendation of an independent data monitoring committee during an interim analysis; Keytruda plus Lynparza did not show an improvement in OS; PFS was not be statistically significant at the second interim analysis, but the data showed a numerical improvement when compared to the control arm | 12/7/2023 | Cancer |
Merck & Co. Inc., of Rahway, N.J., and Eisai Co. Ltd., of Tokyo | Keytruda (pembrolizumab) | Anti-PD-1 therapy | Advanced or recurrent endometrial carcinoma | Leap-001 study in combination with Lenvima did not meet its dual primary endpoints of overall survival (OS) and progression-free survival (PFS); consistent safety profile | 12/8/2023 | Cancer |
Moderna Inc., of Cambridge, U.K., and Merck & Co. Inc., of Rahway, N.J. | mRNA-4157/V940 | Personalized mRNA cancer vaccine | Completely resected stage II, IIIA or IIIB (with nodal involvement [N2]) non-small-cell lung cancer | Initiated phase III study in combination with Keytruda (pembrolizumab); first patient planning to enroll in Australia; planning to enroll 1,089 patients | 12/11/2023 | Cancer |
Novartis AG, of Basel, Switzerland | Kisqali (ribociclib) | CDK4/6 inhibitor | Stage II and III hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) early breast cancer | Data from median follow-up of 33.3 months and following Kisqali treatment completion by 78.3% of patients; reduction in risk of disease recurrence in patients by 25.1% (p=0.0006); distant disease-free survival (25.1% risk reduction) and recurrence-free survival (27.3% risk reduction); low-grade adverse events; no new safety signals | 12/8/2023 | Cancer |
Polaris Group, of Taipei, Taiwan, and San Diego | ADI-PEG 20 (pegargiminase) | Pegylated arginine deiminase | Leiomyosarcoma | First patient dosed | 12/4/2023 | Cancer |
Sanofi SA, of Paris | Sarclisa (isatuximab) | Anti-CD38 humanized monoclonal antibody | Multiple myeloma | Therapy used in combination with standard-of-care bortezomib, lenalidomide and dexamethasone (VRd) hit its primary endpoint of PFS in patients with newly diagnosed MM who are not eligible for a transplant; significantly reduced the risk of disease progression or death compared to VRd alone | 12/7/2023 | Cancer |
Sanofi SA, of Paris | Sarclisa (isatuximab) | Anti-CD38 humanized monoclonal antibody | Multiple myeloma | In combination with carfilzomib, lenalidomide and dexamethasone (KRd) showed a statistically significant improvement in the rate of minimal residual disease (MRD) negativity, compared with KRd alone (77% vs. 67%, p=0.049) in ITT analysis; statistically significant difference in MRD negativity rates after induction with Sarclisa in combination with KRd vs. KRd (45% vs. 26%, p<0.001; 27% vs. 14%, p=0.004); no new safety signals; 3 treatment-related deaths in the Sarclisa combination arm and 1 in the KRd arm | 12/10/2023 | Cancer |
Sanofi SA, of Paris | Tusamitamab ravtansine | Antibody-drug conjugate targeting CEACAM5 | Second-line non-small-cell lung cancer | Discontinuing program based on interim results of the Carmen-LC03 that didn’t meet its dual primary endpoint of improvement in progression-free survival compared to docetaxel | 12/21/2023 | Cancer |
Seagen Inc., of Bothell, Wash. | Tukysa (tucatinib) | Tyrosine kinase inhibitor of the HER2 protein | HER2-positive metastatic breast cancer | Phase III Her2climb-02 trial of Tukysa in combination with Kadcyla (trastuzumab emtansine) showed a statistically significant improvement in progress-free survival, the primary endpoint (9.5 months vs. 7.4 months, p=0.0163); in patients with brain metastases, median PFS was 7.8 months vs. 5.7 months; confirmed objective response rate of 42% vs. 36.1% for placebo; discontinuations were more common in the combination arm but no new safety signals were observed | 12/6/2023 | Cancer |
Sellas Life Sciences Group Inc., of New York | Galinpepimut-S | WT1-targeting peptide immunotherapeutic | Acute myeloid leukemia | Independent data monitoring committee recommended that Regal trial continue without modifications | 12/4/2023 | Cancer |
Cytokinetics Inc., of South San Francisco | Aficamten | Cardiac myosin inhibitor | Symptomatic obstructive hypertrophic cardiomyopathy | In the Sequoia-HCM study, treatment with aficamten increased peak oxygen uptake a least mean difference of 1.74 mL/kg/min (p=0.000002); all 10 prespecified secondary endpoints were statistically significant (p<0.0001); data to be presented at an upcoming medical meeting | 12/27/2023 | Cardiovascular |
Mineralys Therapeutics Inc., of Radnor, Pa. | Lorundrostat | Aldosterone synthase inhibitor | Uncontrolled or resistant hypertension | First subject dosed in Launch-HTN study; top-line data expected in the second half of 2025 | 12/21/2023 | Cardiovascular |
Argenx SE, of Amsterdam, the Netherlands | Efgartigimod subcutaneous Vygart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) | Subcutaneous combination of IgG1 antibody fragment targeting the neonatal Fc receptor and hyaluronidase PH20 | Pemphigus vulgaris and pemphigus foliaceus | In the 222-patient Address study, complete remission rate on a minimal dose of steroids was 35.5% for patients taking the drug, compared to 30.3% for patients on placebo (p=0.5956) | 12/20/2023 | Dermatologic |
Ascletis Pharma Inc., of Hangzhou, China | ASC-40 (denifanstat) | Oral FASN inhibitor | Moderate to severe acne vulgaris | Initiated phase III study and planning to enroll 480 subjects | 12/5/2023 | Dermatologic |
Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C. | Orladeyo (berotralstat) | Decreases activity of plasma kallikrein | For prophylaxis of hereditary angioedema attacks | Open-label extension of Apex-2 study showed rapid and sustained reductions in attacks and improved quality of life over 96 weeks of treatment; well-tolerated; results published in the Journal of Allergy and Clinical Immunology: In Practice | 12/19/2023 | Dermatologic |
Cara Therapeutics Inc., of Stamford, Conn. | Difelikefalin (Korsuva) | Analgesic opioid peptide | Moderate to severe pruritus in adult patients with atopic dermatitis | Study did not demonstrate meaningful clinical benefit compared to TCS alone; discontinued clinical program | 12/18/2023 | Dermatologic |
Demata Therapeutics Inc., of San Diego | DMT-310 | Antimicrobial and anti-inflammatory | Moderate to severe acne | Enrolled first of approximately 550 patients in the Star-1 study; primary endpoints are the mean change from baseline in inflammatory and noninflammatory lesion counts and the Investigator Global Assessment treatment response rate; top-line data expected in first quarter of 2025 | 12/20/2023 | Dermatologic |
Ascendis Pharma Inc., of Copenhagen | Transcon hGH (lonapegsomatropin) | Growth hormone ligand | Growth hormone deficiency | Top-line results achieved primary and secondary endpoints; statistically significant reduction from baseline in trunk fat (p<0.0001) and increase in total body lean mass (p<0.0001) at week 38 compared to placebo; change from baseline in trunk fat mass (p=0.0053); exploratory post-hoc analysis in patients with IGF-1 SDS levels ? 1.75 at week 38 showed same effect on target tissues as daily hGH; safe and well-tolerated; no discontinuations related to study drug | 12/19/2023 | Endocrine/metabolic |
Astrazeneca plc, of London | Lokelma (sodium zirconium cyclosilicate) | Insoluble, non-absorbed sodium zirconium silicate | Hyperkalemia | Discontinued the Stabilize-CKD and Dialize-Outcomes phase III evidence trials; decision was made due to substantially increased enrollment timelines and low event rates, respectively, which made it prohibitive to deliver study results within a timeframe to meaningfully advance clinical practice | 12/1/2023 | Endocrine/Metabolic |
Rezolute Inc., of Redwood City, Calif. | RZ-358 | Human monoclonal antibody that binds to an allosteric site on insulin receptors in the liver, fat, and muscle | Congenital hyperinsulinism | Initiated Sunrize study | 12/14/2023 | Endocrine/Metabolic |
Rhythm Pharmaceuticals Inc., of Boston | Setmelanotide | Melanocortin 4 (MC4) receptor agonist | Bardet-Biedl syndrome, proopiomelanocortin or leptin receptor deficiency obesity | Phase III data in those between ages 2 and younger than 6 showed clinically meaningful reductions in BMI and BMI-Z score; 83.3% of patients (10 of 12) achieved a ?0.2 reduction in BMI-Z score from baseline to week 52; 18.4% mean reduction from baseline in BMI at week 52 (N=12); 3.04 mean reduction from baseline in BMI-Z score at week 52 (N=12); 11 patients completed trial; safety profile consistent with past trials | 12/6/2023 | Endocrine/Metabolic |
Akero Therapeutics Inc., of South San Francisco | Efruxifermin | Differentiated bivalent Fc-FGF21 fusion protein that has been engineered to mimic the balanced biological activity profile of native FGF21 | Biopsy-confirmed precirrhotic nonalcoholic steatohepatitis/metabolic dysfunction-associated steatohepatitis | First doses received in subject | 12/18/2023 | Gastrointestinal |
Inventiva SA, of Daix, France | Lanifibranor | PPAR agonist | Nonalcoholic fatty liver disease | Positive recommendation from the third meeting of the data monitoring committee to continue the Nativ3 phase III study without modification of the protocol; good safety and tolerability profile | 12/4/2023 | Gastrointestinal |
Inventiva SA, of Daix, France, and Long Island City, New York | Lanifibranor | Activates all 3 peroxisome proliferator-activated receptor isoforms | Non-cirrhotic non-alcoholic steatohepatitis and F2/F3 stage of liver fibrosis | First patient randomized in China in the global Nativ3 study that is expected to be fully enrolled in the first quarter of 2024 | 12/20/2023 | Gastrointestinal |
Mithra Pharmaceuticals SA, of Liege, Belgium | Donesta | Orally administered estetrol-based hormone therapy | Symptoms of menopause | Independent data safety monitoring board recommended to continue Donesta trial in Europe following regular safety assessment; last patient-out expected in the first quarter of 2024 | 12/18/2023 | Genitourinary/Sexual Function |
Mithra Pharmaceuticals SA, of Liege, Belgium | Estelle | Contraceptive | Pregnancy prevention | Completed the MIT-Es001-C303 study of adolescent women; data expected in the second quarter of 2024 | 12/20/2023 | Genitourinary/Sexual Function |
Novartis AG, of Basel, Switzerland | Fabhalta (iptacopan) | Oral targeted factor B inhibitor of alternative complement pathway | C3 glomerulopathy | Top-line results from APPEAR-C3G study met its primary endpoint; superiority of iptacopan vs. placebo in proteinuria reduction at 6-month analysis | 12/11/2023 | Genitourinary/Sexual Function |
Travere Therapeutics Inc., of San Diego | Pegtibatinase | Enzyme replacement therapy | Classical homocystinuria | Opened enrollment in the Harmony study to determine the safety and efficacy of pegtibatinase in reducing plasma total homocysteine levels | 12/14/2023 | Genitourinary/Sexual Function |
Lynx Pharmaceuticals Co. Ltd., of Boston, and Hangzhou, China, and Shanghai | LNK-01001 | JAK1 inhibitor | Moderate to severe active rheumatoid arthritis | First patient treated in the study of patients with inadequate response or who are intolerant to biological disease-modifying antirheumatic drugs | 12/20/2023 | Immune |
Merck KGaA, of Darmstadt, Germany | Evobrutinib | BTK inhibitor | Relapsing multiple sclerosis | Did not meet primary endpoints of reducing annualized relapse rates for up to 156 weeks in two phase III Evolution trials comparing it with teriflunomide (0.11 vs. 0.11 in EvolutionRMS 1, and 0.15 vs. 0.14 in EvolutionRMS 2); safety and tolerability consistent with phase II results | 12/6/2023 | Immune |
Allovir Inc., of Waltham, Mass. | Posoleucel | Allogeneic, multivirus-specific T-cell therapy | Virus-associated hemorrhagic cystitis and adenovirus following allogeneic hematopoietic cell transplant | Independent data safety monitoring board reviewed preplanned analyses; recommended stopping its respective trial for futility after a review; not achieved its primary endpoint; no observed safety concerns | 12/22/2023 | Infection |
Cidara Therapeutics Inc., of San Diego | Rezafungin | Once-weekly echinocandin | Candidemia and invasive candidiasis | Completed enrollment in the phase III Restore trial in China | 12/6/2023 | Infection |
Cidara Therapeutics Inc., of San Diego, and Melinta Therapeutics LLC., of Parsippany, N.J. | Rezzayo (rezafungin for injection) | Once-weekly, next-generation echinocandin | Candidemia and invasive candidiasis | Pooled analysis from phase III Restore and phase II Strive study support the non-inferiority of rezafungin vs. caspofungin for all-cause mortality; well-tolerated; similar safety profile to caspofungin; results published in The Lancet Infectious Diseases | 12/5/2023 | Infection |
CSL Ltd., of Melbourne, Australia, and Arcturus Therapeutics Inc., of San Diego | ARCT-154 | Self-amplifying messenger RNA vaccine | COVID-19 prophylaxis | Data published in Lancet Infectious Diseases showed ARCT-154 induced a geometric mean titer (GMT) of Wuhan-Hu-1 neutralizing antibodies of 5641 compared to 3934 for Comirnaty and a GMT of omicron antibodies of 2551 compared to 1958 for Comirnaty | 12/21/2023 | Infection |
CSL Ltd., of Melbourne, Australia, and Arcturus Therapeutics Inc., of San Diego | ARCT-154 | Self-amplifying messenger RNA vaccine | COVID-19 prophylaxis | Booster dose showed as well-tolerated as Comirnaty (Pfizer Inc./Biontech SE) in adult population; no causally associated severe or serious adverse events; 95% and 97% of ARCT-154 and Comirnaty vaccinees, respectively, reported local reactions and 66% and 63%, respectively, had solicited systemic adverse events; mild events; higher immune response against the original Wuhan-Hu-1 virus strain, and a superior immune response against omicron BA.4/5 subvariant of SARS-CoV-2 virus compared to a booster dose of Comirnaty | 12/21/2023 | Infection |
Hyloris Pharmaceuticals SA, of Liège, Belgium | Valacyclovir (HY-029) | Oral suspension formulation of antiviral | Herpes virus infection | Bioavailability of valaciclovir under fasted conditions was similar for the oral suspension compared to Valtrex tablets | 12/26/2023 | Infection |
Invivyd Inc., of Waltham, Mass. | VYD-222 | Neutralizing, half-life extended monoclonal antibody | Prevention of symptomatic COVID-19 | Initial results of ongoing Canopy study showed no study drug-related serious adverse events; mild or moderate in severity in adverse events; produced high serum virus neutralizing antibody titer levels against XBB.1.5 in immunocompromised participants | 12/18/2023 | Infection |
Pfizer Inc., of New York, and Valneva SE, of Saint-Herblain, France | VLA-15 | Multivalent protein subunit vaccine; vaccine against lyme | Lyme disease | Completed recruitment; trial conclusion expected by year-end 2025 | 12/4/2023 | Infection |
Valneva SE, of Saint-Herblain, France | Ixchiq | Single-dose, live attenuated vaccine | Prevention of disease caused by chikungunya virus | Data showed primary endpoint met with 97% seroresponse rate 24 months after a single vaccination; antibody levels remained high and well above the seroresponse threshold; no safety concerns identified in long-term follow-up | 12/4/2023 | Infection |
Verrica Pharmaceuticals Inc., of West Chester, Pa. | TO-208/ VP-102/Ycanth | Drug-device combination that contains a GMP-controlled formulation of cantharidin (0.7% w/v) delivered via a single-use applicator | Molluscum contagiosum | Top-line data showed proportion of subjects achieving complete clearance of all treatable molluscum lesions at the completion of the confirmatory study, statistically significant vs. placebo; well-tolerated | 12/15/2023 | Infection |
Capricor Therapeutics Inc., of San Diego | CAP-1002 | Allogeneic cardiosphere-derived cells | Duchenne muscular dystrophy | Data safety monitoring board recommended to continue the HOPE-3 trial as planned after review of interim futility analysis; top-line data expected in the fourth quarter of 2024; cohort A enrollment completed and initiated for cohort B | 12/11/2023 | Musculoskeletal |
Tonix Pharmaceuticals Inc., of Chatham, N.J. | TNX-102 SL (cyclobenzaprine sublingual tablets) | Antagonist of the 5-HT2A-serotonergic, ?1-adrenergic, H1-histaminergic, and M1-muscarinic cholinergic receptors | Fibromyalgia | TNX-102 SL reduced daily pain compared to placebo (p=0.00005); in a comparison of weeks 1-14 individually, all pain scores for TNX-102 SL were lower than placebo (p<0.01 to p<0.0001) | 12/20/2023 | Musculoskeletal |
Anavex Life Sciences Corp., of New York | ANAVEX2-73 (Blarcamesine) | Activator of the sigma-1 receptor | Rett syndrome | Differential expression of several genes observed in patients treated with drug compared to those in the placebo group | 12/20/2023 | Neurology/Psychiatric |
Annovis Bio Inc., of Berwyn, Pa. | Buntanetap | Translational inhibitor of neurotoxic proteins | Parkinson’s disease | Last patient's last visit completed; top-line results expected in January 2024 | 12/5/2023 | Neurology/Psychiatric |
Apnimed Inc., of Cambridge, Mass. | AD-109 (aroxybutynin/atomoxetine) | Antimuscarinic and a selective norepinephrine reuptake inhibitor | Obstructive sleep apnea | First patient dosed in the 640-patient Synairgy study comparing AD-109 to placebo; primary efficacy endpoint is reduction of airway obstructions; key secondary endpoint is change in PROMIS-Fatigue scale | 12/21/2023 | Neurology/Psychiatric |
Karuna Therapeutics Inc., of Boston | Karxt (xanomeline-trospium) | Muscarinic antipsychotic | Schizophrenia | Karxt demonstrated statistically significant and clinically meaningful improvements in positive and negative symptoms of schizophrenia (PANSS) compared to placebo (p<0.0001 at week 5); ?30% reduction from baseline to week 5 in PANSS total score; statistically significant reductions compared to placebo at week 5 (p<0.05) on each endpoint; well-tolerated; overall discontinuation rates similar to placebo (Karxt 25% vs. placebo 21%); results published in The Lancet | 12/14/2023 | Neurology/Psychiatric |
Teva Pharmaceutical Industries Ltd., of Tel Aviv, Israel | Ajovy (fremanezumab) | Humanized monoclonal antibody that selectively targets the calcitonin gene-related peptide (CGRP) | Migraine and obesity | Post hoc analysis of two phase III studies, Halo-LTS1 and Focus2, showed efficacy was the same in migraine patients with BMI-high and BMI-normal, reducing migraines by 6.8 vs. 7.2 days, respectively, after 6 months of treatment | 12/6/2023 | Neurology/Psychiatric |
Annexon Inc., of Brisbane, Calif. | ANX-007 | C1q and classical complement inhibitor | Geographic atrophy | Plans to run a phase III program consisting of the Archer II study comparing ANX-007 to a sham procedure and the Arrow study comparing ANX-007 to Syfovre (pegcetacoplan, Apellis Pharmaceuticals Inc.); primary endpoint for both studies is the prevention of ?15-letter loss of best corrected visual acuity | 12/20/2023 | Ocular |
Nicox SA, of Sophia Antipolis, France | NCX-470 | Nitric oxide-donating bimatoprost eye drop | Open-angle glaucoma or ocular hypertension | First patient screened in Whistler phase IIIb study; planning to enroll 20 healthy volunteers with ocular hypertension in a double-masked, placebo-controlled study | 12/18/2023 | Ocular |
Oculis Holding AG, of Zug, Switzerland | OCS-01 | Corticosteroid | Inflammation and pain following ocular surgery | First patient in the Optimize-2 study had their first visit; primary endpoints are the absence of anterior chamber cells on day 15 and absence of pain on day 4; data expected by the end of 2024 | 12/20/2023 | Ocular |
Oculis Holding AG, of Zug, Switzerland, and Boston | OCS-01 | High-concentration (15 mg/ml), topical formulation of dexamethasone | Diabetic macular edema | First patient first visit in stage 2 Diamond-1 study; OCS-01 achieved the primary endpoint with robust statistical significance showing improvement in BCVA vs. vehicle at week 6 (p=0.007), following the induction phase (OCS-01: 7.2 letters vs. vehicle: 3.1 letters) and sustained to week 12 with the maintenance dose; 27.4% of patients in the OCS-01 group achieved ?15-letter improvement in BCVA from baseline vs. 7.5% in the vehicle group at week 12 (p=0.009); statistically significant decrease in Central Subfield Thickness; well-tolerated with no unexpected adverse events observed; planning to enroll 350-400 patients in stage 2 | 12/18/2023 | Ocular |
Stuart Therapeutics Inc., of Stuart, Fla. | ST-100 (vezocolmitide) | Patented synthesized polypeptide collagen mimetic peptide platform | Dry eye disease | First patient had the first visit; planning to enroll 320 volunteers in the study | 12/30/2023 | Ocular |
Aquestive Therapeutics Inc., of Warren, N.J. | Anaphylm (epinephrine) | Polymer matrix-based epinephrine prodrug; sublingual film | Anaphylaxis | First patient dosed; topline data expected in first quarter 2024 | 12/5/2023 | Other/Miscellaneous |