|
||||||
| Company | Product | Description | Indication | Status | ||
|
American Society of Hematology (Orlando, Fla.) |
||||||
| AB Science SA, of Paris | AB-8939 | Tubulin inhibitor | Acute myeloid leukemia | In vivo experiments in 3 patient-derived xenograft mouse models and cytarabine(Ara-C)-resistant mouse model suggested study drug, alone or with Ara-C, increased survival and reduced tumor growth vs. Ara-C alone; separate ex vivo and in vivo studies showed study drug had broad antiproliferative activity across AML subtypes and produced antiproliferative effect against blasts isolated from AML patients, with majority of IC50 values ranging from 1.4 nM to 1 µM | ||
| Abbvie Inc., of North Chicago | Imbruvica (ibrutinib) | BTK inhibitor | Chronic lymphocytic leukemia | Extended follow-up analysis of phase III E1912 study showed combination with rituximab vs. standard chemoimmunotherapy regimen (FCR) in previously untreated patients, ages 70 and younger, showed progression-free survival benefits were sustained in combination arm vs. FCR (p-0.009); overall survival continued to favor combo arm (p=0.009) | ||
| Abbvie Inc., of North Chicago | Imbruvica (ibrutinib) | BTK inhibitor | Chronic lymphocytic leukemia | Analysis with up to 6 years of follow-up from Phase III Resonate and Resonate-2 studies in previously untreated and relapsed/refractory patients showed median progression-free survival not reached for first-line or the 1-2 prior lines groups, and median FDA was 40.1 months for the 3 or more prior lines group; greater proportion of patients treated in earlier lines remained progression-free or alive at 60 months (70% for first-line, 60% for 1-2 prior lines and 33% for 3 or more prior lines | ||
| Abbvie Inc., of North Chicago | Imbruvica (ibrutinib) | BTK inhibitor | Chronic lymphocytic leukemia | New data from phase II Captivate study in combination with Venclexta (venetoclax) in previously untreated patients showed those receiving up to 12 cycles of combination regimen achieved high rates of undetectable minimal residual disease in both peripheral blood (75% of patients) and in bone marrow (72% of patients) | ||
| Abbvie Inc., of North Chicago | Imbruvica (ibrutinib) | BTK inhibitor | Relapsed/refractory mantle cell lymphoma | Results from 7.5-year pooled analysis of phase II and III studies showed earlier treatment with monotherapy vs. later lines of therapy extended progression-free survival and increased likelihood of complete response; some patients achieved disappearance of any signs of disease | ||
| Achillion Pharmaceuticals Inc., of Blue Bell, Pa. | Danicopan (ACH-4471) | Oral, small-molecule factor D inhibitor | Paroxysmal nocturnal hemoglobinuria | Top-line data showed dose-finding phase II study in combination with eculizumab in patients who have inadequate response to C5 monotherapy met primary endpoint, showing mean increase of 2.4 g/dL at 24 weeks; treatment also resulted in significant reduction in blood transfusions, with 10 patients receiving 34 infusions (58 units) in 6 months prior to screening and 1 patient receiving 1 transfusion (2 units) during the 24-week trial | ||
| Actinium Pharmaceuticals Inc., of New York | Actimab-A | Antibody radiation conjugate that delivers alpha-emitting radioisotope Actinium-225 via the antibody lintuzumab to cells that express CD33 | Relapsed or refractory acute myeloid leukemia | Phase I combination trial of Actimab-A with the salvage chemotherapy regimen CLAG-M (cladribine, cytarabine and filgrastim with mitoxantrone) showed an 86% overall response rate, which is 56% higher than what was observed with CLAG-M and MEC (etoposide, cytarabine and mitoxantrone hydrochloride) and more than double that of CLAG in a similar patient population; 71% of patients (5 of 7) achieved negative minimal residual disease status in the second dose cohort | ||
| ADC Therapeutics SA, of Lausanne, Switzerland | ADCT-402 (loncastuximab tesirine) | Antibody-drug conjugate composed of humanized monoclonal antibody directed against human CD19 | Relapsed or refractory diffuse large B-cell lymphoma | Interim efficacy and safety data on 52 patients in the ongoing pivotal 145-patient phase II trial showed the overall response rate was 46.2%, including 19.2% complete responses and 26.9% partial responses; stable disease was attained in 19.2% of patients and toxicity was manageable; company plans a BLA submission for the second half of 2020 | ||
| Agios Pharmaceuticals Inc., of Cambridge, Mass. | Tibsovo (ivosidenib) | IDH1 gene inhibitor | Acute myeloid leukemia | As of Feb. 19 data cutoff, in 23 participants with newly diagnosed IDH1-mutant disease ineligible for intensive chemotherapy in ongoing phase I/II study in combination with azacitidine, complete response (CR) rate was 61% and CR + CR with partial hematologic recovery (CRh) rate was 70%; median duration of CR and CR+CRh were not reached; in those with CR, 10 of 14 (71%) had IDH1 mutation clearance in bone marrow mononuclear cells measured by BEAMing digital PCR; most with IDH1 mutation clearance showed measurable residual disease negativity by flow cytometry or next-gen sequencing | ||
| Agios Pharmaceuticals Inc., of Cambridge, Mass. | Tibsovo (ivosidenib) | IDH1 gene inhibitor | Acute myeloid leukemia | Genomic profiling of samples from phase I in IDH1-mutant relapsed/refractory AML found RTK pathway mutations NRAS and PTPN11 at baseline were associated with lower likelihood of clinical response to study drug as monotherapy in relapsed/refractory AML; those with JAK2 mutations were more likely to achieve response | ||
| Agios Pharmaceuticals Inc., of Cambridge, Mass. | Mitapivat (AG-348) | Oral, small-molecule allosteric activator of wild-type and variety of mutated PKR enzymes | Non-transfusion-dependent thalassemia | Data from 8 evaluable patients in phase II study showed 7 achieved hemoglobin increase of ?1 g/dL; for responders, mean hemoglobin increase from baseline was 1.76 g/dL (range, 0.9–3.3 g/dL) during weeks 4-12 | ||
| Aleta Biotherapeutics Inc., of Natick, Mass. | CD19-anti-CD20 bridging protein | Monomeric CD19-ECD-anti-CD20 bridging protein | CD19-negative relapse from CAR-CD19 T-cell treatment | Preclinical results from in vitro study showed CAR19 T cells found and eliminated CD19-negative cells that escaped from CAR-CD19 T- cell treatment; in vivo, CAR-CD19 T cells, plus the injected Aleta bridging protein, controlled tumor cell growth, preventing escape from therapy, while CAR-CD19 T cells alone did not prevent tumor relapse | ||
| Alpine Immune Sciences Inc., of Seattle | ALPN-101 | Dual CD28/ICOS antagonist | Graft-vs.-host disease | Dose-dependent pharmacodynamic activity observed in phase I study in healthy volunteers, including inhibition of T-cell activation, assessed ex vivo based on inhibition of staphylococcal enterotoxin B-induced cytokine production, and inhibition of antibody responses, assessed following immunization with keyhole limpet hemocyanin | ||
| Alx Oncology, of Burlingame, Calif. | ALX-148 | CD47 antagonist | Non-Hodgkin lymphoma | In phase I combination study with rituximab in 29 participants with relapsed/refractory NHL, 21 response-evaluable participants showed objective response rate of 43% and median progression-free survival (mPFS) of 7.3 months; in those with aggressive NHL (n=14), ORR of 36% and mPFS of 3.1 months were observed; in indolent NHL (n=7) ORR of 57% was observed and mPFS was not reached; 2 participants achieved complete response, 1 of whom was refractory to prior rituximab therapy; in those with rituximab-refractory disease (n=9), ORR of 44% was observed; in initial response-evaluable patients with relapsed/refractory NHL (n=3) administered ALX-148 (15 mg/kg once weekly), ORR of 67% was reported and mPFS was not reached | ||
| Amgen Inc., of Thousand Oaks, Calif. | Kyprolis (carfilzomib) | Blocks proteasomes | Relapsed or refractory multiple myeloma | Phase III Candor study of Kyprolis in combination with dexamethasone and Darzalex (daratumumab) (KdD arm) compared to Kyprolis and dexamethasone alone (Kd arm) showed it met the primary endpoint of progression-free survival, resulting in a 37% reduction in the risk of disease progression or death; median PFS was not reached for the KdD arm vs. 15.8 months for the Kd arm | ||
| Amphivena Therapeutics Inc., of South San Francisco | AMV-564 | Bivalent T-cell engager | Relapsed/refractory acute myeloid leukemia | Phase I data provide early evidence of safety and clinical activity and T-cell activation, as well as evidence that it increases in bone marrow T cells and has antileukemic blast activity; of 38 patients, 5 had complete or partial responses, and it was shown to be safe and well-tolerated with no dose-limiting toxicities up to 450 mcg/day | ||
| Aprea Therapeutics Inc., of Boston | APR-246 | Small molecule designed to reactivate mutant and inactivated p53 protein | TP53-mutated myelodysplastic syndromes and acute myeloid leukemia | Data from French phase Ib/II trial in combination with azacitidine showed, as of data cutoff, overall response rate in 24 evaluable MDS patients was 74%, with 66% complete remission rate; with median duration of follow-up of 6.4 months, median overall survival for all enrolled patients not yet reached; data from U.S. phase Ib/II trial showed, as of data cutoff, ORR in 33 evaluable MDS patients was 88%, with 61% CR rate; with median duration of follow-up of 10.8 months, median duration of response was 8.4 months and median duration of CR was 7.3 months; median OS for all enrolled patients was 10.8 months | ||
| Aptose Biosciences Inc., of San Diego | CG-806 | FLT3/BTK inhibitor | Chronic lymphocytic leukemia | Preclinical data comparing CG-806 to ibrutinib showed drug broadly inhibits B-cell receptor signaling in CLL cells, resulting in CLL cell apoptosis and reduced proliferation; CG-806 showed to be more potent than ibrutinib to induce apoptosis of MEC1 CLL cells | ||
| Aptose Biosciences Inc., of San Diego | CG-806 | FLT3/BTK inhibitor | Mantle cell lymphomas | Preclinical data showed superior antilymphoma effects vs. ibrutinib, exerting potent cell growth inhibitory effects in ibrutinib-resistant MCL cells; CG-806 suppresses phosphor-BTK, -Stat3, -AKT, -ERK, -Src, NF-kB and anti-apoptotic protein Mcl1, while up-regulating p53; also increases autophagy in MCL cells and up-regulates CXCR4/E-selectin levels in MCL cells | ||
| Arqule Inc., of Burlington, Mass. | ARQ-531 | Dual BTK/TRK tyrosine kinase inhibitor | B-cell non-Hodgkin lymphoma | Final data from phase I study in 47 participants showed overall response rate of 89% (8/9 evaluable patients, 7/8 harboring BTK-C481S mutation) in relapsed/refractory chronic lymphocytic leukemia (CLL); 11 of 19 dosed at 65 mg once daily remain on study; ORR of 50% (3/6 evaluable) in Richter’s transformation was achieved at 65 mg once daily; 2 additional partial responses observed, including 1 in follicular lymphoma and 1 in diffuse large B-cell lymphoma; 5/5 evaluable CLL patients remain durable confirmed PRs through cycle 9 and remain on therapy; 2/3 of Richter’s patients who achieved PRs came off study after becoming eligible for CAR T therapy The Follicular Lymphoma patient that achieved a PR has been on study for 120 weeks and remains a PR and on therapy |
||
| Ascentage Pharma Group International, of Suzhou, China | HQP-1351 | BCR-ABL inhibitor | Tyrosine kinase inhibitor-resistant chronic myeloid leukemia | In a phase I study, complete hematologic response rate was 95% in 87 patients in the chronic phase (CP) and 85% in 14 patients in the accelerated phase (AP); major molecular response rate was 37% in CP and 36% in AP | ||
| Astex Pharmaceuticals Inc., of Pleasanton, Calif. | ASTX-727 | Fixed-dose combination of cedazuridine and decitabine | Intermediate and high-risk myelodysplastic syndromes | Top-line data from the Ascertain phase III trial show it achieved its primary objective of decitabine systemic exposure equivalence between oral ASTX-727 and intravenous decitabine with an oral/I.V. ratio of about 99%; Astex plans to file NDA by the end of 2019 | ||
| Astrazeneca plc, of Cambridge, U.K. | Calquence (acalabrutinib) | BTK inhibitor | Chronic lymphocytic leukemia | Interim analysis of phase III Elevate TN trial showed study drug, combined with obinutuzumab (Gazyva, Roche Holding AG) or as monotherapy, reduced progression-free survival by 90% and 80%, respectively, vs. chlorambucil + obinutuzumab at median follow-up of 28.3 months; in exploratory analysis, Calquence alone or in combination showed PFS improvements across most prespecified subgroups with high-risk disease characteristics | ||
| Atara Biotherapeutics Inc., of South San Francisco | Tab-cel (tabelecleucel) | Allogeneic T-cell immunotherapy | Epstein-Barr virus-associated post-transplant lymphoproliferative disease | Long-term results from expanded access protocol study showed high overall response rate, short time to response and favorable estimated long-term overall survival rates in patients with EBV+ PTLD following hematopoietic cell transplant (HCT) or solid organ transplant (SOT) who have failed rituximab-based therapy; median time to response of 1 month in both patient cohorts; in responders, 2-year estimated OS rate was 86% for HCT and 100% for SOT | ||
| Aurora Bio Inc., of South San Francisco | AU-R01 (124I-p5+14) | Imaging PET peptide radiotracer | Amyloidosis | Among first 18 participants with light chain amyloidosis (AL) dosed in phase I study, analysis of PET images indicated cardiac uptake in 83%, including 50% with no cardiac symptoms and normal cardiac biomarkers; kidney, spleen and liver uptake was observed in 67%, 42% and 42%, respectively; retention of radiotracer was observed in the nerves, ligaments and lungs in those with transthyretin amyloidosis (ATTR) and in the kidney, spleen, adrenal glands and liver in those with leukocyte cell-derived chemotaxin 2 amyloidosis; 100% of ATTR patients had cardiac uptake, including 50% with no cardiac symptoms and normal cardiac biomarkers and 1 participant with negative pyrophosphate scan confirmed via endomyocardial biopsy | ||
| Autolus Therapeutics plc, of London | AUTO-1 | CD19 CAR | Acute lymphoblastic leukemia | Updated phase I data in recurrent/refractory ALL for 15 evaluable patients showed 13 (87%) achieved minimal residual disease (MRD)-negative complete response at 1 month and all had ongoing CAR T-cell persistence at least follow-up; CD19-negative relapse occurred in 22% (2 of 15); in patients dosed with AUTO-1 manufactured in closed process, 9 of 9 (100%) achieved MRD-negative CR at 1 month and 6-month event-free survival, with overall survival at 100% in that cohort | ||
| Autolus Therapeutics plc, of London | AUTO-1 | CD19 CAR | B-cell acute lymphoblastic leukemia | Data from phase I Carpall trial in pediatric patients showed 19 of 21 (90%) achieved molecular complete remission at 1 month post infusion; CAR T cell expansion was detectable by flow in a number of patients up to 36 months; in cohort 2, 100% achieved molecular complete remission at 1 month post infusion; of 14 patients in cohort 1, overall survival at 6 months was 86% and at 12 months was 71%, while event-free survival was 71% and 54%, respectively; patient in cohort 2 not yet evaluable | ||
| Autolus Therapeutics plc, of London | AUTO-3 | Bicistronic CAR targeting CD19 and CD22 | Relapsed/refractory diffuse large B-cell lymphoma | Data from phase I/II Alexander study showed 5 of 14 had compete response, with 4 of 5 CRs ongoing, the longest at 18 months | ||
| Autolus Therapeutics plc, of London | AUTO-3 | Bicistronic CD19 and CD22 CAR | Pediatric acute lymphoblastic leukemia | Data from phase I/II Amelia study in relapsed/refractory patients showed among 10 CAR T-naïve patients, at median follow-up of 9.7 months, 9 of 10 (90%) achieved complete response and 8 of 10 (80%) achieved complete molecular remission by PCR; estimated overall survival at 12 months was 100% | ||
| Axcella Health Inc., of Cambridge, Mass. | AXA-4010 | Endogenous metabolic modulator | Sickle cell disease | Preclinical data showed plasma amino profiles of adults with SCD were different from those in control group, with 50% reduction in arginine and > 30% reduction in total essential amino acids; endothelial cell cultures treated with TNF-alpha and constituents of AXA-4010 saw reduction in markers of vascular adhesion, inflammation and cell migration vs. cell cultures treated with glutamine and TNF-alpha, which saw increase in adhesion, no change in inflammation and reduction in cell migration; compared to individual amino acids, AXA-4010 constituents showed better improvement in red blood cell deformability | ||
| Beigene Ltd., of Beijing | Brukinsa (zanubrutinib) | BTK inhibitor | Chronic lymphocytic leukemia; small lymphocytic lymphoma | Initial results from arm C in phase III Sequoia monotherapy trial, at Aug. 7 data cutoff with median follow-up of 10 months, showed overall response rate of 92.7% (101/109); partial response rate was 78.9% (86/109) and PR rate with lymphocytosis was 11.9% (13/109); complete response rate was 1.9% (2/109); 4 cases of disease progression occurred; 23.9% (26/109) experienced at least 1 serious adverse event (AE), including 1 fatal AE, pneumonia leading to sepsis and death, considered related to treatment drug | ||
| Beigene Ltd., of Beijing | Brukinsa (zanubrutinib) | BTK inhibitor | Chronic lymphocytic leukemia; small lymphocytic lymphoma | Updated results from global phase I/II trial showed Brukinsa was well-tolerated and active in relapsed/refractory (R/R) or treatment-naive (TN) CLL/SLL, irrespective of del(17p) status (n=123; 101 R/R, 22 TN); at data cutoff of May 8 with median follow-up of 29.5 months, overall response rate was 95.9% (118/123); partial response rate was 73.2% (90/123); PR rate with lymphocytosis was 6.5% (8/123); complete response rate was 16.3% (20/123), including 1 CR with incomplete bone marrow recovery; 2-year progression-free survival was 91% in R/R and 95% in TN disease | ||
| Beigene Ltd., of Beijing | Brukinsa (zanubrutinib) | BTK inhibitor | Non-Hodgkin’s lymphoma | In 54 participants with aggressive disease in phase Ib combination trial with anti-PD-1 antibody tislelizumab (Beigene), at data cutoff of Aug. 31 with median follow-up of 8.1 months, preliminary findings showed overall response rate of 37% (20/54), partial response rate of 20.4% (11/54), complete response rate of 16.7% (9/54) and stable disease rate of 9.3% (5/54) | ||
| Bergenbio ASA, of Bergen, Norway | Bemcentinib (BGB-324) | AXL inhibitor | Acute myeloid leukemia | Phase II combination trial with low-dose cytarabine in older adults showed long duration of response (> 9.9 mo, still maturing), with 50% complete response/complete response with incomplete hematologic recovery (CR/CRi) in 6 evaluable newly diagnosed patients; 1 CR/CRi and 3 stable disease seen in 6 evaluable relapsed/refractory patients with > 2 previous therapies | ||
| Beyondspring Inc., of New York | Plinabulin | Guanine nucleotide exchange factor stimulator; tubulin receptor antagonist | Chemo-induced neutropenia | Data from phase II/III study 105 showed drug's ability to protect granulocyte-monocyte progenitor cells; additional data from study 105 suggested plinabulin protects common lymphoid progenitor cells | ||
| Beyondspring Inc., of New York | Plinabulin | Marine-derived small molecule that sequesters tubulin heterodimers | Chemotherapy-induced neutropenia | Phase III Study 106 trial design provides rationale for combining plinabulin with pegfilgrastim (Neulasta) due to their differing mechanisms of action for preventing CIN | ||
| Bluebird Bio Inc., of Cambridge, Mass. | Lentiglobin | Gene therapy | Sickle cell disease | New data from ongoing phase I/II HGB-206 study showed of 17 patients in cohort C with longest follow-up at 21 months, none required regular red blood cell transfusions post treatment; in those with 6+ months of follow-up, median levels of gene therapy-derived anti-sickling hemoglobin, HbAT87Q, were at least 40% of total hemoglobin; total hemoglobin and HbAT87Q levels ranged from 9.3 – 15.2 g/dL and 2.7 – 9 g/dL, respectively, at last visit; among 9 patients with 6+ months follow-up who had 4 or more vaso-occlusive crises or acute chest syndrome in 2 years prior to treatment, there was 99% reduction in annualized rate of both | ||
| Bluebird Bio Inc., of Cambridge, Mass. | Lentiglobin | Gene therapy | Beta-thalassemia | Clinical data support potential benefits and a consistent safety profile across a broad range of transfusion-dependent ?-thalassemia genotypes and patient populations, including pediatric patients, with the longest duration of follow-up beyond 5 years; patients have achieved and maintained transfusion independence, improvements in multiple markers of bone marrow red blood cell production and reductions in iron overload | ||
| Bluebird Bio Inc., of Cambridge, Mass., and Bristol-Myers Squibb, of Princeton, N.J. | Bb-2217 | BCMA-targeted CAR T-cell therapy | Relapsed/refractory multiple myeloma | Phase I safety and efficacy results show a median duration of response of 11.1 months; CAR T persistence was observed in 8 of 10 evaluable responders at month 6, and 2 of 2 evaluable responders at month 18; adverse events were consistent with known toxicities of CAR T therapies | ||
| Blueprint Medicines Corp., of Cambridge, Mass. | Avapritinib | KIT tyrosine kinase inhibitor; PDGF receptor alpha antagonist | Systemic mastocytosis | In dose-finding part 1 of phase II Pioneer trial that enrolled 39 participants, at Nov. 12 cutoff date with median study time of 12 weeks, mean % change in serum tryptase was -37.72% for 25-mg dose, -54.08% for 50 mg and -56.16% for 100 mg vs. 7.05% for placebo at first post-baseline assessment and -48.24% for 25 mg, -66.67% for 50 mg and -61.83% for 100 mg vs. 0.39% for placebo at 12 weeks | ||
| Blueprint Medicines Corp., of Cambridge, Mass. | Avapritinib | KIT tyrosine kinase inhibitor; PDGF receptor alpha antagonist | Systemic mastocytosis | Top-line results from phase I Explorer trial, at data cutoff of Aug. 30 with median follow-up of 21 months, showed confirmed overall response rate, defined as complete remission with full or partial recovery of peripheral blood counts, partial remission or clinical improvement, of 77% in 48 evaluable participants; median duration of response and median overall survival not reached | ||
| Bristol-Myers Squibb Co., of New York | Lisocabtagene maraleucel (liso-cel) | CD19-directed CAR T-cell therapy | Chronic lymphocytic leukemia; small lymphocytic lymphoma | In phase I/II Transcend CLL 004 study, with 22 participants evaluable for efficacy at data cutoff, at median follow-up of 11 months overall response rate was 81.5% (18/22) with 45.5% (10/22) achieving complete response; in those who failed a BTK inhibitor and venetoclax (Venclexta, Abbvie Inc.), ORR was 89% (8/9), with 67% (6/9) achieving CR; by day 30 following treatment, 68% (15/22) achieved early objective response, with 10/12 responders at 6 months remaining progression-free after at least 9 months and 8 in response at 12 months or >; among 20 participants evaluable for minimal residual disease (MRD), most achieved undetectable MRD in blood (75%) and bone marrow (65%) by next-generation sequencing | ||
| Bristol-Myers Squibb Co., of New York | Lisocabtagene maraleucel (liso-cel) | CD19-directed CAR T-cell therapy | Large B-cell non-Hodgkin's lymphoma | In phase II Pilot study, with 13 participants receiving lymphodepletion followed by liso-cel at data cutoff, all 12 eligible for response evaluation achieved response, including 6 with complete response; 7 of 12 maintained response levels at 3 months following liso-cel infusion | ||
| Bristol-Myers Squibb Co., of Princeton, N.J. | Lisocabtagene maraleucel | CD19-directed CAR T-cell therapy | Relapsed/refractory large B-cell lymphoma | Transcend NHL 001 study met primary endpoint; among evaluable patients, overall response rate was 73% (187/256), with 53% (136/256) achieving complete response; median duration of response was not reached at median follow-up of 12 months; median progression-free survival was 6.8 months and median overall survival was 21.1 months; median PFS and OS for patients who achieved CR was not reached, with 65% progression free and 85.5% alive at 12 months, respectively | ||
| Bristol-Myers Squibb Co., of Princeton, N.J. | CC-486 | Oral hypomethylating agent | Acute myeloid leukemia | Data from the Quazar AML-001 phase III trial showed a significant improvement in overall survival compared with placebo (24.7 months vs. 14.8 months, p=0.0009) in front-line patients; CC-486 had a manageable safety profile | ||
| Bristol-Myers Squibb Co., of Princeton, N.J., and Acceleron Pharma, of Cambridge, Mass. | Reblozyl (luspatercept-aamt) | Erythroid maturation agent | Myelofibrosis-associated anemia | Phase II data show that 14% (cohort 1) and 21% (cohort 3A) of anemia-only patients met the primary endpoint of hemoglobin increase of at least 1.5 g/dL from baseline for at least 12 consecutive weeks at every assessment within the first 24 weeks on the study, in the absence of any red blood cell transfusions | ||
| Bristol-Myers Squibb Co., of Princeton, N.J., and Acceleron Pharma, of Cambridge, Mass. | Reblozyl (luspatercept-aamt) | Erythroid maturation agent | Myelodysplastic syndromes | Pivotal phase III Medalist study data showed that 47.7% of treated patients and 15.8% of placebo patients achieved at least 1 episode of red blood cell transfusion independence lasting at least 8 weeks at any point in the study | ||
| Bristol-Myers Squibb Co., of Princeton, N.J., and Pfizer Inc., of New York | Eliquis (apixaban) | Factor Xa inhibitor | Venous thromboembolism | Real-world analyses vs. low-molecular-weight heparin (LMWH) in patients with VTE and active cancer showed Eliquis use associated with lower rates of major bleeding (MB) (p=0.003), clinically-relevant non-major (CRNM) bleeding (p=0.006) and recurrent VTE (p=0.001) vs. LMWH; Eliquis also associated with lower rate of recurrent VTE (p=0.007) and similar rates of major bleeding (p=0.051) and CRNM bleeding (p=0.145) compared to warfarin | ||
| Catalyst Biosciences Inc., of South San Francisco | Marzeptacog alfa, activated (Marzaa) | Factor VIIa agonist | Hemophilia | As rescue therapy in hemophilia A mouse model of severe bleeding, subcutaneously dosed therapy reduced bleeding and was comparable to intravenous Novoseven (eptacog alfa, activated, Novo Nordisk A/S); separate preclinical study showed Marzaa and Novoseven exhibited comparable characteristics when spiked into hemophilia A plasma containing Hemlibra (emicizumab, Roche Holding AG) at clinically relevant concentrations, suggesting Marzaa expected to be safe in combination with Hemlibra | ||
| Cellectar Biosciences Inc., of Florham Park, N.J. | CLR-131 | Phospholipid ether-drug conjugate | Multiple myeloma | Fractionated dosing data in 19 patients with relapsed/refractory disease from phase I and phase II Clover-1 trials, at cutoff date of July 30, showed overall response rate across 3 dose cohorts was 31.3%, with 100% disease control rate; those receiving higher fractionated 37.5 mCi/m2 dose had 50% ORR with remaining 50% having minimal responses (> 25% reduction in surrogate efficacy marker) | ||
| Cellular Biomedicine Group Inc., of New York | C-CAR088 | Anti-B-cell maturation antigen CAR T-cell therapy | Relapsed or refractory multiple myeloma | Of 5 evaluable patients in ongoing phase I trial, all showed clinical improvement as early as 2 weeks post treatment; by 4 weeks, 1 patient achieved complete response, 3 patients reached very good partial response and 1 reached partial response; C-CAR088 proliferation and expansion in peripheral blood correlated with decrease of tumor burden | ||
| Celularity Inc., of Warren, N.J. | PNK-007 | Allogeneic, placental-derived CD19 CAR T cell-based product | Multiple myeloma | Phase I data showed single infusion was well-tolerated after autologous stem cell transplant for up to 1 year | ||
| Chimerix Inc., of Durham, N.C. | Dstat (formerly CX-01) | Dociparstat sodium | Refractory myelodysplastic syndrome and acute myeloid leukemia | Phase II data show that of 15 evaluable patients that received a median of 3 cycles of Dstat and azacitidine, there was 1 complete remission and 3 bone marrow complete remissions, 9 stable disease and 2 progressive disease for an overall response rate of 27%; the median overall survival was 221 days and not significantly different between AML (221 days) and MDS (248 days) | ||
| Constellation Pharmaceuticals Inc., of Cambridge, Mass. | CPI-0610 | BET protein inhibitor | Myelofibrosis | Data from phase II Manifest trial in combination with Jakafi (ruxolitinib, Incyte Corp.) in JAK inhibitor-naïve patients showed 12 of 15 in first-line treatment arm achieved at least at 35% spleen volume response at 12 weeks | ||
| Corvus Pharmaceuticals Inc., of Burlingame, Calif. | CPI-818 | ITK inhibitor | T-cell lymphomas | Phase I/Ib data for first 7 patients showed pharmacokinetics and occupancy studies in line with expectations; CPI-818 shown to bind covalently to IT at low nanomolar concentrations without reacting with other kinases; in vitro data showed selective cytotoxicity to Sezary cells, while sparing normal T cells, in 3 subjects not enrolled in study; preclinical data in murine models of lymphoproliferative and autoimmune disease showed CPI-818 inhibited development of lymph node and spleen enlargement by preventing proliferation of abnormal T cells and treatment led to regression of lymphadenopathy and splenomegaly in animals with established disease | ||
| Cstone Pharmaceuticals, of Suzhou, China | CS-1001 | Monoclonal antibody directed against PD-L1 | Relapsed or refractory extranodal natural killer T-cell lymphoma | Phase II CS1001-201 data show that 13 (40.6%) of 32 enrolled patients remained on treatment, and 19 (59.4%) discontinued treatment due to radiographic disease progression, adverse events and nonradiographic symptomatic progression; 33.3% achieved a complete response and 3 achieved a partial response for an overall response rate of 43.3% | ||
| CTI Biopharma Corp., of Seattle | Pacritinib | Oral kinase inhibitor with specificity for JAK2, FLT3, IRAK1 and CSF1R | Thrombocytopenic myelofibrosis | PAC203 data demonstrate pacritinib 200 mg twice-daily is well-tolerated with clinical benefit in the highest-risk patient population; mutational analyses of phase II PAC203 patients demonstrate benefit in patients with high mutational risk and low JAK2 allele burden; Pacifica phase III trial is underway | ||
| Cyclacel Pharmaceuticals Inc., of Berkeley Heights, N.J. | CYC-065 | Dual CDK2/CDK9 inhibitor | Acute myeloid leukemia; myelodysplastic syndromes; chronic lymphocytic leukemia | In phase I combination study (CYC065-03) with venetoclax (Venclexta, Abbvie Inc./Roche Holding AG), 3 of 9 participants with relapsed/refractory (R/R) AML/MDS who received doses from 64 mg/m2 to 150 mg/m2 achieved decreases in leukemia blast cells in peripheral blood as reported by investigators; in separate phase I combination study (CYC065-02), first 2 R/R CLL patients achieved shrinkage of enlarged lymph nodes by CT scan | ||
| Editas Medicine Inc., of Cambridge, Mass. | EDIT-301 | Gene editing medicine that uses CRISPR/Cpf1 (formerly Cas12a) | Sickle cell disease and beta-thalassemia | In vivo proof-of-concept data show HbF levels in human red blood cells were increased by about 50 percentage points above background at 16 weeks post-engraftment with pancellular distribution and no lineage skewing; the elevated levels were observed after editing with Cas12a | ||
| Eli Lilly and Co., of Indianapolis | LOXO-305 | Highly selective, non-covalent BTK inhibitor | Chronic lymphocytic leukemia and mantle cell lymphoma | Phase I/II data showed it delivered objective responses at all doses studied in patients who had received diverse prior therapies and who had exhibited varied molecular mechanisms of acquired resistance, including those with BTK resistance, BTK intolerance and BCL2 resistance | ||
| Epizyme Inc., of Cambridge, Mass. | Tazemetostat | Oral EZH2 | Follicular lymphoma | Mature data from ongoing phase II study testing monotherapy inpatients, with or without EZH2 activating mutations, who have received at least 2 prior lines of systemic therapy demonstrated meaningful clinical activity as assessed by both investigators and an independent review committee; objective response rate of 69% for EZH2 mutation and 35% for wild-type EZH2; median duration of response was 11 months and 13 months, respectively, while median-progression-free survival was 14 months and 11 months, respectively; NDA submission in December 2019 | ||
| Fate Therapeutics Inc., of San Diego | FT-596 | Multi-antigen targeting natural killer cell product derived from a clonal master engineered induced pluripotent stem cell line | Lymphoma | Demonstrated comparable antitumor activity to CAR19 T cells in vivo in humanized mouse model of lymphoma; combination with rituximab showed durable tumor clearance in vivo in preclinical lymphoma model; company plans to begin enrollment in clinical trial in early 2020 | ||
| Forma Therapeutics Inc., of Watertown, Mass. | FT-4202 | Selective red blood cell pyruvate kinase-R activator | Sickle cell disease | Phase I data in healthy volunteers showed linear and time-independent pharmacokinetics demonstrating proof of mechanism based on mean maximum % of change in key pharmacodynamic measures from baseline, achieved within 24 hours of single dose and sustained for the multiple ascending-dose 14-day dose period; showed maximal 2,3-DPG response at doses ? 150 mg twice daily or 400 mg once daily in HV RBCs | ||
| Forma Therapeutics Inc., of Watertown, Mass. | Olutasidenib | Next-generation inhibitor of mutated IDH1 | IDH1m acute myeloid leukemia and IDH1m myelodysplastic syndrome | Phase I/II results from ongoing study showed drug was well-tolerated as monotherapy and in combination with azacitidine; demonstrated clinical activity in a high-risk phase I AML population and induced IDH1 mutation clearance in percentage of patients with TN and R/R AML regardless of IWG response | ||
| Forty Seven Inc., of Menlo Park, Calif. | Magrolimab | Monoclonal antibody against CD47 | Myelodysplastic syndrome and acute myeloid leukemia | Phase Ib data showed a 50% complete response rate and a 92% overall response rate in untreated patients with higher-risk MDS, as well as a 55% CR with complete blood count recovery rate and a 64% ORR rate in patients with untreated AML who are ineligible for induction chemotherapy; median duration of response is not yet reached in those on treatment for more than 6.4 and 8.8 months for MDS and AML, respectively; clinical program design may support accelerated approval in higher-risk MDS, with a BLA filing expected for the fourth quarter of 2021 | ||
| Forty Seven Inc., of Menlo Park, Calif. | FSI-174 and magrolimab | Anti-cKIT antibody and anti-CD47 antibody | Conditioning regimen for stem cell transplantation | Preclinical data in nonhuman primates showed all-antibody conditioning regimen significantly depleted hematopoietic stem cells from bone marrow, with no dose-limiting toxicities | ||
| Gamida Cell Ltd., of Boston | GDA-201 | Natural killer cell-based cancer immunotherapy | Non-Hodgkin lymphoma and multiple myeloma | Phase I data show GDA-201 in combination with monoclonal antibodies was generally well-tolerated and demonstrated early evidence of clinical activity in heavily pretreated patients, including 5 complete responses and 1 partial response observed among 9 patients with NHL, and 1 complete response and 5 stable disease patients were seen in the MM group; company plans to start a phase I/II multidose study in patients with NHL in 2020 | ||
| Genentech, a unit of the Roche Group, of South San Francisco | Mosunetuzumab | CD20-CD3 T-cell engaging bispecific antibody | Relapsed or refractory B-cell non-Hodgkin lymphoma | Phase I/Ib GO29781 study, which includes people previously treated with CAR T-cell therapy, showed an objective response rate of 62.7% in slow-growing NHL and 37.1% in aggressive NHL; data demonstrated a complete response rate of 43.3% and 19.4%, respectively; in those who received prior CAR T-cell therapy, the ORR was 38.9%, and 22.2% achieved a CR | ||
| Genentech, a unit of the Roche Group, of South San Francisco | CD20-TCB | CD20-CD3 T-cell engaging bispecific antibody | Relapsed or refractory B-cell non-Hodgkin's lymphoma | Phase I/Ib NP30179 study of CD20-TCB in combination with Gazyva (obinutuzumab) showed an objective response rate of 54% and a complete response rate of 46%, including an ORR and CR of 66.7% in those with follicular lymphoma and an ORR of 50% and a CR of 40.9% in aggressive NHL | ||
| Geron Corp., of Menlo Park, Calif. | Imetelstat | Telomerase inhibitor | Non-del(5q) lower risk Myelodysplastic syndromes | Of the 38 evaluable patients from the phase II Imerge study, 42% achieved ?8-week red blood cell-transfusion independence (RBC-TI) and 28% achieved ?24-week RBC-TI; median duration of TI was 85.9 weeks | ||
| Geron Corp., of Menlo Park, Calif. | Imetelstat | Telomerase inhibitor | Preclinical (myelofibrosis) | Treatment of malignant myelofibrosis cells with JAK inhibitor Jakafi (ruxolitinib, Incyte Corp.) followed by imetelstat produced a greater reduction in malignant progenitor and stem cells compared to simultaneous treatment or either drug alone | ||
| Gracell Biotechnologies Co. Ltd., of Suzhou, China | Fastcar-19 | Genetically modifies a patient's T cells to express CD19-specific CAR | B-cell acute lymphoblastic leukemia | Treatment efficacy showed 34 (97.1%) of patients achieved a complete remission with or without complete blood count recovery on day 28; 32 (91.4%) achieved minimum residual disease-negative complete remission; all 37 patients tolerated the single infusion at different dose levels with no dose-limiting toxicities observed | ||
| Gracell Biotechnologies Co. Ltd., of Suzhou, China | Dual CAR-19-22 | GC022, targets CD19 and CD22 | B-cell acute lymphoblastic leukemia | Treatment efficacy in 20 patients with a 28-day follow-up showed 4 in the low-dose group reported no response and 15 of 16 (93.8%) in the mid- and high-dose groups achieved complete remission | ||
| Gracell Biotechnologies Co. Ltd., of Suzhou, China | Dual CAR-BCMA-19 | GC012, targets BCMA and CD19 | Multiple myeloma | Demonstrated it was effective in eliminating MM tumor cells both in vitro and in vivo; the first-in-human study showed a good safety profile | ||
| Humanigen Inc., of Burlingame, Calif. | GM-CSF k/o CART19 | CAR T with granulocyte-macrophage colony-stimulating factor knocked out | Preclinical (acute lymphoblastic leukemia) | In a xenograft model for relapsed acute lymphoblastic leukemia, therapy improved overall survival compared to wildtype CART19 | ||
| Iaso Biotherapeutics Co. Ltd., of Nanjing, China, and Innovent Biologics Inc., of Suzhou, China | CT-103A | Anti-BCMA CAR T | Relapsed/refractory multiple myeloma | Objective response rate was 100% in 17 evaluable patients in a phase I study; 70.6% of patients achieved a best response of stringent complete or complete response and 88.2% achieved a best response of very good partial response or better | ||
| Immunogen Inc., of Waltham, Mass. | IMGN-632 | CD123-targeting antibody-drug conjugate | Relapsed/refractory acute myeloid leukemia | Phase I data show it displays a well-tolerated safety profile and activity at doses up to and including 0.09 mg/kg per cycle; 38 (54%) of evaluable patients had a reduction in bone marrow blasts and 13 (18%) achieved an objective response, including 2 complete remissions and 10 with incomplete recovery and 1 morphologic leukemia-free state in heavily pretreated patients; 92% had failed prior intensive therapies | ||
| IMV Inc., of Dartmouth, Nova Scotia | DPX-Survivac | Immunotherapy consisting of survivin-based peptides formulated in IMV's DPX drug delivery platform | Recurrent/refractory diffuse large B-cell lymphoma | Phase II Spriel study data show that 7 of 9 (77.8%) evaluable subjects exhibited clinical benefit, including 3 complete responses and 2 partial responses; all 7 had reproducible survivin-specific T-cell responses and favorable safety profiles; top-line results and the launch of an IMV-sponsored study are planned in 2020 | ||
| Innate Pharma SA, of Marseille, France | Lumoxiti (moxetumomab pasudotox-tdfk) | CD22-directed immunotoxin | Relapsed/refractory hairy cell leukemia | Long-term data from pivotal phase III study showed 36% (29/80) of patients achieved durable complete response at day 181, compared to primary analysis in which 30% durable CR rate was reported; there was a 61% probability that patients who achieved CR would maintain it after 5 years | ||
| Innovent Biologics Inc., of Suzhou, China | IBI-326 | Fully human BCMA-targeting CAR T cells | Relapsed/refractory multiple myeloma | With 17 evaluable patients, the objective response rate was 100% in the investigator-initiated trial; 70.6% of patients achieved a best response of stringent complete or complete response, and 88.2% achieved a best response of very good partial response or better | ||
| Janssen Pharmaceutical Cos. of New Brunswick, N.J.-based Johnson & Johnson | JNJ-4528 | BCMA-directed CAR T-cell therapy | Relapsed or refractory multiple myeloma | Initial results from phase Ib/II Cartitude-1 study showed early and deep responses, with median of 5 prior treatment regimens (range 3-18), with 100% achieving a response at median 6-month follow-up; overall response rate included 69% of patients achieving complete response or better, 86% achieving very good partial response or better and 14% achieving partial response; 100% achieved early minimal residual disease-negative status at day 28 post infusion; at 6-month follow-up, 27 of 29 were progression-free | ||
| Janssen Pharmaceutical, unit of Johnson & Johnson, of New Brunswick, N.J. | Darzalex (daratumumab) | CD38-directed antibody | Multiple myeloma | Phase III Alcyone study showed the addition of Darzalex to bortezomib, melphalan and prednisone (D-VMP) improved the overall survival rate (75% vs. 62%) in newly diagnosed, transplant-ineligible patients, with a 40% reduction in the risk of death compared to VMP alone; the addition of Darzalex resulted in higher rates of minimal residual disease negativity | ||
| Jasper Therapeutics, of Menlo Park, Calif. | JSP-191 (formerly AMG-191) | Humanized antibody targeting CD117; conditioning agent to enable stem cell transplantation | Severe combined immunodeficiency | Phase I dose-escalation study data were encouraging and company plans to expand clinical development beyond SCID into clinical trials for acute myeloid leukemia, myelodysplastic syndrome and Fanconi anemia | ||
| Kezar Life Sciences Inc., of South San Francisco | KZR-261 | Sec61 translocon inhibitor | Tumors | Preclinical data demonstrated high degrees of potency against large number of therapeutically relevant oncology and immuno-oncology targets that are Sec61 client proteins, translating into broad antitumor activity; Sec61 inhibitors showed efficacy in vitro and in vivo against multiple hematologic tumor types without inducing cell death in normal cells or significant toxicity in animals | ||
| Kite, a company of Foster City, Calif.-based Gilead Sciences Inc. | Yescarta (axicabtagene ciloleucel) | CAR T therapy targeting CD19 | Refractory large B-cell lymphoma | New data from Zuma-1 trial showed, at minimum follow-up of 3 years, 47% of patients were alive, and median overall survival OS was 25.8 months; updated results from separate Zuma-1 safety management study showed earlier steroid use appeared to decrease percentage of patients with grade ?3 cytokine release syndrome (2%) and neurologic events (17%); objective response rate in cohort 4 was 73%, with 51% achieving complete response | ||
| Kite, a unit of Gilead Sciences Inc., of Foster City, Calif. | KTE-X19 | CD19 CAR T-cell therapy | Relapsed/refractory mangle cell lymphoma | Results from phase II Zuma-2 trial showed, after single infusion, the best objective response via independent radiologic central review was 93%, with 67% having achieved complete response; 12-month estimates of progression-free survival and overall survival were 61% and 83%, respectively | ||
| Kite, a unit of Gilead Sciences Inc., of Foster City, Calif. | Yescarta (axicabtagene ciloleucel) | CD19-targeting CAR T therapy | Relapsed or refractory large B-cell lymphoma | Findings from ongoing postmarketing study showed efficacy and safety of Yescarta comparable to that observed in ZUMA-1 trial, despite a larger proportion of older, more difficult-to-treat patients in the real-world setting | ||
| Kura Oncology Inc., of San Diego | Tipifarnib | Inhibitor of farnesyl transferase | Angioimmunoblastic T-cell lymphoma | Further phase II data from 20 evaluable patients with relapsed/refractory AITL showed 5 achieved complete response and 5 achieved partial response, for objective response rate of 50% on evaluable basis and 38% on an intent-to-treat basis; 3 patients experienced disease stabilization; next-generation sequencing of 19 available biopsies showed 10 (53%) carried C336R/Q386E variants in the killer-cell immunoglobulin-like receptor 3DL2, an immune checkpoint receptor | ||
| Kymera Therapeutics Inc., of Cambridge, Mass. | KYM-003 | Selective degrader of STAT3 | Hematologic malignancies | Preclinical data showed activity across multiple hematologic malignancies, including ALK-positive anaplastic large cell lymphoma, acute myelogenous leukemia and diffuse large B-cell lymphoma; treatment resulted in rapid, potent and highly selective STAT3 degradation with similar activity against both mutant and wild-type STAT3, and sustained degradation of 90% or greater led to apoptosis induction and cancer cell death within 48 hours in vitro and in vivo | ||
| Maat Pharma SA, of Lyon, France | MaaT-013 | Full-ecosystem microbiome restoration biotherapeutic | Gastrointestinal-predominant acute graft-vs.-host disease | Clinical data on compassionate use after allogeneic hematopoietic stem cell transplantation showed all 8 patients experienced at least partial response, with 3 achieving complete response, 2 with very good partial response and 3 with partial response | ||
| Macrogenics Inc., of Rockville, Md. | Flotetuzumab | Bispecific CD123 x CD3 DART molecule | Primary induction failure and early relapsed acute myeloid leukemia | Updated phase I/II data showed response rate of 30% in intent-to-treat population, including complete remission of 16.6% | ||
| Magenta Therapeutics Inc., of Cambridge, Mass. | MGTA-145 | Stem cell mobilizer | Hematopoietic stem cell transplant | Data from healthy volunteers showed drug engages CXCR2 on neutrophils to mobilize CD34+ cells into peripheral blood with limited neutrophil activation; 5 of 6 subjects who received a single dose of MGTA-145 at 0.03 dose level and plerixafor mobilized more than 20 CD34+ cells/microliter, the clinically accepted threshold for successful mobilization, in a single day | ||
| Magenta Therapeutics, of Cambridge, Mass. | CD117-ADC | Targets CD117; antibody-drug conjugate | Sickle cell disease and beta-thalassemia | Preclinical results show a single dose of CD117-ADC achieves the same level of depletion as 4 doses of busulfan chemotherapy to enable successful engraftment and persistence of stem cells modified with the ?-globin gene, which in its mutated form causes sickle cell disease and beta-thalassemia | ||
| Medigene AG, of Munich/Martinsried, Germany | DC vaccine | Dendritic cell vaccine | Acute myeloid leukemia | 1-year interim assessment of phase I/II trial show that, upon in vitro stimulation with WT-1 or PRAME, T cells from peripheral blood of 75% (6 of 8) of relapsing patients were capable of producing the pro-inflammatory cytokine IFNgamma, while such a response was detected in only in 25% (3 of 12) of the patients in remission | ||
| Molecular Partners AG, of Zurich, Switzerland | MP-0250 | Trispecific multi-DARPin candidate neutralizing VEGF-A and HGF | Relapsed/refractory multiple myeloma | Updated data from phase II study in combination with bortezomib and dexamethasone in patients previously exposed to proteasome inhibitors and immunomodulatory drugs showed 1 of 20 patients achieved complete response, 3 achieved very good partial response and 5 achieved partial response, for an overall response rate of 45% | ||
| Molecular Templates Inc., of Austin, Texas | MT-3724 | CD20-targeted immunotoxin | Relapsed/refractory diffuse large B-cell lymphoma | Of 13 serum rituximab-negative DLBCL or mixed DLBCL/FL subjects, 5 responded (38% objective response rate) across range of 5 to 100 ?g/kg doses; of the 5 responses, 2 were complete responses and 3 were partial responses; 3 had stable disease (including 2 patients with 49% and 47% tumor reductions), and 5 patients had progressive disease; of 5 patients receiving maximum tolerated dose of 50 ?g/kg, 3 responded (2 CRs, 1 PR) | ||
| Mustang Bio Inc., of New York | MB-107 | Lentiviral gene therapy | X-linked severe combined immunodeficiency | Phase I/II data presented by St. Jude's Children's Research Hospital and the NIH show MB-107 preceded by low-dose busulfan conditioning continues to be well-tolerated and results in development of functional immune system in newly diagnosed infants; enhanced transduction procedure is demonstrating improvements in older patients who received prior hematopoietic stem cell transplantation | ||
| Nektar Therapeutics Inc., of San Francisco | NKTR-255 | IL-15 agonist | Multiple myeloma and non-Hodgkin lymphoma | Preclinical data showed drug enhanced number and function of NK and CD8+ effector memory T-cell populations in peripheral blood and bone marrow from MM patients and increased expression of activating receptors found on those NK cells; drug also prevented tumor growth and increased survival of CAR T cells when added to a CD19-targeted CAR T-cell regimen in models of B-cell lymphoma | ||
| Novartis AG, of Basel, Switzerland | Kymriah tisagenlecleucel) | CAR T therapy | Relapsed/refractory diffuse large B-cell lymphoma and r/r B-cell acute lymphoblastic leukemia | 2 analyses of real-world experience showed overall response rate of 58% for 80 patients in Juliet trial in DLBCL, including 40% who achieved complete response; among 146 children and young adults in Eliana r/r ALL trial, complete response was 85% | ||
| Oncolytics Biotech Inc., of San Diego | Pelareorep | Intravenously delivered immuno-oncolytic virus | Multiple myeloma | Preclinical and clinical data demonstrate synergies between pelareorep and proteasome inhibitor carfilzomib through inflammation, apoptosis and tumor responses, supporting scientific rationale of the ongoing phase Ib trial; pelareorep treatment selectively infected multiple myeloma cells and not normal bone marrow cells | ||
| Onconova Therapeutics Inc., of Newtown, Pa. | Rigosertib | Small-molecule Ras mimetic | Myelodysplastic syndromes | At entry of the Inspire trial, 50 different mutations were identified at baseline prior to patients receiving rigosertib or physician's choice; phase II data of oral rigosertib combined with azacitidine as a first-line therapy produced an overall response rate of 90% and a complete response rate of 34% | ||
| Oncopeptides AB, of Stockholm | Melflufen | Peptide-drug conjugate | Relapsed/refractory multiple myeloma | Follow-up data from pivotal phase II Horizon study showed overall response rate of 29% and clinical benefit rate of 37% in patients with late-stage disease; NDA expected in first half of 2020 | ||
| Oncopeptides AB, of Stockholm | Melflufen | Peptide-drug conjugate | Relapsed/refractory multiple myeloma | Phase II Anchor (OP-104) triple-combination study data of melflufen and dexamethasone with daratumumab or bortezomib demonstrated positive efficacy; with daratumumab, the overall response rate was 76% with a median progression-free survival of 14.3 months; with bortezomib the ORR was 67%; both combinations were well-tolerated | ||
| Oncternal Therapeutics Inc., of San Diego | Cirmtuzumab | Antibody that binds to ROR1 | Chronic lymphocytic leukemia and mantle cell lymphoma | Phase I/II interim data of cirmtuzumab and ibrutinib show patients with CLL achieved overall best objective response rate of 85% (29 of 34) with progression-free survival of 100%; 1 patient achieved a complete response and 5 had stable disease; cirmtuzumab was well-tolerated; inhibition of ROR1 signaling was shown to reverse gene expression signatures associated with cancer-cell-stemness, oncogenic dedifferentiation and inflammation | ||
| Oryzon Genomics SA, of Madrid, Spain | Iadademstat | Small-molecule LSD1 inhibitor | Acute myeloid leukemia | New data from phase II Alice trial in elderly patients in combination with azacitidine show 6 of 8 (75%) achieving objective responses; of those, 2 were complete remission, 3 were complete remissions with incomplete hematologic recovery and 1 was partial remission; 2 of 5 patients (40%) receiving more than 3 cycles also have become transfusion independent | ||
| Portola Pharmaceuticals Inc., of South San Francisco | Cerdulatinib | Oral SYK/JAK inhibitor | Non-Hodgkin lymphoma subtypes | Interim phase IIa data showed good tolerability and a 22% complete response in patients with relapsed/refractory peripheral T-cell lymphoma, as well as an 8% complete response in patients with cutaneous T-cell lymphoma; partial responses were seen in 15% and 35% of patients, respectively; there was a 52% overall response rate observed with angioimmunoblastic T-cell lymphoma | ||
| Portola Pharmaceuticals Inc., of South San Francisco | Cerdulatinib | Oral SYK/JAK inhibitor | Relapsed/refractory follicular lymphoma | Data from 42 patients in cerdulatinib-only cohort showed overall response rate of 48%; 7 patients (17%) achieved complete response, 13 (31%) achieved partial response and 10 (24%) achieved stable disease; among 21 patients in cerdulatinib and rituximab combination cohort, the ORR was 76%; 5 patients (24%) achieved CR, 11 (52%) achieved PR and 5 (24%) achieved SD; of 11 patients in the combination cohort who have been on 1 to 3 prior therapies, ORR was 91%, with a CR rate of 36% | ||
| Precision Biosciences Inc., of Durham, N.C. | PBCAR-0191 | Targets the cancer cell surface protein CD19 | Relapsed/refractory non-Hodgkin lymphoma and R/R B-cell precursor acute lymphoblastic leukemia | Phase I data showed the NHL cohort achieved day 28-plus objective response rate of 66% (4 of 6) including 1 complete response and 3 partial responses; B-ALL cohort achieved ORR of 33% with 1 CR (1 of 3) | ||
| Principia Biopharma Inc., of South San Francisco | PRN-1008 | Oral, reversible, covalent inhibitor of BTK | Immune thrombocytopenia | Data from phase I/II trial in 31 highly treatment-resistant and refractory patients showed 39%, irrespective of dose and duration of treatment, achieved primary endpoint of ?2 consecutive platelet counts of ?50,000/µL, separated by at least 5 days, and increased by ?20,000/µL from baseline, without requiring rescue medication; 45% achieved any 2 platelet counts ?50,000/µL; most patients who achieved primary endpoint had a platelet count >30,000/µL by the first week of treatment | ||
| Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y. | REGN-5458 | BCMAxCD3 bispecific antibody | Relapsed or refractory multiple myeloma | Initial clinical data showed responses in 4 of 7 (57%) patients, including 3 of 4 (75%) in the 6-mg dose group | ||
| Roche Holding AG, of Basel, Switzerland | Venclexta/Venclyxto (venetoclax) | Binds and inhibit BCL-2 protein | Chronic lymphocytic leukemia | Updated data from pivotal phase III CLL14 study in combination with Gazyva (obinutuzumab) in previously untreated patients who had co-existing medical conditions showed, at median follow-up of more than 3 years, higher rates of minimal residual disease (MRD)-negativity in peripheral blood (76% vs. 35%; p<0.001) and bone marrow (57% vs. 17; p<0.001%) at the end of treatment vs. Gazyva/chlorambucil, respectively; MRD-negativity observed in 42% of those treated with combination who achieved complete response in peripheral blood vs. 14% for those in Gazyva/chlorambucil group (p<0.001); in bone marrow, MRD-negativity was observed in 34% vs. 11% (p<0.001) | ||
| Roche Holding AG, of Basel, Switzerland | Venclexta/Venclyxto (venetoclax) | Binds and inhibit BCL-2 protein | Chronic lymphocytic leukemia | 4-year follow-up data from pivotal phase III Murano study in combination with rituximab in relapsed/refractory patients showed reduced risk of disease progression or death by 81% vs. bendamustine/rituximab (p<0.0001); 4-year progression-free survival estimates were 57.3% vs. 4.6%, respectively | ||
| Rocket Pharmaceuticals Inc., of New York | RP-L102 | Lentiviral vector-based gene therapy | Fanconi anemia | Preliminary phase I results from 2 pediatric patients (ages 5 and 6) treated with Process B RP-L102 prior to development of severe bone marrow failure showed both exhibited early signs of engraftment based on peripheral blood, vector copy number and/or MMC-resistance; preliminary phenotypic correction also apparent in both | ||
| Rocket Pharmaceuticals Inc., of New York | RP-L102 | Lentiviral vector-based gene therapy | Leukocyte adhesion deficiency-1 | Preliminary phase I/II data from first pediatric patient show early evidence of safety and potential efficacy; early signs of engraftment with myeloid-lineage vector copy number levels of 1.5 at 3 months and CD18 expression of 45%, compared to pre-treatment CD18 expressions of <1%.; patient also displayed visible improvement of multiple disease-related skin lesions | ||
| Sangamo Therapeutics Inc., of Brisbane, Calif., and Pfizer Inc., of New York | SB-525 | Gene therapy | Severe hemophilia A | Updated data from phase I/II Alta study showed SB-525 was generally well-tolerated and demonstrated sustained increased factor VIII levels through to 44 weeks, the extent of follow-up for longest treated patient in 3e13 vg/kg dose cohort | ||
| Sangamo Therapeutics Inc., of Richmond, Calif. | ST-400 | Ex vivo gene-edited cell therapy | Transfusion-dependent beta-thalassemia | First 3 patients in phase I/II Thales study showed for patient 1, on-target indels in the infused ST-400 product were 23%, and the CD34+ cell dose was 5.4 x 106 cells/kg; for patient 2, on-target indels in the ST-400 product were 73%, with a CD34+ cell dose of 3.9 x 106 cells/kg; for patient 3, on-target indels in the ST-400 product were 54%, with a CD34+ cell dose of 10.3 x 106 cells/kg | ||
| Sanofi SA, of Paris | Sutimlimab | Monoclonal antibody targeting C1s | Primary cold agglutinin disease | In the phase III Cardinal study, 54% of 13 patients met the composite endpoint criteria, with 62.5% of 15 patients achieving a hemoglobin ? 12 g/dL or an increase of at least 2 g/dL and 71% of 17 patients remaining transfusion-free after week 5 | ||
| Seattle Genetics Inc., of Bothell, Wash. | Adcetris (brentuximab vedotin) | Antibody-drug conjugate directed to CD30 | Front-line Hodgkin lymphoma | Long-term follow-up analyses from phase II study in combination with Opdivo (nivolumab, Bristol-Myers Squibb Co.) in patients 60 and older showed 18 of 19 evaluable patients (95%) had objective response, including 13 (68%) with complete response and 5 (26%) with partial response; all response-evaluable patients experienced tumor reduction; median duration of response not yet reached | ||
| Seattle Genetics Inc., of Bothell, Wash. | Adcetris (brentuximab vedotin) | Antibody-drug conjugate directed to CD30 | Relapsed/refractory classical Hodgkin lymphoma | 2-year follow-up from phase I/II study in combination with Opdivo (nivolumab, Bristol-Myers Squibb Co.) showed of 91 treated patients, 85% (77/91) had objective response, including 67% (61/91) with complete response, 16 with partial response and 6 with stable disease; of 91 treated patients, 67 received autologous stem cell transplant per protocol with no additional salvage therapy | ||
| Seattle Genetics Inc., of Bothell, Wash., and Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Adcetris (brentuximab vedotin) | CD30-targeting antibody-drug conjugate | Stage III or IV frontline classical Hodgkin lymphoma | 4-year post-hoc exploratory analysis show progression-free survival rate for patient receiving Adcetris plus adriamycin, vinblastine and dacarbazine was 81.7% vs. 75.1% in adriamycin, bleomycin, vinblastine and dacarbazine arm, a difference of 6.6%, representing a 31% reduction in the risk of progression or death; median follow-up time was 48.4 months | ||
| Seattle Genetics Inc., of Bothell, Wash., and Takeda Pharmaceutical Co. Ltd., of Osaka, Japan | Adcetris (brentuximab vedotin) | CD30-targeting antibody-drug conjugate | CD30+ peripheral T-cell lymphoma | Analyses from Echelon-2 trial in subset of patients who underwent consolidative stem cell transplant showed combination with cyclophosphamide, doxorubicin, prednisone) vs. cyclophosphamide, doxorubicin, vincristine, prednisone showed progression-free survival estimate favored the use of stem cell transplant; after a median follow-up time of 35.9 months, the 3-year PFS rate for the 38 patients who received a stem cell transplant was 76.1%, while after a median follow-up time of 41.6 months, the 3-year PFS rate for the 76 patients who did not receive a stem cell transplant was 53.3% | ||
| Sierra Oncology Inc., of Vancouver, British Columbia | Momelotinib | JAK1/2 and ACVR1 inhibitor | Myelofibrosis | New phase III analyses show patients had significantly decreased transfusion requirements vs. those treated with Jakafi (ruxolitinib, Incyte Corp.), including nearly 10-fold higher odds of receiving no transfusion during 24-week study period (p<0.0001); patients had significantly reduced chance of receiving 1 transfusion, 2 transfusion events or 3 transfusion events; mean cumulative number of red blood cell (RBC) units received for typical patient was about half of that for patients on Jakafi (p<0.0001); Kaplan-Meier time-to-first RBC unit transfused analysis indicated immediate and sustained treatment effect vs. ruxolitinib (p<0.0001) | ||
| Springworks Therapeutics Inc., of Stamford, Conn., and Glaxosmithkline plc, of London | Nirogacestat (PF-03084014) | Gamma secretase inhibitor | Multiple myeloma and other lymphomas | GSK presented preclinical data in combination with its anti-BCMA antibody-drug conjugate, belantamab mafodotin, showing treatment of BCMA-expressing cancer cell lines with nirogacestat led to significantly increased levels of cell surface expression of BCMA and corresponding decreases in shedding of BCMA, as measured by levels of soluble BCMA; the combination resulted in synergistic increases in cancer cell killing vs. belantamab mafodotin alone, with an up to ~3,000-fold improvement in cytotoxicity | ||
| Stemline Therapeutics Inc., of New York | Elzonris (tagraxofusp) | CD123-directed cytotoxin | Intermediate or high-risk relapsed/refractory myelofibrosis | Phase I/II data showed 45% (9/20) of patients had symptom burden reduction, including 3 with symptom response per IWG-MRT 2013 MF response criteria; 53% (8/15) of patients with baseline splenomegaly >= 5cm experienced spleen size reduction; 20% (3/15) had reduction >35% (specifically 100%, 47% and 46% reductions) | ||
| Stemline Therapeutics Inc., of New York | Elzonris (tagraxofusp) | CD123-directed cytotoxin | Relapsed/refractory multiple myeloma | Phase I/II data showed combination with pomalidomide and dexamethasone produced partial responses in 56% (5/9) of patients; those patients notably had decreases in their plasmacytoid dendritic cells, a cell type implicated in myeloma growth and aggressiveness, and cell of origin of blastic plasmacytoid dendritic cell neoplasm | ||
| Synimmune GmbH, of Tubingen, Germany | Flysyn | Fc-optimized antibody | Acute myeloid leukemia | Interim phase I data of 21 patients treated in 5 cohorts showed 7 (33%) achieved a minimal residual disease response and 1 patient achieved complete molecular remission for more than 1 year | ||
| Syros Pharmaceuticals Inc., of Cambridge, Mass. | Fetal hemoglobin repressor | Nuclear factor IX | Sickle cell disease | Preclinical work discovered and validated nuclear factor I X, using company’s gene control platform, showing how gamma-globin gene, which leads to production of fetal hemoglobin, is controlled and points to new potential targets for therapeutic intervention in SCD | ||
| Takeda Pharmaceutical Co., of Osaka, Japan | Ninlaro (ixazomib) | Oral proteasome inhibitor | Relapsed/refractory systemic light-chain amyloidosis | Additional data from phase III Troumaline-AL1 study in combination with dexamethasone, which reported not meeting first of 2 primary endpoints in June 2019, showed complete response rate of 26% vs. 18% in physician’s choice arm; other endpoint data showed median duration of hematologic response was 46.5 months vs. 20.2 months in physician’s choice; vital organ progression-free survival was 18 months and 11 months, respectively, and hematologic PFS was 20.1 months and 16.7 months, respectively; time to treatment failure was 10.1 months and 5.2 months, respectively | ||
| TG Therapeutics Inc., of New York | TG-1701 | Oral BTK inhibitor | B-cell malignancies | Phase I data show single-agent treatment produced partial responses at multiple dose levels across multiple B-cell diseases; 86% (6/7) of patients treated with 100 mg TG-1701 plus U2 have achieved a response | ||
| TG Therapeutics Inc., of New York | Ublituximab (TG-1101) and umbralisib (TGR-1202) | Glycoengineered monoclonal antibody targeting CD20 and dual PI3K delta and CK1 epsilon inhibitor | Relapsed/refractory chronic lymphocytic leukemia | Phase I/II data testing triple combination therapy with venetoclax (Roche Holding AG) showed overall response rate of 87% 20/23) after U2 induction period at cycle 3, prior to introduction of venetoclax, in relapsed/refractory CLL patients, including patients refractory to ibrutinib | ||
| TG Therapeutics Inc., of New York | Ublituximab (TG-1101) and umbralisib (TGR-1202) | Glycoengineered anti-CD20 monoclonal antibody and dual inhibitor of PI3K delta and CK1 epsilon | Relapsed/refractory chronic lymphocytic leukemia | Triple-therapy data from phase I/II study in combination with venetoclax showed patients followed for at least 12 months had 100% objective response rate and all achieved minimal residual disease (MRD) negativity in peripheral blood; 7 of 9 patients also achieved MRD negativity in bone marrow | ||
| Trovagene Inc., of San Diego | Onvansertib | Oral adenosine triphosphate competitive inhibitor of serine/threonine polo-like-kinase 1 enzyme | Relapsed/refractory acute myeloid leukemia | Phase Ib data showed 6 (17%) patients had complete response; 9 (25%) had an objective response across LDAC and decitabine arms and doses | ||
| UCB SA, of Brussels, Belgium | Rozanolixizumab | Subcutaneous antibody binding to FcRn | Primary immune thrombocytopenia | Phase II data showed clinically relevant improvements (i.e., reaching ?50x109/L) in platelet count and decreases in immunoglobin G levels observed across all dose groups, with higher response rate (55%–67% in 1 x 15-mg/kg and 1 x 20-mg/kg dose groups vs. 36$–45% in 5 x 4-mg/kg, 3 x 7-mg/kg and 2 x 10-mg/kg dose groups) and shorter time to response achieved by the 1 x 15-mg/kg and 1 x 20-mg/kg doses | ||
| Unum Therapeutics Inc., of Cambridge, Mass. | ACTR-707 | Antibody-coupled T-cell receptor engineered T-cell therapy | Relapsed/refractory CD20+ non-Hodgkin lymphoma | Ongoing phase I trial in combination with rituximab showed complete response achieved in 40% (8 of 20) patients in cohorts 1-4; of 8 complete responders, 4 remained in completed response at 6 months follow-up, 2 remain in complete response but have not yet reached 6-month timepoint for evaluation and 2 progressed before 6 months | ||
| Unum Therapeutics Inc., of Cambridge, Mass. | ACTR-087 | Antibody-coupled T-cell receptor engineered T-cell therapy | Relapsed/refractory CD20+ non-Hodgkin lymphoma | Data from phase I ATTCK-20-2 trial in combination with rituximab generated complete responses lasting greater than 6 months in 20% (4 of 20) of evaluable patients | ||
| Verastem Inc., of Boston | Copiktra (duvelisib) | PI3K inhibitor | Relapsed/refractory peripheral T-cell lymphoma | Results from dose-optimization portion of phase II Primo study showed objective response rates of 35% and 54% in cohorts 1 and 2, respectively, with complete response rates of 25$ and 31%, respectively | ||
| Viracta Therapeutics Inc., of San Diego | Nstat (nanatinostat) | Oral HDAC inhibitor | EBV-associated relapsed/refractory lymphomas | Phase Ib/IIa data in combination with valganciclovir showed overall response rate in phase Ib portion of 56% (10/18), with 28% (5/18) complete repose and clinical benefit rate of 78%; median duration of treatment for responders of 6.5 months; in HIV-negative patients, ORR was 67% (10/15), with a 33% (5/15) CR and a CBR of 93% (14/15) | ||
| Windmil Therapeutics Inc., of Baltimore | CAR-MILs | Chimeric antigen receptor-engineered MILs | Multiple myeloma | Preclinical data showed CAR-MILs possess superior antitumor immunity vs. traditional CAR T cells engineered using peripheral blood lymphocytes; in vitro studies showed superior killing of tumor target cells; CAR-MILs also showed greater degree of cytokine and chemokine secretion consistent with a more polyfunctional cytokine profile | ||
| Xencor Inc., of Monrovia, Calif. | XmAb®13676 | CD20 x CD3 bispecific antibody | B-cell malignancies | Data from ongoing phase I study showed XmAb13676 was generally well-tolerated, and a priming dose of 45 mcg/kg was chosen for continued dose escalation in part B for patients with non-Hodgkin lymphoma | ||
| Xenikos BV, of Nijmegen, the Netherlands | T-Guard | 2 toxin-conjugated monoclonal antibodies targeting CD3 and CD7 molecules on T cells and NK cells | Steroid-refractory acute graft-vs.-host disease | Data from expanded access program as second- or third-line treatment to 12 high-risk adults following allogeneic stem cell transplantation for a myeloid or lymphoid malignancy showed 75% achieved clinical response by day 28, with 5 of 9 achieving complete remission; 6-month and 1-year overall survival rate was 75% and 58%, respectively | ||
| Ziopharm Oncology Inc., of Boston | T-cell therapies | Sleeping Beauty-modified T cells expressing membrane bound IL-15 | Tumors | Preclinical data showed T cells can be genetically modified with Sleeping Beauty system to express TCR and mbIL-15; T cells expressing TCR and mbIL-15 exhibited superior antitumor effects vs. TCR-modified T cells without mbIL15; T cells expressing TCR and mbIL-15 persist in mouse model | ||