Company Product Description Indication Status
Phase I
Actinium Pharmaceuticals Inc., of New York Actimab-A CD33 antagonist Relapsed/refractory acute myeloid leukemia Completed planned dose escalation and patient enrollment in study testing drug with salvage chemotherapy CLAG-M in patients who are fit for intensive therapy
Avidity Biosciences Inc., of La Jolla, Calif. AOC-1001 Antibody oligonucleotide conjugate Myotonic dystrophy type 1 Dosed first participants in phase I/II Marina trial
Bicycle Therapeutics plc, of Cambridge, U.K. BT-7480 Fully synthetic Bicycle tumor-targeted immune cell agonist targeting Nectin-4 and agonizing CD137 Advanced solid tumors associated with Nectin-4 expression Dosed first patient in dose-escalation portion of phase I/II study
Codexis Inc., of Redwood City, Calif., and Nestlé Health Science, of Lausanne, Switzerland CDX-7108 Lipase variant Exocrine pancreatic insufficiency Dosed first subject in the healthy volunteer portion of the study that will test the safety, tolerability and pharmacokinetics of single and multiple doses of CDX-7108; study also includes a cohort testing the pharmacodynamics, safety, tolerability and immunogenicity of CDX-7108 in patients
Epizyme Inc., of Cambridge, Mass. EZM-0414 SETD2 inhibitor T(4;14) multiple myeloma, non t(4;14) multiple myeloma and diffuse large B-cell lymphoma Started study designed to test the safety and determine the optimal dose of EZM-0414; following the dose-ranging phase, 3 cohorts will enroll patients with the different blood cancers
Kazia Therapeutics Ltd., of Sydney EVT-801 VEGFR3 inhibitor Renal cell carcinoma and soft-tissue sarcoma Enrolled first patient in the first stage of the study testing multiple ascending doses to determine the maximum tolerated dose and recommended phase II dose (RP2D); second stage will treat an expanded cohort of patients treated at the RP2D; study will measure safety, efficacy, pharmacokinetics and tissue and blood biomarker analyses
Medivir AB, of Stockholm, and IGM Biosciences Inc., of Mountain View, Calif. Birinapant + IGM-8444 Synthetic small-molecule peptidomimetic of SMAC and inhibitor of IAP + DR5 agonist antibody Relapsed and/or refractory solid cancers Started study testing IGM-8444 as a single agent and in combination with FOLFIRI, bevacizumab, venetoclax and/or birinapant; study has a dose-escalation stage and an expansion stage; primary outcome measures are safety and to determine the recommended phase II dose; objective response rate, duration of response and progression-free survival will be measured as secondary endpoints
Nervgen Pharma Corp., of Vancouver, British Columbia NVG-291 Protein tyrosine phosphatase modulator Nerve injury In healthy volunteers, NVG-291 was well-tolerated and had favorable pharmacokinetic properties at the highest single ascending dose; safety review committee recommended proceeding to the multiple ascending-dose portion of the trial
Nodthera Ltd., of Cambridge, U.K. NT-0796 NLRP3 inflammasome inhibitor Healthy volunteers (eventually chronic inflammatory diseases of the brain) First subject dosed in the study testing single and multiple ascending doses of NT-0796; primary objective is safety and tolerability; secondary objectives include pharmacokinetics and pharmacodynamics and cerebrospinal fluid sampling to assess NLRP3 target engagement and compound exposure
PDC*Line Pharma SA, of Liege, Belgium PDC*lung01 Irradiated human plasmacytoïd dendritic cells loaded separately with 7 distinct synthetic human leukocyte antigen serotype-restricted peptide encoded by a tumor antigen Non-small-cell lung cancer First patient dosed with PDC*lung01 plus pembrolizumab in the fourth and final 42-patient cohort; no dose-limiting toxicity or other safety signals observed in the first 3 cohorts
Sangamo Therapeutics Inc., of Brisbane, Calif. Isaralgagene civaparvovec (ST-920) Gene therapy expressing alpha-galactosidase A Fabry disease The 4 patients treated in the first 2 dose cohorts (0.5e13 vg/kg and 1e13 vg/kg) had alpha-galactosidase A activity 2-fold to 15-fold above mean normal that was maintained for up to 1 year for the first patient treated and through 14 weeks for the most recently treated patient; 1 patient stopped enzyme replacement therapy (ERT) and the other patient on ERT plans to stop; fifth patient dosed at the third dose level (3e13vg/kg)
Vaccitech plc, of Oxford, U.K. ChAdOx1 MERS Vaccine Middle Eastern respiratory syndrome prophylaxis Data published in The Lancet Microbe showed the vaccine was generally well-tolerated and induced both humoral and cellular immune responses that lasted through the 6-month follow-up period
Zealand Pharma A/S, of Copenhagen ZP-8396 Amylin analogue Obesity First subject dosed in the single ascending-dose study testing the safety, tolerability, pharmacokinetics and pharmacodynamics in healthy volunteers
Phase II
Ambrx Inc., of San Diego ARX-788 Anti-HER2 antibody drug conjugate HER2-positive metastatic breast cancer First patient dosed in the Ace-breast-03 study testing the efficacy and safety of ARX-788
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 Mild to moderate Alzheimer’s disease After 52 weeks of treatment, Alzheimer’s Disease Assessment Scale, Cognitive Subscale, 13-item Version declined by 1.17 points and 0.76 points for patients treated with 10 mg and 30 mg, respectively, compared to an expected decline of 5.5 points based on treatment with placebo in other studies; treatment did not significantly affect scores on the Alzheimer’s Disease Cooperative Study – Clinical Global Impression of Change scale compared to placebo over 26 or 52 weeks
Chimerix Inc., of Durham, N.C. ONC-201 Dopamine receptor D2 antagonist and caseinolytic protease agonist Recurrent H3 K27M-mutant glioma Objective response rate was 20% by Response Assessment in Neuro-Oncology Criteria for High Grade Gliomas criteria; median duration of response was 11.2 months; median time to response was 8.3 months; full analysis will be presented at the Society for Neuro-Oncology Annual Meeting
Talaris Therapeutics Inc., of Boston FCR-001 Allogeneic cell therapy Kidney transplant Long-term follow-up of 26 patients weaned off chronic immunosuppression (IS) showed all have continued to remain off IS without rejecting their donated kidney
Phase III
Amylyx Pharmaceuticals Inc. of Cambridge, Mass. AMX-0035 Sodium phenylbutyrate and taurursodiol Amyotrophic lateral sclerosis First of approximately 600 patients dosed in the Phoenix study; primary efficacy outcome of the 48-week study is a joint assessment of Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised and survival; secondary endpoints include slow vital capacity, measured both at home using a self-administered spirometer to support virtual data collection and at clinic sites using standard spirometry, quality of life patient-reported outcome assessments, ventilation-free survival rates and other measures
Eyenovia Inc., of New York Microline Formulation of pilocarpine Presbyopia First of approximately 140 patients enrolled in the Vision-2 study; primary endpoint is improvement in high contrast binocular distance corrected near visual acuity measured in low light conditions 2 hours after treatment; top-line results expected in the second quarter of 2022
Talaris Therapeutics Inc., of Boston FCR-001 Allogeneic cell therapy Kidney transplant First 2 patients treated for more than 12 months have been successfully weaned off all chronic immunosuppression without evidence of rejection and with stable kidney function; 3 patients treated for at least 3 months achieved and maintained >50% T-cell chimerism
Xoma Corp., of Emeryville, Calif., and Novartis AG, of Basel, Switzerland NIS-793 Monoclonal antibody targeting TGF beta First-line metastatic pancreatic ductal adenocarcinoma Started the study comparing NIS-793 plus gemcitabine and nab-paclitaxel compared to gemcitabine and nab-paclitaxel alone; primary efficacy endpoint is overall survival; progression-free survival, overall response rate, disease control rate and time to response will be measured as secondary endpoints

Notes

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