Company Product Description Indication Status
Phase I
Prevail Therapeutics Inc., of New York PR-006 AAV9 gene therapy Frontotemporal dementia First of up to 15 participants with GRN mutations dosed in phase I/II Proclaim trial; biomarker and safety analysis on subset of participants expected in 2021
Puretech Health plc, of Boston LYT-200 Galectin-9 inhibitor Metastatic solid tumors Adaptive phase I/II trial initiated, with phase I assessing escalating doses to identify phase II dose, with top-line results expected in fourth quarter of 2021; phase II expansion cohort expected to assess recommended dose as single agent or in combination with chemotherapy and anti-PD-1 therapy in multiple solid tumor types, including pancreatic cancer and cholangiocarcinoma
Phase II
Akari Therapeutics plc, of New York  Nomacopan Dual complement C5 and leukotriene inhibitor Paroxysmal nocturnal hemoglobinuria Data accrued from 19 participants treated for >30 cumulative patient-years showed self-administered study drug was well-tolerated and led to transfusion independence of 79% for 14 formerly transfusion dependent patients treated for at least 6 months, with median time since last transfusion of 13.8 months
Puma Biotechnology Inc., of Los Angeles Nerlynx (neratinib) EGFR antagonist; ERBB2/ERBB4 tyrosine kinase receptor inhibitor Breast cancer Updated results from ongoing Summit basket trial of neratinib + trastuzumab + fulvestrant in 37 efficacy evaluable participants with HER2-mutant, hormone receptor-positive, metastatic disease showed 17 (45.9%) had confirmed objective response, including 1 complete response (CR) and 16 partial responses (PRs), and 20 (54.1%) had clinical benefit (confirmed CR or PR or stable disease for at least 24 weeks); median duration of response was 10.9 months and median progression-free survival was 8.3 months
Sellas Life Sciences Group Inc., of New York Nelipepimut-s ERBB2 tyrosine kinase receptor modulator Breast cancer Data from up to 6 months follow-up from Vadis combination study with GM-CSF in ductal carcinoma in situ disease showed relative change in cytotoxic T-lymphocytes percentage mean values was +1,300+450% vs. 250+150% for GM-CSF alone, for highly statistically significant difference of duration of immune response (p=0.000094)
Phase III
Geron Corp., of Foster City, Calif. Imetelstat Telomerase inhibitor Myelofibrosis ImpactMF trial opened to screen and enroll ~320 people with refractory disease to assess study drug vs. best available therapy; primary efficacy endpoint is overall survival
Greenwich Lifesciences Inc., of Stafford, Texas GP-2 9 amino acid transmembrane peptide of HER2/neu protein Breast cancer Single registration trial will seek conditional FDA approval based on planned interim analysis, which will trigger BLA submission; phase III aims to reproduce findings from phase IIb trial that found first 6 intradermal injections of GP-2 + GM-CSF elicited immune response and reduced recurrence rates to 0% in HER2/neu 3+ patients who received standard trastuzumab after surgery
Scyneis Inc., of Jersey City, N.J. Ibrexafungerp Antifungal agent Difficult-to-treat mucocutaneous, invasive fungal infections and multidrug-resistant Candida auris infections Data review committee is analyzing data from an additional 43 patients in the Furi and Cares open-label studies; data from the 84 total patients expected in the first quarter of 2021
Tychan Pte Ltd., of Singapore TY-027 Anti-SARS-CoV-2 monoclonal antibody COVID-19 infection Trial to begin recruiting 1,305 COVID-19-positive patients to assess effectiveness of slowing disease progression and accelerating recovery
Phase IV
Mitsubishi Tanabe Pharma America Inc., of Jersey City, N.J. Radicava (edaravone)  Neuroprotectant Amyotrophic lateral sclerosis Real-world analysis based on observational, retrospective cohort study of 167 people with ALS treated with study drug for at least 3 consecutive months showed changes in ALS Functional Rating Scale-Revised (ALSFRS-R) scores were consistent with those observed in pivotal phase III trial, which showed study drug slowed loss of physical function over 24-week period by 33% or 2.49 ALSFRS-R points (p=0.0013) vs. placebo


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