Company Product Description Indication Status
Phase I
Menarini Group, of Pomezia, Italy MEN-1611 Phosphatidylinositol 3-kinase inhibitor PIK3CA mutated, RAS and RAF wild-type metastatic colorectal cancer after failing regimens containing irinotecan, oxaliplatin, 5-FU and anti-EGFR Plans to launch the Precise-01 study in second half of 2020 to determine the recommended phase II dose for MEN-1611 plus cetuximab and test the safety, tolerability and pharmacokinetics of the combination  
Moleculin Biotech Inc., of Houston Annamycin  Next-generation anthracycline Elapsed or refractory acute myeloid leukemia During the end-of-phase I meeting with the FDA, the company agreed to expand testing for cardiotoxicity throughout the remainder of its European phase I study where the dose is being escalated to find the recommended phase II dose
Phase II
4D Pharma plc., of Leeds, U.K. MRx-4DP0004 Single-strain live biotherapeutic Hospitalized COVID-19 Opened enrollment in study measuring the mean change in clinical status score as measured by the WHO Ordinal Scale for Clinical Improvement; secondary endpoints include need for, and duration of, ventilation, as well as safety and tolerability
Abivax SA, of Paris, France ABX-464 Up-regulates miR-124 Elderly or high-risk COVID-19 patients Treated first of 1,034 patients in the study measuring the ability of ABX-464 to limit viral replication and severe inflammation that leads to acute respiratory distress syndrome
Pfizer Inc., of New York Glasdegib  Hedgehog pathway inhibitor Acute myeloid leukemia Temporary recruitment hold for trial in older patients with poor-risk AML due to COVID-19
Vaccibody A/S, of Oslo, Norway VB-10.16 DNA vaccine Advanced or recurrent, non-resectable human papillomavirus type 16-positive cervical cancer Dosed first of up to 50 patients in the VB C-02 study testing VB-10.16 plus Tecentriq (atezolizumab, Roche Holding AG)
Phase III
Apellis Pharmaceuticals, Inc., of Waltham, Mass. Pegcetacoplan (APL-2) C3 inhibitor Currently untreated paroxysmal nocturnal hemoglobinuria Completed enrollment in the Prince study; top-line results expected in early 2021
Biocardia Inc., of San Carlos, Calif. Cardiamp cell therapy Autologous bone marrow-derived stem cell therapy + Helix device Chronic myocardial ischemia Cardiamp CMI study activated with the completion of the first site initiation visit; primary endpoint of the 343-patient study is change from baseline in total exercise time on the treadmill using the Modified Bruce Protocol
Brainstorm Cell Therapeutics Inc., of New York Nurown Glial- and brain cell line-derived neurotrophic factor-producing stem cells Amyotrophic lateral sclerosis Last of 200 participants dosed in pivotal trial; top-line data expected on schedule in fourth quarter of 2020
Helsinn Healthcare SA, of Lugano, Switzerland, and MEI Pharma Inc., of San Diego Pracinostat Histone deacetylase inhibitor Acute myeloid leukemia Interim futility analysis by independent data monitoring committee of ongoing combination study with azacitidine in individuals unable to receive standard intensive chemotherapy concluded trial was unlikely to meet primary endpoint of overall survival vs. control group; recruitment to be halted and study terminated
Rhythm Pharmaceuticals Inc., of Boston Setmelanotide Melanocortin-4 receptor agonist Obesity Among participants enrolled in supplemental cohorts, mean reduction in baseline body weight for pro-opiomelanocortin (POMC) deficiency obesity patients (n=4) was -26.3% and mean reduction in body weight for leptin receptor (LEPR) deficiency obesity patients (n=4) was -13.2%; estimated mean % reduction in most hunger score for evaluable patients in supplemental cohorts was -57.3%; combining data from supplemental and pivotal cohorts, 12 of 14 with POMC deficiency obesity and 9 of 15 with LEPR deficiency obesity achieved primary endpoints of > 10% weight loss over about 1 year
Rhythm Pharmaceuticals Inc., of Boston Setmelanotide Melanocortin-4 receptor agonist Obesity As of April 16, 2020, 15 participants from pivotal trials enrolled in long-term extension study maintained durable weight loss with study drug for up to 155 weeks, with stable hunger scores 
Strongbridge Biopharma plc, of Dublin  Recorlev (levoketoconazole)  Hydroxylase inhibitor; lanosterol-14 demethylase inhibitor Cushing's syndrome Last of 43 participants with endogenous disease completed last visit in randomized withdrawal phase of Logics study; top-line data expected in third quarter of 2020 


For more information about individual companies and/or products, see Cortellis.

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