Company Product Description Indication Status
Phase I
Arbutus Biopharma Corp., of Warminster, Pa. AB-729 GalNac-delivered RNAi therapeutic  Hepatitis B infection Phase Ia/Ib AB-729-001 trial in individuals with chronic infection showed mean 60 mg HBsAg reduction of 0.99 log10 IU/mL at week 12 following single 60-mg injection, with normal ALT and AST values throughout follow-up period; 90-mg single-dose and 60-mg multidose cohorts initiated, with data expected in second half of 2020
Phase II
Amplyx Pharmaceuticals Inc., of San Diego Fosmanogepix (APX-001) GPI anchored wall transfer protein 1 inhibitor Candida infection Study completed enrollment; top-line data expected by July 2020
Hepion Pharmaceuticals Inc., of Edison, N.J. CRV-431 Peptidyl-prolyl cis-trans isomerase A inhibitor Nonalcoholic steatohepatitis fibrosis With submission of phase I data memo and phase IIa protocol to FDA completed, phase IIa trial expected to begin in June 2020, enrolling 18 adults with presumed NASH F2 or F3 fibrosis; in addition to safety, tolerability and pharmacokinetics, trial will assess antifibrotic activity measured by quantification of selected fibrosis biomarkers 
Lidds AB, of Uppsala, Sweden Liproca Depot Androgen receptor antagonist Prostate cancer Final data from phase IIb LPC-004 study showed best efficacy occurred in group that received 16-ml injection, where 95% showed decrease in PSA, 57% were classified as responders at month 5 and PSA maximum response rate during study period was 67%
MGB Biopharma Ltd., of Glasgow, U.K. MGB-BP-3 DNA minor groove binder Clostridium difficile infection Dose-ranging study met endpoints; 250-mg dose twice daily for 10 days achieved initial cure and sustained cure of 100%
Phase III
Agios Pharmaceuticals Inc., of Cambridge, Mass. Tibsovo (ivosidenib)  IDH1 gene inhibitor Cholangiocarcinoma Data from ClarIdhy study in previously treated patients with IDH1 mutation, which met primary endpoint of statistically significant improvement in progression-free survival vs. placebo, published in The Lancet Oncology
Biogen Inc., of Cambridge, Mass. Vumerity (diroximel fumarate); Tecfidera (dimethyl fumarate) Monomethyl fumarate prodrug; Kelch-like ECH associated protein 1 modulator; nuclear erythroid 2-related factor 2 stimulator Multiple sclerosis Data from Evolve-MS-2 study showed those treated with either drug (n=295) had reduction in gadolinium-enhancing (Gd+) lesion counts as early as 7 weeks after treatment initiation; after continuing with Vumerity in ongoing open-label Evolve-MS-1 study (n=82), 96.3% were free from Gd+ lesions after 1 year; exploratory analysis of Evolve-MS-2 (n=502) on GI symptoms in relapsing-remitting MS showed those taking Vumerity were less likely than those on Tecfidera to score 2 or higher, the primary endpoint, for 2 or more consecutive days on Individual GI Symptom and Impact Scale (17.4% vs. 29.3%, respectively) and to report concomitant medications for GI tolerability (19% vs. 31%)
Covis Pharma BV, of Luxembourg Alvesco (ciclesonide) Glucocorticoid receptor agonist COVID-19 infection Began recruiting for study to assess the asthma drug in non-hospitalized patients 12 and older with symptomatic infection; primary endpoint is reduction in hospital admission or mortality rate by day 30
Mezzion Pharma Co. Ltd., of Seoul  Udenafil PDE5 inhibitor Single ventricle heart disease Fuel trial showed statistically significant results for efficacy endpoint of myocardial performance index (MPI), including statistically significant improvement in ventricular performance measured by MPI (p=0.028) vs. placebo


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