Liver Disorders

April 13, 2015

GENFIT reported results of a phase IIb trial of GFT505 for nonalcoholic steatohepatitis (NASH). The 52-week trial enrolled 274 subjects (double-blind, placebo-controlled; three arms: placebo, 80mg, 120mg) with centrally-read, liver biopsy proven NASH. Treatment with GFT505 provided a significant beneficial effect on the primary endpoint (GFT505 120mg v. placebo, p=0.016, RR=2.03) in the global randomized population (n=274, full analysis set), in which patients without an end-of-treatment biopsy were considered as non-responders. The primary endpoint also was achieved in the evaluable population of patients who underwent both baseline and end-of-study liver biopsies (n=237, ITT; p=0.027 v. placebo; RR=1.94). In the evaluable patient population, GFT505 120mg also had a beneficial effect of a decrease of NAS-score =2 (p=0.04 v. placebo). The data provide the basis for upcoming phase III trials.

April 6, 2015

Conatus Pharmaceuticals issued results of a phase II study of emricasan for nonalcoholic fatty liver disease (NAFLD), including the subset of NAFLD patients with nonalcoholic steatohepatitis (NASH). The double-blind, placebo-controlled trial enrolled 38 patients. The trial met its primary endpoint, showing a statistically significant (p<0.05) reduction in alanine amino transferase (ALT) in patients treated for 28 days with emricasan at 25mg twice per day dosing compared to patients in the placebo control group. Reductions from baseline in ALT at day 28 of approximately 39% in the emricasan treatment arm and approximately 14% in the placebo arm were similar to results observed in previous trials. Elevated baseline levels of three key serum biomarkers—caspase-cleaved cytokeratin 18 (cCK18), full length cytokeratin 18 and caspase 3/7—also showed statistically significant reductions from baseline in emricasan-treated patients at day 28. A reduction from baseline in cCK18 at Day 28 of approximately 30% in the emricasan treatment arm and an increase from baseline of approximately 4% in the placebo arm were similar to results observed in previous trials. Emricasan was safe and well tolerated in the NAFLD/NASH trial, with no dose-limiting toxicities and no drug-related serious adverse events.