- French drugmaker GenFit is running a large clinical trial to test whether its main drug can treat a liver disease known as NASH. Initial data was expected last year, then pushed back to sometime this quarter. On Thursday, GenFit delayed unveiling the data once again, saying the study will remain blinded for several more weeks to incorporate feedback from the Food and Drug Administration.
- Analysts at SVB Leerink said they spoke to company management after the announcement and were told the FDA's feedback wasn't related to any change in the trial's primary endpoint analysis, but instead was about secondary measures discussed by the agency and GenFit at a late 2019 meeting. The delay, according to SVB Leerink, is thus tied to the amount of time FDA officials said it could take before they send GenFit their latest feedback.
- Others took a more skeptical view of the update, with investors sending GenFit shares down almost 6% Friday morning. "We certainly do not think today's announcement is confidence inspiring given the already high degree of skepticism around this program," wrote analysts at Stifel in a Feb. 20 note to clients.
NASH, or nonalcoholic steatohepatitis, is a disease in which the buildup of liver fat progressively damages the organ, leading in the most severe cases to cirrhosis and the need for a transplant. Though limited, research suggests millions of patients in the U.S. have NASH, making it an attractive target for drug companies.
Yet there's much about the development of NASH that these companies are still figuring out. Only one developer, Intercept Pharmaceuticals, has been able to successfully guide a drug through late-stage testing — and even then, the drug reduced scarred liver tissue in just about a quarter of NASH patients.
Gilead, which brought to market medicines that effectively cure hepatitis C, has had trouble finding success in this next big liver disease. Its experimental treatment missed the mark in multiple studies, causing investors to question whether NASH would remain a focus for the big biotech.
"Gilead is sort of the bellwether for liver diseases. And so, to the extent they struggle, it doesn't bode well for some of the smaller players," Brian Lian, CEO of NASH drug developer Viking Therapeutics, said in an interview with BioPharma Dive early this year.
Against this background, analysts maintain a cautious view of GenFit's drug, elafibranor, and its place in NASH treatment. The drug missed the primary goal of a mid-stage trial back in 2015 — though GenFit said the results were affected by unexpected circumstances — and some see a significant chance for another miss in the late-stage RESOLVE-IT trial. SVB Leerink, which has an "Outperform" rating on GenFit, models a 55% probability of success for elafibranor in NASH.
"This introduces a great deal of uncertainty to the potential success of elafibranor," analysts at JMP Securities wrote about the delayed unblinding. The investment bank noted, however, that GenFit's update has limited readthrough to fellow drug developers working in NASH.