By summer solstice, we'll know the fate of Intercept's NASH drug
Intercept Pharmaceuticals has another date with the FDA, as the regulator will decide for a second time whether to approve the biotech’s drug for patients with pre-cirrhotic liver fibrosis due to nonalcoholic steatohepatitis, or NASH.
The agency set an action date of June 22. FDA said no around the same time in 2020.
At stake is an accelerated approval for obeticholic acid, or OCA, which is already on the market for primary biliary cholangitis. Intercept and ex-US rights owner Advanz market the drug as Ocaliva for the bile duct-destroying disease.
The drug developer touted two interim analyses from the Phase III REGENERATE study, which has brought mixed results in the past four years. The once-daily 25 mg group experienced a win on the primary endpoint at an 18-month analysis, Intercept said last July, touting 22.4% of patients saw at least one stage of fibrosis improvement without worsening of the fatty liver disease. The placebo group was 9.6%.
Patients in the 10 mg group didn’t have the same experience. That portion of the trial did not meet the primary endpoint. On a second primary endpoint, Intercept said the drug didn’t meet the bar on resolution of NASH with no worsening of liver fibrosis. At the time, Jefferies analysts said it wasn’t a surprise.
Then, in September, Intercept reported a fail on another Phase III trial of the drug in NASH. The study, dubbed REVERSE, was not mentioned in Intercept’s Thursday morning press release announcing the FDA’s acceptance of the NDA.
With safety data on nearly 2,500 patients, including 1,000 on the drug for four years, Intercept continues to think it can be the first to market with a NASH drug.
“This regulatory milestone brings us one step closer to reaching our goal of delivering the first available therapy for patients living with pre-cirrhotic fibrosis due to NASH – the most rapidly growing cause of liver transplantation in the U.S.,” Jerry Durso, CEO and president of Intercept, said in a statement.
Biopharmas have long been tripped up by R&D in NASH, but readouts in recent months might paint a rosier picture in the near future, with Madrigal saying it will file for accelerated approval early this year after a Phase III win that sent shares skyrocketing and led to a $300 million capital infusion. Akero Therapeutics also buoyed the NASH field with wins on primary and multiple secondary goals in its Phase IIb study last fall. Meanwhile, Chemomab Therapeutics said it cleared a mid-stage test but doesn’t have immediate plans to stay in NASH because of the costs associated with Phase III studies in the space.