Still riding high on some strong early-year data for its liver disease treatment, Intercept Pharmaceuticals' ($ICPT) shares shot up once more as its lead drug came through in Phase IIb, dismissing some earlier safety worries and setting the table for late-stage study.
In a 283-patient study, Intercept's drug for nonalcoholic steatohepatitis (NASH) met its primary endpoint of improving fatty-liver disease symptoms without worsening scarring, with 46% of those in the treatment arm showing meaningful improvement compared with 21% on placebo. The treatment, called obeticholic acid (OCA), also met its secondary endpoints of broadly reducing disease scores and significantly improving liver scarring, with 35% of OCA patients reporting a reduction in fibrosis compared with 19% taking placebo.
That last endpoint likely stood out to investors concerned about safety signals in the drug's earlier mid-stage results, and the new data sent Intercept up about 60% in after-hours trading. The company's share value has surged more than 500% since the start of the year, when it first unveiled encouraging results against a disease with no approved treatments.
Now Intercept is gearing up for a Phase III OCA trial in NASH, planning to start enrollment in the first half of 2015.
At the same time, the biotech is working through a Phase III program studying OCA in primary biliary cirrhosis (PBC), looking to launch a confirmatory trial by year's end and complete an FDA filing next year. Intercept picked up the agency's fast-track designation for that indication, guaranteeing a shorter trip through regulatory review.
Beyond NASH and PBC, Intercept is developing its lead bile acid analog for alcoholic hepatitis and primary sclerosing cholangitis. The company's pipeline also includes the preclinical fibrosis treatment INT-767 and an early-stage diabetes therapy dubbed INT-777.
- read the filing
Special Report: The top 15 late-stage blockbusters in the pipeline - Obeticholic acid (OCA), Intercept Pharmaceuticals