On Friday night Intercept Pharmaceuticals managed to rattle investors with a 10-K filing flagging a collection of serious cardiac adverse events recorded in a study of its lead drug OCA. But come Sunday morning, the biotech balanced out that bitter taste with an announcement that its Phase III trial had ended with positive data, setting up a regulatory filing for approval.
Both dosages used in the Phase III POISE trial met the primary endpoint for a reduction in serum alkaline phosphatase in patients suffering from primary biliary cirrhosis (PBC). While only 10% of the placebo arm hit that endpoint, 47% in the 10-mg arm and 46% in the 5-titrated-to-10-mg OCA (obeticholic acid) group achieved the goal. David Shapiro, the chief medical officer at Intercept ($ICPT), said that this was the third successful study for this indication, setting the stage for a regulatory filing later in the year.
"These POISE trial results indicate that OCA clearly produced clinically meaningful improvements, not only in the primary endpoint but also across a broad range of biochemical liver function parameters," said Professor Frederik Nevens, the lead investigator in POISE, in a statement. "While ursodiol has been the mainstay of PBC therapy for the past 20 years, a significant proportion of patients fail to get an adequate response with this drug and we need new therapies to prevent their disease progressing to cirrhosis and liver failure. I believe that the POISE data indicate OCA will provide a meaningful clinical improvement in these patients."
Shares of Intercept Pharmaceuticals went into overdrive at the beginning of the year, soaring more than 250% on the surprise news that a Phase IIb clinical study for its lead drug funded largely by the NIH ended early after achieving the primary endpoint.
The bile acid analog--which mimics human bile acid and is an agonist for the farnesoid X receptor--is in development for PBC, nonalcoholic steatohepatitis and other liver and intestinal diseases. The biotech plans to release the detailed data at an upcoming conference.
The Friday night frights, which pushed down Intercept's shares by 16%, related to the 10-K's remarks on adverse events in a study of OCA dubbed FLINT. As noted in the 10-K, and highlighted by Adam Feuerstein at TheStreet, there was also no statistical difference from the SAEs seen in the placebo arm.
- read the release
- here's the 10-K