Intercept Pharmaceuticals Inc. announced that its international Phase 3 POISE trial of obeticholic acid (OCA) for the treatment of primary biliary cirrhosis (PBC) demonstrated that OCA, at both a 10 mg dose and a 5 mg dose titrated to 10 mg, met the trial’s primary endpoint of achieving a reduction in serum alkaline phosphatase (ALP) to less than 1.67x ULN with a reduction of greater than 15% from baseline and a normal bilirubin level after 12 months of therapy.
The proportion of patients meeting the POISE primary endpoint was: 10% in the placebo group, 47% in the 10 mg OCA group and 46% in the 5-10 mg OCA group in an intention to treat analysis. The placebo group experienced a mean decrease in ALP from baseline of 5%, compared to a significant mean decrease of 39% in the 10 mg OCA dose group and 33% in the 5-10 mg OCA titration group. In addition, both OCA dose groups met pre-specified secondary endpoints of improvements in other liver function parameters, including GGT, ALT, AST and total bilirubin.
OCA, Intercept’s lead product candidate, is a bile acid analog and first-in-class agonist of the farnesoid X receptor (FXR) in development for PBC, nonalcoholic steatohepatitis (NASH) and other liver and intestinal diseases.
“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, chairman of the Department of Hepatology at the University of Leuven, Belgium and the lead investigator in POISE. “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.”
“POISE is Intercept’s third successful international, placebo controlled trial of OCA in PBC patients conducted over the past seven years, setting the stage for our anticipated filing for approval of OCA in the U.S., Europe and other countries,” said David Shapiro, chief medical officer of Intercept. “With the results of POISE and our ongoing long-term study of PBC patients on therapy for more than four years, we have shown that OCA produces a significant durable response and believe this will result in better clinical outcomes for many patients. We would like to thank the many investigators and patients who participated in POISE and our other PBC trials.”
Date: March 16, 2014
Source: Intercept Pharmaceutical