Intercept Pharmaceuticals reported positive results from its international Phase III POISE trial evaluating obeticholic acid (OCA) for the treatment of primary billiary cirrhosis (PBC).
The drug was assessed in both a 10 mg dose and a 5 mg dose titrated to 10 mg and achieved the trial's primary endpoint of reducing serum alkaline phosphatase (ALP) to < 1.67x ULN with a ≥ 15% reduction from baseline and normal bilirubin level after 12 months of treatment.
“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. 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,” said Professor Frederik Nevens, M.D., Ph.D., Chairman of the Department of Hepatology at the University of Leuven, Belgium and the lead investigator in POISE study.
Obeticholic acid (OCA) is a bile acid analog and first-in-class agonist of the farnesoid X receptor (FXR) under development for primary billiary cirrhosis (PBC), nonalcoholic steatohepatitis (NASH) and other liver and intestinal diseases.
Primary billiary cirrhosis is an autoimmune liver disease that can lead to cirrhosis and liver failure. The disease is currently the fifth leading indication for liver transplant in the U.S. Disease progress is assessed by measuring blood levels of alkaline phosphatase (ALP) and bilirubin. Ursodiol is currently the only approved drug treatment for PBC, but up to 50% of patients fail to respond adequately and remain at risk of adverse outcomes.
David Shapiro, M.D., Chief Medical Officer of Intercept, said “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. 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.”