Intercept's Phase 3 Trial Of Obeticholic Acid Meets Primary Endpoint

Intercept Pharmaceuticals, Inc. announced in a press release that its Phase 3 POISE trial of obeticholic acid (OCA) showed positive results. OCA is being studied for the treatment of primary biliary cirrhosis (PBC). The results met the primary endpoint of the trial by reducing serum alkaline phosphatase (ALP). 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.”

Primary Biliary Cirrhosis

Primary biliary cirrhosis (PBC) is a chronic disease with an unknown cause, but is most likely due to an autoimmune condition. The immune system goes awry, causing inflamed, damaged, and blocked bile ducts. The liver produces bile, a liquid that digests fats and fat-soluble vitamins A, D, E, and K. Damaged bile ducts cause bile to stay in the liver, damaging liver tissue. Scarred liver tissue cannot process nutrients, regulate hormones, and metabolize drugs. Additionally, the liver cannot remove toxins, bacteria, and infections. Women between the ages of 40 and 60 are most commonly affected. Alkaline phosphatase (ALP) and bilirubin blood levels are primary measures as they indicate adverse outcomes.

Obeticholic Acid

Obeticholic acid (OCA) is an agonist of the farnesoid X receptor (FXR). The novel drug is orally administered and has potential to treat a variety of liver diseases. The POISE trial was a placebo controlled trial, international in scope. 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 said, “OCA clearly produced clinically meaningful improvements, not only in the primary endpoint but also across a broad range of biochemical liver function parameters.”

At both the 5 mg dose and the 10 mg dose, OCA achieved serum alkaline phosphatase reduction (ALP), a 15% reduction from baseline. Patients achieved a normal bilirubin level twelve months after starting therapy. Ten percent of the placebo group reduced ALP levels, 47% of the 10 mg OCA groups reduced ALP levels, and 46% of the 5 mg OCA groups achieved lower ALP levels. Other measures of liver health improved including total bilirubin, GGT, ALT, and AST. Intercept plans to present results at the International Liver Congress of the European Association for the Study of the Liver (EASL) in April 2014.