Intercept Phase III POISE trial published in the New England Journal of Medicine

Intercept Pharmaceuticals Inc. this week announced the publication of its Phase III POISE trial in the New England Journal of Medicine. 
 
The POISE trial evaluated Ocaliva (obeticholic acid) in treating primary biliary cholangitis (formerly known as primary biliary cirrhosis or PBC) in patients with elevated levels of alkaline phosphatase (ALP) and bilirubin. The participants had inadequate responses to ursodeoxycholic acid (UDCA) or could not tolerate UDCA. Ninety-three percent of the participants also received UDCA treatment during the study.
 
The trial found that the participants treated with 5 to 10 mg or 10 mg of Ocaliva had a better response than those treated with the placebo. In general, the patients who received Ocaliva had lowered levels of ALP and bilirubin while the placebo patients' levels rose.  
 
After the double-blind phase of the trial, 97 percent of the participants continued with the five-year open-label extension. The Ocaliva participants showed continued improvements in ALP and bilirubin while the placebo patients' ALP and bilirubin levels dropped to baseline levels after initiating treatment with Ocaliva in the open-label extension.
 
"The POISE data support Ocaliva as an important new treatment option for the substantial group of PBC patients who have an inadequate response to, or cannot tolerate, UDCA and therefore remain at risk of adverse outcomes," University Hospitals Leuven & KU Leuven, Belgium, and the lead author of the paper Dr. Frederik Nevens said. “For nearly 20 years, we only had one option to help our PBC patients. The introduction of Ocaliva provides physicians and patients with an opportunity to rethink treatment goals and take action when ALP and/or bilirubin remain elevated despite UDCA therapy.”

The U.S. Food and Drug Administration (FDA) granted accelerated approval for Ocaliva as a combination or monotherapy on May 27. 

Primary biliary cholangitis  is a rare, autoimmune cholestatic liver disease that puts patients at risk for life-threatening complications.