Results of Phase III POISE trial of Ocaliva (obeticholic acid) for primary biliary cholangitis published in New England Journal of Medicine- Intercept Pharmaceuticals

Intercept Pharmaceuticals has announced that the New England Journal of Medicine published the key results of the Phase III POISE trial of Ocaliva (obeticholic acid) for the treatment of patients with primary biliary cholangitis, formerly known as primary biliary cirrhosis (PBC). On a background of standard of care or given as monotherapy, Ocaliva met the primary endpoint of the POISE trial and improved multiple biochemical disease markers as compared to placebo with high statistical significance.

The POISE trial evaluated the safety and efficacy of once-daily treatment with Ocaliva in PBC patients with an inadequate therapeutic response to, or who are unable to tolerate, ursodeoxycholic acid (UDCA). The trial's primary endpoint was a reduction in alkaline phosphatase (ALP) to below a threshold of 1.67 times the upper limit of the normal range, with a reduction of at least 15% from baseline, and a total bilirubin level at or below the upper limit of the normal range after 12 months of Ocaliva therapy. These liver biomarkers have been shown to be reasonably likely to predict progression to liver failure and resulting liver transplant or premature death in patients with PBC. Ocaliva 5-10 mg (46%) and Ocaliva 10 mg (47%) were both statistically superior to placebo (10%) in achieving the primary endpoint (p<0.001). most ocaliva-treated patients had liver biochemical improvements even if they did not achieve the composite primary endpoint, with a significantly higher percent of patients achieving ?15% alp reduction with ocaliva 5-10 mg and ocaliva 10 mg (both groups 77%) compared to placebo (29%) (p><0.001). the majority of patients (93%) continued receiving udca therapy during the trial.>

See: "A Placebo-Controlled Trial of Obeticholic Acid in Primary Biliary Cholangitis" Frederik Nevens et al. N Engl J Med 2016; 375:631-643 August 18, 2016 DOI: 10.1056/NEJMoa1509840