Intercept announced the publication of key data from the Phase 3 POISE trial of Ocaliva (obeticholic acid) for the treatment of patients with primary biliary cholangitis (PBC) in the New England Journal of Medicine.
POISE was a 12-month, double-blind, placebo-controlled trial (n=217) that assessed the safety and efficacy of once-daily Ocaliva in patients with PBC with inadequate therapeutic response to, or who are unable to tolerate, ursodeoxycholic acid (UDCA). The study’s primary endpoint was a reduction in alkaline phosphatase (ALP) <1.67 times the upper limit of normal, with ≥15% reduction from baseline, and a total bilirubin level at or below the upper limit of normal after 12 months of Ocaliva treatment.
The data showed both Ocaliva 5 to 10mg and Ocaliva 10mg were both statistically superior to placebo in meeting the primary endpoint (46% and 47% vs. 10%; P<0.001). Most patients treated with Ocaliva experienced improvements in liver biochemistry even if they did not meet the composite primary endpoint. There was a significantly higher percentage of patients achieving ≥15% ALP reduction with Ocaliva 5 to 10mg and Ocaliva 10mg vs. placebo (77% and 77% vs. 29%; P<0.001).
Researchers concluded that Ocaliva given with ursodiol or as monotherapy for 12 months in patients with PBC led to significant decreases in ALP and total bilirubin levels compared to placebo.
Ocaliva, a farnesoid X receptor (FXR) agonist, was granted accelerated approval by the Food and Drug Administration (FDA) in May 2016. An improvement in survival or disease-related symptoms has not been established. Ocaliva is available as 5mg and 10mg strength tablets in 30-count bottles.
For more information visit nejm.org.