Intercept Initiates REGENERATE Trial For Obeticholic Acid In NASH With ALF

(RTTNews) - Intercept Pharmaceuticals Inc. (ICPT) announced the initiation of the REGENERATE trial, an international Phase 3 trial of obeticholic acid or OCA, the company's lead FXR agonist, in patients with non-cirrhotic nonalcoholic steatohepatitis or NASH with advanced liver fibrosis.

OCA is the first NASH therapy to have demonstrated reversal of liver fibrosis with a significantly greater therapeutic response versus placebo in patients at higher risk of progression to cirrhosis.

The U.S. Food and Drug Administration has designated OCA as a breakthrough therapy in NASH patients with fibrosis and REGENERATE was designed in accordance with advice from the FDA and European Medicines Agency.

The REGENERATE study population will be primarily comprised of NASH patients with stage 2 or stage 3 liver fibrosis. Advanced liver fibrosis has been shown to be the best predictor of liver transplant and mortality in patients with NASH. In addition, the trial will include an exploratory cohort of NASH patients with early stage 1 liver fibrosis and concomitant diabetes, obesity or elevated alanine aminotransferase (ALT) who are at increased risk of progression to cirrhosis.

REGENERATE has been designed as a double-blind, placebo-controlled Phase 3 clinical trial expected to enroll approximately 2,000 NASH patients at up to 300 qualified centers worldwide and assess the potential benefits of OCA treatment on liver-related and other clinical outcomes.

The trial will include a pre-planned interim histology analysis after 72 weeks of treatment in 1,400 patients, which is intended to serve as the basis for seeking initial U.S. and international marketing approvals of OCA for the treatment of NASH patients with fibrosis. Two co-primary endpoints will be assessed in the interim analysis: (i) fibrosis improvement with no worsening of NASH and (ii) NASH resolution with no worsening of fibrosis.