NDA supported by positive interim analysis results from REGENERATE Phase 3 study demonstrating OCA’s improvement of liver fibrosis without worsening of NASH

NEW YORK, NY, USA I September 27, 2019 I Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT), a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, today announced that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Adminstration (FDA) for obeticholic acid (OCA) for the treatment of patients with fibrosis due to nonalcoholic steatohepatitis (NASH).

OCA is the only investigational therapy to meet the primary endpoint of a Phase 3 study in patients with NASH and is the only such therapy that the FDA has designated a Breakthrough Therapy for NASH with fibrosis. As such, Intercept has requested a Priority Review for the NDA, which, if granted, would result in an anticipated six-month review period.

The submission is based on positive interim analysis results from the pivotal Phase 3 REGENERATE study in patients with liver fibrosis due to NASH. In the study, OCA 25 mg achieved its primary endpoint by demonstrating robust improvement in liver fibrosis (by ≥1 stage) without worsening of NASH at 18 months (p=0.0002 vs placebo).

“Our submission of the first NDA for the treatment of fibrosis due to NASH is a very important milestone for the field and the culmination of more than a decade of hard work,” said Mark Pruzanski, M.D., President and Chief Executive Officer of Intercept. “I am grateful to the thousands of NASH patients participating in our clinical studies, the investigators and study personnel at our study sites around the globe, and the entire Intercept team for bringing us to this point. We look forward to continuing to work with the FDA through the NDA review period and believe that, if approved, OCA has the potential to become an essential treatment for people living with advanced fibrosis due to NASH.”

Intercept also intends to file a marketing authorization application (MAA) with the European Medicines Agency in the fourth quarter of this year.

About Liver Fibrosis due to NASH

Nonalcoholic steatohepatitis (NASH) is a serious progressive liver disease caused by excessive fat accumulation in the liver that induces chronic inflammation, resulting in progressive fibrosis (scarring) that can lead to cirrhosis, eventual liver failure, cancer and death. Advanced fibrosis is associated with a substantially higher risk of liver-related morbidity and mortality in patients with NASH and, as early as 2020, the disease is projected to become the leading cause of liver transplants in the United States. There are currently no medications approved for the treatment of NASH.

About the REGENERATE Study

REGENERATE is a Phase 3, randomized, double-blind, placebo-controlled, multicenter study assessing the safety and efficacy of obeticholic acid (OCA) on clinical outcomes in patients with liver fibrosis due to NASH. A pre-specified 18-month analysis was conducted to assess the effect of OCA on liver histology comparing month 18 biopsies with baseline. REGENERATE has completed target enrollment for the clinical outcomes cohort, with more than 2,400 adult NASH patients randomized across 339 qualified centers worldwide, and will continue through clinical outcomes on a post-marketing basis to confirm clinical benefit. The end-of-study analysis will evaluate the effect of OCA on all-cause mortality and liver-related clinical outcomes, as well as its long-term safety.

About Intercept

Intercept is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat progressive non-viral liver diseases, including primary biliary cholangitis (PBC) and nonalcoholic steatohepatitis (NASH). Founded in 2002 in New York, Intercept has operations in the United States, Europe and Canada. For more information, please visit www.interceptpharma.com or connect with the company on Twitter and LinkedIn.

SOURCE: Intercept