Intercept Announces Publication of Results from the Interim Analysis of the Phase 3 REGENERATE Study of OCA for the Treatment of Fibrosis Due to NASH in The Lancet

NEW YORK, Dec. 05, 2019 (GLOBE NEWSWIRE) — 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 the positive results from the interim analysis of the Phase 3 REGENERATE study of obeticholic acid (OCA) in patients with fibrosis due to nonalcoholic steatohepatitis (NASH) have been published in The Lancet. This is the first peer-reviewed publication of positive results from a pivotal study evaluating an investigational therapy for NASH.
“The first positive Phase 3 study results in NASH represent a real watershed moment for the hepatology field,” said Zobair M. Younossi, M.D., Professor and Chairman of the Department of Medicine at Inova Fairfax Medical Campus, Chair of the REGENERATE Steering Committee and a lead author of the publication. “The antifibrotic efficacy observed with just 18 months of OCA treatment in REGENERATE is particularly meaningful because fibrosis is the most important histological predictor of liver failure and death in patients with NASH.”REGENERATE is an ongoing study that will continue through clinical outcomes for verification and description of clinical benefit. Data from the 18-month interim analysis of the study served as the basis for the New Drug Application (NDA) for OCA for the treatment of fibrosis due to NASH, which was accepted by the U.S. Food and Drug Administration (FDA) in November 2019. As previously announced, Intercept also plans to file a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) in the fourth quarter of 2019.About Liver Fibrosis due to NASHNonalcoholic 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 all-cause 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 StudyREGENERATE 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 interim analysis was conducted to assess the effect of OCA on liver histology comparing month 18 biopsies with baseline. The intent-to-treat population for the interim analysis included 931 patients with stage 2 and 3 fibrosis (placebo, n=311; OCA 10 mg, n=312; OCA 25 mg, n=308). REGENERATE has completed target enrollment for the clinical outcomes cohort, with 2,480 adult NASH patients randomized at 339 qualified centers worldwide, and will continue through clinical outcomes for verification and description of 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.The safety population of the interim analysis included 1,968 randomized patients who received at least one dose of investigational product (OCA or placebo). Adverse events were generally mild to moderate in severity and the most common were consistent with the known profile of OCA. The frequency of serious adverse events was similar across treatment arms (11% in placebo, 11% in OCA 10 mg and 14% in OCA 25 mg). The most common adverse event reported was dose-related pruritus (placebo, 19%; OCA 10 mg, 28%; OCA 25 mg, 51%). The large majority of pruritus events were mild to moderate, with severe pruritus occurring in a small number of patients.About InterceptIntercept 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.Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements, including, but not limited to, statements regarding the progress, timing and results of our clinical trials, including our clinical trials for the treatment of nonalcoholic steatohepatitis (“NASH”), the safety and efficacy of our approved product, Ocaliva (obeticholic acid or “OCA”) for primary biliary cholangitis (“PBC”), and our product development candidates, including OCA for NASH, the timing and acceptance of our regulatory filings and the potential approval of OCA for NASH or any other indications in addition to PBC, the timing and potential commercial success of OCA and any other product candidates we may develop and our strategy, future operations, future financial position, future revenue, projected costs, financial guidance, prospects, plans, objectives of management and expected market growth.For more information about Intercept, please contact:Lisa DeFrancesco
+1-646-565-4833
investors@interceptpharma.com
                                                                                                           
Christopher Frates
+1-646-757-2371
media@interceptpharma.com