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Intercept Pharmaceuticals Announces Initiation of REGENERATE Trial for Obeticholic Acid in NASH Patients With Advanced Liver Fibrosis

NEW YORK, Sept. 28, 2015 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT) (Intercept), a clinical stage biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat chronic underserved liver diseases, today announced the initiation of the REGENERATE trial, an international Phase 3 trial of obeticholic acid (OCA), the company's lead FXR agonist, in patients with non-cirrhotic nonalcoholic steatohepatitis (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 (FDA) 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 (EMA).

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.

"We are excited to embark on the REGENERATE Phase 3 trial of OCA in NASH and build on the exciting results from FLINT that demonstrated OCA's potential to reverse advanced liver fibrosis in NASH patients," said Mark Pruzanski, M.D., Chief Executive Officer and President of Intercept. "NASH is projected to become the leading indication for liver transplant in the U.S. by 2020 and, with no approved treatments, the disease imposes a tremendous public health burden. With REGENERATE, we hope to show that OCA treatment will prevent NASH patients with fibrosis from progressing to cirrhosis and cement its value as a breakthrough therapy for this underserved patient population."

About Nonalcoholic Steatohepatitis

NASH is a serious chronic 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. There are currently no drug therapies approved for the treatment of NASH. Patients with early disease but with risk factors such as diabetes, obesity or elevated ALT are at increased risk of progression to cirrhosis. The proportion of liver transplants attributable to NASH has increased rapidly in past years and by 2020 the disease is projected to become the leading indication for liver transplant ahead of chronic hepatitis C and alcoholic liver disease.

About Intercept

Intercept is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat chronic underserved liver diseases. The Company's lead product candidate, obeticholic acid (OCA), is an agonist of the farnesoid X receptor (FXR). OCA is being developed for a variety of chronic liver diseases, including primary biliary cirrhosis (PBC), nonalcoholic steatohepatitis (NASH), primary sclerosing cholangitis (PSC) and biliary atresia. The FDA has granted OCA breakthrough therapy designation for the treatment of NASH with liver fibrosis and granted OCA fast track designation for the treatment of patients with PBC. OCA has also received orphan drug designation in both the United States and Europe for the treatment of PBC and PSC. Intercept owns worldwide rights to OCA outside of Japan, China and Korea, where it has out-licensed the product candidate to Sumitomo Dainippon Pharma. For more information about Intercept, please visit the Company's website at: www.interceptpharma.com.

Safe Harbor Statements

This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding our plans and ability to seek marketing approval for OCA in NASH with liver fibrosis on the basis of a pre-planned interim analysis, the clinical relevance and utility of the endpoints to be studied in the REGENERATE trial, the link between fibrosis and outcomes in NASH, the ability of past results to predict future results, the prevalence of NASH and the potential size of the NASH market, and our strategic directives under the caption "About Intercept." These "forward-looking statements" are based on management's current expectations of future events and are subject to a number of important risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: the initiation, cost, timing, progress and results of our development activities, preclinical studies and clinical trials; the timing of and our ability to obtain and maintain regulatory approval of OCA, INT-767 and any other product candidates we may develop, particularly the possibility that regulatory authorities may require clinical outcomes data (and not just results based on achievement of a surrogate endpoint) as a condition to any marketing approval for OCA, and any related restrictions, limitations, and/or warnings in the label of any approved product candidates; our plans to research, develop and commercialize our product candidates; the election by our collaborators to pursue research, development and commercialization activities; our ability to attract collaborators with development, regulatory and commercialization expertise; our ability to obtain and maintain intellectual property protection for its product candidates; our ability to successfully commercialize our product candidates; the size and growth of the markets for our product candidates and our ability to serve those markets; the rate and degree of market acceptance of any future products; the success of competing drugs that are or become available; regulatory developments in the United States and other countries; the performance of third-party suppliers and manufacturers; our need for and ability to obtain additional financing; our estimates regarding expenses, future revenues and capital requirements and the accuracy thereof; our ability to retain key scientific or management personnel; and other factors discussed under the heading "Risk Factors" contained in our annual report on Form 10-K for the year ended December 31, 2014 filed on March 2, 2015 as well as any updates to these risk factors filed from time to time in our other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intercept undertakes no duty to update this information unless required by law.

CONTACT: For more information about Intercept Pharmaceuticals, please contact: Intercept Pharmaceuticals: Mark Vignola +1-646-747-1000 investors@interceptpharma.com Media inquiries: media@interceptpharma.com Investor inquiries: investors@interceptpharma.com

Source:Intercept Pharmaceuticals, Inc.