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Intercept Pharmaceuticals Submits Applications in the U.S. and Europe for Marketing Approval of Obeticholic Acid for the Treatment of Primary Biliary Cirrhosis

NEW YORK, June 29, 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 achievement of two important regulatory milestones for obeticholic acid (OCA) in primary biliary cirrhosis (PBC): submission of a New Drug Application (NDA) for accelerated approval to the U.S. Food and Drug Administration (FDA) and acceptance of the Marketing Authorization Application (MAA) by the European Medicines Agency (EMA). Intercept is seeking approval of OCA for the treatment of PBC in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA.

UDCA is currently the only approved medication for the treatment of PBC and is the standard of care for all PBC patients. However, a majority of patients continue to experience persistent elevations above the upper limit of normal in the serum marker alkaline phosphatase (ALP), corresponding with increased risk of liver failure, need for liver transplant and death. Thus, there continues to be a critical need for new treatments for patients with PBC.

The NDA and MAA submissions include a total of 1,507 subjects exposed to at least a single dose of OCA. Of these subjects, 432 were patients with PBC, with 290 treated for at least six months and 232 treated for at least one year. The key efficacy and safety data are derived from three randomized double-blind, placebo-controlled clinical trials in patients with PBC evaluating the effect of OCA on ALP and bilirubin. All three trials met their primary endpoints with high statistical significance and improvements were seen in secondary endpoints including markers of liver injury, immunity, inflammation and apoptosis. OCA treatment was generally well-tolerated, with primarily mild or moderate pruritus being the most common adverse event.

The regulatory submissions are also supported by two clinical databases that include more than 10,000 patients from the Global PBC Study Group and UK-PBC Group, both independently confirming that achieving lower ALP and/or bilirubin levels is significantly correlated with increased transplant-free survival.

"Over the past several years, the medical community has gained a deeper appreciation of the extent of the unmet medical need in PBC," said Mark Pruzanski, M.D., President and Chief Executive Officer. "In each of our PBC clinical trials, OCA has demonstrated the ability to rapidly and sustainably lower ALP and improve bilirubin levels, both when added to UDCA and as monotherapy. If approved, we believe OCA will become an important new treatment option that will help patients with PBC."

OCA has received orphan drug designation for PBC in both the United States and Europe and fast track designation for PBC in the United States. In December 2014, Intercept initiated its rolling NDA submission for accelerated approval of OCA in PBC and began a Phase 3b confirmatory clinical outcomes trial in PBC, in accordance with FDA accelerated approval regulations. This trial is anticipated to enroll approximately 350 patients with advanced PBC at 150 centers in more than 20 countries, and is expected to be completed on a post-marketing basis.

About Primary Biliary Cirrhosis

PBC is a rare liver disease that primarily results from autoimmune destruction of the bile ducts that transport bile acids out of the liver, resulting in cholestasis. It is primarily a disease of women, afflicting approximately one in 1,000 women over the age of 40. PBC is the second leading cause of liver transplant among women in the United States. In Europe, PBC accounts for 50 percent of liver transplants due to cholestatic diseases and 6% of all liver transplants.

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, a population representing potentially more than 14 million patients in the United States, 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 the clinical relevance of the Global PBC Study Group and/or the UK-PBC Group data and the applicability thereof to OCA in PBC, the potential relationship between ALP and bilirubin and adverse clinical outcomes, the clinical utility of the POISE trial selected endpoints and any potential consensus relating thereto, the anticipated need for additional therapies in PBC, the epidemiology and natural history of PBC, clinical, preclinical and regulatory developments for our product candidates, the anticipated timetable for our clinical, regulatory, development and commercialization activities, the anticipated results of our clinical and preclinical trials and other development activities and the timing thereof, 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: Barbara Duncan or Mark Vignola +1-646-747-1000 investors@interceptpharma.com Media inquiries: media@interceptpharma.com Investor inquiries: investors@interceptpharma.com

Source:Intercept Pharmaceuticals, Inc.