Intercept Pharmaceuticals Announces Positive Initial Results from Ongoing Phase 2a Trial in Chronic Bile Acid Diarrhea

NEW YORK, May 20, 2013 /PRNewswire/ -- Intercept Pharmaceuticals, Inc. ("Intercept", NASDAQ: ICPT), a clinical stage biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat chronic liver diseases, today announced positive initial results from OBADIAH, an ongoing Phase 2a trial of obeticholic acid (OCA) as a treatment for primary bile acid diarrhea (PBAD) presented at the Digestive Diseases Week conference. The initial results from the OBADIAH trial demonstrate that treatment with OCA is associated with statistically significant increased levels of fibroblast growth factor 19 (FGF19) and improvement in clinical symptoms in patients with PBAD.

OBADIAH is an investigator-initiated and sponsored open label Phase 2a trial being conducted by Professor Julian Walters at the Imperial College of London in the United Kingdom. The trial is investigating whether OCA can stimulate the release of FGF19 in patients with PBAD. This disease, also known as idiopathic bile acid malabsorption, is a common chronic diarrheal condition due to excessive bile acid production and loss. PBAD is estimated to affect approximately one percent of the population and about one-third of patients diagnosed with diarrhea-predominant irritable bowel syndrome (IBS-D). Patients with PBAD have low levels of FGF19, a hormone released in the ileum in response to FXR activation and regulates bile acid production by the liver. As a result, excess bile acids spill into the gut and produce diarrhea by overstimulating intestinal secretions. All three of Intercept's previously completed Phase 2 trials in other indications demonstrated that OCA markedly and dose dependently stimulates the release of FGF19.

"These data represent an encouraging first step in a new therapy for many patients thought to have IBS-D, who in actuality have primary bile acid diarrhea associated with impaired release of FGF19," stated Professor Walters. "As obeticholic acid is a potent FXR agonist and stimulator of FGF19 release, it is a logical therapeutic approach which we are keen to evaluate further given the need for more effective treatments in patients with this disorder."

The primary outcome measure of the open-label OBADIAH trial is to assess changes in FGF19 levels over a two-week period in ten patients with PBAD and in two other groups, one consisting of patients with secondary bile acid diarrhea due to Crohn's disease and the other consisting of IBS-D patients who have normal FGF19 levels. Secondary outcome measures include clinical symptom scores, biochemical response and tolerability. Data from ten PBAD patients, the first group studied in OBADIAH, indicate that a 25 mg daily oral dose of OCA resulted in a statistically significant increase in median fasting FGF19 from 133 to 237 pg/mL, with most patients achieving a >60% increase (p=0.007). In addition, clinical improvements were seen in all patients with reductions in median stool frequency from 23 to 14 per week (p=0.03) and an improvement in the median Bristol Stool Form Scale assessing stool type from 5.15 to 4.34 (p=0.05). Notably, during the two week follow up period after stopping OCA therapy, stool frequency returned to pre-treatment baseline values. OCA was well tolerated in all patients.

Intercept anticipates final results for all three study groups to be available in the second half of 2013.

The slides for the presentation made by Professor Walters are available on the "Presentations" section of Intercept's website at www.interceptpharma.com.

About Intercept
Intercept is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat orphan and more prevalent liver diseases utilizing its expertise in bile acid chemistry. The company's lead product candidate, obeticholic acid, or OCA, is a bile acid analog and first-in-class agonist of the farnesoid X receptor (FXR). OCA is initially being developed for the second line treatment of primary biliary cirrhosis (PBC) in patients with an inadequate response to, or who are unable to tolerate, ursodiol, the only approved therapy for this indication. PBC is a chronic autoimmune liver disease that may progress to cirrhosis and liver failure, and it is currently the fifth leading indication for liver transplant in the United States. OCA has orphan drug designation in both the United States and Europe for the treatment of PBC. Intercept owns worldwide rights to OCA outside of Japan and China, where it has out-licensed the product candidate to Dainippon Sumitomo Pharma (DSP).

Safe Harbor Statement
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 potential effect of OCA in PBAD such as its impact on FGF19, the anticipated enrollment of OBADIAH, the availability of results from the trial, and the timing of the anticipated enrollment and availability of results, 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 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 Intercept's development activities, preclinical studies and clinical trials; the timing of and Intercept's ability to obtain and maintain regulatory approval of OCA and any other product candidates it may develop, and any related restrictions, limitations, and/or warnings in the label of any approved product candidates; Intercept's plans to research, develop and commercialize future product candidates; the election by Intercept's collaborators to pursue research, development and commercialization activities; Intercept's ability to attract collaborators with development, regulatory and commercialization expertise; Intercept's ability to obtain and maintain intellectual property protection for its product candidates; Intercept's ability to successfully commercialize its product candidates; the size and growth of the markets for Intercept's product candidates and its 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; Intercept's ability to obtain additional financing; Intercept's use of the proceeds from its recently completed initial public offering; the accuracy of Intercept's estimates regarding expenses, future revenues, capital requirements and the need for additional financing; the loss of key scientific or management personnel; and other factors discussed under the heading "Risk Factors" contained in Intercept's annual report on Form 10-K for the year ended 2012, quarterly reports on Form 10-Q, as well as any updates to these risk factors filed from time to time in Intercept's 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.

SOURCE Intercept Pharmaceuticals, Inc.