April 8, 2013
Data Support Strong Statistical Correlation of PBC Biochemical Endpoint With Clinical Outcomes
NEW YORK, April 8, 2013 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT), a clinical stage biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat chronic liver diseases, today announced that an analysis by the Global Primary Biliary Cirrhosis (PBC) Study Group (also known as the PBC Supergroup) confirms that the endpoint used by Intercept in its ongoing Phase 3 POISE trial is strongly predictive of adverse clinical outcomes in PBC patients. The analysis reflects data from more than 2,100 patients. An abstract from the group will be presented at the annual meeting of the European Association for the Study of the Liver (EASL) taking place April 24-28, 2013 in Amsterdam.
The Global PBC Study Group
PBC is an autoimmune chronic liver disease that typically affects women. The disease progresses in patients with an inadequate response to therapy who as a result may require a liver transplant or die. Biochemical assessments of liver function, particularly by measuring plasma levels of alkaline phosphatase (ALP) and bilirubin, are typically used by physicians to diagnose and manage PBC patients. Accordingly, the relationship between these biochemical assessments of liver function and adverse clinical outcomes is highly important in the selection of appropriate endpoints in therapeutic clinical trials.
The Global PBC Study Group is currently comprised of a group of 15 academic medical centers from eight countries that are pooling their data to investigate these relationships. Intercept is sponsoring this independent academic research program but is not involved in the data collection and analysis, which are being conducted by Dr. Henk van Buuren and Dr. Bettina Hansen of Erasmus University Medical Centre in Rotterdam, The Netherlands. The data demonstrate that the primary endpoint being used in Intercept's Phase 3 POISE trial (ALP < 1.67x upper limit of normal and normal bilirubin) is highly statistically predictive of liver transplant-free survival in PBC patients. The risk of adverse outcomes increases from 2-10x in patients with ALP and/or bilirubin levels above these thresholds as compared to patients with biochemical values below them (p < 0.001). Further, the data show that ALP and bilirubin assessed independently are each highly predictive of adverse outcomes.
"This collaborative, international effort has been designed to allow a detailed analysis of both the risks and the natural history of this rare but important disease," noted Dr. van Buuren. "The data show that biochemical assessments predict a patient's risk of requiring a liver transplant or, worse, dying from the disease. More centers have joined the group and we plan to increase the size of the database in the coming months. We believe that we have assembled the largest international PBC study group and database ever, which will prove very useful to both patients and their physicians in the coming years."
Intercept anticipates final data from at least 4,000 patients will be collected and analyzed as part of the study.
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 PBC in patients with an inadequate response to, or who are unable to tolerate, ursodiol (ursodeoxycholic acid), 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. For more information about Intercept, please visit the Company's website at www.interceptpharma.com.
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 relationship between ALP and bilirubin and adverse clinical outcomes, the clinical utility of the POISE trial selected endpoints and any potential consensus relating thereto, clinical, preclinical and regulatory developments for our product candidates, the anticipated results of our clinical and preclinical trials and other development activities, 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 filed with the Securities and Exchange Commission on April 1, 2013, 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.
For more information about Intercept, please contact Mark Pruzanski, M.D., or Barbara Duncan, both of Intercept Pharmaceuticals, at 1-646-747-1000.