Intercept Pharmaceuticals to Study the Role of Gut Microbiota in the Treatment of Non-Viral Liver Diseases by Obeticholic Acid

NEW YORK, March 16, 2016 (GLOBE NEWSWIRE) — Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT), a clinical stage biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat non-viral, progressive liver diseases, today announced a new research initiative with the University of Pennsylvania’s PennCHOP Microbiome Program, a collaboration between the Perelman School of Medicine at the University of Pennsylvania and the Children’s Hospital of Philadelphia (CHOP) to evaluate the effects of obeticholic acid (OCA) and other product candidates on the microbiome in a variety of chronic liver diseases, beginning with nonalcoholic steatohepatitis (NASH).

PennCHOP will analyze samples from Intercept’s ongoing international Phase 3 REGENERATE trial in NASH patients with fibrosis and evaluate how OCA influences the composition and activity of the resident microbiota in the gastrointestinal tract. Analyses of additional patient samples in other diseases are expected to follow as part of the seven-year collaboration. Financial details of the collaboration will not be disclosed.

“This collaboration with Intercept, which gives us access to patient samples and data from the largest NASH clinical program to date, is anticipated to provide new insights into the relationship between gut microbiota, NASH and a modified bile acid like OCA,” said Gary Wu, M.D., the Ferdinand G. Weisbrod Professor in Gastroenterology at the Perelman School of Medicine at the University of Pennsylvania, and co-director of the PennCHOP Microbiome Program. “It also opens the door to future research in diseases such as primary sclerosing cholangitis and primary biliary cholangitis.”

Data from both animal and human studies have demonstrated that changes in the gut microbiome can be an important factor leading to the development and progression of liver disease,1 and it has been hypothesized that restoring a healthier microbiome may reverse or improve a disease state. A recent study2 in cirrhotic animals showed that OCA treatment modified the distinctive mucosal microbiota of cirrhosis to one associated with less severe disease. In addition, OCA treated animals showed inhibition of bacterial translocation from the ileum, normalized intestinal permeability and reduced inflammation of the lymph nodes and spleen. 

“The PennCHOP program is at the forefront of microbiome research, and we are looking forward to collaborating with Penn on this important project,” said David Shapiro, M.D., Intercept’s Chief Medical Officer and Executive Vice President, Development. “This is an exciting area of emerging research and we are committed to determining how OCA’s interaction with the gut-liver axis may help patients with NASH and other non-viral liver diseases.”

Editor’s note: Dr. Wu has received honoraria from Intercept.

About Intercept
Intercept is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat chronic non-viral, progressive liver diseases. Intercept’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, recently renamed primary biliary cholangitis (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. Intercept’s pipeline of product candidates includes other novel bile acid analogs such as INT-767, which is in clinical development. For more information about Intercept, please visit Intercept’s website at:

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 anticipated commercialization of OCA in PBC, the continued development of OCA and Intercept’s other product candidates, the development of OCA in NASH, including the timing and results of the REGENERATE trial, the potential for gut microbiota to provide any meaningful data regarding the effects of OCA in liver disease or the utility of the results of the collaboration, 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; 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, which may be affected by the reimbursement that our products receive from payors; 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 collaborators’ election to pursue research, development and commercialization activities; our ability to attract collaborators with development, regulatory and commercialization expertise; 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, 2015 filed on February 29, 2016 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.


  1. Schnabel B, Brenner DA. Gastroenterology. 2014.
  2. Úbeda et al. J Hepatol. 2016.


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