Intercept Announces Ocaliva™ (Obeticholic Acid) Data in PBC to be Presented at DDW 2016

NEW YORK, May 18, 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, announced today that five abstracts evaluating obeticholic acid (OCA) in patients with primary biliary cholangitis (PBC) and nonalcoholic steatohepatitis (NASH) will be presented at Digestive Disease Week® (DDW) in San Diego, California from May 21-24, 2016. OCA is an investigational product and has not been granted marketing authorization or approval from any regulatory authority.  

“We look forward to sharing additional data from the Phase 3 POISE trial of OCA in PBC patients, including further results from our long-term safety extension and an analysis of the titration strategy used to address treatment-induced pruritus in POISE,” said David Shapiro, M.D., Intercept's Chief Medical Officer & Executive Vice President, Development.  “We hope these presentations will provide gastroenterologists and hepatologists with greater insight into future treatment for PBC patients who have an inadequate response to the current standard of care. In addition, we have several presentations with our academic collaborators that provide further information about our clinical study results in both PBC and NASH.”

At DDW, Intercept will present four analyses of data from the POISE trial, along with an evaluation of the effects of concomitant medications and associated comorbidities on NASH patients who participated in the Phase 2 FLINT trial.

Intercept will be exhibiting at booth #4253 throughout the meeting. Presentations at DDW include:

Oral Presentation in PBC

Tuesday, May 24, 5:00 - 5:15 p.m., Room 8 - SDCC
“Baseline Factors Predicting Obeticholic Acid Induced Pruritus in Patients with PBC” (Abstract 1111)
Elizabeth Smoot Malecha, Ulrich Beuers, Alexander Liberman, Roya Hooshmand-Rad, Yvette Peters, Richard Pencek

Poster Presentations in PBC

Saturday, May 21, 9:30 a.m. - 4:00 p.m., Hall C - SDCC
“Mitigation of Pruritus During Obeticholic Acid Treatment in Patients With Primary Biliary Cirrhosis: Strategies and Successes” (Abstract Sa1575)
Marlyn Mayo, Andreas E. Kremer, Ulrich Beuers, Tonya Marmon, Roya Hooshmand-Rad, Richard Pencek, David Shapiro

Saturday, May 21, 9:30 a.m. - 4:00 p.m., Hall C - SDCC
“Exposure-Response Relationship of Obeticholic Acid for Alkaline Phosphatase and Total Bilirubin in Patients with Primary Biliary Cirrhosis” (Abstract Sa1576)
Jeffrey E. Edwards, Carl LaCerte, Leng Hong Pheng, Thomas Peyret, Nathalie Gosselin, JF Marier, David Shapiro

Saturday, May 21, 9:30 a.m. - 4:00 p.m., Hall C - SDCC
“Durable Response in the Markers of Cholestasis through 18 Months of Open-Label Long Term Safety Extension Study of Obeticholic Acid in Primary Biliary Cirrhosis” (Abstract Sa1579)
Michael H. Trauner, Frederik Nevens, Pietro Andreone, Giuseppe Mazzella, Simone Strasser, Christopher L. Bowlus, Pietro Invernizzi, Joost Drenth, Paul J. Pockros, Jaroslaw Regula, Annarosa Floreani, Simon Hohenester, Velimir A. Luketic, Mitchell L. Shiffman, Karel J. Van Erpecum, Victor Vargas, Catherine Vincent, Bettina Hansen, Leigh MacConell, Tonya Marmon, David Shapiro

Poster Presentation in NASH

Monday, May 23, 9:30 a.m. - 4:00 p.m., Hall C - SDCC
“Evaluation of Effects of Concomitant Medications for NASH and Associated Comorbidities on Histological Improvements with Obeticholic Acid” (Abstract Mo1557)
Kris V. Kowdley, Manal F. Abdelmalek, Arthur McCullough, Rohit Loomba, Bilal Hameed, Naga P. Chalasani, Brent Neuschwander-Tetri, Saswati Hazra, Jianfen Chen, Reshma Shringapure, David Shapiro, Arun J. Sanyal

Educational PBC Presentation

Sunday, May 22, 2:15 – 3:00 p.m., Exhibit Hall A
“The ABCs of PBC: An Overview of Primary Biliary Cirrhosis, Also Known as Primary Biliary Cholangitis”
Fred Poordad

A full list of sessions at DDW, including symposia relating to OCA, is available on the Digestive Disease Week website.

OCA is not approved for use by FDA, EMA or any other regulatory body. No conclusions can be drawn concerning the safety or efficacy of OCA at this time. The brand name Ocaliva has been provisionally approved by the FDA and European Medicines Agency for OCA in PBC.

About Digestive Disease Week
Digestive Disease Week (DDW) is the largest international gathering of physicians, researchers and academics in the fields of gastroenterology, hepatology, endoscopy and gastrointestinal surgery. Jointly sponsored by the American Association for the Study of Liver Diseases (AASLD), the American Gastroenterological Association (AGA) Institute, the American Society for Gastrointestinal Endoscopy (ASGE) and the Society for Surgery of the Alimentary Tract (SSAT), DDW take place May 21-24, 2016, at the San Diego Convention Center, San Diego, CA. The meeting showcases more than 5,000 abstracts and hundreds of lectures on the latest advances in GI research, medicine and technology. More information can be found at

About Primary Biliary Cholangitis, Formerly Known as 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. Since 1988, PBC has been the second-leading overall cause of liver transplant in women in the United States, behind hepatitis C. In Europe, the disease accounts for approximately half of liver transplants due to cholestatic diseases and 6% of all liver transplants.

About the POISE Trial
The POISE trial studied the safety and efficacy of a once-daily treatment with OCA in PBC patients with an inadequate therapeutic response to, or who are unable to tolerate, UDCA. The POISE data showed that OCA, at both a 10 mg dose and a 5 mg dose titrated to 10 mg, met the trial's primary endpoint of achieving a reduction in serum ALP, to below a threshold of 1.67 times upper limit normal, with a minimum of 15% reduction in ALP level from baseline, and a normal bilirubin level after 12 months of therapy. Pruritus, generally mild to moderate, was the most frequently reported adverse event associated with OCA treatment. Decreases in HDL-C and transient increases in LDL-C were observed during treatment. Apart from pruritus, the incidence of adverse events was generally similar across both OCA and placebo groups.

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 alanine aminotransferase (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 non-viral, progressive 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, 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 the Company'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 regulatory approval and 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 clinical relevance and utility of the endpoints and results of Intercept’s clinical trials, 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 use of cash and short term investments; 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.

CONTACT: Contact
For more information about Intercept Pharmaceuticals, please contact:

Mark Vignola

Christopher Frates