New Analyses of the Phase 3 POISE Trial of Ocaliva™ (Obeticholic Acid) for the Treatment of PBC Presented at EASL

NEW YORK, April 14, 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 the results of two new analyses of the POISE trial, the Phase 3 trial of Ocaliva (obeticholic acid) for the treatment of primary biliary cirrhosis, recently renamed primary biliary cholangitis (PBC). The analyses, presented at the International Liver Congress 2016, the 51st Annual Meeting of the European Association for the Study of the Liver (EASL), examined a risk model to predict estimated liver transplant-free survival of patients in the POISE trial and assessed a titration strategy for managing treatment-related pruritus. Ocaliva is an investigational product and has not been granted marketing authorization or approval from any regulatory authority.

The effects of Ocaliva on patient outcomes in PBC have not been directly evaluated, but in the first analysis, researchers from Erasmus University Medical Center used a risk model developed by the Global PBC Study Group to predict liver transplant-free survival in patients treated with Ocaliva or placebo. Of 216 randomized patients in the POISE trial, 93% continued receiving ursodeoxycholic acid (UDCA), the current standard of care and only approved medication for PBC, upon initiating Ocaliva or placebo. The trial's primary endpoint was defined as a reduction in serum alkaline phosphatase (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. The Global PBC Study Group used data collected from more than 4,000 patients to construct and validate its risk model, with age at the beginning of UDCA therapy, total bilirubin, ALP, platelet count, and albumin following 12 months of treatment with UDCA identified as independent predictors of liver transplant-free survival. After one year, the model suggests patients treated with Ocaliva ±UDCA had significantly improved estimated liver transplant-free survival compared to the group treated with placebo ±UDCA (p<0.0001).

The second analysis utilizing the POISE study data examined the titration strategy used in the trial to address treatment-related pruritus. Pruritus is a common symptom of PBC and mild to moderate pruritus was the most common adverse event observed in POISE. The titration strategy – initiating Ocaliva at 5 mg and titrating up to 10 mg based on clinical response at six months – helped mitigate both the incidence and severity of pruritus, resulting in a single patient discontinuing Ocaliva in the titration group over 12 months of treatment. Despite an early increase at two weeks, patient-reported pruritus severity improved over the course of the study and by month six the Ocaliva 10 mg group and placebo ±UDCA group were similar. Additionally, titration appeared to delay time to onset in the subset of patients who experienced pruritus, with a median of 24 days in the titration group vs. nine days in the group initiating Ocaliva treatment at 10 mg. The authors noted that the AASLD and EASL Clinical Practice Guidelines for the treatment of cholestatic pruritus may also be used for management of Ocaliva-induced pruritus.

“The POISE trial has given us a number of important insights about Ocaliva’s potential role as a new therapeutic option for patients with PBC,” said Marlyn Mayo, M.D., Associate Professor, Internal Medicine, University of Texas Southwestern Medical Center, and lead author of the titration analysis. “Ocaliva treatment reduced ALP, a biochemical marker we use to track disease progression and a component of risk models used to predict long-term outcomes in patients with PBC. Additionally, our analysis suggests that the titration approach used in POISE may provide physicians and patients with a strategy to help reduce drug-induced pruritus.”

Ocaliva is not approved for use by FDA, EMA or any other regulatory body. No conclusions can be drawn concerning the safety or efficacy of Ocaliva at this time.

About Primary Biliary Cirrhosis, recently renamed Primary Biliary Cholangitis
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 Ocaliva in PBC patients with an inadequate therapeutic response to, or who are unable to tolerate, UDCA. The POISE data showed that Ocaliva, 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 Ocaliva 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 Ocaliva and placebo groups.

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: 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 anticipated commercialization of Ocaliva in PBC, the clinical relevance and utility of the endpoints used in the Phase 3 POISE trial, the anticipated prevalence of PBC, the continued development of Ocaliva and Intercept's other product candidates, 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; and our ability to attract and 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
For more information about Intercept Pharmaceuticals, please contact:

Mark Vignola
+1-646-747-1000
investors@interceptpharma.com

Christopher Frates
+1-646-757-2371
media@interceptpharma.com

Source:Intercept Pharmaceuticals, Inc.