New Analyses of Data From POISE -- the First Phase 3 Trial in PBC in Two Decades -- Examine the Efficacy, Safety and Tolerability Profile of OCA
NEW YORK, Nov. 8, 2014 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT) (Intercept), a clinical stage biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat chronic liver and intestinal diseases, announced today new analyses of data from clinical trials on obeticholic acid (OCA) in patients with primary biliary cirrhosis (PBC). Six posters, including posters with new analyses of data from POISE – the first Phase 3 trial in PBC in two decades – are being presented at today's poster session at the American Association for the Study of Liver Disease (AASLD) Annual Meeting.
OCA, Intercept's lead product candidate, is a bile acid analog and first-in-class agonist of the farnesoid X receptor (FXR) in development for PBC, nonalcoholic steatohepatitis (NASH), primary sclerosing cholangitis (PSC) and other chronic liver diseases.
In March 2014, Intercept announced that OCA met the primary endpoint of the POISE trial with high statistical significance (p < 0.0001). The posters to be presented at AASLD provide the following information relating to OCA in PBC:
- Poster 318 provides new subgroup analyses of the treatment effect for OCA across a range of patient characteristics associated with greater risk of adverse clinical outcomes, including age at diagnosis, duration of PBC and baseline alkaline phosphatase (ALP).
- Poster 295 presents analyses on the effects of OCA treatment versus placebo on markers of cholestasis (ALP, bilirubin) and hepatobiliary damage (GGT, AST, and ALT), as well as markers of inflammation (C-reactive protein or CRP) and apoptosis (cytokeratin-18 or CK-18).
- Poster 309 presents POISE results in relation to the proportion of patients achieving a response defined by different response criteria, other than the POISE endpoint, such as Paris I, Paris II and Rotterdam.
- Posters 305 and 310 cover new information relating to the titration arm of POISE, where approximately half the patients in the titration arm had their OCA dose increased from 5 mg to 10 mg after six months of treatment based on clinical response. These posters present new data regarding the potential utility of titration as a dosing strategy in the context of managing pruritus, the primary tolerability issue of OCA treatment, while investigating the effects of titration on the efficacy of OCA treatment.
- Poster 319 describes the long-term safety and efficacy results from open-label treatment of OCA for over four years as part of the long-term safety extension trial on patients who participated in a Phase 2 PBC trial that evaluated OCA monotherapy.
Linie Moore, President of the PBCers patient advocacy group, commented on the new PBC research being presented at AASLD: "In the time since I was first diagnosed with PBC almost 20 years ago, there have been no new medications introduced to treat my disease, so it is both gratifying and exciting to see so much progress being made with OCA as a potential new PBC therapy. We hope the PBC community's efforts to encourage research and awareness will continue to inspire new advances in the years to come."
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, ursodiol. The primary endpoint of the 12-month double-blind portion of the trial, completed in March 2014, was the achievement of both an ALP level < 1.67x ULN with a ≥ 15% reduction from baseline and a normal bilirubin level, as compared to placebo. Patients with ALP and bilirubin levels below these thresholds have been shown in long-term clinical studies to have a significantly lower risk of progressing to liver transplant and death. There were 217 patients randomized to one of three groups in the trial: placebo, 10 mg OCA, or 5 mg OCA for six months titrated to 10 mg OCA based on clinical response; 216 patients were dosed.
Patients completing the double-blind phase had the option to continue in an open-label, long-term safety extension (LTSE) phase for another five years, during which all patients receive OCA treatment with daily doses starting at 5 mg and potentially titrating up to 25 mg a day, as clinically indicated. Of the 198 patients who completed the double-blind phase, more than 95% continued in the LTSE phase of the trial.
Additional information regarding the POISE trial can be found on the NIH clinical study listing web site: http://clinicaltrials.gov/ct2/show/NCT01473524.
About Primary Biliary Cirrhosis
PBC is an autoimmune liver disease that may progress to cirrhosis and liver failure, and it is currently the second leading indication for liver transplant among women in the United States. It is primarily a disease of women, afflicting approximately one in 1,000 women over the age of 40. Ursodiol is the only approved drug treatment for PBC and studies have shown that up to 50% of PBC patients may have an inadequate response, thereby remaining at risk of adverse outcomes.
Intercept is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat orphan and more prevalent chronic liver and intestinal diseases utilizing its expertise in bile acid chemistry. The company's lead product candidate, obeticholic acid (OCA), is a bile acid analog and first-in-class agonist of the farnesoid X receptor (FXR). OCA is being developed for a variety of chronic liver diseases including primary biliary cirrhosis (PBC), nonalcoholic steatohepatitis (NASH), and primary sclerosing cholangitis (PSC). OCA has received Fast Track Designation in the United States and orphan drug designation in both the United States and Europe for the treatment of PBC and PSC. Several large, randomized, controlled studies of OCA in the treatment of chronic liver disease have been completed. These include Intercept's Phase 3 POISE trial for the treatment of patients with PBC and the FLINT trial for the treatment of patients with NASH. Intercept owns worldwide rights to OCA outside of Japan, China and Korea, where it has out-licensed the product candidate to Sumitomo Dainippon Pharma. 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 utility of the selected endpoint for POISE or the other clinical data presented at AASLD; the acceptance by regulatory authorities of the POISE trial endpoint or results; the anticipated results of our clinical 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 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, INT-777 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; the election by our collaborators to pursue research, development and commercialization activities; our ability to attract collaborators with development, regulatory and commercialization expertise; 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; 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 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, 2013 filed on March 14, 2014 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.