Research Demonstrates Scope of Unmet Need in PBC
New Analysis of Phase 3 POISE Study Uses Risk Algorithm to Examine Potential of OCA to Lower Patients' Long-Term Risk of Liver Transplant and Liver-Related Death
NEW YORK, Nov. 14, 2015 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT), a clinical stage biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat chronic underserved liver diseases, today announced results from three studies in primary biliary cirrhosis, recently renamed primary biliary cholangitis (PBC), that will be presented at the American Academy for the Study of Liver Diseases (AASLD) Annual Meeting (The Liver Meeting®) in San Francisco, CA from November 14-16. The studies evaluate investigational use of obeticholic acid (OCA), Intercept's lead farnesoid X receptor (FXR) agonist, for the treatment of PBC, the epidemiology of PBC, and both patient and physician perceptions of PBC care. Taken together, the following oral and poster presentations provide more information about the unmet need and treatment goals for PBC.
Clinical Epidemiology of Primary Biliary Cirrhosis based on a Large U.S. Laboratory Database: Incidence and Trends in Serum Alkaline Phosphatase
In an oral presentation, researchers will share results from an analysis of a clinical database of more than 575,000 patients who received an anti-mitochondrial antibody (AMA) test, the principal autoimmune marker used to diagnose PBC in patients with elevated alkaline phosphatase (ALP) levels. Of those patients, 6,107 were classified as having probable PBC based on a positive AMA test and ALP greater than the upper limit of normal at any time prior to AMA testing or up to one month following AMA testing. The study found that the majority (69%) of these likely PBC patients continued to have elevated ALP two years after the first positive AMA test.
"Large-scale epidemiological data on PBC are scarce, so this analysis provides a unique window into ALP trends in this population," said W. Ray Kim, M.D., Stanford University Medical Center, Stanford, CA. "Because PBC patients with elevated ALP levels are at risk of negative outcomes, including potential liver transplant or death, these results suggest a larger unmet need than previously thought for better management of PBC."
A Trial-Based Model of Liver Transplant and Liver-Related Death in Patients with Primary Biliary Cirrhosis
In this oral presentation, researchers will discuss an analysis of data from the Phase 3 POISE trial of OCA in PBC using the UK-PBC predictive model of transplant-free survival based on ALP, bilirubin, alanine transaminase (ALT), albumin and platelet count. Risk was assessed at 5, 10 and 15 years based on a 12-month change from baseline in patients treated with OCA ± ursodiol or placebo ± ursodiol at the end of the POISE study. In the analysis, the UK-PBC risk algorithm showed a significantly lower risk of liver transplant or liver-related death in OCA-treated patients compared to placebo ± ursodiol.
"This analysis is the first to evaluate the potential of OCA to lower patients' long-term risk of liver transplant and liver-related death using the risk algorithm of the UK-PBC research group," said Marco Carbone, M.D., Academic Department of Medical Genetics, University of Cambridge, Cambridge, U.K. "There is an urgent need for new therapy options in PBC that can help patients who have been unable to achieve treatment goals with the current standard of care."
Physician versus Patient Perceptions of Medical Care Quality in Primary Biliary Cirrhosis
This poster will highlight results from a survey examining perceptions of PBC among gastroenterologists (262), hepatologists (60) and patients (214). Patient and physician responses to questions about PBC-related information, PBC diagnosis and treatment, and patient quality of life were broadly similar. However, only approximately one in three PBC patients surveyed knew their most recent ALP score, despite 64% of physicians stating they rely on ALP to monitor the disease. Additionally, although most physicians reported discussing the relationship between PBC symptoms and disease progression with their patients, just one in three patients surveyed understood that worsening PBC symptoms are not an indication of worsening disease. The researchers concluded that improving communication between physicians and patients could enhance patient care.
"These survey results underscore an important opportunity to improve the dialogue about PBC treatment goals between physicians and patients," said David Shapiro, M.D., Intercept's Chief Medical Officer & Executive Vice President, Development. "A large and growing body of research shows that lowering ALP can help reduce the risk of liver transplant and death in PBC, and we are committed to helping patients access both the treatment and educational support needed to improve care."
About Primary Biliary Cirrhosis, also known as 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 Practice to Policy Health Awards Program
Intercept recently launched the Practice to Policy Health Awards Program to address the unmet needs of patients and families impacted by PBC. These awards will support innovations in patient support, health technology, and service design. Through this program, Intercept hopes to enable the healthcare community to introduce new practices and generate evidence that will advance patient care and create the momentum for long-term health improvement. Grant applications can be submitted until January 31, 2016 and information about the Practice to Policy Health Awards Program can be found at: www.InterceptPracticeToPolicy.com.
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, ursodiol. 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. Apart from pruritus, the incidence of adverse events was generally similar across both OCA and placebo groups.
About Intercept
Intercept is a biopharmaceutical company focused on the development and commercialization of novel therapeutics to treat chronic underserved 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.
As announced earlier this year, OCA is progressing along regulatory pathways in both the U.S. and EU. The U.S. Food and Drug Administration has granted Priority Review for OCA for the treatment of PBC and set a target date of February 29, 2016 to take action on the PBC application under the Prescription Drug User Fee Act (PDUFA). In the EU, Intercept continues to plan for approval and launch in the second half of 2016.
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: http://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 clinical, preclinical and regulatory developments for Intercept's product candidates, the anticipated results of Intercept's clinical trials and preclinical studies and other development activities and the timing thereof, Intercept's potential development and regulatory milestones and the timeframes under which it anticipates such milestones may be achieved, the utility of surrogate endpoints and other markers in predicting outcomes in PBC, the ability of past results to predict future results, the clinical utility of our selected endpoint and any potential consensus relating thereto, and Intercept's 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 Intercept's development activities, preclinical studies and clinical trials; the timing of and Intercept's ability to obtain and maintain regulatory approval of OCA, INT-767 and any other product candidates it 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; Intercept's plans to research, develop and commercialize its 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 need for and ability to obtain additional financing; Intercept's estimates regarding expenses, future revenues and capital requirements and the accuracy thereof; Intercept's ability to retain 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 December 31, 2014 filed on March 2, 2015 as well as any updates to these risk factors filed from time to time in 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.