Additional Results of Global Primary Biliary Cirrhosis Study Group Analysis to be Presented at AASLD Annual Meeting

Additional Results of Global Primary Biliary Cirrhosis Study Group Analysis to
                     be Presented at AASLD Annual Meeting

Data Support Strong Statistical Association of PBC Biochemical Endpoint with
Clinical Outcomes

PR Newswire

NEW YORK, Oct. 1, 2013

NEW YORK, Oct. 1, 2013 /PRNewswire/ -- Intercept Pharmaceuticals, Inc.
(NASDAQ: ICPT)(Intercept), a clinical stage biopharmaceutical company focused
on the development and commercialization of novel therapeutics to treat
chronic liver diseases such as primary biliary cirrhosis, today announced that
two analyses by the Global Primary Biliary Cirrhosis (PBC) Study Group (also
known as the PBC Supergroup) have been accepted for oral presentation at the
64^th Annual Meeting of the American Association for the Study of Liver
Diseases (AASLD), taking place November 1 – 5 in Washington, D.C.

Data from over 3,895 PBC patients collected and pooled by an independent group
of 15 academic medical centers across eight countries have been analyzed by
the Global PBC Study Group. These analyses are expected to further confirm
that the surrogate biochemical endpoint used by Intercept in its ongoing Phase
3 POISE trial (i.e., alkaline phosphatase (ALP) < 1.67x upper limit of normal
and normal bilirubin) is strongly predictive of adverse clinical outcomes in
PBC patients.

"This international collaboration has assembled data from the largest group of
PBC patients to have ever been evaluated since PBC was first described over
150 years ago," commented Dr. Henk van Buuren, one of the principal
investigators at Erasmus University Medical Centre. "The data show that
abnormal biochemical values predict a higher risk of adverse clinical
outcomes. We believe that our results will facilitate the development of new
drugs by clearly establishing meaningful biochemical goals for therapy and
enable physicians to determine if a given treatment is effective. PBC is a
rare disease and we are very grateful to our colleagues at the many
participating institutions around the world for sharing their data."

The two abstracts from the Global PBC Study Group can be accessed through the
AASLD website, www.aasld.org.

Primary Biliary Cirrhosis and the Global PBC Study Group
PBC is an autoimmune chronic liver disease that typically affects women. The
disease progresses in those patients who have an inadequate response to
therapy and may require a liver transplant or die. Biochemical assessments of
liver function, particularly by measuring plasma levels of ALP and bilirubin,
are typically used by physicians to diagnose and manage PBC patients. There
is a log-linear relationship between ALP levels and transplant-free survival;
higher levels are associated with a worse prognosis. Accordingly, the
relationship between these biochemical assessments of liver function and
adverse clinical outcomes is highly important in the selection of appropriate
endpoints in therapeutic clinical trials.

Data from the Global PBC Study Group are being analyzed under the direction of
Dr. Bettina Hansen, Dr. Henk van Buuren and colleagues at Erasmus University
Medical Centre in Rotterdam, The Netherlands. Intercept is sponsoring this
independent academic research program but is not involved in the data
collection and analysis. In April 2013, the Global PBC Study Group presented
an analysis of data from over 2,100 patients at the annual meeting of the
European Association for the Study of the Liver (EASL). These preliminary
data demonstrated that the primary endpoint being used in Intercept's Phase 3
POISE trial is highly statistically predictive of liver transplant-free
survival in PBC patients. 

About Intercept
Intercept is a biopharmaceutical company focused on the development and
commercialization of novel therapeutics to treat orphan and more prevalent
liver 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 initially
being developed for the second line treatment of primary biliary cirrhosis
(PBC) in patients with an inadequate response to, or who are unable to
tolerate, ursodiol, the only approved therapy for this indication. OCA has
received orphan drug designation in both the United States and Europe for the
treatment of PBC. Intercept owns worldwide rights to OCA outside of Japan and
China, where it has out-licensed the product candidate to Dainippon Sumitomo
Pharma. For more information about Intercept, please visit the Company's
website at: www.interceptpharma.com.

Safe Harbor Statement
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 analyses of the Global PBC Study Group
data and the results thereof, the relationship between ALP and bilirubin and
adverse clinical outcomes, the clinical utility of the POISE trial selected
endpoints and any potential consensus relating thereto, clinical, preclinical
and regulatory developments for our product candidates, the anticipated
results of our clinical and preclinical 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 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 and any
other product candidates it may develop, and any related restrictions,
limitations, and/or warnings in the label of any approved product candidates;
Intercept's plans to research, develop and commercialize future 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 ability to obtain additional financing; Intercept's use of the
proceeds from its recently completed initial public offering; the accuracy of
Intercept's estimates regarding expenses, future revenues, capital
requirements and the need for additional financing; the loss of 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 2012, as well as any updates to these risk factors filed from time to
time in Intercept's 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.

For more information about Intercept, please contact Barbara Duncan or Senthil
Sundaram, both of Intercept Pharmaceuticals, at 1-646-747-1000.

SOURCE Intercept Pharmaceuticals
 
Press spacebar to pause and continue. Press esc to stop.