Intercept Announces NASH Primary Endpoint Met: FLINT Trial Stopped Early for Efficacy Based on Highly Statistically Significa...
09 January 2014 - 11:32PM
Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT) (Intercept) today
announced that the FLINT trial of obeticholic acid (OCA) for the
treatment of nonalcoholic steatohepatitis (NASH) has been stopped
early for efficacy based on a planned interim analysis showing that
the primary endpoint of the trial has been met. FLINT is a
multi-center, double-blind, placebo-controlled clinical trial
assessing the safety and efficacy of a 25 mg oral dose of OCA
administered daily to biopsy-proven adult NASH patients over a
72-week treatment period. The trial has been sponsored and
conducted by the National Institute of Diabetes & Digestive
& Kidney Diseases (NIDDK), a part of the National Institutes of
Health, at eight leading US academic hepatology centers comprising
the NIDDK's NASH clinical research network (CRN).
The decision to stop FLINT has been based on the recommendation
of the Data Safety Monitoring Board (DSMB) which reviewed liver
biopsy data from before and at the end of the treatment period in
approximately half of the 283 randomized patients, in accordance
with a planned interim efficacy analysis. This analysis
demonstrated that OCA treatment resulted in a highly statistically
significant improvement (p=0.0024 on an intention-to-treat [ITT]
basis) in the primary histological endpoint, defined as a decrease
in the NAFLD Activity Score (NAS) of at least two points with no
worsening of fibrosis, as compared to placebo. Those patients who
had not yet completed the trial and therefore did not have a second
biopsy were treated as non-responders in the ITT analysis. The
pre-defined threshold of statistical significance for stopping
FLINT was p < 0.0031.
"The unexpected early stopping of FLINT due to OCA meeting the
primary endpoint with such high significance is a major milestone,"
said Mark Pruzanski, M.D., Chief Executive Officer of Intercept.
"NASH has grown to epidemic proportions worldwide, having become a
leading cause of cirrhosis and liver failure. On its current
trajectory, the disease is projected to become the leading
indication for liver transplant. We are deeply grateful to the
NIDDK and the NASH CRN for their longstanding commitment both to
improving our understanding of the disease and to sponsoring
ambitious trials like FLINT in their quest to identify novel
treatments for patients suffering from NASH."
Intercept will discuss NASH and the FLINT trial during the
previously announced conference call and audio webcast scheduled to
take place today at 4:30 p.m. ET. The live event will be available
on the investor page of the Intercept website at
http://ir.interceptpharma.com or by calling (855) 232-3919
(domestic) or (315) 625-6894 (international) five minutes prior to
the start time. A replay of the call will be available on the
Intercept website approximately two hours after the completion of
the call and will be archived for two weeks.
About FLINT
The Farnesoid X Receptor Ligand Obeticholic Acid in Nonalcoholic
Steatohepatitis Treatment (FLINT) trial has been sponsored and
conducted by the National Institute of Diabetes & Digestive
& Kidney Diseases (NIDDK). FLINT enrolled 283 adult NASH
patients at eight US centers comprising the NIDDK's NASH clinical
research network. Patients were randomized to receive either a 25
mg dose of OCA or placebo for 72 weeks. Patients enrolled in the
trial were qualified based on a diagnosis determined by liver
biopsy at the start of the trial with a NAFLD Activity Score (NAS)
of four or greater and with a score of at least one in each
component of the NAS eight point scale (steatosis 0-3, lobular
inflammation 0-3, ballooning 0-2). End of study biopsies were
conducted in patients after the 72-week treatment period, with all
biopsies centrally scored in a blinded fashion. Further details can
be found at http://clinicaltrial.gov/ct2/show/NCT01265498.
Intercept's collaborator Dainippon Sumitomo Pharma is currently
conducting a NASH trial in Japan. This trial is evaluating the
safety and efficacy of a once-daily dose of OCA as compared to
placebo, with a targeted enrollment of 200 patients. Enrollment is
projected to be completed by the end of January 2014 with top-line
results expected by the end of 2015.
About NASH
NASH is a serious chronic liver disease caused by excessive fat
accumulation in the liver that, for reasons that are still
incompletely understood, induces chronic inflammation which leads
to progressive fibrosis (scarring) that can lead to cirrhosis,
eventual liver failure and death. There are currently no drugs
approved for the treatment of NASH. Studies have shown that over a
ten year period at least 10% of NASH patients will develop
cirrhosis, and liver-related mortality due to this disease is
ten-fold that of the general population. According to recent
epidemiological studies, it is estimated that approximately 12% of
the U.S. adult population has NASH, while 2.7% (potentially more
than six million patients) are believed to have advanced liver
fibrosis or cirrhosis due to progression of the disease. The
proportion of liver transplants attributable to NASH has increased
rapidly in past years and over the next decade 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
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 being developed
for a variety of chronic liver diseases including NASH, primary
biliary cirrhosis (PBC), portal hypertension and bile acid
diarrhea. 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 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
trajectory of the growth of the market for NASH; the utility of the
selected endpoint; the acceptance by regulatory authorities of the
trial endpoint or results; clinical, preclinical and regulatory
developments for Intercept's product candidates; the anticipated
timeframe for the commencement, completion and receipt of results
from the clinical trials in OCA and for the making of regulatory
submissions; 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, INT-767 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
initial public offering in October 2012 and follow-on offering in
June 2013; 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 December 31, 2012 filed on April 1, 2013 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.
CONTACT: For more information about Intercept,
please contact Barbara Duncan or Senthil Sundaram, both of
Intercept Pharmaceuticals at 1-646-747-1000.
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