Intercept Pharmaceuticals Announces Positive Initial Results from Ongoing
Phase 2a Trial in Chronic Bile Acid Diarrhea
NEW YORK, May 20, 2013
NEW YORK, May 20, 2013 /PRNewswire/ -- Intercept Pharmaceuticals, Inc.
("Intercept", NASDAQ: ICPT), a clinical stage biopharmaceutical company
focused on the development and commercialization of novel therapeutics to
treat chronic liver diseases, today announced positive initial results from
OBADIAH, an ongoing Phase 2a trial of obeticholic acid (OCA) as a treatment
for primary bile acid diarrhea (PBAD) presented at the Digestive Diseases Week
conference. The initial results from the OBADIAH trial demonstrate that
treatment with OCA is associated with statistically significant increased
levels of fibroblast growth factor 19 (FGF19) and improvement in clinical
symptoms in patients with PBAD.
OBADIAH is an investigator-initiated and sponsored open label Phase 2a trial
being conducted by Professor Julian Walters at the Imperial College of London
in the United Kingdom. The trial is investigating whether OCA can stimulate
the release of FGF19 in patients with PBAD. This disease, also known as
idiopathic bile acid malabsorption, is a common chronic diarrheal condition
due to excessive bile acid production and loss. PBAD is estimated to affect
approximately one percent of the population and about one-third of patients
diagnosed with diarrhea-predominant irritable bowel syndrome (IBS-D). Patients
with PBAD have low levels of FGF19, a hormone released in the ileum in
response to FXR activation and regulates bile acid production by the liver. As
a result, excess bile acids spill into the gut and produce diarrhea by
overstimulating intestinal secretions. All three of Intercept's previously
completed Phase 2 trials in other indications demonstrated that OCA markedly
and dose dependently stimulates the release of FGF19.
"These data represent an encouraging first step in a new therapy for many
patients thought to have IBS-D, who in actuality have primary bile acid
diarrhea associated with impaired release of FGF19," stated Professor Walters.
"As obeticholic acid is a potent FXR agonist and stimulator of FGF19 release,
it is a logical therapeutic approach which we are keen to evaluate further
given the need for more effective treatments in patients with this disorder."
The primary outcome measure of the open-label OBADIAH trial is to assess
changes in FGF19 levels over a two-week period in ten patients with PBAD and
in two other groups, one consisting of patients with secondary bile acid
diarrhea due to Crohn's disease and the other consisting of IBS-D patients who
have normal FGF19 levels. Secondary outcome measures include clinical symptom
scores, biochemical response and tolerability. Data from ten PBAD patients,
the first group studied in OBADIAH, indicate that a 25 mg daily oral dose of
OCA resulted in a statistically significant increase in median fasting FGF19
from 133 to 237 pg/mL, with most patients achieving a >60% increase (p=0.007).
In addition, clinical improvements were seen in all patients with reductions
in median stool frequency from 23 to 14 per week (p=0.03) and an improvement
in the median Bristol Stool Form Scale assessing stool type from 5.15 to 4.34
(p=0.05). Notably, during the two week follow up period after stopping OCA
therapy, stool frequency returned to pre-treatment baseline values. OCA was
well tolerated in all patients.
Intercept anticipates final results for all three study groups to be available
in the second half of 2013.
The slides for the presentation made by Professor Walters are available on the
"Presentations" section of Intercept's website at www.interceptpharma.com.
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, or 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. PBC is a
chronic autoimmune liver disease that may progress to cirrhosis and liver
failure, and it is currently the fifth leading indication for liver transplant
in the United States. OCA has 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 (DSP).
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 potential effect of OCA in PBAD such as
its impact on FGF19, the anticipated enrollment of OBADIAH, the availability
of results from the trial, and the timing of the anticipated enrollment and
availability of results, 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, quarterly reports on Form 10-Q, 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.
SOURCE Intercept Pharmaceuticals, Inc.
Contact: For more information about Intercept, please contact Mark Pruzanski,
M.D., or Barbara Duncan, both of Intercept Pharmaceuticals, at 1-646-747-1000.
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