NEW YORK, Dec. 14, 2016 (GLOBE NEWSWIRE) --
Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT) (Intercept), a
biopharmaceutical company focused on the development and
commercialization of novel therapeutics to treat progressive
non-viral liver diseases, today announced that the European
Commission has granted conditional approval for Ocaliva
(obeticholic acid) for the treatment of primary biliary cholangitis
(PBC) in combination with ursodeoxycholic acid (UDCA) in adults
with an inadequate response to UDCA or as monotherapy in adults
unable to tolerate UDCA. Ocaliva is a potent and selective agonist
of the farnesoid X receptor (FXR), which is expressed at high
levels in the liver and intestine and thought to be a key regulator
of bile acid, inflammatory, fibrotic and metabolic pathways.
"The approval of Ocaliva in Europe provides a new
therapeutic option for a substantial group of PBC patients who are
not achieving treatment goals with UDCA alone or who cannot
tolerate UDCA," said Frederik Nevens, M.D., Ph.D., University
Hospitals Leuven & KU Leuven, Belgium, and the lead
investigator of the Phase 3 POISE clinical study. "Despite the
availability of UDCA, many patients have remained at significant
risk of adverse outcomes with no alternative treatment option
available. Ocaliva can now help fill an important unmet need for
these patients."
"We are delighted to be introducing the first new
therapeutic option for PBC in nearly 20 years in Europe where this
disease is a major reason for liver failure and a leading cause of
liver transplant in women," said Lisa Bright, Intercept's
President, International. "Following approval in the U.S. earlier
this year, Ocaliva's marketing authorization in Europe represents
another big step in Intercept's mission to provide patients with
worldwide access to our innovative therapy. This great achievement
will motivate us further to continue developing solutions that
improve the lives of people with progressive non-viral liver
diseases."
The marketing authorization allows Intercept to
market Ocaliva in 28 countries that are member states of the
European Union, as well as 3 additional European Economic Area
member states. As conditions of the approval, Intercept is required
to provide post-approval updates on safety and efficacy analyses
for Ocaliva from the ongoing Phase 4 COBALT outcomes study and a
short-term study in patients with hepatic impairment.
"As a community, our priority is to advocate for
changes which ensure that people diagnosed with PBC have the best
possible prognosis," said Tatjana Reic, President of the European
Liver Patients Association (ELPA). "With this in mind, we are
excited about this advance for patients with an inadequate response
or intolerability to the current available treatment. Such patients
will soon have access to a new treatment option to manage their
PBC."
The marketing authorization was based on efficacy
and safety data derived from three randomized double-blind,
placebo-controlled clinical trials evaluating the effect of Ocaliva
on alkaline phosphatase (ALP) and bilirubin in patients with PBC.
It was also supported by two clinical databases that include more
than 10,000 patients from the Global PBC Study Group and UK-PBC
Group, both independently confirming that achieving lower ALP
and/or bilirubin levels is significantly correlated with increased
transplant-free survival.
In the Phase 3 POISE study, nearly half of
patients (46%) in the titration group treated with Ocaliva in
combination with UDCA achieved the primary endpoint compared to 10%
in the control group (placebo added to UDCA) (p<0.0001).
Additionally, 77% of patients taking Ocaliva in combination with
UDCA achieved a reduction of more than 15% in ALP at 12 months,
compared to 29% taking UDCA alone.
The most commonly reported adverse reactions were
pruritus (63%) and fatigue (22%). Adverse reactions leading to
discontinuation were 1% in the Ocaliva titration arm and 11% in the
Ocaliva 10 mg arm. The most common adverse reaction leading to
discontinuation was pruritus. The majority of pruritus occurred
within the first month of treatment and tended to resolve over time
with continued dosing.
About Primary Biliary
Cholangitis
Primary biliary cholangitis (PBC) is a rare, autoimmune cholestatic
liver disease that puts patients at risk for life-threatening
complications. PBC is primarily a disease of women, afflicting
approximately one in 1,000 women over the age of 40. If left
untreated, survival of PBC patients is significantly worse than the
general population.
About Ocaliva® (obeticholic
acid)
Ocaliva (obeticholic acid) is a potent and highly selective agonist
of the farnesoid X receptor (FXR), a nuclear receptor expressed in
the liver and intestine. FXR is a key regulator of bile acid,
inflammatory, fibrotic and metabolic pathways.
In December 2016, Ocaliva received conditional
marketing authorization in Europe for the treatment of PBC in
combination with ursodeoxycholic acid (UDCA) in adults with an
inadequate response to UDCA or as monotherapy in adults unable to
tolerate UDCA, conditional to the company providing further data
post-approval to confirm benefit. In May 2016, the U.S. Food and
Drug Administration granted accelerated approval to Ocaliva for the
treatment of PBC. For full prescribing information in the U.S.,
visit Ocaliva.com.
EU IMPORTANT SAFETY
INFORMATION
Contraindications
Hypersensitivity to the active substance or to any of the
excipients and complete biliary obstruction.
Warnings and
Precautions
Elevations in alanine amino transferase (ALT) and aspartate
aminotransferase (AST) have been observed in patients taking
obeticholic acid. Clinical signs and symptoms of hepatic
decompensation have also been observed. These events have occurred
as early as within the first month of treatment. Liver-related
adverse events have primarily been observed at doses higher than
the maximum recommended dose of 10 mg once daily. Patients should
be monitored during treatment with Ocaliva for elevations in liver
biochemical tests and for the development of liver-related adverse
events. Dosage adjustments are needed for patients with moderate
(Child-Pugh Class B) or severe (Child-Pugh Class C) hepatic
impairment.
Severe pruritus was reported in 23% of patients
treated with Ocaliva 10 mg arm, 19% of patients in the Ocaliva
titration arm and 7% of patients in the placebo arms. The median
time to onset of severe pruritus was 11, 158 and 75 days for
patients in the Ocaliva 10 mg, Ocaliva titration and placebo arms,
respectively. Management strategies include the addition of bile
acid binding resins or antihistamines, dose reduction, reduced
dosing frequency and/or temporary dose interruption.
Adverse
Reactions
The most commonly reported adverse reactions were pruritus (63%)
and fatigue (22%). Other common adverse reactions observed in
clinical trials (> 5%) were abdominal pain and discomfort, rash,
oropharyngeal pain, dizziness, constipation, arthralgia, thyroid
function abnormality and eczema.
Drug
Interaction
Bile acid binding resins such as cholestyramine, colestipol or
colesevelam adsorb and reduce bile acid absorption and may
reduce efficacy of obeticholic acid. When concomitant bile acid
binding resins are administered, obeticholic acid should be
taken at least 4-6 hours before or 4-6 hours after taking
a bile acid binding resin, or at as great an interval as
possible.
For detailed safety information for Ocaliva
(obeticholic acid) 5 mg and 10 mg tablets including posology and
method of administration, special warnings, drug interactions and
adverse drug reactions, please see the European Summary of Product
Characteristics that can be found
on www.ema.europa.eu once posted.
About the
POISE Study
The POISE trial studied the safety and efficacy of once-daily
treatment with Ocaliva in PBC patients with an inadequate
therapeutic response to, or who are unable to tolerate, UDCA, the
current standard of care. Of 216 patients randomized to three
treatment arms-placebo, Ocaliva 5 mg titrated to 10 mg or Ocaliva
10 mg-93% continued receiving UDCA. The Ocaliva 5-10 mg titration
group received Ocaliva 5 mg for six months, after which dosing was
increased to 10 mg based on tolerability and biochemical response.
The study's primary endpoint was a reduction in ALP to below a
threshold of 1.67 times the upper limit of normal, with a minimum
of 15% reduction in ALP level from baseline, and a normal bilirubin
level after 12 months of therapy.
About
Intercept
Intercept is a biopharmaceutical company focused on the development
and commercialization of novel therapeutics to treat progressive
non-viral liver diseases, including primary biliary cholangitis
(PBC), nonalcoholic steatohepatitis (NASH), primary sclerosing
cholangitis (PSC) and biliary atresia. Founded in 2002 in New York,
Intercept now has operations in the United States, Europe and
Canada. For more information about Intercept, please
visit 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 relevance and utility of ALP and the
surrogate endpoint used in the Phase 3 POISE trial to predict
clinical outcomes, the acceptance of Ocaliva® (obeticholic
acid) as a treatment for PBC by healthcare providers, patients and
payors, the potential approval of OCA in PBC by regulatory bodies
outside the United States and Europe and the timelines related
thereto, the availability of OCA for the treatment of PBC other
jurisdictions outside the United States and Europe and timelines
related thereto, the anticipated prevalence of and other
epidemiological estimates and market data related to PBC, the
continued development of OCA and Intercept's other product
candidates, 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: Intercept's
ability to successfully commercialize Ocaliva in PBC, and
Intercept's ability to maintain its regulatory approval of Ocaliva
in the United States for Ocaliva in PBC; the initiation, cost,
timing, progress and results of Intercept's development activities,
preclinical studies and clinical trials, including Intercept's
development program in NASH; the timing of and Intercept's ability
to obtain and maintain regulatory approval of OCA in PBC in
countries outside the United States and in indications other than
PBC and any other product candidates it may develop such as
INT-767; conditions that may be imposed by regulatory authorities
on Intercept's marketing approvals for its product candidates such
as the need for clinical outcomes data (and not just results based
on achievement of a surrogate endpoint), 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; Intercept's ability to
obtain and maintain intellectual property protection for its
product candidates; Intercept's ability to successfully
commercialize OCA in indications other than PBC and its other
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 of
Intercept's products, which may be affected by the reimbursement
that it may receive for its products from payors; the success of
competing drugs that are or become available; 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; 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 use of cash, short-term investments and the proceeds
from the offering; Intercept's ability to attract and 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, 2015 filed on February
29, 2016 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.
Contact
For more information about Intercept Pharmaceuticals, please contact:
Mark Vignola
+1-646-747-1000
investors@interceptpharma.com
Christopher Frates
+1-646-757-2371
media@interceptpharma.com