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2016-12-14

Intercept Pharmaceuticals, Inc.: European Commission Grants Intercept's Ocaliva® (obeticholic acid) Marketing Authorization for the Treatment of Primary Biliar

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 can...

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