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2016-10-14

Intercept Pharmaceuticals, Inc.: Intercept Pharmaceuticals Receives Positive CHMP Opinion for Ocaliva® (Obeticholic Acid) for the...

Intercept Pharmaceuticals Receives Positive CHMP Opinion for Ocaliva® (Obeticholic Acid) for the Treatment of Primary Biliary Cholangitis in the European UnionNEW YORK, Oct. 14, 2016 (GLOBE NEWSWIRE) -- Intercept Pharmaceuticals, Inc.
(Nasdaq:ICPT) (Intercept), a biopharmaceutical company focused on the
development and commercialization of novel therapeutics to treat non-viral,
progressive liver diseases, today announced that the European Medicines
Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted
a positive opinion recommending marketing authorization of the Company's
Marketing Authorization Application (MAA) for obeticholic acid (OCA), an FXR
agonist, for the treatment of primary biliary cholangitis (PBC) conditional
to the company providing further data post-approval to confirm benefit.

Ursodeoxycholic acid (UDCA) is currently the only approved medication for the
treatment of PBC in Europe and is the standard of care for all PBC patients.
However, a substantial percentage of patients treated with UDCA continue to
experience persistent elevations above the upper limit of normal in the serum
marker alkaline phosphatase (ALP), which has been shown to correspond with
increased risk of liver failure, need for liver transplant and death.
Patients with PBC also face a risk of experiencing adverse outcomes when
bilirubin levels are elevated. Total bilirubin levels, even within the normal
range, have been shown to predict clinical outcomes in PBC.

"Although it is a rare disease, PBC remains one of the most common indications
for liver transplant among women in Europe," said David Jones, M.D., Ph.D.,
Professor of Liver Immunology at Newcastle University and Consultant
Hepatologist at Newcastle upon Tyne Hospitals Trust, which hosts one of
Europe's leading clinical services in the disease. "There is substantial
unmet need in this disease and a real urgency around the need for new
therapies to help the many PBC patients who are either intolerant of the
single existing approved therapy ursodeoxycholic acid or don't respond to it
sufficiently to protect their livers and prevent the development of cirrhosis
and the need for transplant."

The MAA submission included data from more than 1,500 subjects exposed to at
least a single dose of OCA. The positive opinion of the CHMP was based on
efficacy and safety data derived from three randomized double-blind,
placebo-controlled clinical trials in patients with PBC evaluating the effect
of OCA on ALP and bilirubin. The MAA submission 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.

The CHMP opinion will form the basis for a European Commission (EC) decision
as to whether to formally grant the conditional marketing authorization for
OCA with unified labelling in the 28 countries that are Member States of the
European Union, as well as European Economic Area members Iceland,
Liechtenstein and Norway. As the conditions for approval, Intercept is
required to provide post-approval updates on safety and efficacy analyses for
OCA from the ongoing COBALT outcomes trial and a short-term trial in patients
with hepatic impairment.

"We owe a tremendous debt to the many patients and physicians whose
participation in the research program for OCA led to this positive outcome,"
said Lisa Bright, Intercept's President, International. "In addition to
playing a critical role in the development of OCA, the PBC community in
Europe has been the driving force in establishing the two major patient
databases that have been so central to recent advances in our understanding
of the disease. There is a palpable sense of excitement about the growth of
PBC awareness in Europe, and the CHMP's positive opinion on OCA brings us one
step closer to introducing the first new therapy for PBC in approximately two
decades."

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 Obeticholic Acid (OCA)
Obeticholic acid is an 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.

May 2016, the U.S. Food and Drug Administration (FDA) granted accelerated
approval to obeticholic acid for the treatment of PBC under the brand name
Ocaliva® based on a reduction in ALP. An improvement in survival or
disease-related symptoms has not been established. Continued approval for
this indication may be contingent upon verification and description of
clinical benefit in confirmatory trials. The brand name Ocaliva has been
provisionally approved by the EMA.

U.S. IMPORTANT SAFETY INFORMATION

Contraindications
Ocaliva is contraindicated in patients with complete biliary obstruction.

Warnings and Precautions

Liver-Related Adverse Reactions
In two 3-month, placebo-controlled clinical trials, a dose-response
relationship was observed for the occurrence of liver-related adverse
reactions including jaundice, ascites and primary biliary cholangitis flare
with dosages of Ocaliva of 10 mg once daily to 50 mg once daily (up to
5-times the highest recommended dosage), as early as one month after starting
treatment with Ocaliva.

In a pooled analysis of three placebo-controlled trials in patients with PBC,
the exposure-adjusted incidence rates for all serious and otherwise
clinically significant liver-related adverse reactions, and isolated
elevations in liver biochemical tests, per 100 patient exposure years (PEY)
were: 5.2 in the Ocaliva 10 mg group (highest recommended dosage), 19.8 in
the Ocaliva 25 mg group (2.5 times the highest recommended dosage) and 54.5
in the Ocaliva 50 mg group (5 times the highest recommended dosage) compared
to 2.4 in the placebo group.

Monitor patients during treatment with Ocaliva for elevations in liver
biochemical tests and for the development of liver-related adverse reactions.
Weigh the potential risks against the benefits of continuing treatment with
Ocaliva in patients who have experienced clinically significant liver-related
adverse reactions. The maximum recommended dosage of Ocaliva is 10 mg once
daily. Adjust the dosage for patients with moderate or severe hepatic
impairment.

Discontinue Ocaliva in patients who develop complete biliary obstruction.

Severe Pruritus
Severe pruritus was reported in 23% of patients in the Ocaliva 10 mg arm, 19%
of patients in the Ocaliva titration arm and 7% of patients in the placebo
arm in the POISE trial, a 12-month double-blind randomized controlled trial
of 216 patients. Severe pruritus was defined as intense or widespread
itching, interfering with activities of daily living, or causing severe sleep
disturbance, or intolerable discomfort, and typically requiring medical
interventions. In the subgroup of patients in the Ocaliva titration arm who
increased their dosage from 5 mg once daily to 10 mg once daily after 6
months of treatment (n=33), the incidence of severe pruritus was 0% from
months 0 to 6 and 15% from months 6 to 12. 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 resins or
antihistamines, Ocaliva dosage reduction and/or temporary interruption of
Ocaliva dosing.

Reduction in HDL-C
Patients with PBC generally exhibit hyperlipidemia characterized by a
significant elevation in total cholesterol primarily due to increased levels
of high density lipoprotein-cholesterol (HDL-C). In the POISE trial,
dose-dependent reductions from baseline in mean HDL-C levels were observed at
2 weeks in Ocaliva-treated patients, 20% and 9% in the 10 mg and titration
arms, respectively, compared to 2% in the placebo arm. At month 12, the
reduction from baseline in mean HDL-C level was 19% in the Ocaliva 10 mg arm,
12% in the Ocaliva titration arm and 2% in the placebo arm. Nine patients in
the Ocaliva 10 mg arm and six patients in the Ocaliva titration arm, versus
three patients in the placebo arm, had reductions in HDL-C to less than 40
mg/dL.

Monitor patients for changes in serum lipid levels during treatment. For
patients who do not respond to Ocaliva after one year at the highest
recommended dosage that can be tolerated (maximum of 10 mg once daily), and
who experience a reduction in HDL-C, weigh the potential risks against the
benefits of continuing treatment.

Adverse Reactions
The most common adverse reactions from subjects taking Ocaliva (greater than
or equal to 5%) were pruritus, fatigue, abdominal pain and discomfort, rash,
oropharyngeal pain, dizziness, constipation, arthralgia, thyroid function
abnormality and eczema.

Drug Interaction

Bile Acid Binding Resins
Bile acid binding resins such as cholestyramine, colestipol or colesevelam
absorb and reduce bile acid absorption and may reduce the absorption,
systemic exposure and efficacy of Ocaliva. If taking bile acid binding
resins, take Ocaliva at least 4 hours before or 4 hours after (or at as great
an interval as possible) taking a bile acid binding resin.

Please see Full Prescribing Information for Ocaliva (obeticholic acid) 5 mg
and 10 mg tablets.

About Intercept

Intercept is a biopharmaceutical company focused on the development and
commercialization of novel therapeutics to treat non-viral, progressive 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 the European Commission and other regulatory bodies
and the timelines related thereto, the availability of OCA for the treatment
of PBC in Europe and other juri...

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