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 it has received a
2017 Industry Innovation Award from the National Organization for
Rare Disorders (NORD) for the development of Ocaliva (obeticholic
acid).
This award recognizes Intercept’s commitment to the rare disease
community and the impact Ocaliva has made in the lives of patients
with primary biliary cholangitis (PBC), a rare, autoimmune
cholestatic liver disease that puts patients at risk for
life-threatening complications. David Shapiro, M.D., Intercept's
Chief Medical Officer, accepted the award on behalf of
Intercept.
“We are honored to be a recipient of the 2017 Industry
Innovation Award for the development of Ocaliva, the first
medication approved for patients with PBC in nearly 20 years,” said
Dr. Shapiro. “We’d like to thank the PBC community for their
tremendous efforts supporting innovative research and for being an
invaluable partner to Intercept as we have worked to bring Ocaliva
to patients in need.”
Ocaliva is indicated in the United States and the European Union
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. Intercept is also
evaluating obeticholic acid for the treatment of patients with
primary sclerosing cholangitis (PSC) and biliary atresia, two rare
liver diseases that currently have no approved medications.
“We are proud to present the team at Intercept with a 2017
Industry Innovation Award for the important work they have done to
address the serious unmet need in PBC,” said Peter L. Saltonstall,
NORD’s President and CEO. “We hope the research programs evaluating
obeticholic acid for the treatment of patients with other rare
liver diseases lead to further advances for the rare disease
community.”
About NORDThe National Organization for Rare
Disorders (NORD)® is the leading independent advocacy organization
representing all patients and families affected by rare
diseases. NORD is committed to the identification, treatment
and cure of the 7,000 rare diseases that affect 30 million
Americans, or 1 in every 10 people. NORD began as a small
group of patient advocates that formed a coalition to unify and
mobilize support to pass the Orphan Drug Act of 1983. For more than
30 years, NORD has led the way in voicing the needs of the rare
disease community, driving supportive policies and education,
advancing medical research, and providing patient and family
services for those who need them most. NORD represents more
than 260 disease-specific member organizations and their
communities and collaborates with many other organizations in
specific causes of importance to the rare disease patient
community. www.rarediseases.org.
The Rare Impact Awards serves as a NORD’s signature fundraiser
to support the organization’s mission of providing education,
advocacy, research, and patient support for the 1 in 10 Americans
who are battling rare diseases. More than 350 distinguished guests
are expected to attend.
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 is indicated in the
United States 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.
This indication is approved under accelerated approval based on
a reduction in alkaline phosphatase (ALP), as a surrogate endpoint
which is reasonably likely to predict clinical benefit, including
an improvement in liver transplant free-survival. 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. Intercept is currently enrolling COBALT, a Phase 4 clinical
outcomes trial of Ocaliva in patients with PBC with the goal of
confirming clinical benefit on a post-marketing basis.
In December 2016, Ocaliva received conditional marketing
authorization in the European Union for the treatment of PBC in
combination with 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. 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.
U.S. Important Safety
InformationContraindications
Ocaliva is contraindicated in patients with complete biliary
obstruction.
Warnings and PrecautionsLiver-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 (HDLC). 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
(≥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 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 the U.S. Full Prescribing Information for Ocaliva
(obeticholic acid) 5 mg and 10 mg tablets.
About InterceptIntercept 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. Intercept’s
International headquarters are located in London. 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 activities anticipated to
be undertaken by Intercept regarding Ocaliva® in PBC; the potential
approval of OCA in PBC by regulatory bodies outside of the United
States and the European Union and the timelines related thereto;
the initiation, enrollment, conduct and completion of clinical
trials and the timelines related thereto; the anticipated
regulatory process and timetable with respect to OCA and
Intercept's product candidates; the continued development of OCA
and Intercept's other product candidates in diseases such as
primary sclerosing cholangitis and biliary atresia; and Intercept's
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 in jurisdictions in
which Ocaliva is approved for use 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 ones in which it is approved 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
products and 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 products and 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 products and
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 products and 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, 2016 filed on
March 1, 2017 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
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