Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT), a biopharmaceutical
company focused on the development and commercialization of novel
therapeutics to treat progressive non-viral liver diseases, today
announced that multiple abstracts regarding the treatment of
primary biliary cholangitis (PBC) and nonalcoholic steatohepatitis
(NASH) with OCA will be presented at the Digital International
Liver Congress™ 2020, the 55th Annual Meeting of the European
Association for the Study of the Liver (EASL), to be held virtually
from August 27, 2020 to August 29, 2020.
“The new data to be presented at this year’s International Liver
Congress add to the already substantial body of evidence supporting
the anti-fibrotic efficacy of OCA in patients with advanced
fibrosis due to NASH,” said Mark Pruzanski, M.D., President and CEO
of Intercept. “We are also excited to see that several Ocaliva® PBC
abstracts that deepen the understanding of our drug’s long-term
safety and efficacy profile will be presented, including one
presenting the six-year data from the open label extension of our
Phase 3 POISE trial. On behalf of all my colleagues at Intercept,
I’d like to thank EASL for providing this virtual forum for
important new hepatology research and scientific exchange during
the COVID-19 pandemic.”
Presentations at the Digital International Liver Congress
include:
Late-Breaker Poster Presentation
“Obeticholic acid demonstrates sustained improvements at
month 24 in transaminases and non-invasive markers of fibrosis:
results of a post hoc analysis from the interim analysis of the
REGENERATE study” (LBP19)Rohit Loomba, Vlad Ratziu,
Quentin M. Anstee, Stephen Harrison, Arun Sanyal, Mary Rinella,
Zobair Younossi, Zachary Goodman, Pierre Bedossa, Reshma
Shringarpure, Huafeng Zhou, Aditya Venugopal, Mazen Noureddin
Oral Presentation
“Obeticholic acid (OCA) improves experimental
non-invasive markers of NASH and advanced fibrosis: results of a
secondary analysis from the month-18 interim analysis of the
REGENERATE study”
(AS075)Jerome Boursier,
Rohit Loomba, Quentin M. Anstee, Stephen Harrison, Arun Sanyal,
Mary Rinella, Zobair Younossi, Zachary Goodman, Pierre Bedossa,
Céline Fournier, Michael Stenkilsson, Reshma Shringarpure, Luna
Zaru, Aditya Venugopal, Leigh MacConell, Vlad Ratziu
General Poster Presentations
“The burden of disease associated with non-alcoholic
steatohepatitis patients under standard of care”
(THU048)Raluca Pais,
William Green, Stuart Mealing, Aldo Trylesinski, Sandrine Cure,
Heather Davies
“Predicted risk of end stage liver disease utilizing the
UK-PBC risk score with continued standard of care and subsequent
addition of obeticholic acid for 60 Months in patients with primary
biliary cholangitis”
(THU114)David E. Jones,
Marco Carbone, George Mells, Alexander Liberman, Elizabeth Smoot
Malecha, Leigh MacConell
“Noninvasive tests for assessing fibrosis in patients
with non-alcoholic fatty liver disease: an evaluation of combining
test results”
(FRI009)Catherine Vick,
Andrew Joyce, Amy Law, Molly Sherwood, Essy Mozaffari, Bruce
Wong
“Obeticholic acid improves hepatic fibroinflammation as
assessed by multiparametric magnetic resonance imaging: interim
results of the REGENERATE trial”
(FRI066)Rohit Loomba,
Quentin M. Anstee, Stephen Harrison, Arun Sanyal, Vlad Ratziu,
Zobair Younossi, Zachary Goodman, Pierre Bedossa, Rajarshi
Banerjee, Michael Stenkilsson, Reshma Shringarpure, Luna Zaru,
Aditya Venugopal, Leigh MacConell, Mary Rinella
“Obesity-specific health-related quality of life in
patients with non-alcoholic steatohepatitis: results from the
REGENERATE study”
(FRI080)Zobair Younossi,
Maria Stepanova, Fatema Nader, Rohit Loomba, Quentin M. Anstee,
Vlad Ratziu, Stephen Harrison, Arun Sanyal, Jacob George, Susanne
Beckebaum, David Orr, Giuseppe Mazzella, Victor Vargas, Lise Lotte
Gluud, Rifaat Safadi, James Trotter, Jaideep Behari, David
Sheridan, Muhammad Y. Sheikh, Martin Bonacci, Gail Cawkwell, Bruce
Wong, Pierre Bedossa, Zachary Goodman, Mary Rinella, on behalf of
the REGENERATE Study Investigators
“Durability of biochemical improvements through six
years of open label treatment with obeticholic acid in patients
with primary biliary cholangitis
who did not achieve the POISE criteria”
(FRI146)Gideon M.
Hirschfield, Marco Carbone, David E. Jones, Bettina E. Hansen,
Andreas E. Kremer, Michael Trauner, Alexander Liberman, Elizabeth
Smoot Malecha, Leigh MacConell
“Efficacy and tolerance of obeticholic acid in
patients with primary biliary cholangitis and inadequate response
to ursodeoxycholic acid in real life: interim analysis of the
OCARELIFE study”
(FRI180)Vincent Leroy,
Christophe Corpechot, Jérôme Dumortier, Laurent Alric, Dominique
Larrey, Sébastien Dharancy, Olivier Chazouilleres, Alexandra
Heurgue, François Boer, Aldo Trylesinski
A full list of sessions at the Digital International Liver
Congress™ 2020 is available at https://ilc-congress.eu.
About Liver Fibrosis due to NASH
Nonalcoholic steatohepatitis (NASH) is a serious progressive
liver disease caused by excessive fat accumulation in the liver
that induces chronic inflammation, resulting in progressive
fibrosis (scarring) that can lead to cirrhosis, eventual liver
failure, cancer and death. Advanced fibrosis is associated with a
substantially higher risk of liver-related morbidity and mortality
in patients with NASH. In the United States, NASH is currently the
second leading cause for liver transplantation overall, and in
females, the leading cause. NASH is anticipated to become the
leading indication for liver transplantation in Europe within the
next decade. There are currently no medications approved for the
treatment of NASH.
About the REGENERATE Study
REGENERATE is a Phase 3, randomized, double-blind,
placebo-controlled, multicenter study assessing the safety and
efficacy of obeticholic acid (OCA) on clinical outcomes in patients
with liver fibrosis due to NASH. A pre-specified 18-month analysis
was conducted to assess the effect of OCA on liver histology
comparing month 18 biopsies with baseline. REGENERATE has completed
target enrollment for the clinical outcomes cohort, with 2,480
adult NASH patients randomized over 300 qualified centers
worldwide, and is expected to continue through clinical outcomes
for verification and description of clinical benefit. The
end-of-study analysis is designed to evaluate the effect of OCA on
all-cause mortality and liver-related clinical outcomes, as well as
its long-term safety.
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) and nonalcoholic steatohepatitis (NASH). Founded
in 2002 in New York, Intercept has operations in the
United States, Europe and Canada. For more
information, please visit www.interceptpharma.com or
connect with the company
on Twitter and LinkedIn.
About Primary Biliary Cholangitis
Primary biliary cholangitis (PBC) is a chronic, progressive
liver disorder that mostly affects 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 the accelerated approval
pathway 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. We are conducting a Phase
4 clinical outcomes trial, which we refer to as our COBALT trial,
of OCA 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 Europe 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, conditioned
upon us providing further data post-approval to confirm benefit.
For detailed safety information for Ocaliva 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 INFORMATION FOR OCALIVA IN
PBC
WARNING: HEPATIC DECOMPENSATION AND FAILURE IN
INCORRECTLY DOSED PBC PATIENTS WITH CHILD-PUGH CLASS B OR C
OR DECOMPENSATED CIRRHOSIS
- In postmarketing reports, hepatic decompensation and
failure, in some cases fatal, have been reported in patients with
Primary Biliary Cholangitis (PBC) with decompensated cirrhosis or
Child-Pugh Class B or C hepatic impairment when OCALIVA was
dosed more frequently than recommended.
- The recommended starting dosage of OCALIVA is 5 mg once
weekly for patients with Child-Pugh Class B or C hepatic
impairment or a prior decompensation event.
Contraindications
OCALIVA is contraindicated in PBC patients with complete biliary
obstruction.
Warnings and Precautions
Hepatic Decompensation and Failure in Incorrectly-Dosed
PBC Patients with Child-Pugh Class B or C or Decompensated
Cirrhosis
In postmarketing reports, hepatic decompensation and failure, in
some cases fatal, have been reported in PBC patients with
decompensated cirrhosis or Child-Pugh B or C hepatic impairment
when OCALIVA was dosed more frequently than the recommended
starting dosage of 5 mg once weekly. Reported cases typically
occurred within 2 to 5 weeks after starting OCALIVA and were
characterized by an acute increase in total bilirubin and/or ALP
concentrations in association with clinical signs and symptoms of
hepatic decompensation (e.g., ascites, jaundice, gastrointestinal
bleeding, worsening of hepatic encephalopathy).
Routinely monitor patients for progression of PBC disease,
including liver-related complications, with laboratory and clinical
assessments. Dosage adjustment, interruption or discontinuation may
be required. Close monitoring is recommended for patients at an
increased risk of hepatic decompensation. Severe intercurrent
illnesses that may worsen renal function or cause dehydration
(e.g., gastroenteritis), may exacerbate the risk of hepatic
decompensation. Interrupt treatment with OCALIVA in patients with
laboratory or clinical evidence of worsening liver function
indicating risk of decompensation, and monitor the patient’s liver
function. Consider discontinuing OCALIVA in patients who have
experienced clinically significant liver-related adverse reactions.
Discontinue OCALIVA in patients who develop complete biliary
obstruction.
Liver-Related Adverse Reactions
Dose-related, liver-related adverse reactions including
jaundice, worsening ascites and primary biliary cholangitis flare
have been observed in clinical trials, as early as one month after
starting treatment with OCALIVA 10 mg once daily up to 50 mg once
daily (up to 5-times the highest recommended dosage). Monitor PBC
patients during treatment with OCALIVA for elevations in liver
biochemical tests and for the development of liver-related adverse
reactions.
Severe Pruritus
Severe pruritus was reported in 23% of PBC patients in the
OCALIVA 10 mg arm, 19% of PBC patients in the OCALIVA titration
arm, and 7% of PBC patients in the placebo arm in a 12-month
double-blind randomized controlled trial of 216 PBC 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. Consider clinical evaluation of
PBC patients with new onset or worsening severe pruritus.
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).
Dose-dependent reductions from baseline in mean HDL-C levels were
observed at 2 weeks in OCALIVA-treated PBC patients, 20% and 9% in
the 10 mg and titration arms, respectively, compared to 2% in the
placebo arm. Monitor PBC patients for changes in serum lipid levels
during treatment. For PBC patients who do not respond to OCALIVA
after 1 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
for PBC were pruritus, fatigue, abdominal pain and discomfort,
rash, oropharyngeal pain, dizziness, constipation, arthralgia,
thyroid function abnormality, and eczema.
Drug Interactions
Bile Acid Binding Resins
Bile acid binding resins such as cholestyramine, colestipol, or
colesevelam adsorb and reduce bile acid absorption and may reduce
the absorption, systemic exposure, and efficacy of OCALIVA. If
taking a bile acid binding resin, take OCALIVA at least 4 hours
before or 4 hours after taking the bile acid binding resin, or at
as great an interval as possible.
Warfarin
The International Normalized Ratio (INR) decreased following
coadministration of warfarin and OCALIVA. Monitor INR and adjust
the dose of warfarin, as needed, to maintain the target INR range
when coadministering OCALIVA and warfarin.
CYP1A2 Substrates with Narrow Therapeutic
Index
Obeticholic acid, the active ingredient in OCALIVA, may increase
the exposure to concomitant drugs that are CYP1A2 substrates.
Therapeutic monitoring of CYP1A2 substrates with a narrow
therapeutic index (e.g. theophylline and tizanidine) is recommended
when coadministered with OCALIVA.
Inhibitors of Bile Salt Efflux Pump
Avoid concomitant use of inhibitors of the bile salt efflux pump
(BSEP) such as cyclosporine. Concomitant medications that inhibit
canalicular membrane bile acid transporters such as the BSEP may
exacerbate accumulation of conjugated bile salts including taurine
conjugate of obeticholic acid in the liver and result in clinical
symptoms. If concomitant use is deemed necessary, monitor
serum transaminases and bilirubin.
Please see Full Prescribing Information, including Boxed
WARNING and Medication Guide for OCALIVA.
To report SUSPECTED ADVERSE REACTIONS,
contact Intercept Pharmaceuticals, Inc. at 1-844-782-ICPT
or FDA at 1-800-FDA-1088
or www.fda.gov/medwatch.
Cautionary Note Regarding Forward-Looking
Statements
This press release contains forward-looking statements,
including, but not limited to, statements regarding the progress,
timing and results of our clinical trials, including our clinical
trials for the treatment of nonalcoholic steatohepatitis (“NASH”),
the safety and efficacy of our approved product, Ocaliva
(obeticholic acid or “OCA”) for primary biliary cholangitis
(“PBC”), and our product candidates, including OCA for liver
fibrosis due to NASH, the timing and acceptance of our regulatory
filings and the potential approval of OCA for liver fibrosis due to
NASH, the review of our New Drug Application for OCA for the
treatment of liver fibrosis due to NASH by the U.S. Food and Drug
Administration (FDA), our intent to work with the FDA to address
the issues raised in the complete response letter (CRL), the
potential commercial success of OCA, as well as our strategy,
future operations, future financial position, future revenue,
projected costs, financial guidance, prospects, plans and
objectives.
These statements constitute forward-looking statements within
the meaning of Section 27A of the Securities Act of 1933, as
amended, and Section 21E of the Securities Exchange Act of 1934, as
amended. The words “anticipate,” “believe,” “estimate,” “expect,”
“intend,” “may,” “plan,” “predict,” “project,” “target,”
“potential,” “will,” “would,” “could,” “should,” “possible,”
“continue” and similar expressions are intended to identify
forward-looking statements, although not all forward-looking
statements contain these identifying words. Readers are cautioned
not to place undue reliance on these forward-looking statements,
which speak only as of the date of this release, and we undertake
no obligation to update any forward-looking statement except as
required by law. These forward-looking statements are based on
estimates and assumptions by our management that, although believed
to be reasonable, are inherently uncertain and subject to a number
of risks. The following represent some, but not necessarily all, of
the factors that could cause actual results to differ materially
from historical results or those anticipated or predicted by our
forward-looking statements: our ability to successfully
commercialize Ocaliva for PBC; our ability to maintain our
regulatory approval of Ocaliva for PBC in the United
States, Europe, Canada, Israel, Australia and
other jurisdictions in which we have or may receive marketing
authorization; our ability to timely and cost-effectively file for
and obtain regulatory approval of our product candidates on an
accelerated basis or at all, including OCA for liver fibrosis due
to NASH following the issuance of the CRL by the FDA; any advisory
committee recommendation or dispute resolution determination
that our product candidates, including OCA for liver fibrosis due
to NASH, should not be approved or approved only under certain
conditions; any future determination that the regulatory
applications and subsequent information we submit for our
product candidates, including OCA for liver fibrosis due to NASH,
do not contain adequate clinical or other data or meet applicable
regulatory requirements for approval; conditions that may be
imposed by regulatory authorities on our marketing approvals for
our products and product candidates, including OCA for liver
fibrosis due to NASH, such as the need for clinical outcomes data
(and not just results based on achievement of a surrogate
endpoint), any risk mitigation programs such as a REMS, and any
related restrictions, limitations and/or warnings contained in the
label of any of our products or product candidates; any potential
side effects associated with Ocaliva for PBC, OCA for liver
fibrosis due to NASH or our other product candidates that could
delay or prevent approval, require that an approved product be
taken off the market, require the inclusion of safety warnings or
precautions, or otherwise limit the sale of such product or product
candidate; the initiation, timing, cost, conduct, progress and
results of our research and development activities, preclinical
studies and clinical trials, including any issues, delays or
failures in identifying patients, enrolling patients, treating
patients, retaining patients, meeting specific endpoints in the
jurisdictions in which we intend to seek approval or completing and
timely reporting the results of our NASH or PBC clinical trials;
our ability to establish and maintain relationships with, and the
performance of, third-party manufacturers, contract research
organizations and other vendors upon whom we are substantially
dependent for, among other things, the manufacture and supply of
our products, including Ocaliva for PBC and, if approved, OCA for
liver fibrosis due to NASH, and our clinical trial activities; our
ability to identify, develop and successfully commercialize our
products and product candidates, including our ability to
successfully launch OCA for liver fibrosis due to NASH, if
approved; our ability to obtain and maintain intellectual property
protection for our products and product candidates, including our
ability to cost-effectively file, prosecute, defend and enforce any
patent claims or other intellectual property rights; the size and
growth of the markets for our products and product candidates and
our ability to serve those markets; the degree of market acceptance
of Ocaliva for PBC and, if approved, OCA for liver fibrosis due to
NASH or our other product candidates among physicians, patients and
healthcare payors; the availability of adequate coverage and
reimbursement from governmental and private healthcare payors for
our products, including Ocaliva for PBC and, if approved, OCA for
liver fibrosis due to NASH, and our ability to obtain adequate
pricing for such products; our ability to establish and maintain
effective sales, marketing and distribution capabilities, either
directly or through collaborations with third parties; competition
from existing drugs or new drugs that become available; our ability
to prevent system failures, data breaches or violations of data
protection laws; costs and outcomes relating to any disputes,
governmental inquiries or investigations, regulatory proceedings,
legal proceedings or litigation, including any securities,
intellectual property, employment, product liability or other
litigation; our collaborators’ election to pursue research,
development and commercialization activities; our ability to
establish and maintain relationships with collaborators with
development, regulatory and commercialization expertise; our need
for and ability to generate or obtain additional financing; our
estimates regarding future expenses, revenues and capital
requirements and the accuracy thereof; our use of cash and
short-term investments; our ability to acquire, license and invest
in businesses, technologies, product candidates and products; our
ability to attract and retain key personnel to manage our business
effectively; our ability to manage the growth of our operations,
infrastructure, personnel, systems and controls; our ability to
obtain and maintain adequate insurance coverage; the impact of
COVID-19, including any impact on our results of operations or
financial position, related quarantines and government actions,
delays relating to our regulatory applications, disruptions
relating to our ongoing clinical trials or involving our contract
research organizations, study sites or other clinical partners,
disruptions relating to our supply chain or involving our
third-party manufacturers, distributors or other distribution
partners, facility closures or other restrictions, and the extent
and duration thereof; the impact of general U.S. and
foreign economic, industry, market, regulatory or political
conditions, including the potential impact of Brexit; and the other
risks and uncertainties identified in our periodic filings filed
with the U.S. Securities and Exchange Commission, including
our Annual Report on Form 10-K for the year ended December 31,
2019 and our Quarterly Report on Form 10-Q for the quarter ended
June 30, 2020.
Contact
For more information about Intercept, please contact:
Lisa DeFrancesco+1-646-565-4833investors@interceptpharma.com
Christopher Frates+1-646-757-2371media@interceptpharma.com
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