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 obeticholic acid (OCA) will be presented at the Digital
International Liver Congress™ 2021, the 56th Annual Meeting of the
European Association for the Study of the Liver (EASL), to be held
virtually from June 23 to June 26, 2021.
“The REGENERATE data that Intercept will be presenting at this
year’s International Liver Congress provide important new insight
into short-term and longer-term measures of OCA treatment response
in patients with fibrosis due to NASH,” said Gail Cawkwell, M.D.,
Ph.D., Intercept’s Senior Vice President, Medical Affairs, Safety
& Pharmacovigilance. “Specifically, the early reductions in
aminotransferases seen with OCA therapy show a correlation with
fibrosis improvement over time. This is relevant because the early
conversations between a clinician and patient about improvements in
routine laboratory measures like ALT in the period after initiating
therapy can help set expectations for longer term benefit.
“We are also pleased to be presenting new real-world data in PBC
at this year’s Congress,” continued Dr. Cawkwell. “Using a large
administrative claims database, we examined biochemical response in
more than 300 patients treated with Ocaliva who had varying levels
of underlying disease risk at baseline. Across groups, Ocaliva
produced marked reductions in alkaline phosphatase and bilirubin
after both one year and two years of treatment. It is reassuring to
see that the benefits of Ocaliva from the controlled environment of
clinical trials are largely consistent with these results seen with
real-world use."
The use of OCA for NASH is investigational and has not been
approved by the U.S. Food and Drug Administration, the European
Commission or any other health authority.
In addition to new analyses of OCA data, the International Liver
Congress 2021 will also include research from Intercept and
collaborators that provides new perspective on the natural history
of PBC and fibrosis due to NASH, data supporting the role of
noninvasive tests to risk-stratify patients with fibrosis due to
NASH, and results from a multi-country survey examining
patient-provider dialogue and perceptions of stigma associated with
fibrosis due to NASH.
Presentations at the Digital International Liver Congress
include:
Oral Presentation
“Incidence rates of select outcomes among patients with
non-alcoholic steatohepatitis (NASH) and evidence of fibrosis or
cirrhosis” (Abstract #1780)Monica L. Bertoia, Erik Ness,
Thomas Capozza, Lina Titievsky, John D. Seeger
Fibrosis due to NASH Poster Presentations
“Aminotransferase level improvement in patients with
non-alcoholic steatohepatitis are associated with fibrosis
regression in the REGENERATE study” (Abstract #2290)Mary
Rinella, Jean-Francois Dufour, Quentin M. Anstee, Zobair Younossi,
Rohit Loomba, Arun J. Sanyal, Tom Capozza, Tanya Granston, Martin
Bonacci, Aldo Trylesinki, Vlad Ratziu
“Incidence rates of hepatobiliary outcomes among
patients with non-alcoholic steatohepatitis based on fibrosis-4
score severity at baseline” (Abstract
#980)Lina Titievsky, Aziza Jamal-Allial, Kerrin Gallagher,
Tom Capozza, Stephen Dodge, Simo Du, Amy Law, Macky Natha, Erik
Ness, Mindie H. Nguyen, Yuval A. Patel, Amarita Randhawa, Daina B.
Esposito
“Comorbidity and healthcare utilization burden of
patients diagnosed with non-alcoholic steatohepatitis based on
fibrosis-4 score severity” (Abstract #1588)Lina Titievsky,
Aziza Jamal-Allial, Kerrin Gallagher, Thomas Capozza, Stephen
Dodge, Simo Du, Amy Law, Macky Natha, Erik Ness, Mindie H. Nguyen,
Yuval A. Patel, Amarita Randhawa, Daina B. Esposito
“The unmet needs of living with NASH in Europe and
Canada: results from a multicountry survey” (Abstract
#1990)Jeffrey V. Lazarus, Fabienne Marcellin, Camelia
Protopopescu, Aldo Trylesinski, José Luis Calleja, Patrizia
Carrieri
“Disclosure of NASH Diagnosis and fear of
discrimination: results from a multicountry
survey”(Abstract #2032) Jeffrey V
Lazarus, Fabienne Marcellin, Achim Kautz, Vanessa Heditch, Aldo
Trylesinski, José Luis Calleja, Patrizia Carrieri
“NASH Dynamics – The prognosis is impaired in NAFLD
patients with diabetes despite negative Fib4 <1.30” (Abstract
#313) Jerome Boursier, Hannes Hagstrom, Mattias Ekstedt,
Clemence Moreau, Martin Bonacci, Sandrine Cure, Aldo Trylesinski,
Manuel Romero-Gomez
PBC Poster Presentations
“Incidence of hepatic outcomes in patients with
cirrhosis due to primary biliary cholangitis (PBC): A
population-based epidemiology study” (Abstract #1664)Lina
Titievsky, Erik Ness, Amy Law, Ellie Goldman, Darren Wheeler,
Monica Bertoia, John Seeger, Mindie Nguyen, Yuval Patel, Femi
Adekunle, Chiara Bassanelli
“Effectiveness of obeticholic acid in patients with
primary biliary cholangitis stratified by biochemical marker status
in the real-world setting in the United States” (Abstract
#254)Robert G. Gish, Darren Wheeler, Jay Lin, Femi
Adekunle, Melissa Lingohr-Smith, Chiara Bassanelli, Amy Law
A full list of sessions at the Digital International Liver
Congress™ 2021 is available at https://ilc-congress.eu.
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 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 at 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 will evaluate the effect of OCA on all-cause
mortality and liver-related clinical outcomes, as well as long-term
safety.
About Ocaliva® (obeticholic
acid)
OCALIVA, a farnesoid X receptor (FXR) agonist,
is indicated for the treatment of adult patients with primary
biliary cholangitis (PBC)
- without cirrhosis or
- with compensated cirrhosis who do
not have evidence of portal hypertension,
either in combination with ursodeoxycholic acid
(UDCA) with an inadequate response to UDCA or as monotherapy in
patients unable to tolerate UDCA.
This indication is approved under accelerated
approval based on a reduction in alkaline phosphatase (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.
IMPORTANT SAFETY INFORMATION
WARNING: HEPATIC DECOMPENSATION AND
FAILURE IN PRIMARY BILIARY CHOLANGITIS PATIENTS WITH
CIRRHOSIS
- Hepatic decompensation and
failure, sometimes fatal or resulting in liver transplant, have
been reported with OCALIVA treatment in primary biliary cholangitis
(PBC) patients with either compensated or decompensated
cirrhosis.
- OCALIVA is contraindicated
in PBC patients with decompensated cirrhosis, a prior
decompensation event, or with compensated cirrhosis who have
evidence of portal hypertension.
- Permanently discontinue
OCALIVA in patients who develop laboratory or clinical evidence of
hepatic decompensation; have compensated cirrhosis and develop
evidence of portal hypertension, or experience clinically
significant hepatic adverse reactions while on
treatment.
Contraindications
OCALIVA is contraindicated in patients with:
- decompensated cirrhosis (e.g., Child-Pugh Class B or C) or a
prior decompensation event
- compensated cirrhosis who have evidence of portal hypertension
(e.g., ascites, gastroesophageal varices, persistent
thrombocytopenia)
- complete biliary obstruction
Warnings and Precautions
Hepatic Decompensation and Failure in PBC Patients with
Cirrhosis
Hepatic decompensation and failure, sometimes
fatal or resulting in liver transplant, have been reported with
OCALIVA treatment in PBC patients with cirrhosis, either
compensated or decompensated. Among postmarketing cases reporting
it, median time to hepatic decompensation (e.g. new onset ascites)
was 4 months for patients with compensated cirrhosis; median time
to a new decompensation event (e.g. hepatic encephalopathy) was 2.5
months for patients with decompensated cirrhosis.
Some of these cases occurred in patients with
decompensated cirrhosis when they were treated with higher than the
recommended dosage for that patient population; however, cases of
hepatic decompensation and failure have continued to be reported in
patients with decompensated cirrhosis even when they received the
recommended dosage.
Hepatotoxicity was observed in the OCALIVA
clinical trials. A dose-response relationship was observed for the
occurrence of hepatic adverse reactions including jaundice,
worsening 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 two 3-month,
placebo-controlled clinical trials in patients with primarily early
stage PBC.
Routinely monitor patients for progression of
PBC, including hepatic adverse reactions, with laboratory and
clinical assessments to determine whether drug discontinuation is
needed. Closely monitor patients with compensated cirrhosis,
concomitant hepatic disease (e.g., autoimmune hepatitis, alcoholic
liver disease), and/or with severe intercurrent illness for new
evidence of portal hypertension (e.g., ascites, gastroesophageal
varices, persistent thrombocytopenia), or increases above the upper
limit of normal in total bilirubin, direct bilirubin, or
prothrombin time to determine whether drug discontinuation is
needed. Permanently discontinue OCALIVA in patients who develop
laboratory or clinical evidence of hepatic decompensation (e.g.,
ascites, jaundice, variceal bleeding, hepatic encephalopathy), have
compensated cirrhosis and develop evidence of portal hypertension
(e.g., ascites, gastroesophageal varices, persistent
thrombocytopenia), experience clinically significant hepatic
adverse reactions, or develop complete biliary obstruction. If
severe incurrent illness occurs, interrupt treatment with OCALIVA
and monitor the patient’s liver function. After resolution of the
intercurrent illness, consider the potential risks and benefits of
restarting OCALIVA treatment.
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 a 12-month
double-blind randomized controlled clinical 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. Consider clinical evaluation of
patients with new onset or worsening severe pruritus. Management
strategies include the addition of bile acid binding 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 patients, 20% and 9% in the 10 mg and titration
arms, respectively, compared to 2% in the placebo arm. Monitor
patients for changes in serum lipid levels during treatment. For
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 (≥5%) are:
pruritus, fatigue, abdominal pain and discomfort, rash,
oropharyngeal pain, dizziness, constipation, arthralgia, thyroid
function abnormality, and eczema.
Drug Interactions
- Bile Acid Binding ResinsBile 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.
- WarfarinThe 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 co-administering
OCALIVA and warfarin.
- CYP1A2 Substrates with Narrow
Therapeutic IndexObeticholic acid 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
co-administered with OCALIVA.
- Inhibitors of Bile Salt Efflux
PumpAvoid 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 click here for
Full Prescribing Information, including
Boxed WARNING.
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
StatementsThis 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 a 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, including in connection with the newly identified safety
signal relating to Ocaliva identified by the FDA in May 2020
and with respect to patients with PBC with decompensated cirrhosis,
a prior decompensation event or with compensated cirrhosis who have
evidence of portal hypertension; 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; the outcomes of ongoing discussions with the FDA
and European Medicines Agency regarding the feasibility
of the COBALT and 401 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 attract and
retain key personnel to manage our business effectively; 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, cash
equivalents and short-term investments; our ability to acquire,
license and invest in businesses, technologies, product candidates
and products; our ability to manage the growth of our operations,
infrastructure, personnel, systems and controls; our ability to
obtain and maintain adequate insurance coverage; continuing threats
from COVID-19, including additional waves of infections, and their
impacts including quarantines and other 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 and
facility closures or other restrictions, and the impact of the
foregoing on our results of operations and financial position; the
impact of general U.S. and foreign economic, industry,
market, regulatory or political conditions, including the 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, 2020.
CONTACT
For more information about Intercept, please contact:
For investors:investors@interceptpharma.com
For media:media@interceptpharma.com
Source: Intercept Pharmaceuticals, Inc.
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