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 additional data indicating that obeticholic acid (OCA)
helped patients with nonalcoholic steatohepatitis (NASH) achieve
sustained improvements in liver biochemistry and noninvasive
markers of liver fibrosis over two years of treatment. The new
results based upon a post hoc review of the interim analysis data
from the Phase 3 REGENERATE study are being presented at the
virtual International Liver Congress™ 2020, the 55th Annual Meeting
of the European Association for the Study of the Liver (EASL).
“The primary goal of a medicine to treat patients with advanced
fibrosis due to NASH is to halt or reverse the progression to
cirrhosis and its devastating complications,” said Rohit Loomba,
M.D., director, U.C. San Diego NAFLD Research Center and director
of hepatology at U.C. San Diego School of Medicine. “These new
noninvasive data from REGENERATE provide further evidence that OCA
can help patients achieve this goal. It is encouraging to see a
consistent and sustained effect across multiple noninvasive tests
that clinicians use in practice every day to monitor and manage
their patients. The marked improvement in measurements of liver
stiffness observed with OCA therapy was particularly notable.
Additionally, these data give us greater confidence that OCA
continues to provide meaningful and durable benefit beyond the
histologic benefit already established at 18 months.”
As previously reported, once-daily OCA 25 mg met the primary
endpoint of fibrosis improvement (≥1 stage) with no worsening of
NASH at the planned 18-month interim analysis of REGENERATE with
high statistical significance (p=0.0002 vs. placebo). The new
analysis included patients from the interim analysis
intent-to-treat (ITT) population randomized early enough to have
both evaluable Month 18 biopsies (N = 251–263 per treatment arm)
and Month 24 data at the time of the interim analysis (N = 120–125
per arm). Changes from baseline to Month 24 in alanine
aminotransferase (ALT), aspartate aminotransferase (AST),
gamma-glutamyl transferase (GGT), serum markers of fibrosis (FIB-4,
AST to platelet ratio index [APRI]), and liver stiffness
(FibroScan® vibration-controlled transient elastography [VCTE];
subset, N = 64‒70 per arm) were analyzed.
Mean values of transaminases and other serum-based tests
improved rapidly in patients treated with OCA and were sustained
beyond 18 months of therapy compared with placebo. FibroScan VCTE
also demonstrated improvement in liver stiffness in patients
treated with OCA versus placebo after 24 months of therapy, with a
mean difference of 2.7 kPa between OCA 25 mg and placebo. At
baseline, median liver stiffness values were in the advanced
fibrosis range; at 24 months, median values of patients treated
with OCA 25 mg were below the threshold of 7.9 kPa, and most
patients treated with OCA 25 mg had moved from advanced to moderate
fibrosis.
Changes in transaminases and noninvasive markers of fibrosis
were associated with changes in histologic fibrosis, with the
greatest improvements observed in patients who had a ≥1 stage
improvement in fibrosis stage at 18 months. Moreover, early changes
in these markers were more pronounced in patients who had
histologic fibrosis improvement at Month 18. Overall, these
noninvasive data suggest that longer treatment duration with OCA
will likely result in greater fibrosis reduction beyond 18
months.
The overall adverse event profile of OCA 25 mg in the subgroup
of the ITT population with 24 months of follow-up at the time of
the interim analysis was generally consistent with that observed in
the overall ITT population. The most common adverse event was
pruritus. The incidence of serious adverse events was balanced
across the placebo and OCA 25 mg groups. Few serious adverse events
occurred in more than one patient and no consistent pattern of
serious adverse events was observed.
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 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 is designed to evaluate the effect of OCA on
all-cause mortality and liver-related clinical outcomes, as well as
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.
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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