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 it has submitted its Marketing Authorization
Application (MAA) to the European Medicines Agency (EMA) for
obeticholic acid (OCA) for the treatment of fibrosis due to
nonalcoholic steatohepatitis (NASH). The MAA submission is
supported by the positive interim analysis results from the pivotal
Phase 3 REGENERATE study in patients with liver fibrosis due to
NASH.
Intercept also announced that the U.S. Food and Drug
Administration (FDA) has notified the company of the tentative date
for the previously announced advisory committee meeting (AdCom)
related to Intercept’s New Drug Application (NDA) for OCA in liver
fibrosis due to NASH. The FDA has tentatively scheduled the AdCom
for April 22, 2020. Intercept anticipates that the FDA accordingly
will extend the recently announced March 26, 2020 Prescription Drug
User Fee Act (PDUFA) target action date for Intercept’s NDA.
Intercept previously announced the FDA’s acceptance of the NDA and
granting of priority review.
“NASH is quickly becoming one of the most significant public
health challenges in Europe and costs associated with advanced
fibrosis and cirrhosis are estimated to represent approximately 95%
of the total NASH related costs to health care systems,” said Mark
Pruzanski, M.D., President and Chief Executive Officer of
Intercept. “We believe that OCA has the potential to become an
essential treatment for people living with advanced fibrosis due to
NASH and we look forward to working with the EMA during this review
period. Separately, we are pleased that FDA has provided us with
the tentative AdCom date and we look forward to working
collaboratively with the agency during its review of the NDA as we
continue to prepare for our anticipated NASH launch, if approved,
within the first half of 2020.”
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 and, as early as 2020, the disease is
projected to become the leading cause of liver transplants in the
United States. 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 interim
analysis was conducted to assess the effect of OCA on liver
histology comparing month 18 biopsies with baseline. The
intent-to-treat population for the interim analysis included 931
patients with stage 2 and 3 fibrosis (placebo, n=311; OCA 10 mg,
n=312; OCA 25 mg, n=308). REGENERATE has completed target
enrollment for the clinical outcomes cohort, with 2,480 adult NASH
patients randomized at 339 qualified centers worldwide, and will
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 its long-term safety.
The safety population of the interim analysis included 1,968
randomized patients who received at least one dose of
investigational product (OCA or placebo). Adverse events were
generally mild to moderate in severity and the most common were
consistent with the known profile of OCA. The frequency of serious
adverse events was similar across treatment arms (11% in placebo,
11% in OCA 10 mg and 14% in OCA 25 mg). The most common adverse
event reported was dose-related pruritus (placebo, 19%; OCA 10 mg,
28%; OCA 25 mg, 51%). The large majority of pruritus events were
mild to moderate, with severe pruritus occurring in a small number
of patients.
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
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 development candidates, including OCA for
NASH, the timing and acceptance of our regulatory filings and the
potential approval of OCA for NASH or any other indications in
addition to PBC, the timing and potential commercial success of OCA
and any other product candidates we may develop and our strategy,
future operations, future financial position, future revenue,
projected costs, financial guidance, prospects, plans, objectives
of management and expected market growth. 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; 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 timely and cost-effectively file for and obtain
regulatory approval of our product candidates, including the
regulatory approval of our New Drug Application for NASH; any
advisory committee recommendation that our product candidates,
including OCA for NASH, should not be approved or approved only
under certain conditions; any determination that the regulatory
applications and subsequent information we submit for our product
candidates, including OCA for 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, 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 contained in the
label of any of our products or product candidates; any potential
side effects associated with Ocaliva for PBC, OCA for 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; 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 NASH, and our clinical
trial activities; our ability to identify, develop and successfully
commercialize our products and product candidates, including our
ability to timely and successfully launch OCA for 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 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 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, 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
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, 2018.
Contact
For more information about Intercept, please contact:
Lisa
DeFrancesco+1-646-565-4833investors@interceptpharma.com
Christopher Frates+1-646-757-2371media@interceptpharma.com
Intercept Pharmaceuticals (NASDAQ:ICPT)
Historical Stock Chart
From Aug 2024 to Sep 2024
Intercept Pharmaceuticals (NASDAQ:ICPT)
Historical Stock Chart
From Sep 2023 to Sep 2024