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 formally notified the European Medicines
Agency (EMA) of its decision to withdraw its Marketing
Authorization Application (MAA) for obeticholic acid (OCA) for the
treatment of liver fibrosis due to nonalcoholic steatohepatitis
(NASH).
The initial MAA filing in December 2019 was supported by the
positive interim analysis results from the pivotal Phase 3
REGENERATE study which remains ongoing. Intercept is in the process
of generating additional efficacy and safety data from REGENERATE,
including a full reread of Month 18 biopsies using a new consensus
biopsy reading approach, as well as a robust body of adjudicated
safety data, to support a potential refiling in the U.S. As
disclosed previously, Intercept has been working with the Committee
for Medicinal Products for Human Use (CHMP) to potentially include
this data in the current MAA review process. However, the
established application timeline could not be extended any further
to allow for submission of the additional data – which is expected
in the early part of 2022 – and based on their review of data
submitted to date, the CHMP was not able to determine a positive
benefit-risk. As a result, the Company has made the decision to
withdraw the MAA.
“Advanced fibrosis due to NASH remains a significant unmet need
in Europe. We are in the midst of producing the largest data
package in the NASH field, which will include significantly more
evidence regarding the benefit/risk of OCA in NASH,” said Jerry
Durso, President and Chief Executive Officer of Intercept. “Once we
have the results of these new analyses, we will assess the
possibility of submitting a new application to the EMA.”
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 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. 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 will evaluate the effect of OCA on all-cause
mortality and liver-related clinical outcomes, as well as long-term
safety.
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, including in connection with our
update to Ocaliva prescribing information in May 2021
contraindicating Ocaliva for patients with PBC and decompensated
cirrhosis, a prior decompensation event, or compensated cirrhosis
with 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 interactions with regulators
(e.g., the FDA and the European Medicines Agency) regarding our
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 attract and
retain key personnel to manage our business effectively; our
ability to prevent or defend against system failures or security or
data breaches due to cyber-attacks, or cyber intrusions, including
ransomware, phishing attacks and other malicious intrusions; our
ability to comply with 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 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:
Investor inquiries:
investors@interceptpharma.com
Media inquiries:
media@interceptpharma.com
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