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 the U.S. Food and Drug Administration (FDA) has
issued a Complete Response Letter (CRL) regarding the New Drug
Application (NDA) for obeticholic acid (OCA) for the treatment of
fibrosis due to nonalcoholic steatohepatitis (NASH).
The CRL indicated that, based on the data the FDA has reviewed
to date, the Agency has determined that the predicted benefit of
OCA based on a surrogate histopathologic endpoint remains uncertain
and does not sufficiently outweigh the potential risks to support
accelerated approval for the treatment of patients with liver
fibrosis due to NASH. The FDA recommends that Intercept submit
additional post-interim analysis efficacy and safety data from the
ongoing REGENERATE study in support of potential accelerated
approval and that the long-term outcomes phase of the study should
continue.
“At no point during the review did the FDA communicate that OCA
was not approvable on an accelerated basis, and we strongly believe
that the totality of data submitted to date both meet the
requirements of the Agency’s own guidance and clearly support the
positive benefit-risk profile of OCA,” said Mark Pruzanski, M.D.,
President and CEO of Intercept. “We are disappointed to see the
determination the Agency has reached based on an apparently
incomplete review, and without having provided medical experts and
patients the opportunity to be heard at the anticipated Adcom on
the merits of OCA, which is a designated Breakthrough Therapy. The
FDA has progressively increased the complexity of the histologic
endpoints, creating a very high bar that only OCA has so far met in
a pivotal Phase 3 study. On behalf of the hepatology community, we
are very concerned that the Agency’s apparently still evolving
expectations will make it exceedingly challenging to bring
innovative therapies to NASH patients with high unmet medical need.
We plan to meet as soon as possible with the FDA to review the CRL
and discuss options for an efficient path forward to
approval.”
Intercept had previously disclosed that, based on the FDA’s
decision to postpone a tentatively scheduled advisory committee
meeting (Adcom), it was expected that the Agency’s review of its
NDA would extend beyond the PDUFA goal date and that the FDA would
move forward with rescheduling the Adcom.
The NDA submission for OCA is the first for NASH and was based
on data from 35 clinical trials and more than 1,700 NASH patients
treated with the drug. OCA is the only investigational NASH drug
with Breakthrough Therapy designation and has uniquely demonstrated
reproducible ability to reverse or otherwise stabilize liver
fibrosis in patients with advanced fibrosis due to NASH. According
to the FDA draft guidance for NASH fibrosis, of the histologic
features of NASH, fibrosis is considered the strongest predictor of
adverse clinical outcomes, including liver-related death. There is
currently no approved therapy for this devastating disease, which
has become a leading cause of liver failure and resulting poor
clinical outcomes.
Conference Call on June 29, 2020 at 8:30
a.m. ET
The Company will host a conference call today, June 29, at 8:30
a.m. ET to discuss the Complete Response Letter. The conference
call will be available on the investor page of our website at
http://ir.interceptpharma.com or by calling (855) 232-3919
(toll-free domestic) or (315) 625-6894 (international) passcode
5586301. A replay of the call will be available on our website
shortly following the completion of the call and will be available
for two weeks.
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 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 review of
our New Drug Application for obeticholic acid (OCA) for the
treatment of fibrosis due to nonalcoholic steatohepatitis (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), 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; 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 net sales, non-GAAP adjusted
operating expenses 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
March 31, 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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