* New Drug Application granted priority review with PDUFA date set for June
10, 2024
* European Medicines Agency (EMA) has also validated the Marketing
Authorization Application (MAA) for elafibranor
* Investigational elafibranor is the first novel second-line treatment for
primary biliary cholangitis (PBC) to be filed in E.U. and U.S. in nearly a
decade
PARIS, FRANCE, 07 December 2023 - Ipsen (Euronext: IPN; ADR: IPSEY) and GENFIT
(Nasdaq and Euronext: GNFT) today announced that the U.S. Food and Drug
Administration (FDA) has accepted the New Drug Application (NDA) for
investigational elafibranor. An oral, once-daily dual peroxisome activated
receptor alpha/delta (PPAR ?,?) agonist, investigational elafibranor could
potentially be the first novel second-line treatment for the rare, cholestatic
liver disease, PBC, in nearly a decade. The target FDA PDUFA date under priority
review is June 10, 2024.
The European Medicines Agency (EMA) has also validated Ipsen's Marketing
Authorization Application (MAA) for elafibranor and the review of the submission
to the EMA's Committee for Medicinal Products for Human Use (CHMP) began on 26
October 2023. Furthermore, a third simultaneous regulatory filing of elafibranor
has been validated for review by the UK Medicines and Healthcare products
Regulatory Agency (MHRA).
"We are delighted to have achieved simultaneous filings for elafibranor, which
is in line with our ambition to be able to bring a new and much needed medicine
to as many people living with PBC as rapidly as possible," said Christelle
Huguet, EVP and Head of Research & Development, Ipsen. "This is a condition
where many patients are living with worsening disease and debilitating symptoms
despite being on treatment. Elafibranor, if approved, has the potential to
change the management of this challenging condition for people living with PBC,
offering a new second line treatment choice, where the number of effective
options are currently limited."
PBC is a rare, progressive, autoimmune cholestatic liver disease(1) in which
bile ducts in the liver are gradually destroyed.(2) The damage to bile ducts can
inhibit the liver's ability to rid the body of toxins, and can lead to scarring
of liver tissue, known as cirrhosis.(1,2,3) Common symptoms of PBC include
fatigue and pruritus (itch), which can be severely debilitating.(4) Untreated,
PBC can lead to liver failure, or in some cases death.(1 )It primarily affects
women, with nine women diagnosed for every man.(3) A significant proportion of
people living with PBC do not benefit from existing therapies.(5,6,7)
"These simultaneous regulatory submission acceptances are another important step
in the elafibranor journey. We are pleased to be partnering with Ipsen, who we
know has a good understanding of the rare-disease regulatory process," said
Pascal Prigent, Chief Executive Officer of GENFIT. "We know they share the same
goal as GENFIT, to bring a new, much needed treatment option to people living
with PBC as fast as possible; we look forward to elafibranor's progress through
the regulatory review processes."
ENDS
Elafibranor
Elafibranor is an oral, once-daily, dual peroxisome activated receptor (PPAR)
alpha/delta (?,?) agonist, currently under investigation as a treatment for
patients with PBC, a rare cholestatic liver disease. Elafibranor, through
activation of PPAR ?,? targets multiple cell types and biological processes
involved in the pathophysiology of PBC, including cholestasis (impairment of
bile flow in the liver), bile toxicity, inflammation and fibrosis and bile acid
output. In 2019, elafibranor was granted a Breakthrough Therapy Designation by
the U.S Food and Drug Administration in adults with PBC who have an inadequate
response to ursodeoxycholic acid (UDCA) the existing first-line therapy for PBC.
Elafibranor has not received approval by regulatory authorities anywhere in the
world.
ELATIVE
ELATIVE is a multi-center, randomized, double-blind, placebo-controlled Phase
III clinical trial, with an open-label long-term extension (NCT04526665).
ELATIVE evaluated the efficacy and safety of elafibranor 80mg once daily versus
placebo for the treatment of patients with PBC with an inadequate response or
intolerance to UDCA. The trial enrolled 161 patients who were randomized 2:1 to
receive either elafibranor 80mg once daily or placebo. Patients with an
inadequate response to UDCA would continue to receive UDCA in combination with
elafibranor or placebo, while patients unable to tolerate UDCA would receive
only elafibranor or placebo. Data confirmed the potential for elafibranor to be
an effective new treatment option for PBC, with 13 times more patients achieving
a biochemical response, suggesting an improvement in disease progression, when
treated with elafibranor compared with patients on placebo: 47% placebo-adjusted
difference, elafibranor 80mg (51%) compared with placebo (4%) (P10% of patients and more frequently on
elafibranor versus placebo included abdominal pain, diarrhea, nausea, and
vomiting.(8)
Ipsen
We are a global biopharmaceutical company with a focus on bringing
transformative medicines to patients in three therapeutic areas: Oncology, Rare
Disease and Neuroscience.
Our pipeline is fueled by external innovation and supported by nearly 100 years
of development experience and global hubs in the U.S., France and the U.K. Our
teams in more than 40 countries and our partnerships around the world enable us
to bring medicines to patients in more than 100 countries.
Ipsen is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored
Level I American Depositary Receipt program (ADR: IPSEY). For more information,
visit ipsen.com (http://www.ipsen.com).
GENFIT
GENFIT is a late-stage biopharmaceutical company dedicated to improving the
lives of patients with rare and life-threatening liver diseases characterized by
high unmet medical needs. GENFIT is a pioneer in liver disease research and
development with a rich history and strong scientific heritage spanning more
than two decades. Today, GENFIT has a growing and diversified pipeline with
programs at various development stages. The Company's area of focus is Acute on
Chronic Liver Failure (ACLF). Its ACLF franchise consists of five assets in
development: VS-01, NTZ, SRT-015, CLM-022 and VS-02-HE. These are all based on
differentiated mechanisms of action leveraging complementary pathways. Other
assets target other life-threatening disease indications such as
cholangiocarcinoma (CCA) and Urea Cycle Disorders (UCD)/Organic Acidemias (OA).
GENFIT's track record in bringing early-stage assets with high potential to late
development and pre-commercialization stages is highlighted in the successful
52-week Phase 3 ELATIVE® trial evaluating elafibranor in PBC. Beyond
therapeutics, GENFIT's pipeline also includes a diagnostic franchise focused on
MASH (previously known as NASH) and ammonia. GENFIT has facilities in Lille and
Paris (France), Zurich (Switzerland) and Cambridge, MA (USA). GENFIT is a
publicly traded company listed on the Nasdaq Global Select Market and on
compartment B of Euronext's regulated market in Paris (Nasdaq and Euronext:
GNFT). In 2021, IPSEN became one of GENFIT's largest shareholders and holds 8%
of the company's share capital. For more information, visit www.genfit.com
Ipsen contacts
Investors
Craig Marks | + 44 (0)7584 34 91 93 | craig.marks@ipsen.com
Nicolas Bogler | + 33 6 52 19 98 92 | nicolas.bogler@ipsen.com
Media
Amy Wolf | + 41 79 576 07 23 | amy.wolf@ipsen.com
Ioana Piscociu | + 33 6 69 09 12 96 | Ioana.piscociu@ipsen.com
Anna Gibbins | + 44 7717 80 19 00| anna.gibbins@ipsen.com
GENFIT contacts
Investors
GENFIT Investors | + 33 3 20 16 40 00 | investors@genfit.com
Media
Stephanie Boyer | + 33 3 20 16 40 00 | stephanie.boyer@genfit.com
Ipsen Forward-Looking Statements
The forward-looking statements, objectives and targets contained herein are
based on Ipsen's management strategy, current views and assumptions. Such
statements involve known and unknown risks and uncertainties that may cause
actual results, performance or events to differ materially from those
anticipated herein. All of the above risks could affect Ipsen's future ability
to achieve its financial targets, which were set assuming reasonable
macroeconomic conditions based on the information available today. Use of the
words 'believes', 'anticipates' and 'expects' and similar expressions are
intended to identify forward-looking statements, including Ipsen's expectations
regarding future events, including regulatory filings and determinations.
Moreover, the targets described in this document were prepared without taking
into account external-growth assumptions and potential future acquisitions,
which may alter these parameters. These objectives are based on data and
assumptions regarded as reasonable by Ipsen. These targets depend on conditions
or facts likely to happen in the future, and not exclusively on historical data.
Actual results may depart significantly from these targets given the occurrence
of certain risks and uncertainties, notably the fact that a promising medicine
in early development phase or clinical trial may end up never being launched on
the market or reaching its commercial targets, notably for regulatory or
competition reasons. Ipsen must face or might face competition from generic
medicine that might translate into a loss of market share. Furthermore, the
research and development process involves several stages each of which involves
the substantial risk that Ipsen may fail to achieve its objectives and be forced
to abandon its efforts with regards to a medicine in which it has invested
significant sums. Therefore, Ipsen cannot be certain that favorable results
obtained during preclinical trials will be confirmed subsequently during
clinical trials, or that the results of clinical trials will be sufficient to
demonstrate the safe and effective nature of the medicine concerned. There can
be no guarantees a medicine will receive the necessary regulatory approvals or
that the medicine will prove to be commercially successful. If underlying
assumptions prove inaccurate or risks or uncertainties materialize, actual
results may differ materially from those set forth in the forward-looking
statements. Other risks and uncertainties include but are not limited to,
general industry conditions and competition; general economic factors, including
interest rate and currency exchange rate fluctuations; the impact of
pharmaceutical industry regulation and healthcare legislation; global trends
toward healthcare cost containment; technological advances, new medicine and
patents attained by competitors; challenges inherent in new-medicine
development, including obtaining regulatory approval; Ipsen's ability to
accurately predict future market conditions; manufacturing difficulties or
delays; financial instability of international economies and sovereign risk;
dependence on the effectiveness of Ipsen's patents and other protections for
innovative medicines; and the exposure to litigation, including patent
litigation, and/or regulatory actions. Ipsen also depends on third parties to
develop and market some of its medicines which could potentially generate
substantial royalties; these partners could behave in such ways which could
cause damage to Ipsen's activities and financial results. Ipsen cannot be
certain that its partners will fulfil their obligations. It might be unable to
obtain any benefit from those agreements. A default by any of Ipsen's partners
could generate lower revenues than expected. Such situations could have a
negative impact on Ipsen's business, financial position or performance. Ipsen
expressly disclaims any obligation or undertaking to update or revise any
forward-looking statements, targets or estimates contained in this press release
to reflect any change in events, conditions, assumptions or circumstances on
which any such statements are based, unless so required by applicable law.
Ipsen's business is subject to the risk factors outlined in its registration
documents filed with the French Autorité des Marchés Financiers. The risks and
uncertainties set out are not exhaustive and the reader is advised to refer to
Ipsen's latest Universal Registration Document, available on ipsen.com
(https://www.globenewswire.com/Tracker?data=cRbeTYiLm_i554EQ7ITgGkjFSkEO7CuADsdG
INJYRhuLZP1b2RRGQKaNMQhqDBefwdyOcUcRf2VYKHI9RWUEWA==).
GENFIT Forward-Looking Statements
This press release contains certain forward-looking statements, including those
within the meaning of the Private Securities Litigation Reform Act of 1995 with
respect to GENFIT, including, but not limited to statements about the potential
of elafibranor as a safe and effective second-line treatment for PBC, the
opportunity to manage the disease progression and the potential of elafibranor
to improve pruritus, reduce cholestatic injury and improve liver function. The
use of certain words, including "believe", "potential," "expect", "target",
"may" and "will" and similar expressions, is intended to identify forward-
looking statements. Although the Company believes its expectations are based on
the current expectations and reasonable assumptions of the Company's management,
these forward-looking statements are subject to numerous known and unknown risks
and uncertainties, which could cause actual results to differ materially from
those expressed in, or implied or projected by, the forward-looking statements.
These risks and uncertainties include, among other things, the uncertainties
inherent in research and development, including in relation to safety of drug
candidates, cost of, progression of, and results from, our ongoing and planned
clinical trials, review and approvals by regulatory authorities in the United
States, Europe and worldwide, of our drug and diagnostic candidates, potential
commercial success of elafibranor if approved, exchange rate fluctuations, our
continued ability to raise capital to fund our development, as well as those
risks and uncertainties discussed or identified in the Company's public filings
with the AMF, including those listed in Chapter 2 "Main Risks and Uncertainties"
of the Company's 2022 Universal Registration Document filed with the AMF on
April 18, 2023, which is available on the Company's website (www.genfit.com) and
on the website of the AMF (www.amf-france.org) and public filings and reports
filed with the U.S. Securities and Exchange Commission ("SEC") including the
Company's 2022 Annual Report on Form 20-F filed with the SEC on April 18, 2023
and subsequent filings and reports filed with the AMF or SEC, including the
Half-Year Business and Financial Report at June 30, 2023 or otherwise made
public, by the Company. In addition, even if the Company's results, performance,
financial condition and liquidity, and the development of the industry in which
it operates are consistent with such forward-looking statements, they may not be
predictive of results or developments in future periods. These forward-looking
statements speak only as of the date of publication of this document. Other than
as required by applicable law, the Company does not undertake any obligation to
update or revise any forward-looking information or statements, whether as a
result of new information, future events or otherwise.
References
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biliary cholangitis. J Hepatol. 67(1):145-172.
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8. Kowdley. K.V, et al. NEJM. 2023. DOI: 10.1056/NEJMoa2306185
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