GENFIT Highlights ACLF Development Strategy at “ACLF Day” during
AASLD The Liver Meeting® 2023
- GENFIT highlighted new focus and development strategy
in Acute On-Chronic Liver Failure (ACLF)
- ACLF is a very serious condition affecting
~294,0001 2
patients with chronic liver diseases in the USA and Europe
every year and is associated with multi-organ failure and high
short-term mortality
- It is an underserved medical condition with currently
no approved treatment and few programs under development
globally
- GENFIT has developed a unique pipeline of 5 different
drug candidates targeting key pathophysiological pathways of
ACLF
- Phase 2 interim data readout from lead program VS-01 in
ACLF targeted for 2Q24
Lille (France), Cambridge
(Massachusetts, United States), Zurich (Switzerland), November 16,
2023 – GENFIT (Nasdaq and Euronext:
GNFT), a late-stage biopharmaceutical company dedicated to
improving the lives of patients with rare and life-threatening
liver diseases, today announced the highlights from its “ACLF Day”
held on November 11, 2023 during AASLD The Liver Meeting ® in
Boston, MA (USA).
GENFIT is now mainly focused on the development
of therapies for ACLF which is an area of very high unmet medical
need. ACLF is a potentially deadly, but reversible, condition in
patients with chronic liver diseases, which is associated with
multi-organ failure and high short-term mortality. There is
currently no approved treatment for ACLF. The presentation from the
event is available on the company website in the investors
section.
The development of the company’s new, expanded
and diversified pipeline targeting rare and life-threatening liver
diseases with high unmet medical needs will be supported by
potential milestone payments and royalties deriving from the
licensing of elafibranor3 to its partner, Ipsen. At AASLD, Ipsen
presented additional data from the ELATIVE® Phase 3 study in
primary biliary cholangitis (PBC) and announced its publication in
the prestigious New England Journal of Medicine (November 2023)4.
Data from ELATIVE® are being used to support Ipsen's submissions
for elafibranor as a treatment for PBC with regulatory authorities
in the US and EU5.
Pascal Prigent, CEO of GENFIT,
commented: “During AASLD we were excited to further discuss our
development strategy around our new pipeline focused on ACLF, where
we believe multiple programs have the potential to transform the
treatment paradigm for these patients. It was also great to see
more data presented by our partner Ipsen on PBC, which confirm that
elafibranor has a competitive profile and a great potential to help
patients with this disease. This AASLD meeting reinforced our
conviction that Ipsen, as it works through the approval process
with regulatory authorities in the US and EU, is committed to
getting elafibranor to patients as quickly and efficiently as
possible. For GENFIT this could mean significant revenues that will
fund the development of a truly unique portfolio aiming at
providing solutions for healthcare professionals treating patients
suffering from a very challenging condition for which there is
currently no approved options. We look forward to presenting
preliminary data from our lead ACLF program, VS-01, as early as
next year.”
ACLF: high short-term mortality, no
approved treatment
ACLF is a serious, but potentially reversible,
condition in patients with chronic liver disease and cirrhosis,
which is associated with multiple organ failure, and a 23% to 74%
mortality at 28 days6 depending on grade severity. As part of the
“ACLF Day” event during AASLD, the critical need for treatment has
been highlighted by leading experts in ACLF.
Dr Jennifer C. Lai, MD, MBA, Transplant
Hepatologist, Endowed Professor of Liver Health &
Transplantation, University of California, San Francisco
(UCSF) said: “ACLF is a terrifying condition for patients
and their caregivers, but it’s equally terrifying for clinicians,
because we know our patients are in grave and immediate danger, and
we have no approved treatment to help them. It is very frustrating
and therefore it is critical that therapies are developed, ideally
to reverse the course of the disease or at least to give the
patient more time to get a potentially life-saving transplantation.
This is an area of huge unmet medical need and I welcome new
efforts to develop potentially helpful therapies.”
GENFIT’s pipeline in
ACLF: targeted to address the unmet need
GENFIT’s ACLF therapeutic candidates have been
strategically selected based on the pathophysiology of ACLF – as
defined by liver experts and consortiums such as EF-CLIF (European
Foundation for the study of Chronic Liver Failure) – to address the
most relevant pathways. Considerations about the limitations in
current standard of care have also been taken into account. Among
the pathways identified, priority is given to systemic
inflammation, cell death and microbiota.
Dean Hum, PhD, Chief Scientific Officer
of GENFIT, commented: “Our R&D strategy is centered on
understanding the pathophysiology of ACLF and selecting molecules
that we believe are best positioned to impact specific relevant
pathways. This multi-factorial approach will allow us greater
chances of success as well as leveraging potential synergies via
combinations. It also offers the possibility to apply key learnings
across all ACLF programs, to accelerate overall execution.”
The ACLF pipeline overview:
-
VS-01 – Phase 2 initiated (interim data readout
targeted for the end of 2Q24): VS-01 is a liposomal-based
technology designed to remove ammonia and other ACLF toxins from
the blood (peritoneal route of administration)
-
NTZ – Reformulation and Phase 2 under preparation
(proof-of-concept study initiation targeted for the first half of
2025): NTZ is an anti-inflammatory and anti-bacterial agent aiming
to reduce systemic inflammation, and impede release of PAMPs7 and
bacterial translocation (oral route of administration)
- SRT-015 –
First-in-Human study under preparation (expected to be initiated
2H24): SRT-015 is an ASK1 inhibitor, liver-centric, aimed at
inhibiting cell death, inflammation and fibrosis (injectable route
of administration)
- CLM-022 –
Preclinical proof of concept under preparation (expected to start
2024): CLM-022 is an NLRP3 inflammasome inhibitor aimed at
inhibiting systemic inflammation and cell death (pyroptosis)
-
VS-02-HE – Completion of IND enabling studies
expected in 2025: VS-02-HE aims at reducing hyperammonemia,
stabilizing blood ammonia and preventing hepatic encephalopathy
(oral route of administration)
Inaugural Patient
Advocacy Council organized by GENFIT
Patients are at the heart of what GENFIT does.
As we embark on a major new R&D initiative, it was important to
integrate the patient voice very early in the process. This is why
we hosted two Patient Advocacy Council sessions at AASLD. GENFIT’s
ambition is to co-design initiatives with patient representatives
and leading clinicians in order to better understand patients’ and
caregivers’ information needs, gather their insights and
perspective in the context of clinical trial design, and facilitate
access to innovative medicine through clinical trials.
ABOUT 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 Metabolic dysfunction-associated
steatohepatitis (MASH) (previously known as nonalcoholic
steatohepatitis – 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
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
expectations for availability of clinical data in the evaluation of
VS-01 in ACLF, Ipsen’s ability to obtain quickly and efficiently,
marketing authorization for elafibranor in PBC, GENFIT’s ability to
receive milestones and royalties under the collaboration and
licensing agreement with Ipsen with respect to PBC and the use of
those potential revenues to fund GENFIT’s further R&D and start
dates of clinical and pre-clinical development phases for GENFIT’s
other pipeline programs. 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.
CONTACT
GENFIT | Investors
Tel: +33 3 2016 4000 | investors@genfit.com
PRESS RELATIONS | Media
Stephanie Boyer – Press relations | Tel: +333
2016 4000 | stephanie.boyer@genfit.com
GENFIT | 885 Avenue Eugène Avinée, 59120 Loos -
FRANCE | +333 2016 4000 | www.genfit.com
1 Source: Moreau, R., et al., (2013) Supplemental Table 10)2
Therapies targeting ACLF are eligible for Orphan Drug Designation
given low prevalence and lack of therapies3 Elafibranor’s rights
have been licensed to Ipsen in December 2021 and to Terns
Pharmaceuticals in June 2019 for China, Hong Kong and Macau 4
https://www.nejm.org/doi/full/10.1056/NEJMoa23061855
https://www.ipsen.com/websites/ipsen_com_v2/wp-content/uploads/2023/08/11114914/Ipsen-investor-presentation-September-2023.pdf
(slide 9)6 Arroyo V et al., Nat. Rev. Dis. Primers 2 (2016)7
Pathogen-associated molecular patterns
- GENFIT Highlights ACLF Development Strategy at “ACLF Day”
during AASLD The Liver Meeting® 2023
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