GENFIT Updates 2024 Outlook Following Acceptance of Elafibranor
Filings in Primary Biliary Cholangitis (PBC)
- US Food and Drug Administration (FDA) has granted
Priority Review for New Drug Application (NDA) for elafibranor in
PBC, and European Medicine Agency (EMA) has also validated the
Marketing Authorization Application (MAA) for
elafibranor.
- Acceptance triggers a first milestone payment. Further
milestones are expected upon US and European launches which could
now happen in 2Q24 in the US (FDA PDUFA1
action date: June 10, 2024) and 2H24 in Europe. These
milestones total approximately 89M€.
- Launches in the US and Europe will also make GENFIT
eligible for royalty payments.
- Revenues will fund the development of GENFIT’s
pipeline, now mainly focused on Acute On-Chronic Liver Failure
(ACLF) with 5 differentiated assets.
Lille (France), Cambridge
(Massachusetts, United States), Zurich (Switzerland), December 8,
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 announces its revised outlook for 2024 and
reflects on recent progress.
Elafibranor in PBC
Ipsen has made significant progress since the
announcement of positive interim data of ELATIVE® pivotal Phase 3
trial in June 2023:
- Strong leadership presence at AASLD
The Liver Meeting® and additional results from ELATIVE® presented
as late breaker, including data showing statistically significant
improvement (nominal p-value < 0.05) on multidimensional
measures of pruritus (PBC-40 and 5D-itch scores)
- Publication of ELATIVE® Phase 3
data in the New England Journal of Medicine
- Regulatory filing acceptance
obtained in the US, Europe and United Kingdom2 in less than 6
months after topline Phase 3 data, and Priority Review granted for
NDA by the US FDA with PDUFA target action date on June 10,
2024
During its Capital Market Day on December 7,
Ipsen reiterated its confidence in elafibranor as its profile could
be very beneficial for patients suffering from PBC. In the ELATIVE®
Phase 3 trial, significant treatment benefit in the primary
composite endpoint was achieved with elafibranor, with a high
responder rate, and a low placebo effect3. The key secondary
endpoint on ALP normalization was achieved – despite a high
baseline ALP level – with high statistical significance, the
pruritus improvements on PBC-40 and 5D-itch scores were also
statistically significant4 and elafibranor was generally
well-tolerated with a well-documented safety profile consistent
with previous trials.
Acceptance of filings in the US and Europe have
triggered the first milestone payment for GENFIT. We also expect
additional milestones after US and European launches and as a
result should receive a total of approximately 89M€ by the end of
next year. Ipsen also indicated that they were expecting global
peak sales to exceed 500M€ for elafibranor in PBC. With eligibility
for tiered double-digit royalties under the agreement with Ipsen,
this could mean a very significant revenue stream for GENFIT based
on these royalties and additional milestone payments.
Ipsen also indicated that it is developing
elafibranor in Primary Sclerosing Cholangitis (PSC) which could
lead to further revenues for GENFIT under the licensing
agreement.
Pascal Prigent, CEO of GENFIT,
commented: “We continue to be pleased by Ipsen’s commitment and
results already obtained. We believe that their excellent and
proven launch capabilities will allow them, once approved, to
quickly bring elafibranor to the many patients that need it. I have
no doubt that they will maximize the potential of elafibranor, and
this will obviously be very beneficial for GENFIT as it will help
us accelerate the development of our deep and promising
pipeline.”
Pipeline outlook for 2024
GENFIT’s R&D efforts have pivoted from
chronic to acute liver diseases, with a strong focus on ACLF where
the unmet medical need is very important, with currently no
approved therapies. Our therapeutic candidates have been
strategically selected based on the pathophysiology of ACLF to
address the most relevant pathways via differentiated and
complementary modes of action.
In 2024, progress is expected to be made on all
programs of the ACLF franchise:
- VS-01 (liposomal-based technology
designed to remove ammonia and other ACLF toxins from the blood):
Phase 2 initiated with interim data readout targeted for
mid-2024
- NTZ (anti-inflammatory and
anti-bacterial agent aiming to reduce systemic inflammation, and
impede release of PAMPs5 and bacterial translocation):
reformulation and Phase 2 under preparation in 2024 with a
proof-of-concept study initiation targeted for 1H25
- SRT-015 (ASK1 inhibitor,
liver-centric, aimed at inhibiting cell death, inflammation and
fibrosis): first-in-Human study initiation targeted 2H24
- CLM-022 (NLRP3 inflammasome
inhibitor aimed at inhibiting systemic inflammation and cell
death): preclinical Proof of Concept expected to initiate in
2024
- VS-02-HE (small molecule aiming at
reducing hyperammonemia, stabilizing blood ammonia and preventing
hepatic encephalopathy): IND enabling studies starting in 2024 with
completion expected in 2025
GENFIT also develops assets in other rare and
life-threatening liver diseases with high unmet medical needs:
- GNS561 for cholangiocarcinoma (CCA): Phase 1b interim biomarker
data targeted as early as 1H24
- VS-01 for Urea Cycle Disorders (UCDs) and Organic Acidemias
(OA): IND enabling non clinical studies completion expected in
2024
ABOUT ELAFIBRANOR
Elafibranor is an oral, once-daily, dual
peroxisome activated receptor (PPAR) alpha/delta (α,δ) agonist,
currently under investigation as a treatment for patients with PBC,
a rare cholestatic liver disease. Elafibranor, through activation
of PPAR α,δ 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.
ABOUT ELATIVE®
ELATIVE is a multi-center, randomized,
double-blind, placebo-controlled Phase 3 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%)
(P<0.001).
ABOUT ACLF
ACLF presents as a syndrome defined by a
combination of hepatic and extrahepatic organ dysfunctions and
failures and a uniformly poor prognosis. In patients with liver
cirrhosis and acute hepatic decompensation, ACLF can be triggered
by a precipitating event (e.g., an infection) that leads to a
progressive functional deterioration of multiple organs with high
short-term mortality (23% to 74% mortality at 28 days)6.
ACLF represents a significant cost of care for
healthcare systems, as it is characterized by an abrupt
life-threatening worsening of a preexisting advanced chronic liver
disease resulting in acute liver decompensation, liver failure and
extrahepatic organ failure. With a $52k average cost per
hospitalization per patient in the US and a 16-day average length
of hospital stay, estimated annual cost burden in the US was $6.4bn
in 2021, a nearly 4-fold increase since 20117.
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 the potential of elafibranor to
receive marketing authorization in the United States, Europe and
United Kingdom for PBC, expected milestone and royalty payments for
elafibranor in PBC, Ipsen’s expectations regarding global peak
sales for elafibranor in PBC, Ipsen’s ability to effectively
maximize commercialization of elafibranor and the ability of GENFIT
to receive revenues related to development and future
commercialization of elafibranor in PSC. 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.
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
1 Prescription Drug User Fee Act2
https://ir.genfit.com/news-releases/news-release-details/ipsen-confirms-us-fda-grants-priority-review-new-drug3
a 47% placebo-adjusted difference (P<0.001) between patients on
elafibranor 80mg (51%) compared with patients on placebo (4%)
achieving a biochemical response4 nominal p-value < 0.055
pathogen-associated molecular pattern molecules6 Arroyo V et al.,
Nat. Rev. Dis. Primers 2 (2016)7 IQVIA presentation GENFIT’s ACLF
Day Nov 2023
GENFIT | 885 Avenue Eugène Avinée, 59120
Loos - FRANCE | +333 2016 4000 |
www.genfit.com
- 2023.12.08 - PR EN - GENFIT - 2024 outlook - FINAL
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