- Phase 3
data for elafibranor
in primary biliary cholangitis (PBC) expected in
2Q23
-
Expanded pipeline
now covers
five therapeutic areas
with high unmet medical need via six
independent programs exploring
the potential of differentiated mechanisms of
action
-
A regular stream of new
clinical data expected over the next few
years across the spectrum of
development stages (Phase 1, Phase 2, Phase
3)
-
Aggregated global
market size
potential
>$15bn1,
distributed among acute on-chronic liver failure
(ACLF) with
~$6bn,
hepatic encephalopathy
(HE)
with ~$4bn,
cholangiocarcinoma (CCA) with
~$3bn, urea
cycle disorder
(UCD)/organic
acidemia
disorder (OAD) with
~$1.5bn and
PBC with ~$1.5bn
-
Targeted
indications include four
orphan indications offering valuable incentives
and optionality for accelerated market access
- Replay of event now
available on Investors &
Media section of GENFIT website
Lille (France),
Cambridge (Massachusetts, United States), Zurich
(Switzerland) November 17,
2022 – GENFIT (Nasdaq and Euronext:
GNFT), a late-stage biopharmaceutical company dedicated to
improving the lives of patients with liver diseases characterized
by high unmet medical needs, today announced the highlights from
its Pipeline Day events reviewing the Company’s clinical progress
and recent strategic updates.
A video replay of the Pipeline Day events which
took place in Paris (on October 5, 2022) and New York (on October
19, 2022) is now available and can be accessed from the "Events
& Presentations" page under the "Investors & Media" section
of the GENFIT website.
Pipeline and R&D
strategy
Over the last 12 months, GENFIT has successfully
executed its strategic plan.
In December 2021, GENFIT signed a licensing and
collaboration agreement with Ipsen for the elafibranor program,
which consolidated GENFIT’s financial situation (€120M upfront
payment, €28M equity investment with a significant premium, up to
€360M milestones and tiered double-digit royalties up to 20%).
GENFIT also secured patient recruitment for the
ELATIVE® Phase 3 trial in PBC, successfully completing this
activity in June 2022 despite challenges faced with the COVID
pandemic. Topline data are expected in the next few months.
GENFIT then strengthened, diversified and
expanded our Research and Development pipeline with the goal to
capitalize on our expertise in bringing early stage assets up to
pre-commercialization stages in liver diseases. Its new R&D
strategy now focuses exclusively on liver diseases with high unmet
medical need and significant market potential, with the
in-licensing of the rights for a novel asset initially developed by
Genoscience Pharma in CCA in December 2021, followed by the
acquisition of the Swiss-based clinical-stage biopharmaceutical
company Versantis in September 2022, adding three additional assets
positioned in ACLF, in HE and in UCD/OAD.
Pascal
Prigent, Chief Executive Officer
of GENFIT commented: ”GENFIT’s perspectives have
dramatically evolved over the last two years. Although the outcome
was not what we had hoped for, GENFIT has greatly benefited from
its work in NASH. We have acquired great know-how in liver disease
specific research, from target identification to proprietary
models. We also developed expertise in clinical development and
regulatory affairs in emerging diseases. We have built solid
networks with experts, academic institutions, patient associations,
etc. It is this accumulated experience and established
infrastructure that we are able to leverage now. The clinical
development of elafibranor in PBC is nearing completion with high
level results expected in Q2 next year. We see the signature of the
related strategic partnership with Ipsen at the end of 2021 as
further reason to be optimistic about elafibranor’s commercial
outlook. The deal with Ipsen also gave us the financial means to
make significant progress on our pipeline over the past nine
months, as we executed two other deals with Genoscience and
Versantis, continuing to execute our strategic roadmap. During our
Pipeline Day, we were excited to host a deep dive into the new
programs recently integrated into our pipeline and hear from
leading experts, who provided valuable insights into the unmet
medical needs of patients, as well as potential market
opportunities. These presentations also outlined what we believe
has been a transformative year for GENFIT, as we work with
increased momentum to diversify our pipeline with innovative
therapeutic opportunities for rare liver diseases.”
Key Expert
Speakers included:
- Jennifer C. Lai, MD, MBA –
Transplant Hepatologist, University of California, UCSF, USA
- Jonel Trebicka, MD, PhD,
Professor of Medicine, Chair of Department of Internal Medicine,
UKM Uniklinikum Muenster, Germany
- Mark Yarchoan, MD, Associate
Professor of Oncology at John Hopkins Medicine,
Baltimore, USA
- Angela Lamarca, MD – Medical
Oncology, Fundacion Jimenez Diaz University Hospital, Madrid,
Spain
Back Bay Life Science Advisors also shared the
results of a market study conducted in 2021 in ACLF. This study was
based on an extensive review of published literature as well as
in-depth discussions with Key Opinion Leaders (KOLs) managing ACLF
patients, with hospital pharmacists, hospital administrators, and
managed care organizations (payers) in the US.
Pipeline Program
Highlights
NTZ and VS-01-ACLF for
acute on-chronic liver failure
(ACLF)
- ACLF is a
syndrome that is characterized by an abrupt life-threatening
worsening of a pre-existing advanced chronic liver disease
resulting in acute liver decompensation, liver failure and
extrahepatic organ failure (brain, kidneys, cardiovascular and
respiratory). ACLF is an underserved medical condition associated
with short-term mortality (23% to 74% mortality at 28 days,
depending on severity grade) and a significant cost of care for
healthcare systems. No drugs have been approved in this indication
so far. Conservative estimates lead to a current total addressable
population of ~215,000 patients with ACLF across the US and EU5, a
number expected to grow up to ~300,000 patients by 2036. The ACLF
market size is estimated to be as high as $6bn (US and EU) by
20302.
- GENFIT is
currently developing two programs in ACLF. The first one aims at
evaluating the potential of the molecule nitazoxanide (NTZ).
Preclinical data generated via several disease models highlighted
the potential of NTZ with regards to inflammation, liver and kidney
function, brain edema and survival in sepsis, and as such supported
further clinical development. Phase 1 results in patients with
hepatic impairment as well as patients with renal impairment will
be disclosed in the coming months. The second program aims at
evaluating VS-01-ACLF, a first-in-class innovative liposomal-based
therapeutic product candidate for potential first-line therapy. A
Phase 1 trial highlighted the favorable safety and tolerability
profile of VS-01 and provided encouraging preliminary efficacy
results, with >80% of treated patients improving or stabilizing
their disease (Child-Pugh Score assessment). A Phase 2
Proof-of-concept study is expected to launch as early as 4Q22 (a
60-patient, randomized and controlled trial). VS-01-ACLF has been
granted Orphan Drug Designation (ODD) in ACLF by the US Food and
Drug Administration (FDA).
VS-02-HE for hepatic
encephalopathy (HE)
- HE is a nervous
system disorder brought on by advanced chronic liver disease. It is
one of the major complications of advanced liver disease and portal
hypertension. 30%-40% of patients with cirrhosis will experience at
least one episode of HE. The hepatic failure leads to the
accumulation of the neurotoxin ammonia in the blood stream,
ultimately leading to abnormally functioning neurons, and often
coma. HE is associated with increased hospitalizations,
recurrences, healthcare costs and mortality. It is a largely
underdiagnosed and undertreated condition and is associated with
poor quality of life. In the US only, 2 million patients are
believed to be at risk of developing HE and 200,000 patients are
hospitalized yearly3. In Europe, incidence is close to 1 million
patients. Standard of care with current treatments is associated
with side effects and moderate efficacy. The estimated annual
economic burden associated with HE in the US was $7.2bn in 2009 and
around $12bn in 20144. Estimates for global market size are as high
as $4.1bn in 20265.
- GENFIT is
developing VS-02, a urease inhibitor currently in preclinical
stage. It will be developed as a unique oral and colon-active
formulation designed to minimize systemic absorption of ammonia and
to act where ammonia is primarily produced, while reducing
glutamine levels in the brain. The treatment goal is to
reduce/stabilize the accumulation of ammonia in the blood and
prevent rehospitalization. Investigational New Drug-enabling6
nonclinical studies are targeted for completion in 2025.
GNS561 for
cholangiocarcinoma (CCA)
- CCA is a type
of cancer that forms in the bile ducts that carry the digestive
fluid bile and is the second most common primary hepatic malignancy
accounting for approximately 15% of all primary liver tumors. Cases
of CCA are usually asymptomatic in early stages and are therefore
often diagnosed when the disease is already in advanced stages. The
silent presentation of these tumors combined with their highly
aggressive nature and being refractory to chemotherapy contribute
to poor prognosis and high mortality, representing ~2% of all
cancer-related deaths worldwide yearly. Although CCA is a rare
cancer, its incidence (0.3–6 per 100,000 inhabitants per year) and
mortality (1–6 per 100,000 inhabitants per year), have been
increasing in the past few decades worldwide, representing a global
health problem. Although surgery is a potential curative option for
CCA, most patients are diagnosed at late stages due to lack of
specific symptoms. The majority of patients with CCA have
metastatic or locally advanced (i.e. unresectable) disease at
presentation, and only ∼25% are eligible for resection. When
disease is unresectable, the current first-line treatment is
chemotherapy. After progression on first-line chemotherapy,
second-line treatments exist but despite the options presently
available, the unmet need remains high due to limited benefits on
survival. 9,000 new patients are diagnosed every year in the US,
and 10,000 in EU57. Based on available data, the global market size
is evaluated at $1.2bn in 2021 and, with the Compound Annual Growth
Rate (CAGR) at 12.5%, it is expected to reach $3.2bn in 20308.
- GENFIT is
developing GNS561, a small molecule PPT1 inhibitor that blocks
cancer cell proliferation by inhibiting late stage autophagy
leading to cell death9. It received ODD from the FDA in September
2022, confirming its potential in this disease area. First-In-Human
effects of PPT1 inhibition following GNS561 administration in
patients with primary and secondary liver cancers have been
observed. The safety profile, exposure, and preliminary signal of
activity support the investigation of GNS561 in combination. A
Phase 1b/2 study is expected to be launched with a
mitogen-activated protein kinase (MEK) inhibitor in the near future
in patients with KRAS (Kirsten rat sarcoma viral oncogene homolog)
mutated cholangiocarcinoma who have failed treatment with first
line treatment and who do not have an actionable mutation.
VS-01-UCD
for urea cycle disorder
(UCD)/organic
acidemia
disorder (OAD)
- Patients
suffering from UCD and OAD, which are two different groups of
congenital metabolic diseases, have difficulties to metabolize
ammonia. This is due to a deficiency in one of the six enzymes
involved in the urea cycle, creating a situation where ammonia is
not eliminated in the urine, ultimately leading to hyperammonemia
attacks. Patients are usually diagnosed after they are born, via
universal newborn screening tests. While these conditions are
ultra-rare with 1,900 acute hyperammonemic crises in the US and EU5
per year10, the mortality is very high as 75% will die after 5
years, and survivors will often have severe brain injuries. There
is no acute treatment available for early onset crises, and
neonatal hemodialysis is risky, highly invasive and widely
unavailable. 45% of UCD patients remain untreated and no drug is
currently approved for treatment of OAD. Based on available data,
global market size in UCD and OAD is evaluated at $1.5bn in 2021,
based on two drugs that have been approved in UCD but not in acute
hyperammonemia11.
- GENFIT is
developing VS-01-UCD, a potential first-line lifesaving treatment
for acute hyperammonemic crises. As the peritoneal route of
administration is well adapted to pediatric patients, this
treatment should be feasible in all hospitals and should be quickly
implemented. This is a tremendous improvement over neonatal
hemodialysis, which is only possible in specialized centers and is
a long and complex procedure. ODD and Rare Pediatric Disease
designation (RPDD) have been granted to VS-01 by the FDA for this
indication. GENFIT is also potentially eligible to a Priority
Review Voucher upon approval.
ABOUT GENFIT
GENFIT is a late-stage biopharmaceutical company
dedicated to improving the lives of patients with 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. Thanks
to its expertise in bringing early-stage assets with high potential
to late development and pre-commercialization stages, today GENFIT
boasts a growing and diversified pipeline of innovative therapeutic
and diagnostic solutions.
Its R&D pipeline covers five therapeutic
areas via six independent programs which explore the potential of
differentiated mechanisms of action, across a variety of
development stages (Phase 1, Phase 2, Phase 3). These diseases are
acute on-chronic liver failure (ACLF), hepatic encephalopathy (HE),
cholangiocarcinoma (CCA), urea cycle disorder (UCD)/organic
acidemia disorder (OAD) and primary biliary cholangitis (PBC).
Beyond therapeutics, GENFIT’s pipeline also includes a diagnostic
franchise focused on NASH and ACLF.
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.
www.genfit.com
FORWARD LOOKING STATEMENTS
This press release contains certain
forward-looking statements with respect to GENFIT, including those
within the meaning of the Private Securities Litigation Reform Act
of 1995, in relation to expected availability of ongoing or
upcoming clinical data over the coming months and years,
specifically data from the Phase 3 ELATIVE® clinical trial, launch
of a Phase 1b/2 study of GNS561 in CCA, anticipated market
potential in each of the disease areas that GENFIT is
investigating, development costs and timelines for development of
our pipeline. The use of certain words, including “consider”,
“contemplate”, “think”, “aim”, “expect”, “understand”, “should”,
“aspire”, “estimate”, “believe”, “wish”, “may”, “could”, “allow”,
“seek”, “encourage” or “have confidence” or (as the case may be)
the negative forms of such terms or any other variant of such terms
or other terms similar to them in meaning is intended to identify
forward-looking statements. Although the Company believes its
projections are based on reasonable expectations and 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, biomarkers, progression of, and
results from, its ongoing and planned clinical trials, review and
approvals by regulatory authorities of its drug and diagnostic
candidates, the impact of the COVID-19 pandemic, exchange rate
fluctuations, potential synergies related to the acquisition of
Versantis and our capacity to integrate Versantis and to develop
its programs and our continued ability to raise capital to fund its
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 2021 Universal Registration Document filed with the AMF
on April 29 2022 under n° D.22-0400, 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 2021 Annual Report on Form 20-F filed with the SEC on
April 29, 2022 and the 2022 Half-Year Business and Financial
Report. 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 Based on actual figures as well as preliminary market studies
derived from different sources 2 Extrapolated from ‘Time trend in
the healthcare burden and mortality of ACLF in the US’ – Hepatology
20163 Potnis et al., International Journal of Hepatology 2021 4
Stepanova et al., Clin Gast Hep 2012 5 Hepatic Encephalopathy
Market Report by Coherent Market Insights6 The purpose of
IND-enabling studies is to secure approval to conduct the
first-in-human clinical trials with a new drug (IND stands for
Investigational New Drug)7 IQVIA derived data8 Olympus Research
Global9 Harding JJ, Awada A, Roth G, Decaens T, Merle P, Kotecki N,
Dreyer C, Ansaldi C, Rachid M, Mezouar S, Menut A, Bestion EN,
Paradis V, Halfon P, Abou-Alfa GK, Raymond E. First-In-Human
Effects of PPT1 Inhibition Using the Oral Treatment with
GNS561/Ezurpimtrostat in Patients with Primary and Secondary Liver
Cancers. Liver Cancer. 2022 Feb 15;11(3):268-277. doi:
10.1159/00052241810 Summar et al., 2013 | Martin-Hernandez et al.,
2014 | Nettesheim et al., 201711 2021 assessment from longitudinal
and real-world data reference publications (no projections nor
modelling)
- GENFIT Pipeline Day Highlights Diverse Product Portfolio in
Underserved Liver Diseases
Genfit (EU:GNFT)
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