FOR IMMEDIATE RELEASE
GENFIT Announces
FDA Protocol Clearance for Phase 2 Clinical Trial of Elafibranor in
Pediatric NASH
-
FDA accepts study protocol,
providing green light for GENFIT to initiate Phase 2 clinical trial
in pediatric NASH
-
Elafibranor, the first molecule
with positive results on registrational endpoint in Phase 2b
clinical trial in adult NASH to be evaluated in pediatric
NASH
-
12-week randomized trial of 20
pediatric patients to be initiated at U.S. clinical centers
specializing in NASH pediatrics
Lille (France),
Cambridge (Massachusetts, United States), March 11, 2019 -
GENFIT (Euronext: GNFT - ISIN: FR0004163111), a late-stage
biopharmaceutical company dedicated to the discovery and
development of innovative therapeutic and diagnostic solutions in
metabolic and liver related diseases, today announced protocol
clearance by the FDA for a Phase 2 trial evaluating elafibranor in
children and adolescents with non-alcoholic steatohepatitis
(NASH).
The first NASH
pediatric trial based on compelling Phase 2 clinical data in
adults
Elafibranor is ideally positioned
to be evaluated in children and adolescents with NASH, based on its
compelling Phase 2 data in adult NASH[1]:
-
Efficacy on "NASH resolution without worsening
of fibrosis" (26% elafibranor vs. 5% placebo; p-value 0.02), the
biopsy-based regulatory endpoint for marketing approval that
addresses the underlying cause of disease progression;
-
Beneficial cardiovascular profile (reduction of
LDL, TG, HDL, and improved insulin sensitivity), known to be
important for NASH patients;
-
No safety and no tolerability concerns observed,
essential in a chronic and silent condition like NASH.
Based on this clinical evidence,
GENFIT had already obtained the PSP (Pediatric Study Plan)
agreement by the FDA as well as the PIP (Pediatric Investigation
Plan) agreement by the EMA (European Medicines Agency), supporting
the initiation of this first-of-its-kind clinical trial.
Randomization of the first group of patients is expected in the
upcoming weeks.
Trial
design
-
Study to assess the pharmacokinetic and
pharmacodynamic profile and the safety and tolerability of two dose
levels of elafibranor (80 mg and 120 mg);
-
20 patients between 8 to 17 years of age, with
NASH;
-
12-week trial duration;
-
Open-label study;
-
Randomized across two arms;
-
U.S. multicenter study.
The burden of
NASH in the pediatric population
Non-Alcoholic Fatty Liver Disease,
or NAFLD, has become the most common liver abnormality diagnosed in
children and adolescents[2], and is
associated with insulin resistance and
hypertriglyceridemia[3]. NAFLD is
considered as the hepatic manifestation of the metabolic syndrome
and should be suspected in all overweight or obese children and
adolescents[4].
Recent alarming figures, collected
between 1988 and 2010 in more than 8000 American children and
adolescents (NHANES participants), have shown a tripling in the
rates of NAFLD from 3.3% to 10.1%, and a prevalence of NASH with an
almost five-fold increase from 0.7% to 3.4% over the same period,
resulting in serious liver conditions[5].
In addition, studies have shown
that 17% of American children with NAFLD are likely to have
fibrosis[6]. Among
obese children, those with metabolic syndrome are three times as
likely to develop NAFLD compared to those without metabolic
syndrome[7].
Statistically significant differences between NASH and NAFLD
suggest an increased cardiovascular disease risk in children with
NASH[8].
Dr. Joel
Lavine, MD, PhD, Co-Chair NASH CRN (NIDDK), Professor and Chief of
Pediatric GI/Hepatology/Nutrition, Columbia University, NY,
USA, commented: "Childhood obesity is a
problematic diagnosis for children, their families and the medical
community at large. The prevalence of NASH in
children is rapidly rising - the direct consequence of modern
lifestyles, unhealthy eating habits, and limited exercise -
becoming a major concern for hepatologists, gastroenterologists and
diabetologists around the world. The trial evaluating elafibranor
for pediatric NASH is the first study of its kind and a key
milestone for the NASH community. The scientific rationale, based
on adult Phase 2 clinical evidence, is sound. Elafibranor's neutral
safety profile coupled with cardiometabolic efficacy may address
the major medical needs faced by these children. Children
demonstrating metabolic dysfunction are at risk to progress to
advanced fibrosis, type 2 diabetes and cardiovascular disease in
the absence of effective interventions. Thus, the scientific
community eagerly awaits the clinical readout of elafibranor in
this NASH population."
Dean Hum,
Ph.D, Chief Operating Officer of GENFIT, added: "The initiation of a Phase 2 trial of elafibranor in
pediatric NASH is a landmark study, as to our knowledge there has
been no other molecule that has shown clinical evidence in a Phase
2b adult NASH trial and then progressed to clinical evaluation in
the pediatric NASH setting. Given the
substantial number of children from 8 to 17 years old with NASH,
GENFIT is enthusiastic to explore the
potential to provide children and adolescents with a treatment
option in the near future. Beyond the clinical
usage of elafibranor, we believe the scientific data generated
throughout the trial will be highly beneficial for physicians
tackling this disease and impactful for the still immature field of
pediatric NASH."
ABOUT
GENFIT
GENFIT is a biopharmaceutical
company focused on discovering and developing drug candidates and
diagnostic solutions targeting liver diseases, in particular those
of metabolic origin, and hepatobiliary diseases. GENFIT
concentrates its R&D efforts in areas of high unmet medical
needs corresponding to a lack of approved treatments. GENFIT's lead
proprietary compound, elafibranor, is a drug candidate currently
being evaluated in one of the most advanced Phase 3 clinical trials
worldwide ("RESOLVE-IT") in nonalcoholic steatohepatitis (NASH),
considered by regulatory authorities as a medical emergency because
it is often silent, with potentially severe consequences, and with
a prevalence on the rise. Elafibranor has also obtained positive
preliminary results in a Phase 2 clinical trial in primary biliary
cholangitis (PBC), a severe chronic liver disease. As part of its
comprehensive approach to clinical management of NASH patients,
GENFIT is conducting an ambitious discovery and development program
aimed at providing patients and physicians with a blood-based test
for the diagnosis of NASH, i.e. non-invasive and easy-to-access.
With facilities in Lille and Paris, France, and Cambridge, MA
(USA), the Company has approximately 150 employees. GENFIT is a
public company listed in compartment B of Euronext's regulated
market in Paris (Euronext: GNFT - ISIN: FR0004163111).
FORWARD LOOKING
STATEMENT/DISCLAIMER
This press release contains
certain forward-looking statements. Although the Company believes
its expectations are based on reasonable assumptions, these
forward-looking statements are subject to numerous 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 related to safety, progression of, and
results from, its ongoing and planned clinical trials, including
its anticipated pediatric clinical trial of elafibranor and its
RESOLVE-IT Phase 3 trial of elafibranor, the potential for
elafibranor to produce similar results in a pediatric population as
it has in adult populations, review and approvals by regulatory
authorities, such as the FDA or the EMA, of its drug and diagnostic
candidates, as well as those discussed or identified in the
Company's public filings with the AMF, including those listed in
Section 4 "Main Risks and Uncertainties" of the Company's 2018
Registration Document filed with the French Autorité des marchés
financiers on February 27, 2019 under n° D.19-0078, which is
available on GENFIT's website (www.genfit.com) and on the website
of the AMF (www.amf-france.org). Other than as required by
applicable law, the Company does not undertake any obligation to
update or revise any forward-looking information or statements.
This press release and the information contained herein do not
constitute an offer to sell or a solicitation of an offer to buy or
subscribe to shares in GENFIT in any country. This press release
has been prepared in both French and English. In the event of any
differences between the two texts, the French language version
shall supersede.
CONTACT
GENFIT | Investors
Naomi EICHENBAUM - Investor Relations | Tel: +1
(617) 714 5252 | investors@genfit.com
PRESS RELATIONS | Media
Hélène LAVIN - Press relations | Tel: +333 2016
4000 | helene.lavin@genfit.com
GENFIT | 885
Avenue Eugène Avinée, 59120 Loos - FRANCE | +333 2016 4000 |
www.genfit.com
[1] Ratziu, et
al. (2016). Elafibranor, an Agonist of the Peroxisome
Proliferator-Activated Receptor-a and -d, Induces Resolution of
Nonalcoholic Steatohepatitis Without Fibrosis Worsening. Gastroenterology, 150(5), pp.1147-1159.e5.
[2] Schwimmer,
et al. (2006). Prevalence of Fatty Liver in Children and
Adolescents. Pediatrics, 118(4), pp.1388-1393.
[3] Schwimmer,
J., Pardee, P., Lavine, J., Blumkin, A. and Cook, S. (2008).
Cardiovascular Risk Factors and the Metabolic Syndrome in Pediatric
Nonalcoholic Fatty Liver Disease. Circulation, 118(3),
pp.277-283.
[4] Nobili, V.,
et al. (2015). Nonalcoholic Fatty Liver Disease. JAMA Pediatrics,
169/2: 170.
[5] Selvakumar,
et al. (2016). Reduced lysosomal acid lipase activity - A potential
role in the pathogenesis of non alcoholic fatty liver disease in
pediatric patients. Digestive and Liver
Disease, 48/8: 909-913.
[6] Africa et
al., In Children With Nonalcoholic Fatty Liver Disease, Zone 1
Steatosis Is Associated With Advanced Fibrosis. Clin Gastroenterol
Hepatol, 2017.
[7] Papandreou,
D., Karavetian, M., Karabouta, Z., & Andreou, E. (2017). Obese
Children with Metabolic Syndrome Have 3 Times Higher Risk to Have
Nonalcoholic Fatty Liver Disease Compared with Those without
Metabolic Syndrome. International Journal of Endocrinology, 2017:
1-5.
[8] AASLD,
Poster 20-96, Konomi et al., 2017, NASH CRN, Vos, 2017.
2019.03.11 - PR - NASH pediatric
protocol
This
announcement is distributed by West Corporation on behalf of West
Corporation clients.
The issuer of this announcement warrants that they are solely
responsible for the content, accuracy and originality of the
information contained therein.
Source: GENFIT via Globenewswire
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