Results from Ipsen’s ELATIVE® pivotal Phase III trial of
elafibranor in PBC presented as late breaking data at AASLD
congress and published in New England Journal of Medicine
- ELATIVE® Phase III trial confirms
potential for investigational elafibranor as a novel,
first-in-class, dual PPAR α,δ agonist for patients with primary
biliary cholangitis.
- Elafibranor demonstrates
significant improvements in biomarkers of disease progression
versus placebo, including significant treatment benefit with
improvement in biochemical response and alkaline phosphatase (ALP)
normalization, along with patient-reported outcomes data suggesting
a possible improvement in pruritus.
- Elafibranor was generally
well-tolerated with a well-documented safety profile consistent
with previous trials.
PARIS, FRANCE, 13 November,
2023 – Ipsen (Euronext: IPN; ADR: IPSEY) and GENFIT
(Nasdaq and Euronext: GNFT) today announced full results from the
pivotal Phase III ELATIVE® trial, which are being presented in a
late-breaking oral session (Abstract #484, Monday, 13 November at
16.45 EST) at the American Association for the Study of Liver
Disease (AASLD) and simultaneously published in the New England
Journal of Medicine (NEJM). This trial evaluated the efficacy and
safety of investigational elafibranor, an oral, dual PPAR α,δ
agonist, as a potential novel class of treatment for patients with
the rare, autoimmune cholestatic liver disease, primary biliary
cholangitis (PBC).
Results show statistically significant
improvements in biomarkers of disease progression across key
endpoints with a significant treatment benefit achieved in the
primary composite endpoint, demonstrating a 47% placebo-adjusted
difference (P<0.001) between patients on elafibranor 80mg (51%)
compared with patients on placebo (4%) achieving a biochemical
response. In the trial, a biochemical response is defined as
alkaline phosphatase (ALP) <1.67 x upper limit of normal (ULN),
an ALP decrease ≥ 15 percent and total bilirubin (TB) ≤ ULN at 52
weeks. ALP and bilirubin are important predictors of PBC disease
progression. Reductions in levels of both can indicate reduced
cholestatic injury and improved liver function.
Only patients receiving elafibranor achieved
normalization of ALP (upper limit of normal 104 U/L in females and
129 U/L in males) at Week 52 (15% vs 0% placebo, P=0.002), a key
secondary endpoint of the trial. The significant biochemical effect
of elafibranor measured by ALP reduction was further supported by
data demonstrating reductions from baseline in ALP levels were
rapid, seen as early as Week 4 in the elafibranor group, and were
sustained through Week 52, with a decrease in ALP of 41% on
elafibranor compared with placebo.
“When managing PBC our first goal is to
effectively control the disease progression which can lead to liver
failure. The results from ELATIVE provide compelling evidence that
elafibranor has the potential to achieve this goal, with evidence
of a highly significant treatment benefit that is associated with
improved clinical outcomes,” said Dr Christopher Bowlus, Professor
of Gastroenterology and Hepatology, University of California Davis,
U.S. “In addition, our patients need relief from the significant
symptom burden of PBC, particularly those with moderate to severe
itch. Data from ELATIVE demonstrated the possibility of improved
pruritus for patients taking elafibranor compared with those on
placebo. Taken together, these data suggest elafibranor could offer
an effective new treatment opportunity for PBC management.”
ELATIVE investigated the effect of treatment
with elafibranor on pruritus (severe itch) across three separate
patient-reported outcome measures. On the key secondary endpoint
using the PBC Worst Itch NRS score, the reduction of pruritus
observed for elafibranor versus placebo was not statistically
significant (LS mean, –1.93 versus –1.15; difference, –0.78;
95% CI, –1.99 to 0.42; P=0.20). Two other secondary
patient-reported outcome measures were used to assess itch, and
greater reductions in pruritus were observed with elafibranor
compared with placebo at Week 52, according to the itch domain of
PBC-40 quality of life questionnaire (LS mean difference -2.3; 95%
CI, -4.0 to -0.7) and 5-D Itch total score (LS mean difference,
-3.0; 95% CI, -5.5 to -0.5).
“We believe these data suggest that elafibranor
could be a paradigm-changing treatment meeting the unmet need for
an effective second-line option,” said Christelle Huguet, EVP and
Head of Research and Development, Ipsen. “These data from ELATIVE
have provided a better understanding of how we can effectively
manage both disease progression and the symptom burden still
experienced by many people living with PBC. It would not have been
possible for us to investigate the potential for new innovative
treatments without the involvement of the patients and their wider
families and caregivers, to whom we are immensely grateful. We are
also enormously grateful to the study investigators, who have
supported us and provided us with the benefit of their expertise in
designing and running this study.”
PBC is a rare, autoimmune, cholestatic liver
disease, affecting approximately nine women for every one man. A
build-up of bile and toxins (cholestasis) and chronic inflammation
causes irreversible fibrosis (scarring) of the liver and
destruction of the bile ducts. It is a life-long condition that can
worsen over time if not effectively treated, leading to liver
transplant and in some cases, premature death. PBC impacts
patient’s daily lives through debilitating symptoms including most
commonly pruritus and fatigue. Currently, there are no approved
treatments available that can effectively manage both disease
progression and life-impacting symptoms.
“Living with PBC can be very challenging for many people. The
fear of the disease progressing hangs over you, and you have to
manage as best you can with the daily symptom burden, symptoms that
can sometimes be so debilitating it takes every ounce of strength
to get through another day,” explained Mo Christie, Head of Patient
Services, PBC Foundation, UK. “As someone who is living with PBC, I
appreciate the need for clinicians, other patients, and families to
understand the condition and the impact that coming to terms with
living with an incurable condition can have on a person’s life. The
impact can be enormous, so it is vitally important to all aspects
of our lives that we can access knowledge, care and effective
medicines, when we see our clinicians.”
Elafibranor was well tolerated in the trial.
Similar percentages of patients in the treatment group and the
placebo group experienced adverse events, treatment-related adverse
events, severe or serious adverse events or adverse events leading
to discontinuation. Adverse events occurring in >10% of patients
and more frequently on elafibranor versus placebo included
abdominal pain, diarrhea, nausea, and vomiting. Elafibranor has a
well-documented safety profile across a broad patient population
and is consistent with cumulative safety data from past elafibranor
trials in other indications, including NASH.
Data from ELATIVE are being used to support
submissions for elafibranor as a treatment for PBC with regulatory
authorities worldwide.
ENDS
ELATIVEELATIVE is a
multi-center, randomized, double-blind, placebo-controlled Phase
III clinical trial, with an open-label long-term extension
(NCT04526665). ELATIVE is evaluating 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
ursodeoxycholic acid (UDCA), the existing first-line therapy for
PBC. The trial enrolled 161 patients who were randomized 2:1 to
receive 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.
ElafibranorElafibranor is a
novel, 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 liver disease. Concurrent
α,δ activation targets inflammation, cholestasis and fibrosis in
PBC. In 2019, elafibranor was granted a Breakthrough Therapy
Designation by the FDA in adults with PBC who have an inadequate
response to UDCA. Elafibranor has not received approval by
regulatory authorities anywhere in the world.
Ipsen Ipsen is a global,
mid-sized biopharmaceutical company focused on transformative
medicines in Oncology, Rare Disease and Neuroscience. With total
sales of €3.0bn in FY 2022, Ipsen sells medicines in over
100 countries. Alongside its external-innovation strategy, the
Company’s research and development efforts are focused on its
innovative and differentiated technological platforms located in
the heart of leading biotechnological and life-science hubs:
Paris-Saclay, France; Oxford, U.K.; Cambridge, U.S.; Shanghai,
China. Ipsen has around 5,300 colleagues worldwide and 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
GENFITGENFIT 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
For further information:
Ipsen
Contacts Investors |
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Craig
MarksVice President, Investor Relations+44 (0)7584 349 193
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Nicolas
Bogler Investor Relations Manager+33 6 52 19 98 92
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Media |
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Anna
GibbinsGlobal Head of Franchise Communications,Rare
Disease+44 (0)7717801900 Amy WolfVP, Head
of Corporate Brand Strategy & Communications+41 79 576 07 23
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Ioana
PiscociuSenior ManagerGlobal Media Relations+33 6 69 09 12
96 |
GENFIT contacts
GENFIT | Investors Tel: +33 3
2016 4000 | investors@genfit.com
PRESS RELATIONS | Media Stephanie Boyer – Press
relations | Tel: +33 3 2016 4000 | stephanie.boyer@genfit.com
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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
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