- New data
from the ELATIVE® Phase III trial
show 70% of patients treated with elafibranor achieved composite
endpoint of slowing disease progression measured by biochemical
response after 78-weeks
- Data from
the itch domain of the PBC-40 and 5-D Itch questionnaires shows the
potential of elafibranor to improve itch-related quality of life in
patients with moderate-to-severe pruritus
- Significant
unmet need remains for new treatment options in primary biliary
cholangitis that control disease progression and debilitating
symptoms impacting quality of life
PARIS, FRANCE, DD June 2024
Ipsen (Euronext: IPN; ADR: IPSEY) today announced new late-breaking
data at the European Association for the Study of the Liver (EASL)
Congress demonstrating the enduring efficacy of elafibranor in
managing disease progression after 78 weeks of treatment. In the
variable double-blind period of the ELATIVE® Phase III trial in
primary biliary cholangitis (PBC), the potential for elafibranor to
improve itch-related quality of life (QoL) as measured by the itch
domain of the PBC-40 and the 5-D Itch questionnaire was also
demonstrated. Elafibranor is a novel, potential first-in-class,
PPAR agonist. It is currently under review by the U.S. Food and
Drug Administration, the European Medicines Agency and the UK
Medicines and Healthcare Products Regulatory Authority.
“There are a significant proportion of people
living with PBC who experience worsening disease and debilitating
symptoms despite being on treatment. These long-term data from the
Phase III ELATIVE study further demonstrate the potential for
elafibranor to provide an effective treatment option for these
patients,” said Sandra Silvestri, M.D. Executive Vice President and
Chief Medical Officer, Ipsen. “A lack of effective management can
lead to advanced forms of the disease where liver transplantation
may be the only option. Transplants are not trivial, so we must and
can do better to preserve native liver function for people living
with PBC.”
Data presented at EASL for patients who had
their week-78 double-blind visit (30 patients receiving elafibranor
and 13 patients receiving placebo) demonstrated the efficacy of
elafibranor was sustained after 78 weeks of treatment with 70% of
patients on elafibranor meeting the composite endpoint of
biochemical response versus 0% on placebo. Biochemical response was
defined as alkaline phosphatase (ALP) <1.67 x upper limit of
normal (ULN), an ALP decrease ≥ 15 percent and total bilirubin (TB)
≤ ULN. ALP and bilirubin are important predictors of PBC disease
progression. Reductions in levels of both can indicate reduced
liver injury and improved liver function. ALP normalization for
patients on elafibranor was sustained out to week-78 as well as
across other important biomarkers of liver injury, including total
bilirubin and gamma glutamyl transferase.1
New patient-reported outcome data from ELATIVE
at week 52 were also presented, demonstrating the potential
beneficial effect of elafibranor on itch-related quality of life,
including sleep and functioning. Treatment with elafibranor led to
greater reductions in 5-D Itch score which comprises five domains
(degree, duration, dimension, disability and distribution) versus
placebo. A clinically meaningful reduction in the itch domain of
PBC-40 for elafibranor versus placebo was also observed, with a
greater proportion of patients treated with elafibranor
experiencing improvement in itch-related quality of life. These
include across measures of severity of itching, sleep disturbance
and emotional impact of itching, versus placebo. In the 5-D Itch
domain of duration, reduced itching was reported by 58% of patients
receiving elafibranor at week 52, compared with 27% on placebo.
Additionally, 80% of patients receiving elafibranor improved to no
sleep disturbance or only occasional delay, compared with 30% on
placebo. The improvements in 5-D Itch and PBC-40 Itch emphasize the
potential of elafibranor to reduce both the severity of PBC
symptoms and their impact on QoL.2
“When you have a patient with PBC, it’s vital to
manage disease progression, to prevent or delay liver damage or
failure. You also want to provide relief from distressing symptoms
because they can have a very detrimental impact on quality of
life,” said Dr. Christopher Bowlus, Professor of Gastroenterology
and Hepatology, University of California Davis, U.S. “These new
data from ELATIVE provide further evidence that elafibranor has the
potential to address the two priority treatment goals by
demonstrating longer-term improvements in the prognostic markers of
disease progression, as well as potential improvements in
pruritus-symptom severity and impacts on the quality of life.”
PBC is a rare, progressive, autoimmune
cholestatic liver disease, in which the body attacks and gradually
destroys the liver’s small bile ducts.3 If left untreated, bile and
toxins can build up, leading to scarring of the liver and eventual
liver failure.3-5 Symptoms of PBC can have a substantial impact on
a person’s QoL, including fatigue and itching.6,7 However, while
some people living with PBC may not display symptoms, they remain
at risk of disease progression and liver damage, making active
disease management vital.8
Ipsen also presented new data from its growing
rare cholestatic liver disease portfolio at EASL, including data on
its treatment for progressive familial intrahepatic cholestasis and
Alagille syndrome.
ENDS
About ELATIVE
ELATIVE 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. Patients continued their assigned treatment
after Week 52 until all patients had completed their treatment or
for a maximum of 104 weeks. Data was also collected during this
period, and additional analyses were conducted with a focus on Week
78.
About Ipsen
We are a global biopharmaceutical company with a
focus on bringing transformative medicines to patients in three
therapeutic areas: Oncology, Rare Disease and Neuroscience.
Our pipeline is fueled by external innovation
and supported by nearly 100 years of development experience and
global hubs in the U.S., France and the U.K. Our teams in more than
40 countries and our partnerships around the world enable us to
bring medicines to patients in more than 80 countries.
Ipsen 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.
Ipsen contacts
Investors
- Craig
Marks | + 44 7584 349 193
- Nicolas
Bogler | + 33 6 52 19 98 92
Media
- Amy Wolf
| + 41 79 576 07 23 | amy.wolf@ipsen.com
- Anna
Gibbins | + 44 7717 801900 | anna.gibbins@ipsen.com
Disclaimers and/or Forward-Looking
StatementsThe forward-looking statements, objectives and
targets contained herein are based on Ipsen’s management strategy,
current views and assumptions. Such statements involve known and
unknown risks and uncertainties that may cause actual results,
performance or events to differ materially from those anticipated
herein. All of the above risks could affect Ipsen’s future ability
to achieve its financial targets, which were set assuming
reasonable macroeconomic conditions based on the information
available today. Use of the words ‘believes’, ‘anticipates’ and
‘expects’ and similar expressions are intended to identify
forward-looking statements, including Ipsen’s expectations
regarding future events, including regulatory filings and
determinations. Moreover, the targets described in this document
were prepared without taking into account external-growth
assumptions and potential future acquisitions, which may alter
these parameters. These objectives are based on data and
assumptions regarded as reasonable by Ipsen. These targets depend
on conditions or facts likely to happen in the future, and not
exclusively on historical data. Actual results may depart
significantly from these targets given the occurrence of certain
risks and uncertainties, notably the fact that a promising medicine
in early development phase or clinical trial may end up never being
launched on the market or reaching its commercial targets, notably
for regulatory or competition reasons. Ipsen must face or might
face competition from generic medicine that might translate into a
loss of market share. Furthermore, the research and development
process involves several stages each of which involves the
substantial risk that Ipsen may fail to achieve its objectives and
be forced to abandon its efforts with regards to a medicine in
which it has invested significant sums. Therefore, Ipsen cannot be
certain that favorable results obtained during preclinical trials
will be confirmed subsequently during clinical trials, or that the
results of clinical trials will be sufficient to demonstrate the
safe and effective nature of the medicine concerned. There can be
no guarantees a medicine will receive the necessary regulatory
approvals or that the medicine will prove to be commercially
successful. If underlying assumptions prove inaccurate or risks or
uncertainties materialize, actual results may differ materially
from those set forth in the forward-looking statements. Other risks
and uncertainties include but are not limited to, general industry
conditions and competition; general economic factors, including
interest rate and currency exchange rate fluctuations; the impact
of pharmaceutical industry regulation and healthcare legislation;
global trends toward healthcare cost containment; technological
advances, new medicine and patents attained by competitors;
challenges inherent in new-medicine development, including
obtaining regulatory approval; Ipsen’s ability to accurately
predict future market conditions; manufacturing difficulties or
delays; financial instability of international economies and
sovereign risk; dependence on the effectiveness of Ipsen’s patents
and other protections for innovative medicines; and the exposure to
litigation, including patent litigation, and/or regulatory actions.
Ipsen also depends on third parties to develop and market some of
its medicines which could potentially generate substantial
royalties; these partners could behave in such ways which could
cause damage to Ipsen’s activities and financial results. Ipsen
cannot be certain that its partners will fulfil their obligations.
It might be unable to obtain any benefit from those agreements. A
default by any of Ipsen’s partners could generate lower revenues
than expected. Such situations could have a negative impact on
Ipsen’s business, financial position or performance. Ipsen
expressly disclaims any obligation or undertaking to update or
revise any forward-looking statements, targets or estimates
contained in this press release to reflect any change in events,
conditions, assumptions or circumstances on which any such
statements are based, unless so required by applicable law. Ipsen’s
business is subject to the risk factors outlined in its
registration documents filed with the French Autorité des
Marchés Financiers. The risks and uncertainties set out are not
exhaustive and the reader is advised to refer to Ipsen’s latest
Universal Registration Document, available on ipsen.com.
References
- Bowlus et al. J. Hepatol.
2024 ; 80(S1)S80
- Kremer et al. J. Hepatol. 2024;
80(S1)S91
- EASL. J Hepatol. 2017;
67(1):145-172.
- Younossi ZM, et al. Am J
Gastroenterol. 2019; 114(1):48–63.
- Galoosian A, et al. J Clin Transl
Hepatol. 2020; 8(1), pp. 49-60.
- Mells GF, et al. Hepatology. 2013; 58:
273-283.
- C Levy, et al. Abstract presented at
ISPOR, 7-11 May 2023, Boston.
- Prince MI, et al. Gut. 2004; 53(6),
pp.865-870.
- PR_ELATIVE EASL 2024_05062024
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