- Six abstracts to be presented
demonstrating efficacy and tolerability of investigational Bylvay
in select cholestatic liver diseases
- New data emphasizes the consistent
benefit of Bylvay as an investigational drug in Alagille syndrome
and an approved medicine in PFIC, with evidence of rapid,
sustained, and significant improvements in pruritus and sleep, and
reductions in serum bile acids (sBAs)
- Further data shows evidence of
disease modification with longer-term native liver survival in PFIC
patients
PARIS, FRANCE,
17 May 2023 –
Ipsen (Euronext: IPN; ADR: IPSEY) today announced that new data
from its growing rare disease portfolio will be presented at the
55th Annual Meeting of the European Society for Paediatric
Gastroenterology, Hepatology and Nutrition (ESPGHAN), taking place
in Vienna, Austria 17-20 May 2023. The six data presentations, made
up of four oral, one poster, and one e-poster, consistently
demonstrate the efficacy and tolerability of investigational
Bylvay® (odevixibat) for the potential treatment of cholestatic
liver disease patients with Alagille syndrome (ALGS) and the
treatment of patients with progressive familial intrahepatic
cholestasis (PFIC).
“This new data continues to build evidence in
support of the potential impact Bylvay can have on those living
with cholestatic liver diseases, such as ALGS and PFIC, where we
have investigational findings of significant improvements in
pruritus and sleep, and reductions in sBAs. In PFIC, longer native
liver survival suggests there may be a disease-modifying benefit to
this treatment,” said Dr. Howard Mayer, Executive Vice President
and Head of Research and Development, Ipsen.
Highlights from key investigational data on
Bylvay to be presented during the 55th Annual Meeting of ESPGHAN
include:
- Additional data from the ASSERT
Phase III and ASSERT-EXT studies showing Bylvay demonstrated rapid,
sustained, and highly significant improvements in pruritus,
reductions in sBAs, and improvements in sleep in patients with
Alagille syndrome.
- Findings exploring Bylvay usage in
association with PFIC patients keeping their native livers for up
to three years – additional data from the PEDFIC 1 and PEDFIC 2
studies.
- Discoveries from a study with
real-world data exploring diarrhea and quality of life issues in
PFIC1 patients post liver transplant and the impact of Bylvay
usage.
Follow Ipsen on Twitter via @IpsenGroup and keep
up to date with ESPGHAN news and updates by using the hashtag
#ESPGHAN23.
Presentations
Oral (Abstract
#328): Efficacy and
Safety of Odevixibat in Patients with Alagille Syndrome: Top-line
Results from Assert, A Phase III, Double-blind, Randomized,
Placebo-controlled Study Presenter: Dr. Nadia
Ovchinsky, Professor of Pediatrics, Hassenfeld Children’s Hospital
at NYU Langone, NYU Grossman School of MedicineSession
Title: Plenary Session: Highest Scoring
AbstractsDate & Time: 05/18 | 11:45-13:15 |
Hall A
Oral (Abstract
#361): Efficacy and
Safety of Odevixibat in Patients with Alagille Syndrome: Interim
Results from The Open-label, Phase III Assert-EXT
Study Presenter: Dr. Nadia Ovchinsky,
Professor of Pediatrics, Hassenfeld Children’s Hospital at NYU
Langone, NYU Grossman School of MedicineSession
Title: Parallel Session Hepatology – Abstract Session
02Date & Time: 5/19 | 12-13 | Hall G
Oral (Abstract
#369): Native Liver
Survival in Odevixibat Serum Bile Acid Responders: Data from the
PEDFIC Studies in Patients with Progressive Familial Intrahepatic
Cholestasis Presenter:
Prof. Richard J. Thompson, Molecular Hepatology, Institute of Liver
Studies, King’s College London Session Title:
Plenary Session: Highest Scoring AbstractsDate &
Time: 5/19 | 8:30-10 | Hall A
Oral (Abstract
#179): Odevixibat
Treatment Induces Biliary Bile Acid Secretion in Responsive
Patients with Bile Export Pump Deficiency
(PFIC2)Presenter: Dr. Mark
Nomden, Department of Pediatric Surgery, Department of Pediatrics,
University of Groningen, University Medical Center Groningen,
Groningen, The NetherlandsSession
Title: Parallel Session: Hepatology – Abstract
Session 02Date & Time: 5/19 | 12-13 |
Hall G
Poster (Abstract #579): Odevixibat Treatment in
a Patient with Undefined Cholestasis and No Unified Genetic
Diagnosis: A Case
Report Presenter: Dr. Tassos
Grammatikopoulos, Institute of Liver Studies, King’s College
LondonSession Title: Paper Poster
ViewingDate & Time: Available for viewing
during Exhibition opening hours onsite
E-Poster (Abstract
#805): Odevixibat
Therapy After Liver Transplantation in Patients with FIC1-Deficient
Progressive Familial Intrahepatic Cholestasis and Severe Diarrhea:
A Retrospective Case Series Presenter: Dr.
Georg-Friedrich Vogel, Department of Paediatrics I and Institute of
Cell Biology, Medical University of Innsbruck Session
Title: E-Poster Presentations: HEP -
TransplantationDate & Time: 5/20 |
11:50-12:40 | E-Poster Station 1
About the Phase
III PEDFIC & ASSERT
StudiesThe PEDFIC trials represent the largest studies
ever completed in children with PFIC, or progressive familial
intrahepatic cholestasis, a rare genetic disorder that causes
progressive, life-threatening liver disease. PEDFIC 1 was a
randomized, double-blind, placebo-controlled Phase III trial that
evaluated the efficacy and tolerability of Bylvay in reducing
pruritus and serum bile acids (sBAs) in children with PFIC, and
PEDFIC 2 is a long-term, open-label Phase III extension study.
Patients with PFIC have impaired bile flow, or cholestasis, and the
resulting bile build-up in liver cells causes liver disease and
symptoms, such as intense itching, poor sleep, delayed growth, and
diminished quality of life. The harmful impacts of the disease
extend to parents and caregivers, as the 2022 multinational PICTURE
study revealed that PFIC negatively affects caregivers’ quality of
life, relationships, and career prospects.
ASSERT is a gold standard, prospective
intervention trial with 32 sites across North America, Europe,
Middle East, and Asia Pacific. The double-blind, randomized,
placebo-controlled trial was designed to evaluate the safety and
efficacy of 120 µg /kg/day Bylvay for 24 weeks in relieving
pruritus in patients with ALGS. Key secondary endpoints measure
serum bile acid levels and safety and tolerability. The trial
enrolled patients aged 0 to 17 years of age with a genetically
confirmed diagnosis of ALGS. In the primary analysis, the study met
the primary endpoint showing statistically significant reduction in
pruritus as measured by the PRUCISION Observer-Reported Outcome
scratching score (0-4 point scale), from baseline at month 6 (weeks
21 to 24), compared to the placebo arm (p=0.002). Over 90% of
patients were pruritus responders during the study, as defined as
at least a 1-point drop at any time point. The study also met the
key secondary endpoint showing a statistically significant
reduction in serum bile acid concentration from baseline to the
average of weeks 20 and 24 (compared to the placebo arm p=0.001).
Statistically significant improvements in multiple sleep parameters
were observed as early as week 1-4 compared to patients on placebo
with continued improvement through week 24. In the study, there
were no patient discontinuations. Bylvay was well tolerated, with
an overall adverse event incidence similar to placebo and a low
incidence of drug-related diarrhea (11.4% vs. 5.9% placebo).
About Bylvay
(odevixibat)A
potent, once-daily, non-systemic ileal bile acid transport
inhibitor (IBATi), Bylvay has minimal systemic exposure and acts
locally in the small intestine. It is approved in the U.S. for the
treatment of pruritus in patients three months of age and older in
all types of PFIC, where it has orphan exclusivity. Bylvay was
first launched as a treatment option for patients with PFIC in the
U.S. in 2021, where it is supported by a program designed to assist
with access to treatment and patient support. Bylvay is also
approved in the E.U. for the treatment of PFIC in patients aged six
months or older. It has launched in over nine countries and has
secured public reimbursement across several major markets including
Germany, Italy, the U.K., France and Belgium.
View full E.U. prescribing information here:
Bylvay,
INN-odevixibat
(europa.eu)View full U.S. prescribing information
here: label (fda.gov)
About 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 5,000 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.
On March 3rd, 2023, Ipsen completed the
acquisition of Albireo Pharma Inc, a leading innovator in
bile-acid modulators to treat rare liver conditions, and the
marketing authorization holder of Bylvay.
For further information:
Contacts
Investors
Craig MarksVice President,
Investor Relations+44 (0)7584 349 193
Nicolas BoglerInvestor Relations Manager+33 6
52 19 98 92
Media
Anna GibbinsGlobal Head of
Franchise Communications, Rare Disease +44 (0)7717801900
Amy WolfVP, Head of Corporate
Brand Strategy & Communications+41 79 576 07 23
Ioana
PiscociuSenior Manager, Global Media Relations+33
6 69 09 12 96
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