- Seven abstracts to be presented demonstrating treatment effects
of Bylvay® in two cholestatic liver diseases, progressive familial
intrahepatic cholestasis and Alagille syndrome
- Data to be presented from a qualitative trial on patient
validation of pruritus (itch) and fatigue assessment tools for
people living with primary biliary cholangitis (PBC), used as part
of the ELATIVE Phase III registrational trial for elafibranor
PARIS, FRANCE,
12 June
2023 – Ipsen (Euronext: IPN; ADR:
IPSEY) today announced that the company will present data from
across its growing rare liver disease portfolio, at the European
Association for the Trial of Liver (EASL) Congress 2023, 21-24 June
in Vienna, Austria. These include seven abstracts on new clinical
data being presented on Bylvay® (odevixibat) when used in patients
with progressive familial intrahepatic cholestasis (PFIC) and
Alagille syndrome (ALGS). In addition, an abstract on content
validation of patient-reported outcomes assessment tools, used with
patients with primary biliary cholangitis (PBC), is being
presented.
The Bylvay abstracts provide further
understanding of the treatment’s efficacy and safety profile, when
used in sub-groups of both paediatric and adult patients with PFIC.
The data report on outcomes including event free survival,
reduction in serum bile acids (sBAs), pruritus and other quality of
life outcomes, in long-term clinical trial and real-world settings.
In the investigational Bylvay indication for patients with ALGS,
new pooled data from the Phase III ASSERT trial and extension
studies demonstrate significant improvements in pruritus and sBAs
and sleep disturbances.
The patient-reported outcomes assessment tools,
include the PBC Worst Itch Numeric Rating Scale (PBC WI NRS) and
PROMIS Fatigue Short Form 7a (PFSF 7a), are being used to assess
symptoms in ELATIVE, a Phase III clinical trial of elafibranor, an
investigational therapy for PBC. The tools have been designed to
derive meaningful change thresholds among patients with PBC
experiencing symptoms like pruritus (itch) and fatigue. PBC is a
disease where symptoms can be debilitating for patients and have a
significant impact on their quality of life.1, 2
Finally, two abstracts will be presented on
pre-clinical data from two pipeline assets under investigation for
further cholestatic diseases.
“We look forward to presenting additional data
from our Phase III Bylvay trials in both PFIC and ALGS, in addition
to data supporting the validation of the patient report outcome
tools we are using in our Phase III trial ELATIVE, with
investigational elafibranor, amongst the scientific peer group
attending EASL 2023,” said Dr. Howard Mayer, Executive Vice
President and Head of Research and Development, Ipsen. “These data
highlight our ongoing focus on supporting the communities of
patients living with rare liver disease by better understanding
their needs and furthering research on potential treatment
options.”
Highlights from key data to be presented during
the EASL congress 2023 include:
- Analysis of long-term treatment
effects of Bylvay in children with PFIC compared to matched,
non-Bylvay treated patients from the NAtural Course and Prognosis
of PFIC and Effect of Biliary Diversion (NAPPED)
registry.
- Outcomes from a case trial focused
on the treatment of Bylvay in patients with PFIC with the MYO5B
mutation.
- Discoveries from a subgroup in the
PEDFIC 2 trial, showing Bylvay’s efficacy and tolerability in
adults with PFIC.
- A real-world case series
demonstrating Bylvay’s effectiveness and safety in adults with
genetic cholestasis disorders.
- Pooled data from ASSERT Phase III
and ASSERT-EXT studies, showing efficacy and safety outcomes after
36-weeks of treatment.
- Insights from AS03969 and A3907,
early stage ASBT inhibitors, in development for adult liver
diseases.
- Trial results on the relevance and
importance of the Primary Biliary Cholangitis Worst Itch Numerical
Rating Scale (PBC WI NRS) and PROMIS Fatigue Short Form 7a (PFSF
7a), two clinical outcome assessment tools for symptomatic patients
living with PBC.
+++
Full presentation
details:
Poster (Abstract
#1511): Analysis of long-term treatment
effects of odevixibat on clinical outcomes in children with
progressive familial intrahepatic cholestasis in odevixibat
clinical studies vs external controls from the NAPPED
database.Presenter: Bettina Hansen, Department of
Epidemiology & Biostatistics, Erasmus MC, Rotterdam, the
Netherlands; Toronto Centre for Liver Disease & TGHRI,
University Health Network, Canada; IHPME, University of Toronto,
Toronto, CanadaSession title: Poster - Rare liver
diseasesDate and time: June 22, 2023 |
9:00-18:30
Poster (Abstract
#912): Efficacy and safety outcomes with
odevixibat treatment: Pooled data from the Phase 3 ASSERT and
ASSERT-EXT studies in patients with Alagille syndrome
Session title: Poster - Rare liver
diseasesDate and time: June 22, 2023 |
9:00-18:30
Poster
(Abstract
#1031): Outcomes in adult
patients with progressive familial intrahepatic cholestasis treated
with odevixibat: subgroup analysis from the PEDFIC 2
trialPresenter: Henkjan Verkade, Department of
Paediatrics, University of Groningen, Beatrix Children’s
Hospital/University Medical Centre Groningen, Groningen, the
NetherlandsSession title: Poster - Rare liver
diseasesDate and time: June 22, 2023 |
9:00-18:30
Poster (Abstract
#1039): Real-world experience of odevixibat in
adults with genetic disorders of cholestasis
Presenter: Palak Trivedi, National Institute for
Health Research Birmingham Biomedical Research Centre, Centre for
Liver and Gastrointestinal Research, University of Birmingham,
Birmingham, UKSession title: Poster - Rare liver
diseasesDate and time: June 22, 2023 |
9:00-18:30
Poster (Abstract
#1013): Long-term efficacy and safety of
odevixibat in patients with progressive familial intrahepatic
cholestasis: results with 96 weeks or more of
treatmentPresenter: Ekkehard Sturm, Paediatric
Gastroenterology and Hepatology, University Children’s Hospital
Tübingen, Tübingen, GermanySession title: Poster -
Rare liver diseasesDate and time: June 22, 2023 |
9:00-18:30
Poster (Abstract
#1017): Odevixibat therapy following liver
transplantation in patients with FIC1-deficient progressive
familial intrahepatic cholestasis: a retrospective case
seriesPresenter: Georg-Friedrich Vogel, Department
of Paediatrics I, Medical University of Innsbruck, Innsbruck,
Austria; Institute of Cell Biology, Medical University of
Innsbruck, Innsbruck, AustriaSession title: Poster
- Rare liver diseasesDate and time: June 22, 2023
| 9:00-18:30
Poster (Abstract
#1323): Odevixibat therapy in patients with MYO5B
mutations: a retrospective case
seriesPresenter: Emmanuel
Gonzalès, Hépatologie et Transplantation Hépatique Pédiatriques,
Hôpital Bicêtre, Paris, FranceSession title:
Poster - Rare liver diseasesDate and time: June
22, 2023 | 9:00-18:30
Poster (Abstract
1067): Evaluating pruritus and fatigue in patients
with treatment-refractory primary biliary
cholangitisPresenters:
Peter Serafini, Director Global Market Access and Marwan Sleiman,
Global Medical Affairs Director, Ipsen Session
title: Rare liver diseases (including paediatric and
genetic)Date and time: 22nd June, 09:00-18:00
CEST
Poster (Abstract
#1325): Inhibition of the renal apical
sodium-dependent bile acid transporter prevents cholemic
nephropathyPresenter: Ahmed Ghallab, Department of
Toxicology, Leibniz Research Centre for Working Environment and
Human Factors, Technical University Dortmund, Ardeystr 67, 44139,
Dortmund, GermanySession title: Poster - Rare
liver diseasesDate and time: June 22, 2023 |
9:00-18:30
Oral
(ID OS-074-YI): A3907, a systemic
ASBT inhibitor, improves cholestasis in mice by inhibiting
multi-organ bile acid transport and shows translational relevance
to humansPresenter: Francisco J. Caballero-Camino,
Department of Liver and Gastrointestinal Diseases, Biodonostia
Health Research Institute – Donostia University Hospital,
University of the Basque Country (UPV/EHU), San Sebastian,
SpainSession title: Immune-mediated and
cholestatic diseasesDate and time: June 24, 2023 |
15-15:15
Follow Ipsen on Twitter via @IpsenGroup and keep
up to date with EASL news and updates by using the hashtag
#EASLCongress
ENDS
About the Phase
III PEDFIC
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
trial. 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
trial revealed that PFIC negatively affects caregivers’ quality of
life, relationships, and career prospects.
About the Phase
III ASSERT trialASSERT
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 trial 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
trial, as defined as at least a 1-point drop at any time point. The
trial 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 trial, 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)Bylvay
is a potent, once-daily, non-systemic IBATi that acts locally in
the small intestine and has minimal systemic exposure. It is
approved in the U.S. for the treatment of pruritus in patients
three months of age and older with 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 the PBC trial:
Evaluating pruritus and fatigue in patients with
treatment-refractory primary biliary
cholangitisSemi-structured qualitative interviews were
conducted with 20 patients (aged 28-68 years; 19 females) diagnosed
with PBC, (mean 10.7 years since diagnosis) experiencing pruritus
(mild [30%], moderate [45%] or severe [25%]), using Institutional
Review Board-approved materials. The PFSF 7a consists of 7 items
that measure both the experience of fatigue and interference of
fatigue on daily activities over the past 7 days using a Likert
response scale. Patients were asked to evaluate the PBC WI NRS and
PFSF 7a on ease of understanding of instructions and items, ease of
use of scale/response options, and appropriateness of recall period
to capture the patient experience. Interviews were conducted by
experienced qualitative researchers, and audio recordings were
transcribed and analyzed with coding software.
About
ElafibranorElafibranor is a novel, oral,
once-daily, dual peroxisome activated receptor (PPAR) alpha/delta
(α,δ) agonist, currently under investigation as treatment for
patients with PBC, a rare liver disease. In 2019, it was granted a
Breakthrough Therapy designation by the FDA in adults with PBC who
have an inadequate response to ursodeoxycholic acid (UDCA).
Elafibranor has not received approval by regulatory authorities
anywhere in the world.
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 5,400 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
References:
- Mells GF et al. Hepatology.
2013 ; 58 : 273-283
- Poupon RE et al. Hepatology.
2004 ; 40(2) : 489-494
For further information:
Ipsen
Contacts Investors |
|
Craig
MarksVice President, Investor Relations+44 (0)7584 349
193 |
|
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
ManagerGlobal Media Relations+33 6 69 09 12 96 |
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- Ipsen PR_EASL_2023_12062023
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