Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage rare liver
disease company developing novel bile acid modulators, today
announced the presentation of data at the European Association for
the Study of the Liver (EASL) International Liver Congress™ 2021,
being held virtually June 23 – 26. Data to be presented includes
two oral presentations and seven posters across studies in rare
pediatric cholestatic liver disease and in adult liver disease.
Highlights to include data from the Phase 3 PEDFIC 1 study and
PEDFIC 2 long-term extension study of Albireo’s lead product
candidate, Bylvay™ (odevixibat) and data from studies of its
product candidate, A3907. The full list of Albireo presentations is
listed below.
Pediatric Liver Presentations
Oral Presentation:
Rapid and Durable Effect of Odevixibat on Clinical and Biochemical
Parameters of Cholestasis in Children With Progressive Familial
Intrahepatic CholestasisPresenter: Dr. Richard J.
Thompson, Professor of Molecular Hepatology at King's College
London and principal investigator of the
studySession: Rare liver disease (including
paediatric and genetic)Date & Time: Friday,
June 25, 8:15 a.m. CEST
Poster PO-1811:
Odevixibat Effects on Cholestasis-Related Parameters: Analysis of
Pooled Data From the PEDFIC 1 and PEDFIC 2 Studies in Children With
Progressive Familial Intrahepatic
CholestasisPresenter: Dr. Richard J. Thompson,
Professor of Molecular Hepatology at King's College London and
principal investigator of the studySession: Rare
liver diseases (including paediatric and genetic) ePoster
SessionDate & Time: Friday, June 25, 11:30
a.m. CEST
Poster PO-1833:
Odevixibat Therapy Improves Clinically Meaningful Endpoints in
Children With Progressive Familial Intrahepatic Cholestasis: Data
From the PEDFIC 1 and PEDFIC 2 TrialsPresenter:
Lorenzo D’Antiga, M.D., Director of Child Health, Centre for
Paediatric Hepatology, Gastroenterology and Transplantation,
Hospital Papa Giovanni XXIII, Bergamo,
ItalySession: Rare liver diseases (including
paediatric and genetic)Date & Time: Wednesday,
June 23, 8:00 a.m. CEST
Poster PO-1665:
Substantial Clinical Benefits With Odevixibat Treatment Across
Progressive Familial Intrahepatic Cholestasis Genetic Deficiencies:
Subgroup Analysis of Serum Bile Acids, Pruritus, and Safety Using
Pooled Data From the PEDFIC 1 and 2
StudiesPresenter: Florence Lacaille, M.D.,
Pediatric Gastroenterology-Hepatology-Nutrition Unit, Hôpital
Universitaire Necker-Enfants MaladesSession: Rare
liver diseases (including paediatric and genetic)Date &
Time: Wednesday, June 23, 8:00 a.m. CEST
Poster PO-1641:
Long-term Treatment With Odevixibat Improves Multiple Sleep
Parameters in Patients With Progressive Familial Intrahepatic
Cholestasis: A Pooled Responder Analysis From the Phase 3 PEDFIC
StudiesPresenter: Dr. Richard J. Thompson, M.D.,
Professor of Molecular Hepatology at King's College London and
principal investigator of the studySession: Rare
liver diseases (including paediatric and genetic)Date &
Time: Wednesday, June 23, 8:00 a.m. CEST
Poster PO-1843:
Pretreatment Serum Bile Acid Parameters and Predictability of
Response to Odevixibat, an Ileal Bile Acid Transporter Inhibitor,
in Children With Progressive Familial Intrahepatic
CholestasisPresenter: Henkjan Verkade, M.D.,
Ph.D., Professor of Pediatrics at the University Medical Center
GroningenSession: Rare liver diseases (including
paediatric and genetic)Date & Time: Wednesday,
June 23, 8:00 a.m. CEST
Poster PO-1722:
Epidemiology and burden of Progressive Familial Intrahepatic
Cholestasis: Systematic ReviewPresenter: Velichka
Valcheva, M.D., M.Sc., Vice President EU Medical Affairs, Albireo
Pharma, Inc.Session: Rare liver diseases
(including paediatric and genetic)Date & Time:
Wednesday, June 23, 8:00 a.m. CEST
Adult Liver Presentations
Oral Presentation:
A3907, a Novel, Orally Bioavailable Inhibitor of the Apical
Sodium-Dependent Bile Acid Transporter, Improves Liver Injury in a
Mouse Model of Cholestatic Liver DiseasePresenter:
Peter Åkerblad, Ph.D., Senior Director of Early Development
R&D, Albireo Pharma, Inc.Session: Targeting
liver inflammation and fibrosisDate & Time:
Friday, June 25, 2:00 p.m. CEST
Poster PO-1849:
A3907, a Novel Orally Available Inhibitor of the Apical
Sodium-Dependent Bile Acid Transporter, Improves Key Clinical
Markers of Non-Alcoholic Steatohepatitis in Obese Diet-Induced and
Biopsy-Confirmed Mouse ModelsPresenter: Peter
Åkerblad, Ph.D., Senior Director of Early Development R&D,
Albireo Pharma, Inc.Session: NAFLD: Experimental
and pathophysiologyDate & Time: Wednesday,
June 23, 8:00 a.m. CEST
All posters will be available to ILC participants on June 21,
2021 at 10:00 AM CEST (4:00 AM EST).
About Bylvay
(odevixibat)Bylvay is an investigational
product candidate being developed to treat rare pediatric
cholestatic liver diseases, including PFIC, biliary atresia and
ALGS. A potent, once-daily, non-systemic ileal bile acid transport
inhibitor (IBATi), Bylvay acts locally in the small
intestine. Bylvay does not require refrigeration and can be
taken as a capsule for older children, or opened and sprinkled onto
food, which are factors of key importance for adherence in a
pediatric patient population.
The FDA has granted Priority Review and set a PDUFA goal date of
July 20, 2021. The European Medicines Agency's (EMA)
Committee for Medicinal Products for Human Use (CHMP) recently
issued a positive opinion recommending approval of Bylvay for the
treatment of PFIC. Bylvay is the only IBATi granted
accelerated assessment by the EMA. Bylvay also been granted Orphan
Designation, as well as access to the PRIority MEdicines (PRIME)
scheme for the treatment of PFIC. The EMA’s Pediatric Committee has
agreed to Albireo’s Bylvay Pediatric Investigation Plans for PFIC
and biliary atresia. In addition to PFIC, Bylvay has Orphan Drug
Designations for the treatment of Alagille syndrome, biliary
atresia and primary biliary cholangitis. With FDA and EMA
regulatory submissions complete, Bylvay has the potential to become
the first approved drug treatment for patients with PFIC in the U.S
and Europe. The Company anticipates potential regulatory approvals,
issuance of a rare pediatric disease priority review voucher and
launch in the second half of 2021.
The MAA and NDA filings are supported by results from PEDFIC 1
and PEDFIC 2 Phase 3 studies. PEDFIC 1 was the first and largest,
global, pivotal Phase 3 study conducted in PFIC, which evaluated
the efficacy and tolerability of Bylvay in reducing pruritus and
serum bile acids in a randomized, double-blind, placebo-controlled
trial. In the PEDFIC 1 study, Bylvay met both primary endpoints and
was well tolerated with very low incidence of diarrhea/frequent
bowel movements (9.5% of Bylvay treated patients vs. 5.0% of
placebo patients).
ir.albireopharma.com/news-releases/news-release-details/albireo-phase-3-trial-meets-both-primary-endpoints-odevixibat.
PEDFIC 2 is a long-term, open-label Phase 3 extension study. The
Company also provides an Expanded Access Program (EAP) for eligible
patients with PFIC in the U.S., Europe, Canada and Australia.
Bylvay is also currently being evaluated in the BOLD Phase 3 trial
in patients with biliary atresia, and the global Phase 3 ASSERT
trial for ALGS.
About AlbireoAlbireo Pharma is a clinical-stage
biopharmaceutical company focused on the development of novel bile
acid modulators to treat rare pediatric and adult liver diseases.
Albireo’s lead product candidate, Bylvay, is being developed to
treat rare pediatric cholestatic liver diseases with Phase 3 trials
in PFIC, Alagille syndrome and biliary atresia. For PFIC, the FDA
recently granted Priority Review and set a PDUFA goal date of July
20, 2021. In Europe, the EMA validated MAA. Bylvay is the only
IBATi granted accelerated assessment by the EMA. Bylvay has been
provisionally accepted by both the FDA and EMA as the brand name
for odevixibat. The Company has also initiated a Phase 1 clinical
trial for A3907 to advance development in adult cholestatic liver
disease, with IND-enabling studies moving ahead with A2342 for
viral and cholestatic liver disease. Albireo was spun out from
AstraZeneca in 2008 and is headquartered in Boston, Massachusetts,
with its key operating subsidiary in Gothenburg, Sweden. The Boston
Business Journal named Albireo one of the 2020 Best Places to Work
in Massachusetts for the second consecutive year. For more
information on Albireo, please visit www.albireopharma.com.
Forward-Looking Statements This press release
includes “forward-looking statements” within the meaning of the
Private Securities Litigation Reform Act of 1995. Forward-looking
statements include statements, other than statements of historical
fact, regarding, among other things: the plans for, or progress,
scope, cost, initiation, duration, enrollment, results or timing
for availability of results of, development of Bylvay or any other
Albireo product candidate or program; expectations regarding the
impact of the COVID-19 pandemic on our business and our ability to
adapt our plans and activities as appropriate; the pivotal trial
for Bylvay in biliary atresia (BOLD), and the pivotal trial for
Bylvay in Alagille syndrome (ASSERT); the target indication(s) for
development or approval, the size, design, population, location,
conduct, cost, objective, enrollment, duration or endpoints of any
clinical trial, or the timing for initiation or completion of or
availability or reporting of results from any clinical trial,
including the long-term open-label extension study for Bylvay in
PFIC, BOLD, ASSERT and the Phase 1 clinical trial for A3907; the
potential approval and commercialization of Bylvay and the timing
for such potential approval and commercialization; the potential
for Bylvay to become the first approved drug for PFIC patients;
discussions with the FDA or EMA regarding our programs; the
potential benefits or competitive position of Bylvay or any other
Albireo product candidate or program or the commercial opportunity
in any target indication; the potential effects of Bylvay of the
treatment of PFIC patients and its potential to improve the current
standard of care; the potential benefits of an orphan drug
designation; the potential issuance of a rare pediatric disease
priority review voucher; or Albireo’s plans, expectations or future
operations, financial position, revenues, costs or expenses.
Albireo often uses words such as “anticipates,” “believes,”
“plans,” “expects,” “projects,” “future,” “intends,” “may,” “will,”
“should,” “could,” “estimates,” “predicts,” “potential,” “planned,”
“continue,” “guidance,” or the negative of these terms or other
similar expressions to identify forward-looking statements. Actual
results, performance or experience may differ materially from those
expressed or implied by any forward-looking statement as a result
of various risks, uncertainties and other factors, including, but
not limited to: whether the NDA for Bylvay for the treatment of
pruritus in patients with PFIC will be approved by the FDA and
whether the MAA for Bylvay in PFIC will be approved by the EMA;
whether the FDA or EMA will complete their respective reviews
within the target timelines, including the FDA’s PDUFA goal date,
as a potential result of the impact of the COVID-19 pandemic or
otherwise; the risk that the NDA will not be approved despite the
FDA’s acceptance of the NDA for review or that the MAA will not be
approved despite CHMP’s opinion recommending approval of Bylvay for
the treatment of PFIC; whether the FDA or EMA will require
additional information, whether we will be able to provide in a
timely manner any additional information that the FDA or EMA
requests, and whether such additional information will be
satisfactory to the FDA and EMA; other potential negative impacts
of the COVID-19 pandemic, including on manufacturing, supply,
conduct or initiation of clinical trials, or other aspects of our
business; whether favorable findings from clinical trials of Bylvay
to date, including findings in indications other than PFIC, will be
predictive of results from other clinical trials of Bylvay; whether
either or both of the FDA and EMA will determine that the primary
endpoint for their respective evaluations and treatment duration of
the double-blind Phase 3 trial in patients with PFIC are sufficient
to support approval of Bylvay in the United States or the European
Union, to treat PFIC, a symptom of PFIC, a specific PFIC subtype(s)
or otherwise; the outcome and interpretation by regulatory
authorities of the ongoing third-party study pooling and analyzing
of long-term PFIC patient data; the timing for initiation or
completion of, or for availability of data from, clinical trials of
Bylvay or A3907, including BOLD and ASSERT, and the outcomes of
such trials; Albireo’s ability to obtain coverage, pricing or
reimbursement for approved products in the United States or
European Union; delays or other challenges in the recruitment of
patients for, or the conduct of, company’s clinical trials; and
Albireo’s critical accounting policies. These and other risks and
uncertainties that Albireo faces are described in greater detail
under the heading “Risk Factors” in Albireo’s most recent Annual
Report on Form 10-K or in subsequent filings that it makes with the
Securities and Exchange Commission. As a result of risks and
uncertainties that Albireo faces, the results or events indicated
by any forward-looking statement may not occur. Albireo cautions
you not to place undue reliance on any forward-looking statement.
In addition, any forward-looking statement in this press release
represents Albireo’s views only as of the date of this press
release and should not be relied upon as representing its views as
of any subsequent date. Albireo disclaims any obligation to update
any forward-looking statement except as required by applicable
law.
Media Contact:Colleen Alabiso,
857-356-3905, colleen.alabiso@albireopharma.comLisa Rivero,
617-947-0899, lisa.rivero@syneoshealth.com
Investor Contact:Hans Vitzthum, LifeSci
Advisors, LLC., 617-430-7578
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