Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company
developing novel bile acid modulators, today announced that it
presented analyses of its pivotal PEDFIC 1 and PEDFIC 2 studies
which evaluated Bylvay for the treatment of progressive familial
intrahepatic cholestasis (PFIC), at the Digestive Disease Week
Annual Meeting held May 21-24, 2022. Also presented at the meeting,
were new results from a preclinical study showing the potential of
the company’s investigational dual-acting ileal/kidney and liver
bile acid inhibitors to significantly increase bile acid excretion.
All abstracts are available on the DDW website.
“The PEDFIC 1 and PEDFIC 2 Phase 3 clinical trials represent the
largest body of data ever collected in PFIC, a rare disease that
causes tremendous suffering for patients and their families,” said
Ron Cooper, President and Chief Executive Officer of
Albireo. “We’re pleased to share data demonstrating Bylvay’s
efficacy on a wide range of all types of PFIC as well as positive
impact on all levels of pruritus in PFIC.”
Bylvay is a potent, non-systemic once daily ileal bile acid
transport inhibitor (IBATi) which is approved in the U.S. for the
treatment of pruritus in patients 3 months of age and older in all
types of PFIC, and in Europe for the treatment of all types of PFIC
in patients aged 6 months or older, based on the PEDFIC 1 study and
PEDFIC 2 open-label extension study.
Bylvay PEDFIC 1 & 2 Treatment DataThe
following data presentations at DDW provided analyses from PEDFIC
1, 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, and PEDFIC 2, a long-term,
open-label Phase 3 extension study.
E-poster #1229: Relationships Between Decreases
in Serum Bile Acids, Pruritus, and Sleep Disturbance Scores with up
to 72 Weeks of Odevixibat Treatment in Patients with Progressive
Familial Intrahepatic CholestasisLead Author: Dr.
Buket Dalgic, Department of Pediatric Gastroenterology, Gazi
University Faculty of Medicine
E-poster #1230: Efficacy and Safety of
Odevixibat in Children with Progressive Familial Intrahepatic
Cholestasis with Prior Partial External Biliary
DiversionLead Author: Dr. Binita M. Kamath,
Hospital for Sick Children and the University of Toronto
E-poster #1231: Odevixibat Therapy Improves
Clinically Meaningful Endpoints in Children with Progressive
Familial Intrahepatic Cholestasis: Data from the PEDFIC 1 and
PEDFIC 2 TrialsLead Author: Dr. Richard J.
Thompson, Institute of Liver Studies, King’s College London
E-poster #1232: Odevixibat Effects on
Cholestasis-Related Parameters: Analysis of Pooled Data from the
PEDFIC 1 and PEDFIC 2 Studies in Children with Progressive Familial
Intrahepatic CholestasisLead Author: Dr. Richard
J. Thompson, Institute of Liver Studies, King’s College London
E-poster #1233: 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 StudiesLead
Author: Dr. Richard J. Thompson, Institute of Liver
Studies, King’s College London
E-poster #1228: Disease Burden and Natural
History of Progressive Familial Intrahepatic Cholestasis: Baseline
Clinical Characteristics Among Odevixibat-Treated Patients in the
Phase 3 PEDFIC Studies Lead Author: Dr. Girish
Gupte, Liver Unit and Small Bowel Transplantation, Birmingham
Women’s and Children’s NHS Foundation Trust
Presentation on Dual-Acting Bile Acid Transport
Inhibitors E-poster #1327: Dual Acting
Ileal/Renal-Liver Bile acid Transporter Inhibitors Significantly
Increase Urinary Excretion of Non-Sulfated Bile Acids in a
Diethoxy-Carbonyl-Dihydro-Collidine-Induced Mouse Model of
Cholestasis Lead Author: Dr Anuradha Rao,
Department of Pediatrics, Emory University School of Medicine,
Atlanta.
“The ASBT and NTCP bile acid transporters play important roles
in maintaining bile acid homeostasis,” said Dr. Jan Mattsson,
Ph.D., Chief Scientific Officer and Co-Founder of Albireo. “This
study shows that dual-acting ASBT/NTCP with different selectivity
may represent an attractive strategy to reduce bile acid burden in
hepatobiliary diseases, reinforcing the potential of our A3907 and
A2342 programs as well as our earlier staged novel bile acid
modulators.”
About Bylvay (odevixibat)Bylvay is the first
drug approved in the U.S. for the treatment of pruritus
in patients 3 months of age and older in all types of progressive
familial intrahepatic cholestasis (PFIC). Limitation of Use: Bylvay
may not be effective in PFIC type 2 patients with ABCB11 variants
resulting in non-functional or complete absence of bile salt export
pump protein (BSEP-3). The European Commission (EC)
and UK Medicines and Healthcare Products Regulatory
Agency (MHRA) have also granted marketing authorization of
Bylvay for the treatment of PFIC in patients aged 6 months or
older. Bylvay is available in Germany and
the UK and will be available for sale in other European
countries following pricing and reimbursement approval. A potent,
once-daily, non-systemic ileal bile acid transport inhibitor,
Bylvay acts locally in the small intestine. Bylvay can be taken as
a capsule for patients that are able to swallow capsules, or opened
and sprinkled onto food, which is a factor of key importance for
adherence in a pediatric patient population. The most common
adverse reactions for Bylvay are diarrhea, liver test
abnormalities, vomiting, abdominal pain, and fat-soluble vitamin
deficiency. The medicine can only be obtained with a prescription.
For more information about using Bylvay, see the package leaflet or
contact your doctor or pharmacist. For full prescribing
information, visit www.bylvay.com.
In the U.S. and Europe, Bylvay has orphan
exclusivity for its approved PFIC indications, and orphan
designations for the treatment of ALGS, biliary atresia and primary
biliary cholangitis. Bylvay is being evaluated in the ongoing
PEDFIC 2 open-label trial in patients with PFIC, in the BOLD Phase
3 study for patients with biliary atresia and the ASSERT Phase 3
study for ALGS.
Important Safety Information
- The most common
adverse reactions for Bylvay are diarrhea, liver test
abnormalities, vomiting, abdominal pain, and fat-soluble vitamin
deficiency.
- Liver Test
Abnormalities: Patients should obtain baseline liver tests and
monitor during treatment. Dose reduction or treatment interruption
may be required if abnormalities occur. For persistent or recurrent
liver test abnormalities, consider treatment discontinuation.
- Diarrhea: Treat
dehydration. Treatment interruption or discontinuation may be
required for persistent diarrhea.
- Fat-Soluble Vitamin
(FSV) Deficiency: Patient should obtain baseline vitamin levels and
monitor during treatment. Supplement if deficiency is observed. If
FSV deficiency persists or worsens despite FSV supplementation,
discontinue treatment.
About AlbireoAlbireo Pharma is a rare
disease company focused on the development of novel bile acid
modulators to treat rare pediatric and adult liver diseases.
Albireo’s lead product, Bylvay, was approved by
the U.S. FDA as the first drug for the treatment of
pruritus in all types of progressive familial intrahepatic
cholestasis (PFIC), and it is also being developed to treat other
rare pediatric cholestatic liver diseases with Phase 3 trials in
Alagille syndrome (ALGS) and biliary atresia, as well as Open-label
Extension (OLE) studies for PFIC and ALGS. In Europe, Bylvay
has been approved for the treatment of PFIC with pricing listing
in Germany and guidance from the National Institute
for Health and Care Excellence (NICE) recommending Bylvay for
use in the National Health Service in England,
Wales and Northern Ireland. The Company has also
completed a Phase 1 clinical trial for A3907 to advance development
in adult cholestatic liver disease, with IND-enabling studies
progressing 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. 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: Albireo’s commercialization
plans; 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; the PEDFIC 2 open-label trial in patients with PFIC;
the pivotal trial for Bylvay in biliary atresia (BOLD); the pivotal
trial for Bylvay in Alagille syndrome (ASSERT); clinical trials for
A3907 and A2342, the target indication(s) for development or
approval; 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, the BOLD and ASSERT trials, or A3907 or A2342 trials,
potential regulatory approval and plans for potential
commercialization of Bylvay in additional countries; the potential
benefits or competitive position of Bylvay or any other Albireo
product candidate or program or the commercial opportunity in any
target indication; Bylvay’s funding for use in the National Health
Service in England, Wales and Northern Ireland; future price
listings and reimbursement approvals of Bylvay; the length of time
for which Albireo’s cash resources are expected to be sufficient;
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: there are no guarantees that Bylvay will be
commercially successful; we may encounter issues, delays or other
challenges in commercializing Bylvay; whether Bylvay receives
adequate reimbursement from third-party payors; the degree to which
Bylvay receives acceptance from patients and physicians for its
approved indication; challenges associated with execution of our
sales activities, which in each case could limit the potential of
our product; challenges associated with supply and distribution
activities, which in each case could limit our sales and the
availability of our product; results achieved in Bylvay in the
treatment of patients with PFIC may be different than observed in
clinical trials, and may vary among patients; 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; there is no guarantee that Bylvay
will be approved in jurisdictions or for indications beyond the
jurisdictions in which or indications for which Bylvay is currently
approved; there is no guarantee that our other products candidates
will be approved; estimates of the addressable patient population
for target indications may prove to be incorrect; 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, including BOLD and ASSERT and the
clinical trials of A3907 and A2342, and the outcomes of such
trials; Albireo’s ability to obtain coverage, pricing or
reimbursement for approved products in the United States or Europe;
delays or other challenges in the recruitment of patients for, or
the conduct of, the Company’s clinical trials; and the Company’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.comLance Buckley,
917-439-2241, lbuckley@lippetaylor.com
Investor Contact:Hans Vitzthum, LifeSci
Advisors, LLC., 617-430-7578
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