Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease
company developing novel bile acid modulators, today announced the
completion of patient enrollment in the ASSERT study, a Phase 3
pivotal trial of Bylvay (odevixibat) in patients with Alagille
syndrome (ALGS). Topline results are expected to be available by
the end of the year, consistent with guidance, and with the
enrollment of 52 patients versus an original target of 45. ASSERT
is a gold standard, global, double-blind, randomized,
placebo-controlled trial designed to evaluate the safety and
efficacy of Bylvay in patients with ALGS over 24 weeks. Both the
U.S. FDA and EMA have agreed on the Phase 3 study design and have
indicated that this single study would be sufficient for regulatory
filings. Bylvay is a potent, once-daily, non-systemic ileal bile
acid transport inhibitor (IBATi) already 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.
“Families coping with Alagille syndrome are deeply in need of
more therapeutic options, as evidenced by the completed enrollment
for our Phase 3 ASSERT study, consistent with guidance, while
exceeding our original enrollment target,” said Ron Cooper,
President and Chief Executive Officer of Albireo. “Beyond this
important clinical milestone, we continue to deliver on our promise
to increase access to Bylvay for all eligible patients and are
pleased to announce the opening of an Expanded Access Program for
Alagille syndrome patients.”
ALGS is a rare, multisystem genetic disorder that can affect the
liver, heart, skeleton, eyes, central nervous system, kidneys and
facial features. Liver damage is caused by a paucity of bile ducts
preventing bile flow from the liver to the small intestine.
Approximately 95% of patients with the condition present with
chronic cholestasis, usually within the first three months of life,
and as many as 88% also present with severe, intractable
pruritus.
Aligned to the Company’s mission of providing hope for families,
Albireo continues to prioritize access and continued scientific
research for patients living with rare cholestatic liver diseases.
Albireo opened an Expanded Access Program (EAP) with the first
patient already enrolled. The program is available in the U.S. and
Europe, which aims to provide access to Bylvay for patients
suffering from ALGS, prior to the product’s planned approval and
reimbursement. Timing of availability in Europe will vary due to
country-specific regulations and stock availability. This program
is available for patients with a clinical diagnosis of ALGS who
have no other therapeutic options. For full eligibility criteria
and additional information, please contact
odevixibat@tannerpharma.com.
“I am proud of the work and great progress we are making in our
studies of Bylvay in rare cholestatic diseases,” said Jan Mattsson,
Chief Scientific Officer and Head of R&D at Albireo.
“Completing enrollment of the Phase 3 ASSERT study, while
continuing to enroll biliary atresia patients in the Phase 3 BOLD
study, are significant steps in our clinical development and
scientific leadership in bile acid modulation for the treatment of
liver diseases.”
The Company continues to enroll and dose patients in the Phase 3
BOLD study, which is the first and only pivotal trial of an IBATi
in biliary atresia, that passed the 50% enrollment milestone and
remains on track for topline data in 2024. Biliary atresia is the
most common pediatric cholestatic liver disease with no approved
drug treatment.
About ASSERT
ASSERT is a gold standard, prospective intervention trial with
35 sites across North America, Europe, Middle
East and Asia Pacific. The double-blind, randomized,
placebo-controlled trial is designed to evaluate the safety and
efficacy of 120 µg /kg/day Bylvay (odevixibat) for 24 weeks in
relieving pruritus in patients with ALGS. Secondary endpoints will
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. The primary efficacy
endpoint is a change from baseline in scratching to Month 6 (Weeks
21 to 24) as measured by the Albireo ObsRO caregiver instrument.
The key secondary efficacy endpoint is a change in serum bile acid
levels from baseline to the average of Week 20 and Week 24.
After completing the ASSERT trial, study participants have the
option to enroll in an extension study to continue receiving access
to Bylvay while it advances along the regulatory pathway. An EAP
program is also available to provide eligible patients with ALGS
with access to Bylvay (odevixibat) prior to the product’s approval
and reimbursement, subject to authorization by the relevant
competent authority. Learn more about Albireo’s commitment to
access at www.albireopharma.com/responsibility/access/.
EAP for ALGS
Albireo has partnered with Tanner Pharma Group for the ALGS EAP.
Eligible patients with ALGS, that are not in screening in the
ASSERT study, may receive Bylvay on a free-of-charge (FOC) basis,
subject to authorization by the relevant country competent
authority, and meeting of Albireo’s eligibility criteria. If you
are a physician who would like to request ALGS EAP access for your
patient, please send your enquiry to Tanner using
odevixibat@tannerpharma.com, and you will receive a response within
one working day with further information.
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 Albireo
Albireo 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 the England, Wales and Northern Ireland UK. 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. The Boston Business
Journal named Albireo one of the 2019 and 2020 Best Places to Work
in Massachusetts. 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, A3907, A2342
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); the Phase 2 study for A3907 the
IND-enabling or clinical studies for 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, the
Phase 2 study for A3907, and the IND-enabling and clinical studies
for A2342; expectations that biliary atresia is the most common
pediatric cholestatic liver disease with no approved drug
treatment; 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;
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: 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 Phase 2 clinical trial of
A3907, 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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