Albireo Pharma, Inc. (Nasdaq: ALBO), a rare disease company
developing novel bile acid modulators to treat pediatric and adult
liver diseases, today announced the completion of patient
enrollment in the BOLD (
Biliary atresia and the
use of
Odevixibat in treating
Liver
Disease) study, a global
gold standard Phase 3 pivotal trial of Bylvay (odevixibat) in 205
patients with biliary atresia (BA) over 24 months. Topline results
are expected to be available by the end of 2024, consistent with
previous guidance. Recent discussions with health authorities
confirm that an outcome study would be necessary to achieve
approval or to fulfill the requirements of an accelerated filing.
Albireo has engaged in discussions with the FDA and EMA about the
BOLD Phase 3 study design; both have recently indicated that a
positive single study could be sufficient for approval.
“Enrolling 205 children suffering from biliary atresia into the
largest Phase 3 pediatric cholestasis trial ever is a significant
achievement allowing us to evaluate Bylvay in an unprecedented
number of patients across geographies,” said Jan Mattsson, Chief
Scientific Officer and Head of R&D at Albireo. “Biliary atresia
is the most common pediatric cholestatic liver disease and given
our recent successes with Bylvay Phase 3 studies in PFIC and
Alagille syndrome, we feel confident of a positive outcome and look
forward to being able to provide these children the first approved
drug treatment for biliary atresia.”
The BOLD study is the first and only global Phase 3 study of an
ileal bile acid transporter (IBAT) inhibitor in biliary atresia
with 58 clinical trial sites in 19 countries. BOLD is a
double-blind, randomized, placebo-controlled trial that measures
native liver survival with Bylvay over 24 months in children with
biliary atresia. Bylvay has received orphan drug designation for
biliary atresia in the United States and European Union.
“The completion of enrollment for the BOLD trial is a major
milestone for the children with biliary atresia and their
families. I and my pediatric hepatology colleagues from around
the world await completion of the trial in order to evaluate the
results. As such, we expect it to provide important insights into
the mechanisms of disease progression and potentially demonstrate
that we can change the outcome of this disease with a drug for the
first time,” said Saul J. Karpen, M.D., Ph.D., pediatric
hepatologist at Children’s Healthcare of Atlanta/Emory University
School of Medicine and lead investigator of the BOLD trial.
“With approximately 75% of biliary atresia patients needing a liver
transplant during childhood, the medical community is eager to have
a targeted therapeutic that directly addresses the intrahepatic
accumulation of bile acids. If this intervention improves the
response to Kasai portoenterostomy, it could ultimately delay or
prevent liver transplant for babies with this serious and
perplexing disease, one that is currently without any effective
medical therapies.”
Biliary atresia is a rare pediatric liver disease and yet the
most common pediatric cholestatic liver disease and the leading
cause of pediatric liver transplant across all diseases with
symptoms typically developing about two to eight weeks after birth.
Damaged or absent bile ducts result in bile and bile acids being
trapped inside the liver, quickly resulting in cirrhosis and even
liver failure. The disease impacts an estimated 45,000 people
around the globe and is the leading cause of liver transplants
among children. There are no approved pharmacological treatments
for biliary atresia.
“It is terrifying to receive a diagnosis of biliary atresia for
your newborn, and even worse, when you are informed that your child
will likely need a liver transplant. We are very excited that the
BOLD trial is fully enrolled, and we eagerly await the results,
hoping that families will finally have an option that could help
avoid a liver transplant,” said Jennifer Lau, Co-Founder and
President, BARE Inc.
Bylvay is a potent, oral, once-daily, non-systemic ileal bile
acid transport inhibitor (IBATi), with minimal systemic absorption
that acts locally in the small intestine. Bylvay is 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.
In addition, Bylvay met primary endpoint of improvement in pruritus
and key secondary endpoint of reduction in serum bile acids in a
recently announced Phase 3 ASSERT trial in patients with Alagille
syndrome.
About BOLD
BOLD (NCT04336722) is a double-blind, randomized,
placebo-controlled trial to evaluate the efficacy and safety of
odevixibat in children who have biliary atresia and have undergone
a Kasai procedure before the age of three months. Children in the
treatment arm receive odevixibat (120 μg/kg) orally once daily for
24 months. The primary efficacy endpoint is improvement in the
proportion of patients who are alive and have not undergone a liver
transplant after two years of treatment compared to placebo, and
secondary outcome measures include time to onset of any sentinel
events, total bilirubin levels and serum bile acid levels. The
trial enrolled 205 patients at 58 sites globally.
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. A potent,
once-daily, non-systemic ileal bile acid transport inhibitor,
Bylvay has minimal systemic exposure and 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 open-label trial 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 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 a completed Phase 3 trial in
Alagille syndrome (ALGS), an ongoing Phase 3 study in biliary
atresia, as well as Open-label Extension (OLE) studies for PFIC and
ALGS. In Europe, Bylvay is reimbursed for the treatment of PFIC in
Germany, England, Wales & Northern Ireland, Scotland, Italy,
and Belgium. 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 expected cash runway; 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 target indication(s) for development or
approval; the timing for anticipated regulatory filings;
discussions with the FDA or EMA regarding our programs; potential
regulatory approval and plans for potential commercialization of
Bylvay in biliary atresia or ALGS or Albireo’s other product
candidates; the potential benefits or competitive position of
Bylvay or any other Albireo product candidate or program or the
commercial opportunity in any target indication; 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 we will achieve a positive outcome in the BOLD trial on the
timeline we expect, and if so, whether results from the BOLD study
will be sufficient for approval by the FDA and EMA, or whether the
FDA and EMA will require additional information, whether we will be
able to provide in a timely manner any additional information that
the FDA and EMA request, and whether such additional information
will be satisfactory to the FDA and EMA; 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
or other approved indications may be different than observed in
clinical trials, and may vary among patients; 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 PFIC, ALGS and other
indications, 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 (such as biliary
atresia or ALGS) beyond the jurisdictions in which or indications
for which Bylvay is currently approved; there is no guarantee that
our other product 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 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; any repurchase by the Company of Sagard’s interest
in the royalty interest payments under our royalty monetization
agreement with Sagard could materially impact our financial
condition; 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 Contacts: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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