Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage rare liver
disease company developing novel bile acid modulators, today
announced the initiation of its global Phase 3 pivotal trial,
ASSERT, Alagille Syndrome looking at Safety and Efficacy in a
Randomized controlled Trial, which will evaluate odevixibat in
patients with Alagille syndrome. Odevixibat is a potent,
once-daily, non-systemic ileal bile acid transport inhibitor
(IBATi) being investigated for the treatment of rare pediatric
cholestatic liver diseases, including progressive familial
intrahepatic cholestasis (PFIC), biliary atresia and Alagille
syndrome (ALGS). ASSERT is Albireo’s third global trial in rare
cholestatic liver conditions and furthers the Company’s efforts to
deliver life-changing therapies to children and young adults living
with these diseases.
ALGS is a rare multisystem genetic disorder that can affect the
liver, heart and other parts of the body. 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. Currently, there is
no approved drug therapy for the treatment of ALGS.
ASSERT is a gold standard, prospective intervention trial. The
double-blind, randomized, placebo-controlled trial is designed to
evaluate the safety and efficacy of 120 µg/kg/day odevixibat for 24
weeks in relieving pruritus in patients with ALGS. Secondary
endpoints will measure serum bile acid levels and safety and
tolerability. Both the U.S. Food and Drug Administration (FDA) and
European Medicines Agency (EMA) have agreed on the study design and
have indicated that a single study demonstrating safety and
efficacy of odevixibat would be sufficient for regulatory
filings. The trial is expected to enroll approximately 45
patients aged 0 to 17 years of age with a genetically confirmed
diagnosis of ALGS across 35 sites in North America, Europe, Middle
East and Asia Pacific.
“Odevixibat is the first IBAT inhibitor to have demonstrated
efficacy and tolerability in a Phase 3 randomized,
placebo-controlled trial, and this gives us increased confidence
for positive clinical outcomes in Alagille syndrome with
ASSERT,” said Ron Cooper, President and Chief Executive
Officer of Albireo. “We are pleased to initiate the ASSERT study
within guidance and offer hope to children and young adults around
the globe with Alagille syndrome who have no approved therapeutic
options today.”
Albireo recently submitted for a New Drug Application (NDA) to
the U.S. FDA and a Marketing Authorization Application (MAA) to the
EMA seeking approval of odevixibat for the treatment of patients
with PFIC. Odevixibat has previously received Fast Track, Rare
Pediatric Disease and Orphan Drug Designations in the U.S. In
addition to PFIC, odevixibat has Orphan Drug Designations for the
treatment of Alagille syndrome, biliary atresia and primary biliary
cholangitis. The EMA has granted odevixibat accelerated
assessment, 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 odevixibat Pediatric
Investigation Plans for PFIC and biliary atresia. With U.S. and EU
regulatory submissions for odevixibat in PFIC completed, the
Company anticipates potential regulatory approvals, issuance of a
rare pediatric disease priority review voucher and launch in the
second half of 2021.
Odevixibat is also currently being evaluated in the ongoing
PEDFIC 2 Phase 3 open-label trial in patients with PFIC, and the
BOLD Phase 3 trial in patients with biliary atresia. The Company
provides an Expanded Access Program for eligible patients with PFIC
in the U.S., Canada, Australia and Europe.
About Alagille SyndromeAlagille syndrome (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 ALGS present with chronic
cholestasis, usually within the first three months of life, and up
to 88% also present with severe, intractable pruritus. Currently,
there are no approved drug treatments.
About PFICProgressive familial intrahepatic
cholestasis (PFIC) is a rare disorder that causes progressive,
life-threatening liver disease. Patients have impaired bile flow,
or cholestasis, caused by genetic mutations. The resulting bile
build-up in liver cells causes liver disease and symptoms. The most
prominent and problematic ongoing manifestation of the disease is
pruritus, or intense itching, which often results in a severely
diminished quality of life. Other symptoms include jaundice, poor
weight gain and slowed growth. In many cases, PFIC leads to
cirrhosis and liver failure within the first 10 years of life, and
nearly all people with PFIC require treatment before age 30. There
are no drugs currently approved for PFIC, only surgical options
that include partial external biliary diversion (PEBD) and liver
transplantation. Additional information on PFIC is available at
https://www.pficvoices.com.
About Biliary AtresiaBiliary atresia is a rare
pediatric liver disease with symptoms typically developing about
two to eight weeks after birth. Damaged or absent bile ducts
outside the liver result in bile and bile acids being trapped
inside the liver, quickly resulting in cirrhosis and even liver
failure. Children have clay-colored or no color in their stools and
jaundice, among other things, and a few patients are pruritic.
Biliary atresia is the most common pediatric cholestatic liver
disease and is the leading cause of liver transplants among
children as there are no approved drug treatments.
About OdevixibatOdevixibat is an
investigational product candidate being developed to treat rare
pediatric cholestatic liver diseases, including progressive
familial intrahepatic cholestasis (PFIC), biliary atresia and
Alagille syndrome. A potent, once-daily, non-systemic ileal bile
acid transport inhibitor (IBATi), odevixibat acts locally in the
small intestine. Odevixibat 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. Odevixibat is
currently being evaluated in the ongoing PEDFIC 2 open-label trial
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,
and other adult liver diseases and disorders. Albireo’s lead
product candidate, odevixibat, is being developed to treat rare
pediatric cholestatic liver diseases with Phase 3 pivotal trials in
PFIC, Alagille syndrome and biliary atresia. The Company completed
IND-enabling studies for new preclinical candidate A3907 this year
and plans to advance development in adult 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 odevixibat or any other Albireo product candidate or
program, including regarding expectations regarding the impact of
COVID-19 on our business and our ability to adapt our approach as
appropriate; the Phase 3 clinical program for odevixibat in
patients with PFIC, the pivotal trial for odevixibat in biliary
atresia (BOLD), and the pivotal trial for odevixibat 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 odevixibat
in PFIC, the pivotal trial for odevixibat in biliary atresia, the
pivotal trial for odevixibat in Alagille syndrome; the potential
approval and commercialization of odevixibat; discussions with the
FDA or EMA regarding our programs; the potential benefits or
competitive position of odevixibat or any other Albireo product
candidate or program or the commercial opportunity in any target
indication; the potential effects of odevixibat 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,” and 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: 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 odevixibat to
date, including findings in indications other than PFIC, will be
predictive of results from other clinical trials of odevixibat;
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 odevixibat
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 odevixibat, including
the pivotal program in biliary atresia or the pivotal program in
Alagille syndrome, 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., 857-272-6177
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