Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage rare liver
disease company developing novel bile acid modulators, today
announced progress in two key clinical trials designed to further
the Company’s efforts to deliver life-changing drugs to children
and adults living with rare liver diseases. Furthering the
Company’s goal of advancing multiple approaches for modulating bile
acids, Albireo initiated a Phase 1 study with a new compound,
A3907, an oral systemic apical sodium-dependent bile acid
transporter (ASBT) inhibitor. A3907 is being developed for adult
cholestatic liver diseases such as primary sclerosing cholangitis
(PSC) and primary biliary cholangitis (PBC).
Due to high oral bioavailability, A3907 can inhibit ASBT in the
intestine and kidney, with the potential to increase elimination of
bile acids by both fecal and urinary excretion. By using dual
pathway diversion of bile acids, next generation modulators like
A3907 seek to increase efficacy without the dose limiting diarrhea
seen with bile acid transport inhibitors today.
“Reaching a new milestone with the first patients dosed in our
Phase 1 A3907 study represents an important pipeline development
for Albireo, reinforcing our scientific leadership in bile acid
modulation and ambition to expand into adult liver disease,” said
Ron Cooper, President and Chief Executive Officer of Albireo.
“Simultaneously, we are focused on our ambition of building
odevixibat into a globally available billion-dollar product by the
end of the decade, and by dosing our first patient in the ASSERT
study we’ve shown great progress in the mission to provide a new
drug option to treat rare cholestatic liver diseases.”
The Phase 1 study is a first-in-human, double-blind, single and
multiple ascending dose study in healthy adult subjects to
investigate the safety, tolerability, pharmacokinetics and
pharmacodynamics of an A3907 oral formulation. In pre-clinical
studies, A3907 showed high systemic exposure and increased level of
urinary bile acid secretion in mice, and in a mice model of
cholestasis and sclerosing cholangitis, A3907 decreased serum bile
acids, and reduced plasma levels of transaminases as well as
markers for cell damage and fibrosis. Topline data for the Phase 1
study is anticipated in the second half of 2021, with subsequent
initiation of Phase 2 in 2022. Beyond A3907, the Company also
recently selected new development candidate A2342, an oral systemic
sodium-taurocholate co-transporting peptide (NTCP) inhibitor for
viral disease and cholestatic diseases and is moving ahead with
IND-enabling studies.
Additionally, the Company enrolled its first patient in the
ASSERT Study, a global Phase 3 pivotal trial of odevixibat in
patients with Alagille syndrome (ALGS). 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 ALGS. This
milestone is in keeping with Albireo’s plans to achieve full site
activation by mid-year, with topline data expected in 2022.
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.
“I am proud of the work and great progress we are making in our
studies of odevixibat in rare cholestatic diseases,” said Patrick
Horn, Chief Medical Officer of Albireo. “Enrolling our first
Alagille patient and continuing enrollment of biliary atresia
patients in the BOLD study with 46 sites active, while also
progressing A3907 from concept to the clinic, are significant steps
in developing our wholly owned pipeline of products to address
multiple liver indications.”
The U.S. Food and Drug Administration (FDA) granted Priority
Review to Albireo’s New Drug Application for odevixibat in PFIC
with a Prescription Drug User Fee Act (PDUFA) goal date of July
20th of this year, and the Company was informed that there are no
plans for an FDA advisory committee meeting. The European Medicines
Agency (EMA) has granted odevixibat accelerated assessment and
Orphan Designation, as well as access to the PRIority MEdicines
(PRIME) scheme for the treatment of PFIC. The EMA’s Pediatric
Committee has also agreed to Albireo’s odevixibat Pediatric
Investigation Plans for PFIC and biliary atresia. In addition to
PFIC, odevixibat has Orphan Drug Designations for the treatment of
Alagille syndrome, biliary atresia and primary biliary cholangitis.
Albireo anticipates potential regulatory approvals, issuance of a
rare pediatric disease Priority Review Voucher and launch in the
second half of 2021.
About A3907A3907 is a selective inhibitor of
the apical sodium-dependent bile acid transporter (ASBT) with a
dual mechanism of action. Due to oral bioavailability, A3907 acts
on both renal and ileal transporters to increase elimination of
bile acids by both fecal and urinary excretion. This dual
inhibition approach may yield increased efficacy without the dose
limiting diarrhea seen with bile acid transport inhibitors
today.
About Cholestatic Adult Liver DiseasesAdult
cholestatic diseases are a diverse group of disorders known for the
appearance of jaundice, fatigue, pruritus and/or complications of
cirrhosis. The most common adult cholestatic liver diseases are
primary biliary cholangitis and primary sclerosing cholangitis.
Primary biliary cholangitis is a chronic disease in which the bile
ducts in the liver are slowly destroyed. When the bile ducts are
damaged, bile can back up in the liver and sometimes lead to
irreversible scarring of liver tissue (cirrhosis). Primary
sclerosing cholangitis is a disease of the bile ducts where
inflammation causes scars within the bile ducts. These scars make
the ducts hard and narrow, gradually causing serious liver damage
that leads to liver failure, repeated infections and tumors of the
bile duct or liver.
About ASSERTASSERT 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 FDA
and 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. An additional
exploratory cohort of patients ≥18 years of age with genetically
confirmed diagnosis will be enrolled, not to exceed 18 patients in
total. 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. Key secondary efficacy endpoint is a
change in serum bile acid levels from baseline to the average of
Week 20 and Week 24.
About OdevixibatOdevixibat 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), 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. The FDA has granted
Priority Review and set a PDUFA goal date of July 20, 2021. In
Europe, the EMA validated MAA. Odevixibat is the only IBATi granted
accelerated assessment by the EMA.
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 odevixibat in reducing pruritus
and serum bile acids in a randomized, double-blind,
placebo-controlled trial. In the PEDFIC 1 study, odevixibat met
both primary endpoints and was well tolerated with very low
incidence of diarrhea/frequent bowel movements (9.5% of odevixibat
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.
Odevixibat 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, odevixibat, is being developed to
treat rare pediatric cholestatic liver diseases with Phase 3 trials
in PFIC, Alagille syndrome and biliary atresia. The Company has
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 odevixibat or any other Albireo product candidate or
program; including 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
odevixibat in biliary atresia (BOLD), and the pivotal trial for
odevixibat in Alagille syndrome (ASSERT); the Phase 1 trial for
A3907, 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 Phase 1 trial for A3907, the
potential approval and commercialization of odevixibat; the
potential for odevixibat 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 odevixibat,
A3907, A2342 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,” 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 odevixibat for the treatment of pruritus in
patients with PFIC will be approved by the FDA and whether the MAA
for odevixibat 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; whether the FDA will require additional
information, whether we will be able to provide in a timely manner
any additional information that the FDA requests, and whether such
additional information will be satisfactory to the FDA; 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 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 A3907 or 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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