Albireo Pharma, Inc. (Nasdaq: ALBO), a rare disease company
developing novel bile acid modulators to treat pediatric and adult
liver diseases, presented new data at the American Association for
the Study of Liver Diseases (AASLD) The Liver Meeting® 2022, being
held November 4 – 8, 2022 in Washington, D.C. Across three oral
presentations, including two late-breakers, and six posters, the
Company provided evidence of early, rapid, and sustained efficacy
with Bylvay (odevixibat) treatment in patients with progressive
familial intrahepatic cholestasis (PFIC) and Alagille syndrome
(ALGS).
Two key oral presentations on the PEDFIC trials provided
evidence of the disease modifying effects of Bylvay in patients
with PFIC. The first underscored that a decrease in serum bile
acids was strongly associated with native liver survival for up to
three years in PFIC patients treated with Bylvay. A second
late-breaking oral presentation showed that Bylvay restored bile
acid secretion in PFIC patients with bile salt export pump
deficiency. The late-breaking abstract was selected by AASLD for
inclusion as a key presentation in “The Best of The Liver Meeting”
in the Pediatric Hepatology category.
“We are pleased to share long term data in PFIC, providing
evidence that Bylvay could modify the course of disease, preserving
patients’ native liver and improving families’ quality of life by
alleviating disease burden,” said Jan Mattsson, Ph.D., Chief
Scientific Officer and Head of R&D at Albireo. “Furthermore,
showing early, rapid, and consistent improvements in pruritus and
bile acid levels in patients with Alagille syndrome, through the
ASSERT body of evidence, makes for a big year at this year’s AASLD
congress. PFIC and Alagille syndrome are devastating childhood
diseases and our goal at Albireo has always been to relieve the
suffering of these young patients and their families.”
“Patients with PFIC have long needed a disease modifying
treatment,” said Dr. Richard Thompson, Professor of Molecular
Hepatology at King’s College London, and principal investigator of
the PEDFIC 1 and PEDFIC 2 trials. “Sadly, most children with PFIC
end up having a liver transplant, with fewer than half of PFIC1
patients and less than a third of PFIC2 patients keeping their
native liver through their eighteenth birthday. I am encouraged by
long-term data that showed Bylvay reduced serum bile acids after
six months, and helped patients preserve their native liver. This
supports the importance of treating early with Bylvay so children
can benefit from the protective effects we are seeing on the
liver.”
Along with results observed with Bylvay treatment in PFIC
patients, Albireo shared the first detailed results of the Phase 3
ASSERT trial in Alagille syndrome (ALGS) in a late-breaking oral
presentation. The data showed that Bylvay provided early, rapid,
clinically meaningful, and sustained improvements in pruritus, as
well as significant reductions in bile acids and improvements in
sleep quality in patients with ALGS. The ASSERT abstract was
selected by AASLD for inclusion as a key presentation in “The Best
of The Liver Meeting” in the Pediatric Hepatology category.
Analyses of the ASSERT and PEDFIC trials and real-world data on
Bylvay treatment, as well as data on the investigational therapy
A3907, were highlighted in the following presentations at AASLD The
Liver Meeting:
Early, Rapid, Sustained Effects in Alagille
Syndrome
Oral, Late-Breaking Parallel Session Presentation
(Abstract #5005; Publication #38786): Efficacy and Safety
of Odevixibat in Patients with Alagille Syndrome: Top-Line Results
from ASSERT, a Phase 3, Double-Blind, Randomized,
Placebo-Controlled StudyPresenter: Dr. Nadia
Ovchinsky, Children’s Hospital at Montefiore, Albert Einstein
College of Medicine, Bronx, NY, USASession:
Late-Breaking Oral Abstract Session 1; Monday, November 7, 2022,
9:15 AM EST
Top-line, unblinded data from the ASSERT study demonstrated that
Bylvay treatment led to significant and clinically
meaningful improvements in pruritus, as well as reductions in
bile acid levels and improvements in sleep parameters in
patients with ALGS. Over 90% of patients were pruritus responders
and the treatment effects were early, rapid, and
sustained. Bylvay was generally well tolerated; the overall
incidence of treatment emergent adverse events (TEAEs) was similar
to placebo. No patients discontinued the study and 96% of
patients rolled over into the open-label extension study.
Disease Modification in PFIC
Oral Presentation (Abstract #865): Native Liver
Survival in Odevixibat Serum Bile Acid Responders: Data from the
PEDFIC Studies in Patients with Progressive Familial Intrahepatic
CholestasisPresenter: Dr. Richard J.
Thompson, Institute of Liver Studies, King’s College
LondonSession: Genes to Cures: What’s New in
Pediatric Liver Disease; Sunday, November 6, 2:10 PM EST
Pooled data analysis showed that PFIC patients who responded to
Bylvay remained liver transplant free for up to three years. A
decrease in serum bile acids (sBAs) at six months of treatment was
strongly associated with native liver survival (p=0.0050) for sBA
responders vs non-responders.
Oral, Late-Breaking Parallel Session
Presentation (Abstract #5004; Publication #38801):
Odevixibat Treatment in Responsive Patients with Bile Salt Export
Pump Deficiency Restores Biliary Bile Acid Secretion, as Indicated
by Serum Bile Acid CompositionPresenter: Dr. Mark
Nomden, Department of Surgery and Pediatrics, University Medical
Center Groningen, Groningen, the
NetherlandsSession: Late-Breaking Oral Abstract
Session 1; Monday, November 7, 2022, 10:00 AM EST
The analysis found that responders not only had a decrease in
sBA concentration, but also alternations in sBA composition, more
like that of a healthy individual. Data indicated that Bylvay
restored biliary bile acid secretion in treatment-responsive
patients with BSEP deficiency.
Durable Improvement Across Wide Range of PFIC
Patients
Poster (Abstract #37939): Long-term
Efficacy and Safety of Odevixibat in Patients with Progressive
Familial Intrahepatic Cholestasis: Results with 96 Weeks or More of
TreatmentPresenter: Dr. Richard J.
Thompson, Institute of Liver Studies, King’s College
LondonSession: Poster Session
IV; Monday, November 7, 1:00 PM – 2:00 PM
EST
Pooled data analysis showed Bylvay treatment for 96 weeks or
more was associated with durable clinical benefits in patients with
PFIC, with improvements over time in mean sBA and aminotransferase
levels and growth. Mean levels of alanine aminotransferase (ALT)
and aspartate aminotransferase (AST) decreased over time, while
mean total bilirubin levels were relatively unchanged. Mean height
and weight Z scores also increased over time and Bylvay was
generally well tolerated.
Poster (Abstract #37254): Serum Bile Acid
Levels, Pruritus Scores, and Growth Over Time in Odevixibat
Responders: Pooled Data from the PEDFIC Studies in Patients with
Progressive Familial Intrahepatic
CholestasisPresenter: Dr. Lorenzo
D'Antiga, Department of Paediatric Hepatology,
Gastroenterology, and Transplantation, Azienda Ospedaliera Papa
Giovanni XXIII, Bergamo, ItalySession: Poster
Session IV; Monday, November 7, 1:00 PM – 2:00 PM
EST
This analysis of pooled data examined treatment effects in 49
Bylvay responders who had a mean duration of exposure of 110 weeks
and showed mean reductions in sBAs and pruritus were sustained over
time, across PFIC types in general. Patients also had mean
improvements in growth.
Poster (Abstract #37273): Effect of
Odevixibat in Patients with Progressive Familial Intrahepatic
Cholestasis Type 2 with at Least 1 Severe Mutation (BSEP3 Compound
Heterozygotes): Pooled Data from the PEDFIC 1 and PEDFIC 2
StudiesPresenter: Dr. Henkjan J.
Verkade, Department of Paediatrics, University of
Groningen, Beatrix Children’s Hospital/University Medical
Centre Groningen, Groningen, the
NetherlandsSession: Poster Session
IV; Monday, November 7, 1:00
PM – 2:00 PM EST
Partial external biliary diversion (PEBD) is not effective in
PFIC2 patients with BSEP 3 mutations, but this pooled data analysis
showed that treatment with Bylvay provided substantial reductions
in sBAs and/or pruritus severity in most patients with BSEP2/BSEP3
mutations, demonstrating that Bylvay may provide an alternative to
surgical intervention for these patients.
Poster (Abstract #37608): Odevixibat
Therapy in Patients with FIC1-Deficient Progressive Familial
Intrahepatic Cholestasis and Diarrhea Following Liver
Transplantation That Impacted Daily Activities: A Retrospective
Case SeriesPresenter: Georg-Friedrich Vogel,
Assistant Professor, Medical University of Innsbruck,
Innsbruck, AustriaSession: Poster
Session IV; Monday, November 7, 1:00 PM – 2:00 PM
EST
Chologenic diarrhea can be a frequent and severe symptom after
liver transplant in patients with FIC1 deficiency. Real-world data
in three PFIC1 patients indicated that treatment with Bylvay can
improve diarrhea and quality of life in PFIC1 patients with severe
diarrhea after transplant.
Poster (Abstract #850): Odevixibat
Treatment in Patients with Recurrent Episodic Cholestasis and
Biallelic Mutations in ATP8B1: A Retrospective Case
SeriesPresenter: Dr. Angelo Di Giorgio,
Hospital Papa Giovanni XXIII, Bergamo,
ItalySession: Poster Session IV; Monday,
November 7, 1:00 PM – 2:00 PM EST
Recurrent episodic cholestasis is a rare disease characterized
by episodes of cholestasis followed by periods of remission. In a
case series of six patients treated with Bylvay during a
cholestasis episode, most patients experienced clinical
improvement, including improvements in sBA levels, hepatic
laboratory parameters, pruritus, sleep disturbances and impacts on
daily life. Before starting Bylvay treatment, all 6 patients
had high sBAs and severe pruritus leading to sleep or mood
disturbances and/or the inability to attend school, play sports or
work.
ASBTi A3907 for Cholestatic Liver Diseases
The apical sodium-dependent bile acid transporter (ASBT) plays
an important role in regulation of bile acid homeostasis by
promoting the reuptake of bile acids in the ileum, bile ducts, and
proximal tubuli of the kidney. A3907 is an investigational therapy
that is designed to inhibit ASBT to potentially benefit people with
cholestatic liver diseases.
The study showed that A3097 improved the general condition and
liver phenotype of mice with induced obstructive cholestasis by
promoting urinary secretion of bile acids, demonstrating that A3907
may have therapeutic potential for patients with cholestatic liver
diseases. Treatment with A3907 resulted in marked decreases in
serum and bile levels of bile acids relative to vehicle. The A3907
study was selected among the four posters for the Cholestatic and
Autoimmune Liver Diseases SIG (Special Interest Group) debrief.
Poster (Abstract #37733): Systemic ASBT
Inhibition with A3907 Stimulates Urinary Excretion of Bile Acids
and Halts Liver Disease Progression in Bile-Duct–Obstructed
MicePresenters: Drs. Peter Åkerblad and Erik
Lindström, Albireo, Boston, MA,
USASession: Poster Session III; Sunday,
November 6, 1:00 PM – 2:00 PM EST
About the Phase 3 PEDFIC & ASSERT
Studies
The PEDFIC trials represent the largest studies ever completed
in children with PFIC, or progressive familial intrahepatic
cholestasis, a rare genetic disorder that causes progressive,
life-threatening liver disease. PEDFIC 1 was a randomized,
double-blind, placebo-controlled Phase 3 trial that evaluated the
efficacy and tolerability of Bylvay in reducing pruritus and serum
bile acids (sBAs) in children with PFIC, and PEDFIC 2 is a
long-term, open-label Phase 3 extension study. Patients with PFIC
have impaired bile flow, or cholestasis, and the resulting bile
build-up in liver cells causes liver disease and symptoms, such as
intense itching, poor sleep, delayed growth, and diminished quality
of life. The harmful impacts of the disease extend to parents and
caregivers, as the 2022 multinational PICTURE study revealed that
PFIC negatively affects caregivers’ quality of life, relationships,
and career prospects.
ASSERT is a gold standard, prospective intervention trial in
Alagille syndrome, or ALGS, a rare, multisystem genetic disorder
that can affect the liver, heart, skeleton, eyes, central nervous
system, kidneys, and facial features. With 32 sites
across North America, Europe, Middle East,
and Asia Pacific, the double-blind, randomized,
placebo-controlled ASSERT trial was designed to evaluate the safety
and efficacy of 120 µg /kg/day Bylvay for 24 weeks in relieving
pruritus in patients with Alagille syndrome (ALGS). Key secondary
endpoints measure serum bile acid levels, safety, and tolerability.
The Company estimates that ALGS impacts 25,000 people globally. In
people with ALGS, 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 percent also present with severe, intractable
pruritus.
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 AlbireoAlbireo 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; ;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 the regulatory filings to be made for
Bylvay in patients with ALGS will be made on the timelines we
expect and be approved by the FDA and EMA; whether the FDA and EMA
will complete their respective reviews within target timelines,
once determined; 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; 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.com Lance Buckley, 917-439-2241,
lbuckley@lippetaylor.com
Investor Contact: Hans Vitzthum, LifeSci
Advisors, LLC., 617-430-7578
Albireo Pharma (NASDAQ:ALBO)
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