Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company
developing novel bile acid modulators, today announced the
presentation of new data in pediatric and adult liver diseases at
the European Association for the Study of the Liver (EASL)
International Liver Congress™ 2022 being held in London. The
presentations provide new analyses of the Phase 3 PEDFIC 1 and
PEDFIC 2 trials of Bylvay® (odevixibat) in progressive familial
intrahepatic cholestasis (PFIC), as well as data on pipeline
ASBT/NTCP inhibitor compounds, including A2342 in Hepatitis B and
D.
“With the largest data set ever compiled on children and
families affected by PFIC, we’ve been able to quantify the
significant impact of PFIC and illustrate the myriad ways that it
dramatically harms patients’ and families’ quality of life,” said
Jan Mattsson, Ph.D., Chief Scientific Officer and Head of R&D.
“We’ve also shown that when patients with various types of PFIC
find relief from their pruritus with Bylvay, they experience
benefits across sleep, quality of life and hepatic parameters.
Seeing these improvements across the board underscores the urgent
need to assess and treat patients’ pruritus.”
Albireo also launched SPARK, a new grant-based program
established to identify and drive improvements in the quality of
care for rare liver diseases, beginning with PFIC in 2022. SPARK is
designed to encourage new ideas with the potential to demonstrate
best practices in patient-centered care. Healthcare professionals,
advocates, and other PFIC experts are invited to submit
applications at www.spark-grants.com starting July 1st.
“PFIC data and research are important because they reinforce why
we need to prioritize the treatment of patients’ pruritus,” said
Francesca Lombardozzi, PFIC caregiver. “What I hear from families
coping with PFIC is that pruritus impacts all aspects of their
lives, most critically their sleep – leaving the entire family
exhausted at times. I encourage families to advocate for effective
pruritus treatment for their children as it has the potential to
improve many aspects of their daily life.”
Bylvay PEDFIC 1 and PEDFIC 2 DataThe Company
presented data on Bylvay in five posters. Bylvay is a potent,
non-systemic once daily ileal bile acid transport inhibitor (IBATi)
that is the only treatment approved in Europe for the
treatment of all types of PFIC in patients aged 6 months or older
and the only treatment approved in the U.S. for the
treatment of pruritus in patients 3 months of age and older in all
types of PFIC. The PEDFIC 1 trial was the first and largest,
global, pivotal Phase 3 study conducted in PFIC, which evaluated
the efficacy and tolerability of Bylvay in reducing pruritus and
serum bile acids (sBAs) in a randomized, double-blind,
placebo-controlled trial, and PEDFIC 2 is a long-term, open-label
Phase 3 extension study. Key findings from the data include:
Long-Term Efficacy and Safety Data Show Bylvay Improves
Quality of Life for Patients and Families Across PFIC
Subtypes
- Bylvay Responders Experience Improvements in Quality of
Life Measures (poster #763). This analysis of pooled data
from the PEDFIC studies reveals that all patients had large quality
of life deficits at baseline across emotional functioning, social
functioning, physical functioning and school functioning, with mean
scores between 47 and 67 on the PedsQL. Patients who responded to
Bylvay treatment, and particularly their families, experienced mean
quality of life improvements that were sustained over time, while
quality of life for patients who did not respond to Bylvay was
largely unchanged for them and their families. Of 84 total patients
in the pooled population, PedsQL data were available for 53
patients, and PEDsQL Family Impact (FI) data were available for 79
patients. Among patients with available QoL data, both responders
and non-responders had impaired QoL at least some of the time at
baseline. PEDsQL total scores showed improved QoL for responders vs
non-responders over time up to week 72.
- Bylvay Provides Durable Treatment Benefits (poster
#1197). In this pooled analysis of data from the PEDFIC
studies, Bylvay provided durable clinical treatment benefits in
patients with PFIC who received treatment for at least 72 weeks,
with mean improvements over time in sBA levels, pruritus, hepatic
parameters and growth. Bylvay was generally well tolerated during ≥
72 weeks of treatment. Among the subgroup of patients treated with
Bylvay for ≥72 weeks, 96% had a TEAE and 52% experienced
drug-related TEAEs. The most common TEAEs were pyrexia, upper
respiratory tract infection, and diarrhea.
- Improvements in Pruritus on Bylvay Lead to Improvements
in Quality of Life, Hepatic Biomarkers, and Sleep (poster
#865). Results of pooled analysis population of 82
patients treated with Bylvay including 44 pruritus responders
of PEDFIC 1 and PEDFIC 2 show that patients across various types of
PFIC who had a pruritus response with Bylvay experienced
improvements in quality of life, sleep and hepatic biomarkers that
were sustained for up to 72 weeks. From baseline to week
72, pruritus responders had significant quality of life
improvements, as assessed by mean PEDsQL total scores (p=0.048) and
PEDsQL FI total scores (p=0.007). Data also indicated that pruritus
responders had significant improvements in several
caregiver-reported sleep parameters (all P<0.001), including
reductions in days with blood due to scratching, days needing help
falling asleep, days needing soothing, and days sleeping with
caregiver. Among all pruritus responders, 86% had any TEAE,
none of which were drug related serious TEAEs. Two patients
experienced TEAEs that led to study discontinuation, which
included splenomegaly, diarrhoea, jaundice, decreased weight,
and/or hypophagia.
- Bylvay Treatment Impacts Hepatic Parameters, Growth,
Sleep, and Biochemical Markers Across PFIC Types (poster
#850). Patients with across various types of PFIC treated
with Bylvay for up to 72 weeks, with some tracked up to 128 weeks,
experienced reductions in autotaxin levels and increases in C4
levels, as well as variable changes in hepatic parameters, sleep
characteristics and growth. 84 patients received Bylvay during the
pooled analysis period. Patients of almost all PFIC types
experienced mean improvements in caregiver-reported sleep
parameters. Patients of most PFIC types generally had mean
increases in height and/or weight Z scores with Bylvay treatment;
patients with PFIC1 and PFIC3 experienced more variable changes in
weight Z scores. Among all patients, 85% had any TEAE, which
was similar across all PFIC types and mild or moderate in
severity. All serious TEAEs were assessed as unrelated to
study drug.
Evidence of Bylvay Efficacy, With and Without
Concomitant UDCA
- Total, Primary, and Secondary Serum Bile Acid Changes
and Pruritus Improvement During Bylvay Treatment (Poster
#847). A large proportion of patients in the PEDFIC 1
trial were on UDCA at baseline, but still had elevated sBAs and
pruritus. This analysis assessed mean total, primary and secondary
sBAs among Bylvay sBA responders, partial responders (≥ 30%
reduction) and nonresponders, and showed that UDCA treatment
resulted in higher sBAs at baseline, but it did not seem to affect
sBA response to Bylvay. A sBA partial response was not associated
with a mean decrease in pruritus with concomitant UDCA use and
all sBA responder groups included patients for whom pruritus
response did not correlate with sBA response.
Pipeline Data for A2342 and Other Investigational
CompoundsAlbireo is investigating several ASBT and NTCP
inhibitor compounds. The apical sodium-dependent bile acid
transporter (ASBT) and Na+ -taurocholate co-transporting
polypeptide (NTCP) play key roles in maintaining bile acid
homeostasis, and their selective inhibition has shown efficacy in
liver diseases. Key findings from the data presentations
include:
- The Orally
Available Sodium/Taurocholate Co-Transporting Polypeptide Inhibitor
A2342 Blocks Hepatitis B And D Entry In Vitro (Poster
#1226). This preclinical study of A2342 supports its
potential as the first orally bioavailable molecule able to inhibit
Hepatitis B and D entry via NTCP in the nanomolar range.
- Dual
Ileal/Renal-Liver Bile Acid Transporter Inhibitors with Different
Transporter Selectivity in Vitro Differentially Increase Faecal and
Urinary Bile Acid Excretion in Organic Anion-Transporting
Polypeptide 1a/1b Knockout Mice in Vivo (Poster #1238).
This preclinical study showed that dual ASBT/NTCP inhibitors with
different selectivity may have a role as part of precision medicine
strategies to reduce the bile acid burden in forms of hepatobiliary
disease.
Albireo hosted a company-sponsored symposium that will
feature a case blended approach of idiopathic cholestasis and
targeted next-generation sequencing panels.
Symposium: Idiopathic Cholestasis and Targeted
Next-Generation Sequencing Panels: A Case Blended
ApproachExpert panel: Prof. Patrick McKiernan,
Birmingham Children's Hospital, NHS Foundation Trust, UK, Dr.
Christoph Leiskau, Hannover Medical School, Germany, Dr. Angelo Di
Giorgio, Hospital Papa Giovanni XXIII, Bergamo, ItalyDate
& Time: Thursday, 23 June 23, 12:30-13:30 BST at ExCel
London
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 England, Wales and Northern Ireland. 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 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; potential regulatory approval and plans for potential
commercialization of Bylvay in additional countries; 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:
results achieved in Bylvay in the treatment of patients with PFIC
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 indications other than PFIC, will be
predictive of results from other clinical trials of Bylvay; 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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