Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company
developing novel bile acid modulators, today announced the
presentation of new data at the annual meeting of the European
Society for Paediatric Gastroenterology, Hepatology and Nutrition
(ESPGHAN) being held in Copenhagen, Denmark. The data provided new
analyses of the Phase 3 PEDFIC 1 and PEDFIC 2 trials of Bylvay®
(odevixibat), demonstrating that dose escalation can provide
greater benefits in relieving patients’ pruritus, with similar
tolerability, as well as showing that reductions in pruritus are
associated with important improvements in sleep and other quality
of life measures in children with all types of progressive familial
intrahepatic cholestasis (PFIC).
“These data analyses, from the largest studies ever completed in
children with PFIC, reinforce that when you give patients’ relief
from their pruritus, you can dramatically improve many aspects of
their life and that of their families – perhaps most importantly
resulting in better sleep,” said Jan Mattsson, Ph.D., Chief
Scientific Officer and Head of R&D. “The data also provide
physicians insight into dose escalation, providing them confidence
that they can use a higher dose of Bylvay to increase patients’
pruritus relief and help more patients become pruritus responders,
with a tolerability profile that is similar to that seen with a
lower dose.”
Bylvay PEDFIC 1 and PEDFIC 2 DataBylvay 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.
Evidence of Improved Pruritus Response with Bylvay Dose
Escalation
- Oral Presentation: Efficacy and Safety Outcomes in
Patients with Progressive Familial Intrahepatic Cholestasis Who Had
an Odevixibat Dose Escalation: Pooled Results from the PEDFIC 1 and
PEDFIC 2 Studies. In the pooled analysis population
(N=84), 21 patients with PFIC treated with Bylvay who had a
treatment response on 40 μg/kg/day maintained their response and/or
had further improvements with dose escalation to 120 μg/kg/day.
Among those with dose escalation, additional patients became
pruritus responders at the higher dose and some non-responders
became pruritus responders at the higher dose in the PEDFIC
studies. Bylvay was generally well tolerated throughout dose
escalation. In patients who received 40 μg/kg/day of Bylvay during
PEDFIC 1 and escalated to 120 μg/kg/day in PEDFIC 2, the majority
of TEAEs were mild or moderate in severity.
Long-Term Data Demonstrate Efficacy of Bylvay Across
PFIC Subtypes
- Oral Presentation: Analysis of Quality of Life, Hepatic
Biochemical Markers, and Sleep in Patients With Progressive
Familial Intrahepatic Cholestasis Who Had a Pruritus Response With
Odevixibat Treatment. 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.
- Oral
Presentation: Changes in Hepatic Parameters, Growth, Sleep, and
Biochemical Markers With Odevixibat Treatment Across Patients With
Various Types of Progressive Familial Intrahepatic
Cholestasis. Patients with all 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
- e-Poster
Abstract #363: Total, Primary, and Secondary Serum Bile Acid
Concentrations in Patients With Progressive Familial Intrahepatic
Cholestasis With Serum Bile Acid Response or Not With Odevixibat
Treatment: Assessing the Contribution of Ursodeoxycholic Acid
Concentration. 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 whether excluding UDCA
concentrations from total sBA measurements altered the proportion
of patients who had a sBA response to Bylvay and showed no
appreciable changes. Moreover, patients who had a sBA response
experienced large reductions in sBAs, whether or not they were on
concomitant UDCA.
Albireo will also be hosting a company-sponsored symposium that
will feature case-based expert discussion on real-life experience
of treating PFIC with an IBAT inhibitor. Details for
attendance:
Breakfast Symposium: Real-life experience of
treating PFIC with an IBAT inhibitorExpert panel:
Prof. Patrick McKiernan, Birmingham Children's Hospital, NHS
Foundation Trust, UK, Dr. Christoph Leiskau, Hannover Medical
School, Germany, and Dr. Angelo Di Giorgio, Hospital Papa Giovanni
XXIII, Bergamo, ItalyDate & Time: Saturday, 25
June, 7:15-8:15 CEST
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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