Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease
company developing novel bile acid modulators, today announced that
The Lancet Gastroenterology & Hepatology published the results
of the pivotal PEDFIC 1 trial, which evaluated Bylvay® (odevixibat)
for the treatment of progressive familial intrahepatic cholestasis
(PFIC). The study met both of its two primary endpoints,
demonstrating that Bylvay provided statistically significant
improvements in pruritus severity scores and reductions in serum
bile acid levels, with rapid, sustained, and consistent results.
The full manuscript of the PEDFIC 1 study is available on The
Lancet Gastroenterology & Hepatology website.
A potent, once-daily, non-systemic ileal bile acid transporter
inhibitor (IBATi), Bylvay has minimal systemic exposure and acts
locally in the small intestine. Bylvay was recently approved
in the U.S. for the treatment of pruritus in patients 3 months of
age and older with all types of PFIC, and in Europe for the
treatment of all types of PFIC in patients aged 6 months or older,
based on the PEDFIC 1 study and PEDFIC 2 open-label extension (OLE)
study.
“The PEDFIC 1 study is the largest trial ever completed in PFIC
and we are pleased that a prestigious journal has recognized the
quality of the science and importance of the landmark results,”
said Ron Cooper, President and Chief Executive Officer of
Albireo. “The study outcomes have been positively received by
physicians and payors enabling swift pricing and reimbursement
reviews of Bylvay, helping to accelerate access to this much needed
medicine for patients suffering with this cholestatic liver
disease.”
The publication includes data from the randomized, double-blind,
placebo-controlled Phase 3 PEDFIC 1 global study. Within the study
findings:
- The study was conducted from May
2018 to July 2020 and evaluated 62 patients, aged 6 months to 15.9
years, with PFIC type 1 or type 2. Patients were randomized to
receive either a 40 µg/kg/day (n=23) or 120 µg/kg/day (n=19) oral
dose of Bylvay (odevixibat) or placebo (n=20) once daily for 24
weeks.
- The model-adjusted (least squares)
mean proportion of positive pruritus assessments (PPAs) at the
patient level was significantly higher with Bylvay, at 55% for the
all-Bylvay group (58% and 52% in the Bylvay 40 and 120 μg/kg/day
groups, respectively) compared to 30% in the placebo group
(p=0.0038).
- The proportion of patients achieving
a serum bile acid response, defined as a ≥70% reduction from
baseline in fasting serum bile acids or serum bile acids
≤70 μmol/L at week 24, was 33% in the all-Bylvay group
(including 43% and 21% of patients in the Bylvay 40 and 120
μg/kg/day groups, respectively), compared to no patients in the
placebo group (p=0.0030).
- The study also showed meaningful
improvements in secondary endpoints related to pruritus and serum
bile acids, with a greater proportion of patients treated with
Bylvay having a clinically meaningful improvement in pruritus at
week 24, defined as a drop from baseline of 1.0 point or more on
the 0−4 point scale, than patients treated with placebo (43% vs
11%, respectively). Improvement in pruritus among Bylvay-treated
patients was observed by week 4 of treatment.
- Consistent with improvements
observed with pruritus, treatment with Bylvay improved patients’
sleep by weeks 21−24 as reported by caregivers, with reductions
from baseline in the percentage of days needing help falling
asleep, −43% for the all-Bylvay group versus −3% for the placebo
group; soothing, −44% versus −8%, respectively; and sleeping with
the caregiver, −42% versus −5%, respectively.
- Mean serum bile acid levels
decreased by 114.3 μmol/L in the all-Bylvay group at weeks 22
through 24, compared to an increase of 13.1 μmol/L in the placebo
group (p=0.0022). Changes from baseline in serum bile acids were
also observed as early as week 4 of Bylvay treatment.
- In the study, Bylvay
was well tolerated, with an overall adverse event incidence similar
to placebo. There were no drug-related serious adverse events
reported during the study. Drug-related treatment-emergent adverse
events of diarrhea/frequent bowel movements occurred in 10% (4/42)
of Bylvay-treated patients and in 5% (1/20) of placebo-treated
patients. In same study, 31% (13/42) of patients on Bylvay reported
diarrhea compared with 10% (2/20) of placebo.
Patients with PFIC have impaired bile flow, or cholestasis, and
the resulting bile build-up in liver cells causes liver disease and
symptoms including 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.
The results from the PEDFIC studies provide confidence in the
translatability of the data in Alagille syndrome (ALGS) and biliary
atresia and the two Phase 3 studies in progress. The ASSERT study
in ALGS is fully enrolled and topline data is expected by the end
of the year. The BOLD study in biliary atresia is expected to
complete enrollment this year with topline data available in
2024.
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
transporter inhibitor (IBATi), 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 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 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 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, 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
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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