Albireo Pharma, Inc. (Nasdaq: ALBO), a rare disease company
developing novel bile acid modulators to treat pediatric and adult
liver diseases, today announced the presentation of new data at the
NASPGHAN Annual Meeting being held in Orlando, Florida from October
12-15, 2022. A new analysis of pooled data from the landmark Phase
3 PEDFIC 1 and PEDFIC 2 trials demonstrates that Bylvay
(odevixibat) reduces serum bile acids and pruritus in children with
progressive familial intrahepatic cholestasis (PFIC) who have
varying levels of baseline hepatic impairment. Importantly, the
analysis shows that significantly more patients with mild hepatic
impairment at baseline had a serum bile acid response, suggesting
children may benefit from earlier treatment with Bylvay.
Additional data analyses presented at the
meeting show that Bylvay reduced pruritus in patients regardless of
their baseline pruritus scores. Further, pruritus reductions were
associated with improvements in serum bile acids, hepatic
parameters, growth, and disease aspects related to sleep, with
greater reductions in pruritus associated, in general, with greater
improvements across these additional measures.
“These new data provide important evidence that
treating children with Bylvay earlier in their disease course could
result in greater efficacy,” said Jan Mattsson, Ph.D., Chief
Scientific Officer and Head of R&D at Albireo. “Furthermore,
the data we are presenting reinforce the medicine’s efficacy in
patients with mild pruritus, suggesting patients’ benefit from
beginning treatment at the first sign of itching.”
“If a physician is trying to determine when to
start treatment with Bylvay, this new data suggest the earlier the
better,” said Dr. Lorenzo D’Antiga, Department of Paediatric
Hepatology, Gastroenterology, and Transplantation, Azienda
Ospedaliera Papa Giovanni XXIII, Bergamo. “We should consider
treating patients at an early stage of the disease to preserve
their native liver and provide relief from the incessant itching
associated with the condition. When we see babies stop scratching
and sleeping better, we see quality of life improvements for them
and their parents.”
PFIC is a rare genetic disorder that causes
progressive, life-threatening liver disease. 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.
New Analyses of the PEDFIC 1 and PEDFIC
2 TrialsThe global PEDFIC trials represent the largest
studies ever completed in children with PFIC. New pooled data
analyses from PEDFIC 1, 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, a long-term, open-label
Phase 3 extension study, are being shared at NASPGHAN in an oral
presentation and two poster presentations. An additional poster
presents data showing that Bylvay may be mixed in liquids to give
to the youngest patients:
Benefit to Treating Earlier with
Bylvay
Oral Presentation: Hepatic Impairment
Classifications at Baseline in Responders to Odevixibat Therapy in
Children with Progressive Familial Intrahepatic
CholestasisLead Author: Dr. Lorenzo
D’Antiga, Department of Paediatric Hepatology,
Gastroenterology, and Transplantation, Azienda Ospedaliera Papa
Giovanni XXIII, Bergamo, ItalySession Title:
Concurrent Session V – Potent P’s in Hepatology: A daily
doubleDate & Time: Saturday, October 15,
2:00pm – 3:30pm ET
Pooled data analysis showed that treatment with
Bylvay is associated with improvements in serum bile acids and
pruritus and suggests greater efficacy when treatment with Bylvay
occurs earlier in the disease. Significantly more patients with
mild hepatic impairment at baseline, according to Child-Pugh
scores, had a serum bile acid response than patients with more
severe hepatic impairment at baseline. Among patients with mild and
moderate hepatic impairment at baseline, 45% and 24% were Bylvay
responders, respectively (p value=0.039). No patients had
Child-Pugh scores of severe hepatic impairment at baseline. Across
baseline hepatic impairment levels, pruritus response rates were
comparable and Bylvay was generally well tolerated.
Direct Link Between Sustained Level of Pruritus
Reduction and Magnitude of Improvement in Other Disease
Parameters
Poster Abstract #301: Outcomes with Odevixibat
in Patients with Progressive Familial Intrahepatic Cholestasis by
Level of Pruritus Reduction: Pooled Analysis from the PEDFIC Trials
Lead Author: Dr. Ekkehard Sturm, Pediatric
Gastroenterology and Hepatology, University Children’s Hospital
Tübingen, Tübingen, GermanySession Title: Poster
Session IIDate & Time: Friday, October 14,
12:00pm – 2:30pm ET
An analysis of pooled data from 77 patients
treated with Bylvay showed that pruritus reductions were associated
with improvements in serum bile acids, hepatic parameters, growth,
and disease aspects related to sleep and, in general, patients who
had larger-magnitude pruritus reductions had larger-magnitude
improvements in other outcomes. The median percentage change from
baseline to weeks 70–72 in serum bile acid level was –12% in
patients who had pruritus reductions of <1, –54% in patients who
had reductions of ≥1 to <2, –94% in patients who had reductions
of ≥2 to <3,–96% in patients who had reductions of ≥3 to <4,
and –94% in patients who achieved a pruritus score of 0 or 1.
Bylvay was generally well tolerated regardless of the level of
pruritus reduction.
Evidence of Bylvay Efficacy in Patients
with Lower Baseline Pruritus Severity
Poster Abstract #547: Outcomes in Patients with
Progressive Familial Intrahepatic Cholestasis Treated with
Odevixibat who had Medium or Lower Pruritus Severity at Baseline:
Pooled Analysis from the PEDFIC 1 and PEDFIC 2 StudiesLead
Author: Dr. Kathleen M. Loomes, Children’s Hospital of
Philadelphia, Philadelphia, PA, USASession Title:
Poster Session IIIDate & Time: Saturday,
October 15, 12:00pm – 2:30pm ET
A pooled data analysis of 16 patients who had
medium or lower pruritus severity at baseline showed that treatment
with Bylvay was associated with improvement in serum bile acids,
pruritus, hepatic parameters, growth, sleep, and quality of life
(QoL), suggesting even patients with lower baseline pruritus
severity may benefit from Bylvay treatment. Treatment with Bylvay
led to a serum bile acid response in 31% of patients; improvements
in mean percentage of days needing help falling asleep, falling
from baseline of 41% to 18%; needing soothing falling from 37% to
19%; and sleeping with a caregiver falling from 36% to 16%; but not
in percentage of days with scratching associated with bleeding,
which was 9% at baseline and 30% at weeks 61-72. Bylvay was
generally well tolerated.
Stability of Bylvay Mixed in Liquid for Pediatric
Dosing
Poster Abstract #70: Stability of Odevixibat
Oral Capsule Contents in Liquids Lead Author: Dr.
Prince Korah, Albireo, Boston, MA, USASession
Title: Poster Session IDate & Time:
Thursday, October 13, 5:00pm – 7:00pm ET
An in vitro study showed that when Bylvay is
mixed in a range of liquids often used to administer medicines to
young infants, it is stable and provides children with an adequate
dose. As the symptoms of PFIC often begin in infancy, when a child
is not yet eating solid food, it is important that parents have an
option to mix Bylvay in a liquid to give to their young
children.
Albireo will also host a product theater featuring an expert
perspective on treating PFIC patients with Bylvay:
Product Theater: Relief Made Possible with
Bylvay® (odevixibat)Expert: Dr.
Saeed Mohammad, Monroe Carell Jr. Children’s Hospital at
Vanderbilt, Nashville, TN, USADate & Time:
Friday, October 14, 1:45pm -2:15pm ETLocation:
Promenade in front of Exhibit Hall
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
Albireo Albireo 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; the timing for
anticipated regulatory filings; 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 impact of the Expanded
Access Program; 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; any repurchase by the Company of
Sagard’s interest in the royalty interest payments under our
royalty monetization agreement with Sagard could materially impact
our financial condition; 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
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