Albireo Pharma, Inc. (Nasdaq: ALBO), a rare disease company
developing novel bile acid modulators to treat pediatric and adult
liver diseases, today announced the acceptance of two late
breakers, including one late breaker with new data from the Phase 3
ASSERT study, to be presented as an oral presentation at the
American Association for the Study of Liver Disease (AASLD) The
Liver Meeting® 2022, November 4 – 8, 2022. Positive topline results
from ASSERT, a global, double-blind, randomized, placebo-controlled
trial which evaluated the safety and efficacy of Bylvay in ALGS
patients from birth to early adulthood, were announced in October.
A second late breaker was also accepted as an oral presentation
with data showing Bylvay restored biliary bile acid secretion in
treatment-responsive progressive familial intrahepatic cholestasis
(PFIC) patients with bile salt export pump (BSEP) deficiency in the
PEDFIC 1 trial. A third oral presentation was previously announced,
with a pooled data analysis of the PEDFIC trials showing that a
decrease in serum bile acids was strongly associated with native
liver survival in PFIC patients treated with Bylvay.
Earlier this month, Albireo announced that an additional six
abstracts were accepted for presentation at the AASLD meeting,
showcasing data on Bylvay treatment in patients with several types
of PFIC, as well as data for A3907, the Company’s ASBT inhibitor in
clinical development for the treatment of adult liver disease. A
full list of presentations can be found on The Liver Meeting
Digital Experience™ 2022 website.
Oral 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 Title: Late-Breaking Oral Abstract
Session 1 Presentation Type: Oral, Late-Breaking
Parallel Session Date & Time: Monday, November
7, 2022, 9:00 AM - 10:30 AM ESTPresentation Time:
9:15 AM EST
Oral 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 Title:
Late-Breaking Oral Abstract Session 1 Presentation
Type: Oral, Late-Breaking Parallel SessionDate
& Time: Monday, November 7, 2022, 9:00 AM - 10:30 AM
ESTPresentation Time: 10:00 AM EST
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 Title: Genes to Cures: What’s
New in Pediatric Liver DiseaseDate &
Time: Sunday, November 6, 2:10 PM EST
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.
ASSERT Phase 3 Clinical Trial Data
ASSERT is a gold standard, prospective intervention trial with
32 sites across North America, Europe, Middle East, and Asia
Pacific. The double-blind, randomized, placebo-controlled trial was
designed to evaluate the safety and efficacy of 120 µg /kg/day
Bylvay (odevixibat) for 24 weeks in relieving pruritus in patients
with Alagille syndrome (ALGS). Key secondary endpoints measure
serum bile acid levels and safety and tolerability. The trial
enrolled patients aged 0 to 17 years of age with a genetically
confirmed diagnosis of ALGS. The primary efficacy endpoint was a
change from baseline to month 6 (weeks 21 to 24) in pruritus
measured by scratching with the PRUCISION Observer-Reported Outcome
(ObsRO) scratching score caregiver instrument (0-4 point scale).
The key secondary efficacy endpoint was a change in serum bile acid
responses (sBAs) from baseline to the average of weeks 20 and
24.
In the primary analysis, the study met the primary endpoint
showing statistically significant reduction in pruritus as measured
by the PRUCISION Observer-Reported Outcome scratching score (0-4
point scale), from baseline at month 6 (weeks 21 to 24), compared
to the placebo arm (p=0.002). The study also met the key secondary
endpoint showing a statistically significant reduction in serum
bile acid concentration from baseline to the average of weeks 20
and 24 (compared to the placebo arm p=0.001). Statistically
significant improvements in multiple sleep parameters were observed
as early as week 1-4 compared to patients on placebo with continued
improvement through week 24. In the study, there were no patient
discontinuations. Bylvay was well tolerated, with an overall
adverse event incidence similar to placebo and a low incidence of
drug-related diarrhea (11.4% vs. 5.9% placebo).
|
Placebo n=17 |
Odevixibat n=35 |
P-value |
Mean change from baseline in scratching score at month 6
(weeks 21 to 24) |
-0.80 |
|
-1.69 |
|
0.002 |
Mean change in serum bile acid levels from baseline to
average of weeks 20 & 24 |
22.39 |
|
-90.35 |
|
0.001 |
Rate of drug-related diarrhea |
5.9% |
|
11.4% |
|
- |
Number of discontinuations |
0 |
|
0 |
|
- |
The Company continues to enroll patients in the Phase 3 BOLD
study, which is the first and only pivotal trial of an IBATi in
biliary atresia (BA) and remains on track to fully enroll by end of
year, with topline data planned for 2024. BA is the most common
pediatric cholestatic liver disease with no approved drug
treatment. With clinical programs in ALGS and BA, Bylvay has the
potential to be approved for three pediatric cholestatic liver
diseases.
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 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; potential regulatory approval and
plans for potential commercialization of Bylvay in biliary atresia
or ALGS or in additional countries, 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; expectations that
biliary atresia is the most common pediatric cholestatic liver
disease with no approved drug treatment; 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 U.S. Food and Drug Administration (FDA) and the European
Medicines Agency (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.comLance
Buckley, 917-439-2241, lbuckley@lippetaylor.com
Investor Contact:Hans Vitzthum, LifeSci
Advisors, LLC., 617-430-7578
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