Albireo Pharma, Inc. (Nasdaq: ALBO), a rare disease company
developing novel bile acid modulators to treat pediatric and adult
liver diseases, today announced positive topline results from the
Phase 3 ASSERT study evaluating the safety and efficacy of Bylvay
in Alagille syndrome (ALGS) patients from birth to early adulthood.
The global, double-blind, randomized, placebo-controlled trial met
its primary endpoint of improvement in pruritus (p=0.002) and its
key secondary endpoint of reduction in serum bile acids (sBAs)
(p=0.001). There were no patient discontinuations and Bylvay was
well tolerated, with low rates of drug-related diarrhea (11.4% vs.
5.9% placebo). Albireo has engaged in discussions with the FDA and
EMA about the Phase 3 study design, both have indicated that a
successful single study would be sufficient for approval. The
Company plans to immediately submit regulatory filings in the U.S.
and EU.
“Our ASSERT Phase 3 study demonstrated early, rapid, and
sustained effects on reducing pruritus and bile acids in Alagille
syndrome, as it did in the Phase 3 PFIC study,” said Ron Cooper,
President and Chief Executive Officer of Albireo. “We have
successfully completed two of three gold standard pediatric liver
disease studies, look forward to imminent full enrollment of a
third Phase 3 with our BOLD biliary atresia study, and with more
than $270 million in cash, we have sufficient resources to execute
on our plans. At the same time, we are seeing an acceleration in
PFIC revenue and expect Q3 Bylvay sales to be above $7
million.”
A potent, once-daily, non-systemic ileal bile acid transport
inhibitor (IBATi), Bylvay has minimal systemic exposure and acts
locally in the small intestine. Bylvay is already 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), and in Europe for the treatment of all types of PFIC in
patients aged 6 months or older.
“The robust results from the ASSERT Phase 3 trial are important
because physicians urgently need more options in the treatment of
Alagille syndrome. Bylvay reduced the devastating pruritus, which
is so common among this patient population and critical to helping
us improve sleep, among other burdens of the disease,” explained
Nadia Ovchinsky, MD, Pediatric Gastroenterologist and Hepatologist,
Children’s Hospital at Montefiore and ASSERT Principal
Investigator. “The study also showed Bylvay reduced serum bile
acid levels, which could indicate that Bylvay may
diminish the severity of liver disease over time, an important
consideration for me as a treating physician.”
Alagille syndrome, or ALGS, is a rare, multisystem genetic
disorder that the Company estimates impacts 25,000 people globally.
ALGS can affect the liver, heart, skeleton, eyes, central nervous
system, kidneys and facial features. Liver damage is caused by a
paucity of bile ducts preventing bile flow from the liver to the
small intestine. Approximately 95% of patients with the condition
present with chronic cholestasis, usually within the first three
months of life, and as many as 88 percent also present with severe,
intractable pruritus.
“Alagille syndrome is a devastating diagnosis and families of
children with this syndrome need more treatment options,” said
Roberta Smith, President, Alagille Syndrome Alliance. “As a mother
of a child living with Alagille syndrome, I know firsthand the
devastation of pruritus and the terrible impact to a child’s
quality of life and ability to sleep and thrive. Knowing my
physician may have another treatment option is very
meaningful.”
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). Full results from
the Phase 3 clinical trial will be presented at a future scientific
meeting.
|
Placebon=17 |
Odevixibatn=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.
Conference Call
Albireo will host a conference call and live audio webcast
at 8:30 a.m. EDT. To access the live conference call by phone,
dial 1-877-407-0792 (domestic) or 1-201-689-8263
(international), and provide the access code 13733685. A live audio
webcast will be accessible from the Investors page
at ir.albireopharma.com/. To ensure a timely connection to the
webcast, it is recommended that participants register at least 15
minutes prior to the scheduled start time. An archived version of
the webcast will be available for replay on the Events &
Presentations section of the Investors page of Albireo’s website
for 3 months following the event.
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 ALGS Expanded Access Program
Albireo continues to prioritize access and continued scientific
research for patients living with rare cholestatic liver diseases,
with the Expanded Access Program (EAP) for ALGS. Albireo has
partnered with Tanner Pharma Group. Eligible patients with ALGS in
Europe and the U.S., with no other options, may receive Bylvay on a
free-of-charge (FOC) basis, through our existing EAP subject to
authorization by the relevant country competent authority, and
meeting of Albireo’s eligibility criteria. If you are a physician
who would like to request ALGS EAP access for your patient, please
send your enquiry to Tanner using odevixibat@tannerpharma.com, and
you will receive a response within one working day with further
information.
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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