Albireo Achieves Significant Milestones for Odevixibat Across Multiple Diseases
January 29 2020 - 4:01PM
Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage orphan
pediatric liver disease company developing novel bile acid
modulators, today announced a number of advances and new
initiatives in the clinical program for odevixibat, an oral
once-daily capsule in development for the treatment of progressive
familial intrahepatic cholestasis (PFIC), biliary atresia and
Alagille syndrome. The U.S. Food and Drug Administration (FDA) has
cleared the company’s investigational new drug (IND) application to
initiate a global pivotal trial in biliary atresia. Following
scheduled FDA interactions in the first quarter of 2020, the
company plans to commence an additional pivotal program in Alagille
syndrome by the end of 2020. Management also continues to expect
topline data from its Phase 3 trial in PFIC in mid-2020.
“We continue to believe in the potential for odevixibat to
address unmet needs across multiple rare cholestatic liver
diseases, and we remain steadfastly committed to patients and their
families living with these devastating conditions,” said Ron
Cooper, Albireo’s President and Chief Executive Officer. “We expect
our Phase 3 trial in PFIC will be fully randomized in the coming
days, and we expect to report topline data in mid-2020. Today, I am
particularly excited to announce that the FDA has cleared our IND,
and we expect to initiate a precedent-setting pivotal trial in
biliary atresia in the first half of 2020. Following FDA input
later this quarter, we plan to initiate a third odevixibat pivotal
trial, in Alagille syndrome, later this year. We are a growing,
energized and well-prepared organization, and look forward to 2020
being a transformational year for Albireo.”
The PEDFIC 1 trial is studying both high and low dose odevixibat
in both PFIC type 1 and type 2 patients who are 6 months to 18
years of age. Patients randomized to odevixibat are being treated
with high (120µg/kg) or low (40µg/kg) dose once-daily oral capsules
or sprinkles. The trial uses Albireo’s planned commercial
formulation of odevixibat, which does not require refrigeration.
PEDFIC 1 has a target of 60 patients, 59 have been randomized and
the randomization visit for the final patient has been scheduled in
the coming days. Albireo expects top-line data from PEDFIC 1 in
mid-2020 with potential first regulatory approval and odevixibat
launch in the second half of 2021.
Biliary atresia is a rare disease that impacts an estimated
15,000-20,000 people in the United States and European Union and is
the leading cause of liver transplants among children. Albireo’s
planned double-blind, placebo controlled pivotal trial in biliary
atresia is designed to enroll approximately 200 patients at 70
sites globally. Patients will receive either placebo or high-dose
(120µg/kg) odevixibat once daily. The primary endpoint is survival
with native liver after 2 years of treatment. The FDA and European
Commission have granted orphan designations for odevixibat in the
treatment of biliary atresia.
In addition, Albireo has been evaluating a proposed pivotal
study design for odevixibat in Alagille syndrome in consultation
with physicians and regulators, and plans to initiate the trial
later this year. The FDA and European Commission have granted
orphan designations for odevixibat in the treatment of Alagille
syndrome.
The company had cash and cash equivalents at September 30, 2019
of $142.7 million, and continues to anticipate that its current
cash balance will be sufficient to meet its operating needs into
2021.
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:
the plans for, or progress, scope, cost, initiation, duration,
enrollment, results or timing for availability of results of,
development of odevixibat, elobixibat or any other Albireo product
candidate or program, including regarding the Phase 3 clinical
program for odevixibat in patients with PFIC, the planned pivotal
trial for odevixibat in biliary atresia and the planned pivotal
trial for odevixibat in Alagille syndrome; the potential approval
and commercialization of odevixibat; discussions with the FDA
regarding our programs; the potential benefits or competitive
position of odevixibat or any other Albireo product candidate or
program or the commercial opportunity in any target indication; the
potential benefits of an orphan drug designation; that Albireo’s
current cash balance will be sufficient to meet its operating needs
into 2021; 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,” and 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 favorable findings
from clinical trials of odevixibat to date, including findings in
indications other than PFIC, will be predictive of results from the
trials comprising the Phase 3 PFIC program or any other clinical
trials of odevixibat; whether either or both of
the FDA and EMA will determine that the primary endpoint
for their respective evaluations and treatment duration of the
double-blind Phase 3 trial in patients with PFIC are sufficient,
even if the primary endpoint is met with statistical significance,
to support approval of odevixibat in the United States or
the European Union, to treat PFIC, a symptom of PFIC, a
specific PFIC subtype(s) or otherwise; 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 odevixibat, including the trials
comprising the Phase 3 PFIC program, or the pivotal programs in
biliary atresia and Alagille syndrome, and the outcomes of such
trials; Albireo’s completion of discussions with the FDA regarding
the planned pivotal trial for odevixibat in Alagille syndrome;
Albireo’s ability to obtain coverage, pricing or reimbursement for
approved products in the United States or European
Union; delays or other challenges in the recruitment of patients
for, or the conduct of, the double-blind Phase 3 trial or other
pivotal trials; and Albireo’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.
Investor Contact: Hans Vitzthum, LifeSci
Advisors, LLC., 212-915-2568
Media Contact: Heather Anderson, 6 Degrees,
980-938-0260, handerson@6degreespr.com
Source: Albireo Pharma, Inc.
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