Albireo Recognizes PFIC Awareness Day and Highlights Urgent Needs of Progressive Familial Intrahepatic Cholestasis Families
October 03 2019 - 8:00AM
Albireo Pharma, Inc. (NASDAQ: ALBO), a clinical-stage orphan
pediatric liver disease company developing novel bile acid
modulators, announced today its support for PFIC Awareness Day, and
reaffirmed its commitment to patients and families living with
progressive familial intrahepatic cholestasis (PFIC).
PFIC is an ultra-rare, life-threatening, pediatric
liver disease characterized by pruritus (intense itching), jaundice
(elevated serum bilirubin levels that may manifest as yellowing of
the skin), and poor weight gain and growth. People with PFIC are
often diagnosed in infancy or early childhood and can progress to
cirrhosis and liver failure by age 10 without surgical
intervention. Children even undergo liver transplant due to the
relentless pruritus, and this has risks and long-term challenges.On
October 5, 2019, grassroots organization PFIC Advocacy and Resource
Network, Inc. (“PFIC Network,” www.PFIC.org) will hold the first
ever PFIC Awareness Day. Members of the PFIC community all over the
world will host lemonade stand fundraisers to raise awareness in
their communities. In collaboration with Life Gave Lemons, the PFIC
Network is providing families with lemonade stand fundraising
toolkits that empower them to become advocates. Among other
resources, it includes PFIC brochures, stickers and advocacy
bracelets. “These fundraising events bring our community closer and
really empower the kiddos—those that are living with this
unbelievably challenging condition,” said Lisa Crompton,
Co-Director of PFIC Network. “Our children who are affected by PFIC
are resilient, gritty and motivated to build a strong support
system for families while we wait for urgently-needed treatments.”
Albireo shares perspectives about the impact of PFIC and the
critical need for education and treatment through PFIC Voices,
www.PFICvoices.com. Families affected by the condition tell their
perspectives about life with this debilitating condition as part of
the global initiative to build awareness of PFIC. “PFIC Awareness
Day is an opportunity to stand in support of the PFIC community, as
we work to develop a potential new treatment option for people with
PFIC,” said Ron Cooper, President and Chief Executive Officer of
Albireo.Albireo’s Phase 3 PEDFIC clinical trial program for lead
investigational product candidate odevixibat in PFIC consists of
two clinical trials. PEDFIC 1 is a randomized, double-blind,
placebo-controlled, global multicenter clinical trial designed to
enroll approximately 60 patients with PFIC type 1 or type 2. PEDFIC
2 is a long-term, open-label extension study of PEDFIC 1 and
includes a second cohort of PFIC patients who do not meet
eligibility criteria for PEDFIC 1, but have elevated serum bile
acid levels and pruritus. This includes patients with all types of
PFIC, and patients younger than 6 months or older than 18 years of
age. The company expects topline data in mid-2020. There are
currently 45 sites actively recruiting for the PEDFIC
program.About PFICProgressive familial
intrahepatic cholestasis (PFIC) is a rare genetic disorder that is
estimated to affect between one in every 50,000 to 100,000 children
born worldwide and causes progressive, life-threatening liver
disease. People diagnosed with PFIC have impaired bile flow, or
cholestasis, caused by genetic mutations. The resulting bile
build-up in liver cells causes liver disease and symptoms. The most
prominent and problematic ongoing manifestation of the disease is
pruritus (intense itching), which often results in a severely
diminished quality of life. PFIC is also characterized by jaundice
(yellowing of the skin), and poor weight gain and growth. In many
cases, PFIC leads to cirrhosis and liver failure within the first
10 years of life, and nearly all people with PFIC require treatment
before age 30. There are no medicines currently approved for PFIC,
only surgical options, including a procedure known as partial
external biliary diversion (PEBD), and liver transplantation. These
options carry substantial risks. Additional information on PFIC is
available at https://www.pfic.org and
https://www.pficvoices.comAbout Albireo Albireo
Pharma is a clinical-stage biopharmaceutical company focused
on the development of novel bile acid modulators to treat orphan
pediatric liver diseases, and other liver and gastrointestinal
diseases and disorders. Albireo’s lead product candidate,
odevixibat, is being developed to treat rare pediatric cholestatic
liver diseases and is in Phase 3 development in its initial target
indication, progressive familial intrahepatic cholestasis.
Albireo’s clinical pipeline also includes two Phase 2 product
candidates. Albireo’s elobixibat, approved in Japan for
the treatment of chronic constipation, is the first ileal bile
acid transporter (IBAT) inhibitor approved anywhere in the world.
Albireo was spun out from AstraZeneca in
2008. Albireo was spun out from AstraZeneca in 2008. Albireo
Pharma is located in Boston, Mass., and its key operating
subsidiary is located in Gothenburg, Sweden. The Boston Business
Journal named Albireo one of the 2019 Best Places to Work in
Massachusetts. 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: the
plans for, or progress, scope, cost, duration or results or timing
for availability of results of, development of odevixibat,
including regarding the Phase 3 clinical program for odevixibat in
patients with PFIC;, the target indication(s) for development, the
size, design, population, location, conduct, objective, enrollment,
duration or endpoints of any clinical trial, or the timing for
initiation or completion of or reporting of results from any
clinical trial, including the double-blind Phase 3 PFIC trial for
odevixibat,; the potential approval and commercialization of
odevixibat; the size of the PFIC population or any other disease
population for indications that may be targeted by Albireo; the
potential benefits or competitive position of odevixibat or the
commercial opportunity in any target indication. 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, and the outcomes of
such trials; 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;
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.
Media Contact: Bill Berry, Berry
& Company Public Relations, (212) 253-8881,
billberry@berrypr.com
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