Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage orphan
pediatric liver disease company developing novel bile acid
modulators, announced today the launch of PFIC Voices, a new global
campaign to build awareness and understanding of the burden of
progressive familial intrahepatic cholestasis (PFIC), an
ultra-rare, genetic, pediatric liver disease. Through PFIC Voices,
Albireo is joining with leaders from the patient advocacy
community, families and clinicians to share perspectives about the
impact of PFIC, and the critical need for education, treatment and
support.
“Imagine the heartbreak of trying to console a
child who is scratching herself so much day and night that she
tears through her skin to the point of bleeding,” said Emily
Ventura, leader of PFIC Network (www.pfic.org), and mother to a
PFIC patient. “This is the experience of patients living with PFIC,
and it affects the entire family. We are confronted with
life-altering challenges in the present and an uncertain future.
While we have made important progress in raising awareness in
recent years, we must all join in the effort to support research
that could lead to treatment. PFIC Voices is a way for patient and
parent voices to be heard.”
PFIC is rare but devastating, and those affected
are desperate for relief—for their child and for the whole family.
It is an inherited liver disease in which normal bile flow is
disrupted, elevating bile acids to toxic levels. In many cases,
patients diagnosed with PFIC progress to cirrhosis and experience
end-stage liver disease by 10 years old. PFIC is estimated to
affect one in every 50,000 to 100,000 children born worldwide. It
is characterized by pruritus (intense itching), jaundice (yellowing
of the skin), nutrient malabsorption, and poor weight gain and
growth. PFIC is disruptive to sleep, leading to irritability and
daytime fatigue for all family members. Itching may be so severe
that it drives the decision to seek liver transplant. Liver
transplant and other surgical interventions may fail, have
complications, or bring psychological and social issues.
“There are no approved treatments for PFIC, and
healthcare providers have only limited options available to help
manage this disease,” said Dr. Patrick McKiernan, Director,
Pediatric Hepatology Program, UPMC Children’s Hospital of
Pittsburgh. “These children usually need disfiguring surgeries or a
liver transplant in addition to managing severe symptoms. Efforts
to increase awareness of the impact of PFIC can play an important
role in both improving treatment outcomes for patients, and driving
research that could lead to new treatments in the years ahead.”
Albireo has been talking with parents, patients and
physicians around the world to understand their experiences living
with, and managing, this debilitating condition. Many are eager to
share their stories. PFIC Voices includes a new website,
www.PFICvoices.com, which seeks to provide a glimpse into life with
the disease. The site currently features one family’s story, with
more information to come. In addition, Albireo is sponsoring the
first-ever PFIC Family Conference to be held on June 21, 2019, in
Cincinnati, Ohio, in conjunction with the Alagille Syndrome
Alliance’s 8th International Symposium and Scientific Meeting on
Alagille Syndrome. Albireo is a supporter of the PFIC Advocacy and
Resource Network, the only patient advocacy organization
specifically serving the PFIC community. As part of the PFIC Voices
effort, Albireo also provided an unrestricted educational grant to
the American Liver Foundation (www.liverfoundation.org) in support
of a PFIC webinar, which will be hosted on Tuesday, July 9 at 1
p.m. EDT.
“Albireo is committed to research that can lead to
new and innovative treatments for rare cholestatic liver diseases,”
said Ron Cooper, President and Chief Executive Officer of Albireo.
“We are committed to supporting patients, parents and treaters and,
through PFIC Voices, bring their experiences to light.”
About Albireo Albireo
Pharma is a clinical-stage biopharmaceutical company focused
through its operating subsidiary 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 (A4250), 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 Pharma is
in Boston, Massachusetts, 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, duration or
endpoints of the double-blind Phase 3 PFIC trial for odevixibat;
the size of the PFIC population; or the potential benefits or
competitive position of odevixibat. 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 will be predictive of results from the trials
comprising the Phase 3 PFIC program for 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, enrollment, 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.
Investor Contact: Hans Vitzthum,
LifeSci Advisors, LLC., 212-915-2568
Media Contact: Bill Berry, Berry
& Company Public Relations, (212) 253-8881,
bberry@berrypr.com
Source: Albireo Pharma, Inc.
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