Data supports advancement of A4250 into a planned
Phase 3 trial in patients with progressive familial intrahepatic
cholestasis
Albireo Pharma, Inc. (Nasdaq:ALBO), a clinical-stage orphan
pediatric liver disease company developing novel bile acid
modulators, announced that the embargo on its late-breaker abstract
discussing preliminary data from a Phase 2 clinical trial of its
lead product candidate A4250 in children with cholestatic liver
disease has been lifted and the data will be presented today at The
International Liver Congress™ 2017 in Amsterdam. The data
demonstrated improvement in pruritus and reduction in serum bile
acids (sBA) in most patients, particularly patients with
progressive familial intrahepatic cholestasis (PFIC), after four
weeks of treatment with A4250.
“This study represents the first time we have studied A4250 in
children and we are very pleased with the data, especially the
responses seen in patients with the rare, life-threatening liver
disease PFIC,” said Ron Cooper, President and Chief Executive
Officer of Albireo. “We are currently engaging with regulatory
authorities in the United States and Europe with the objective to
design a coordinated Phase 3 program in patients with PFIC, which
we expect to initiate in the second half of 2017.”
A4250 is a highly potent and selective inhibitor of the ileal
bile acid transporter (IBAT) that has minimal systemic exposure.
The open label, multicenter, dose finding Phase 2 trial assessed
the safety and tolerability of A4250 and explored changes in sBA
levels and pruritus. Nineteen patients aged one to 17 years old
with a pediatric cholestatic liver disease, including PFIC (subtype
1, 2 or 3), Alagille syndrome, biliary atresia or intrahepatic
cholestasis, were enrolled in the trial’s first five cohorts. Five
different doses of A4250 were evaluated, ranging from 10 µg/kg to
200 µg/kg.
A4250 reduced mean levels of sBA in all five dose groups, with
substantial sBA reductions observed in seven of nine PFIC patients
(ranging from 43 percent to 98 percent). In addition, 14 of 19
patients showed improvement in pruritus using a visual analogue
scale (VAS-Itch 0-10). The dose with the greatest improvement
showed a mean decrease of 2.86 points from baseline. The trial was
not powered for formal statistical analyses.
“The findings in this study illustrate the potential for A4250
to improve the treatment of children with PFIC or other rare
cholestatic liver diseases,” said Ulrich Baumann, MD, an
investigator in the A4250 Phase 2 clinical trial and professor at
Hannover Medical School in Germany. “There is no drug proven to be
effective in treating these devastating diseases, and surgery, with
its associated limitations and drawbacks, is often the only viable
alternative. A safe and effective new treatment option for these
patients is greatly needed.”
The data showed a significant correlation between reduction in
sBA and improvement of pruritus. In addition, no treatment-related
serious adverse events were observed, and A4250 was well
tolerated.
Subsequent to the submission date for ILC, the trial’s remaining
patients completed the study.
About A4250 A4250 is a first-in-class
product candidate in development for progressive familial
intrahepatic cholestasis (PFIC) and potentially other orphan
pediatric cholestatic liver diseases. A4250 is a highly potent and
selective inhibitor of the ileal bile acid transporter (IBAT), has
minimal systemic exposure and acts locally in the gut. Albireo
anticipates initiating a Phase 3 clinical trial of A4250 in
patients with PFIC in the second half of 2017.
A4250 has been granted orphan drug designation for PFIC in the
United States and the European Union and has been granted access to
the PRIority MEdicines (PRIME) program of the European Medicines
Agency (EMA) for the treatment of PFIC.
About Albireo Albireo 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 clinical pipeline includes two
Phase 2 product candidates and one Phase 3 product candidate.
Albireo was spun out from AstraZeneca in 2008.
Albireo Pharma is located in Boston, Massachusetts, and its key
operating subsidiary, Albireo AB, is located in Gothenburg, Sweden.
For more information on Albireo, please
visit www.albireopharma.com.
Forward-Looking StatementsThis 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: the progress or
scope of development of A4250, including regarding a planned Phase
3 clinical trial of A4250 in patients with PFIC; the timing for
initiation of such planned Phase 3 clinical trial of A4250; the
competitive position of A4250 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,” 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 and uncertainties, including, but not limited to,
risks and uncertainties relating to: whether preliminary data from
the Phase 2 trial of A4250 in children with chronic cholestasis
will be confirmed following database lock; whether the FDA, EMA or
other regulatory authorities will determine clinical results for
A4250 to be sufficient to support advancement into a planned Phase
3 clinical trial in patients with PFIC in the United States and
Europe; whether favorable findings from clinical trials of A4250 to
date will be predictive of results from future clinical trials,
including the planned Phase 3 PFIC trial; the designs, endpoints,
sizes and durations for trials that will be required to support
approval of A4250 to treat patients with PFIC or any other orphan
pediatric liver disease; whether Albireo’s cash resources will be
sufficient to advance A4250 through completion of a planned Phase 3
PFIC trial; the timing for initiation or completion of, or for
availability of data from, ongoing or future trials of A4250,
including the planned Phase 3 PFIC trial, and the outcomes of such
trials; and delays or other challenges in the recruitment of
patients for the planned Phase 3 PFIC trial. 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 and in other 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.
Source: Albireo Pharma, Inc.
Investor Contact:
Hans Vitzthum
LifeSci Advisors, LLC.
212-915-2568
Media Contact:
Heather Anderson
6 Degrees
980-938-0260
handerson@6degreespr.com
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