- Reduction in serum bile acids and pruritus
observed in both populations – - Alagille abstract selected
for inclusion in ’Best of ILC’ – - Albireo plans to initiate
second A4250 pivotal program in biliary atresia second half of 2019
–
Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage orphan
pediatric liver disease company developing novel bile acid
modulators, announced today that results in Alagille syndrome and
biliary atresia patients from its completed Phase 2 clinical trial
evaluating A4250 in pediatric cholestasis were presented at
the European Association for the Study of the Liver
(EASL) The International Liver Congress (ILC) 2019
in Vienna, Austria.
Lead investigator Ulrich Baumann, M.D., presented the Alagille
results today during an oral presentation.
“Liver damage caused by cholestasis is a hallmark of Alagille
syndrome, a rare and life-threatening liver disease, and there is
an urgent need for a non-invasive pharmacologic treatment option,”
said Dr. Baumann, Professor of Pediatric Gastroenterology and
Hepatology, Hannover Medical School in Hannover, Germany. “The
Alagille patient data from this Phase 2 trial, including the
meaningful reductions in serum bile acids and improved pruritus and
sleep scores, indicate that A4250 could be a promising therapy for
cholestatic liver diseases like Alagille, and suggest that further
investigation of A4250 in children with Alagille is warranted.”
Investigator Ekkehard Sturm, M.D., Ph.D., presented the biliary
atresia results yesterday in a poster session.
“Although biliary atresia is an orphan disease, it is the most
common pediatric liver disease for which transplants are done, and
there are no approved pharmacological treatments. The A4250 data,
though limited, are consistent with results from the overall study
population of pediatric patients with chronic CLD and are
encouraging,” said Dr. Sturm, Head of Pediatric
Gastroenterology-Hepatology, Liver and Intestinal Transplantation,
Children's Hospital, University of Tuebingen in Tuebingen, Germany.
“I am pleased to see that Albireo plans to embark on a biliary
atresia pivotal program as the unmet need for these patients is
significant.”
A4250 is a highly potent and selective inhibitor of the ileal
bile acid transporter (IBAT) currently being studied in a Phase 3
clinical trial in children with progressive familial intrahepatic
cholestasis (PFIC), a life-threatening, rare cholestatic liver
disease. Albireo plans to initiate a second A4250 pivotal trial, in
biliary atresia, in the second half of 2019. Albireo estimates that
biliary atresia is one of the most common rare pediatric liver
diseases, with roughly 15,000 to 20,000 patients in the U.S. and EU
combined and about 80% of patients requiring liver transplant
within their first two decades of life.
The open-label, multicenter, dose-finding Phase 2 clinical trial
assessed the safety and tolerability of A4250 in 20 children with
cholestatic liver diseases, including PFIC, Alagille syndrome and
biliary atresia. Efficacy endpoints included change in serum bile
acid (sBA) levels and pruritus. A4250 was administered orally in
doses ranging from 10 µg/kg to 200 µg/kg once daily for 4
weeks.
A4250 demonstrated marked sBA reductions of up to 92% in the
majority of Alagille patients. The majority of Alagille patients
also showed improvement in pruritus as measured by three different
scales. One patient had an elevation in bile acids versus baseline.
The abstract was selected for inclusion in EASL’s
‘Best of ILC’ summary resource highlighting the most
noteworthy contributions to its scientific program, which will be
available at https://ilc-congress.eu/slide-decks/ following the
Congress.
To date, Albireo is the only company to generate positive data
suggesting an effect on bile acids and pruritus in biliary atresia
patients using a pharmacologic approach. A4250 demonstrated
significant sBA reductions of 57.6% and 50.8% in two patients with
high baseline bile acids (> 130 μmol/L) and showed improvement
in pruritus across two different pruritus scales. No effect was
observed in a third patient with a low baseline sBA. These data,
combined with the total dataset of n =24, form the basis for a
pivotal development program in a second indication, biliary
atresia.
In patients with Alagille and biliary atresia, A4250 was
generally well-tolerated. Adverse events,
including some increased transaminases, were mild and transient.
Two Alagille patients with high baseline transaminase levels
experienced further increases, which informed the decision to not
dose escalate.
The data for patients with progressive familial intrahepatic
cholestasis (PFIC) were presented at a previous meeting.
“Albireo’s goal is to develop a pediatric cholestasis medicine
that can benefit people across multiple rare diseases,” said Ron
Cooper, President and Chief Executive Officer of Albireo. “These
data, and our key regulatory designations in biliary atresia,
Alagille and PFIC in the U.S. and the EU, reinforce the potential
of A4250 to achieve that goal.”
Also at EASL, the NAPPED (NAtural course and Prognosis of PFIC
and Effect of biliary Diversion) consortium presented data on the
natural history of PFIC: “Predicting long-term outcome after
surgical biliary diversion in BSEP-deficiency patients: Results
from the NAPPED consortium” (abstract number PS-195). Notably, the
NAPPED consortium data showed that surgical biliary diversion
profoundly decreased sBA in BSEP-deficient patients with mild or
moderate PFIC Type 2 genetic severity. NAPPED is supported by an
unrestricted grant from Albireo.
About A4250 A4250 is a first-in-class
product candidate being developed to treat rare pediatric
cholestatic liver diseases and is in Phase 3 development in its
initial target indication, progressive familial intrahepatic
cholestasis (PFIC). A highly potent and selective inhibitor of the
ileal bile acid transporter (IBAT), A4250 has minimal systemic
exposure and acts locally in the small intestine.
The PFIC A4250 program, or elements of it, have received fast
track, rare pediatric disease and orphan drug designations
in the United States. In addition, the FDA has
granted orphan drug designation to A4250 for the treatment of
Alagille syndrome, biliary atresia and primary biliary cholangitis.
The European Medicines Agency (EMA) has granted A4250
orphan designation, as well as access to the PRIority MEdicines
(PRIME) scheme for the treatment of PFIC. Its Pediatric Committee
has agreed to Albireo's A4250 Pediatric Investigation Plan for
PFIC. EMA also has granted orphan designation to A4250 for the
treatment of Alagille syndrome, biliary atresia and primary biliary
cholangitis.
A4250 is currently being evaluated in a Phase 3 clinical
program, PEDFIC 1, in patients with PFIC, subtype 1 or 2
(NCT03566238). The PEDFIC 1 clinical trial is currently recruiting
in over 35 clinical trial sites in 14 countries worldwide. More
information may be found on www.clinicaltrials.gov.
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, 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 (PFIC). 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 located
in Boston, Massachusetts, and its key operating
subsidiary is located 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:
the plans for, or progress, scope, cost, duration or results or
timing for availability of results of, development of A4250 or any
other Albireo product candidate or program, including regarding the
Phase 3 clinical program for A4250 in patients with PFIC; the
target indication(s) for development, the size, design, population,
location, conduct, objective, 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 A4250; the size of the PFIC population, the
biliary atresia population, the NASH population, or any other
disease population for indications that may be targeted by Albireo;
the potential benefits or competitive position of A4250, or any
other Albireo product candidate or program or the commercial
opportunity in any target indication; the potential benefits of a
rare pediatric disease designation, the potential benefits of an
orphan drug designation, the potential benefits of a fast track
designation, the pricing of A4250 if approved; the period for which
Albireo’s cash resources will be sufficient to fund its operating
requirements (runway); 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
A4250 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 A4250; 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 A4250 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 A4250, 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.
Investor Contact:Hans
Vitzthum
LifeSci Advisors,
LLC
212-915-2568
Media Contact:Heather Anderson 6
Degrees980-938-0260 handerson@6degreespr.com
Source: Albireo Pharma, Inc.
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