Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage orphan
pediatric liver disease company developing novel bile acid
modulators, announced that clinical data from a Phase 2 study of
lead product candidate odevixibat (A4250), a highly potent and
selective inhibitor of the ileal bile acid transporter (IBAT), in
biliary atresia, Alagille syndrome and progressive familial
intrahepatic cholestasis (PFIC) were presented today at
the 2019 European Society for Paediatric Gastroenterology,
Hepatology and Nutrition (ESPGHAN) Annual Meeting in Glasgow,
Scotland.
“The encouraging data presented at ESPGHAN support our further
study of odevixibat as a potential treatment across a range of rare
cholestatic liver diseases,” said Ron Cooper, President and Chief
Executive Officer of Albireo. “We look forward to advancing our
IBAT inhibitors in new indications this year, and sharing topline
Phase 3 data on odevixibat in PFIC by the end of 2019 or early
2020.”
In the Phase 2 clinical trial in pediatric cholestasis,
odevixibat demonstrated marked reductions in serum bile acids (sBA)
in the majority of Alagille patients as high as 92 percent
(presentation number H-O-006). The majority of Alagille patients
also showed improvement in pruritus, as measured by three different
scales. One patient out of the six had an elevation in bile acids
versus baseline.
In the same trial, odevixibat demonstrated significant sBA
reductions of 57.6 percent and 50.8 percent in two biliary atresia
patients with high baseline bile acids (>130 μmol/L), and showed
improvement in pruritus across two different pruritus scales
(poster number H-P-029). No effect was observed in a third patient
with low baseline sBA.
In both the Alagille and biliary atresia patients, odevixibat
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 not to
dose escalate above 200 ug.
“Alagille syndrome and biliary atresia are rare and
life-threatening liver diseases with significant unmet medical
need,” said Ulrich Baumann, M.D., lead investigator and Professor
of Pediatric Gastroenterology and Hepatology, Hannover Medical
School in Hannover, Germany. “The Alagille and biliary atresia
patient data from this Phase 2 trial, while limited, are promising
and suggest that further studies are warranted.”
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. Albireo plans to initiate
a pivotal trial in biliary atresia in the second half of 2019.
In addition to the Phase 2 data, a case study evaluated a
patient with PFIC type 2 who was treated with odevixibat in the
Phase 2 trial in pediatric cholestasis (poster number H-P-130).
After the study period ended, the patient’s pruritus returned, and
partial external biliary diversion (PEBD) was performed. The study
found that, with both odevixibat and PEBD surgery, serum bile acids
were reduced by ≥95 percent and returned to normal levels, and the
patient showed improvement in pruritus and sleep.
PFIC is a serious disease characterized by an accumulation of
bile acids that can cause debilitating pruritus. It often results
in liver disease and failure before adulthood. PEBD is an invasive
surgical approach commonly used to reduce serum bile acids and
pruritus. This is the first case study comparing the effectiveness
of a pharmacologic IBAT inhibitor to the current gold standard
surgical intervention treatment.
Odevixibat is currently being studied in the PEDFIC Phase 3
clinical program in children with PFIC. In the PEDFIC studies, a
proprietary measurement tool is being used to assess pruritus and
sleep disturbance. Data on the development of the proprietary
patient- and observer-reported outcome (PRO and ObsRO) tools also
were presented at ESPHGAN (poster number H-P-138). Based on seven
waves of interviews (N=36 sessions) with 24 patients and 29
caregivers, the tools were designed as diaries completed twice
daily featuring a 5-point (0-4) pictorial response scale with
numeric, color-coded and verbal anchors. The study tested patients’
and caregivers’ understanding of the measures as intended.
About Odevixibat Odevixibat is a 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), odevixibat has minimal systemic exposure and
acts locally in the small intestine.
Odevixibat is being evaluated in a Phase 3 clinical trial,
PEDFIC 1, in patients with PFIC subtype 1 or 2 (NCT03566238). The
PEDFIC 1 clinical trial is recruiting at more than 40 clinical
trial sites worldwide. More information may be found on
www.clinicaltrials.gov.
The odevixibat PFIC program, or elements of it, have received
fast track, rare pediatric disease and orphan drug designations
in the United States. In addition, the the U.S. Food
and Drug Administration (FDA) has granted orphan drug
designation to odevixibat for the treatment of Alagille syndrome,
biliary atresia and primary biliary cholangitis. The European
Medicines Agency (EMA) has granted odevixibat 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 odevixibat Pediatric Investigation Plan for
PFIC. EMA also has granted orphan designation to odevixibat for the
treatment of Alagille syndrome, biliary atresia and primary biliary
cholangitis.
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 (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. 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 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, among other things:
the plans for, or progress, scope, cost, duration or 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, the planned Phase 2 clinical trial for elobixibat
in NAFLD/NASH, 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 planned pivotal trial for odevixibat in biliary
atresia or the planned Phase 2 trial for elobixibat in NAFLD/NASH;
the potential approval and commercialization of odevixibat; 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 odevixibat, elobixibat, or any other
Albireo product candidate or program or the commercial opportunity
in any target indication; the potential benefits of an orphan drug
designation; the pricing of odevixibat 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
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.
Investor Contact: Hans Vitzthum, LifeSci
Advisors, LLC., 212-915-2568Media Contact: Heather
Anderson, 6 Degrees, 980-938-0260, handerson@6degreespr.com
Source: Albireo Pharma, Inc.
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