Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage orphan
pediatric liver disease company developing novel bile acid
modulators, today announced topline results from the Phase 2 proof-
of-concept clinical trial evaluating elobixibat, its ileal bile
acid transporter (IBAT) inhibitor currently approved in Japan for
chronic constipation, for the treatment of nonalcoholic fatty liver
disease (NAFLD) and nonalcoholic steatohepatitis (NASH). Following
promising results in preclinical studies, the Phase 2 study
achieved the primary endpoint of reduction of low-density
lipoprotein cholesterol (LDL-C) but did not achieve
proof-of-concept for other key NASH measures.
Elobixibat achieved the primary endpoint of a reduction in LDL-C
with a clinically meaningful effect of -20.5 mg/dL in serum LDL-C
compared to -11.1 mg/dL, elobixibat vs. placebo (p<0.022).
These reductions were in addition to lipid lowering treatment where
56.5% of the elobixibat and 45.8% of the placebo patients were on
stable lipid lowering therapy at baseline. Liver fat
reduction in the elobixibat group was -2.6% as measured by MRI
PDFF. The baseline alanine aminotransferase (ALT) levels were
within the normal range and there was no meaningful change observed
in the study. Overall elobixibat was well tolerated, with adverse
event incidence similar to placebo and with no serious adverse
events or discontinuations due to treatment. The study showed
an acceptable gastrointestinal tolerability with 3 of 23 elobixibat
patients reporting mild to moderate transient diarrhea attributable
to drug treatment.
The proof-of-concept Phase 2, multicenter, placebo-controlled
trial enrolled 47 patients across 15 U.S. sites, and was designed
to assess the safety and efficacy of a once-daily 5 mg dose of
elobixibat over 16 weeks in adult patients with biopsy-confirmed
NASH or a diagnosis of suspected NAFLD or NASH based on metabolic
syndrome definitions. The primary endpoint was change from baseline
in LDL-C. Secondary endpoints included change in liver fat by
imaging, and in ALT and aspartate aminotransferase levels.
Exploratory endpoints included measures of glucose and insulin
homeostasis, which are biomarkers for inflammation and
fibrosis.
“We wanted to investigate the potential of elobixibat in NASH
and allocated minimal resources to an exploratory Phase 2 study.
Based on the results of this study, we have made the decision
not to pursue further development of elobixibat in NASH,”
said Ron Cooper, President and Chief Executive Officer of
Albireo. “Our main focus continues to be on odevixibat in
rare pediatric liver diseases and we look forward to our Phase 3
topline data in the coming weeks.”
Albireo is focused and remains on track with several milestones
for the remainder of 2020, including topline results from the
PEDFIC 1 pivotal Phase 3 trial of odevixibat in progressive
familial intrahepatic cholestasis (PFIC) in the coming weeks,
potential regulatory approval, issuance of a rare pediatric disease
priority review voucher and odevixibat launch anticipated in the
second half of 2021. Other anticipated events include initiation of
a pivotal Phase 3 trial of odevixibat in Alagille syndrome by the
end of 2020, and continued enrollment of patients in the BOLD
pivotal Phase 3 trial of odevixibat in biliary atresia. Albireo
also continues to progress development of a new preclinical
candidate and expects to complete IND-enabling studies this
year.
About Elobixibat Elobixibat is a
first-in-class, once-daily, oral ileal bile acid transporter (IBAT)
inhibitor and is approved in Japan for the treatment of
patients with chronic constipation (excluding constipation caused
by organic disease). It is marketed and sold
in Japan under the trade name GOOFICE®.
About AlbireoAlbireo 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 progressive familial intrahepatic cholestasis (PFIC) and biliary
atresia, with a third Phase 3 trial being planned in Alagille
syndrome.
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 2020 Best Places to Work in Massachusetts for the second
consecutive year. 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, initiation, duration,
enrollment, results or timing for availability of results of,
development of odevixibat or any other Albireo product candidate or
program, including regarding expectations regarding the impact of
COVID-19 on our business and our ability to adapt our approach as
appropriate; the Phase 3 clinical program for odevixibat in
patients with PFIC, the pivotal trial for odevixibat in biliary
atresia (BOLD), the planned pivotal trial for odevixibat in
Alagille syndrome and Phase 2 trial for elobixibat being conducted
through EA Pharma in Japan; the target indication(s) for
development or approval, the size, design, population, location,
conduct, cost, objective, enrollment, duration or endpoints of any
clinical trial, or the timing for initiation or completion of or
availability or reporting of results from any clinical trial,
including the Phase 3 PFIC trial for odevixibat, and the long-term
open-label extension study, the pivotal trial for odevixibat in
biliary atresia, the planned pivotal trial for odevixibat in
Alagille syndrome; the potential approval and commercialization of
odevixibat; discussions with the FDA or EMA regarding our programs;
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 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: negative impacts of the COVID-19 pandemic,
including on manufacturing, supply, conduct or initiation of
clinical trials, or other aspects of our business; 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, the pivotal program
in biliary atresia or the planned pivotal program in Alagille
syndrome, 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 or other pivotal trials; 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, 617-430-7578Media Contact: Claire
LaCagnina, 6 Degrees,
315-765-1462, clacagnina@6degreespr.com
Source: Albireo Pharma, Inc.
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