Albireo Pharma, Inc. (Nasdaq: ALBO), a clinical-stage orphan
pediatric liver disease company developing novel bile acid
modulators, provided an update on the progress of the development
of odevixibat for pediatric cholestatic liver diseases and
elobixibat for NASH.
The odevixibat PEDFIC program in progressive
familial intrahepatic cholestasis (PFIC) consists of two clinical
trials. PEDFIC 1 is a randomized, double-blind, placebo-controlled,
global multicenter clinical trial designed to enroll approximately
60 patients with PFIC type 1 or type 2. PEDFIC 2 is an open-label
extension study designed to assess long-term safety and durability
of response in a cohort of patients rolled over from PEDFIC 1, and
it includes a second cohort of PFIC patients who are not eligible
for PEDFIC 1.
“We are extremely encouraged with the recruiting
efforts by the participating sites in our Phase 3 trial for
odevixibat in PFIC, as we have now enrolled over half of the study.
We also have a potentially sufficient number of patients currently
in screening and prescreening to complete the study, and we expect
to have topline data mid-2020. Approximately one third of the
recruited patients to-date have been screen failures, which is
higher than expected, but importantly, this provides greater
homogeneity of the PFIC type 1 or type 2 patients who ultimately
enroll in the trial, which preserves the integrity of the study,”
said Ron Cooper, President and Chief Executive Officer of Albireo.
“In addition, we’re excited to have screened the first patients in
the Phase 2 study with once-daily elobixibat in NAFLD/NASH, as we
believe elobixibat has the potential to find a significant place in
the emerging NASH treatment landscape.”
PEDFIC 1 is a global study with 44 sites
actively recruiting. The company expects topline data in mid-2020
and will provide a further update when the study has been fully
enrolled.
PEDFIC 2 is an open-label extension study for
patients to roll into after completing PEDFIC 1. Many of the
patients not randomized into PEDFIC 1 will potentially be eligible
for the PEDFIC 2 expanded cohort, which has a broader entry
criteria. Patients are given high-dose odevixibat, 120 (µg/kg/day)
and will be studied for 18 months. Sites are active for this global
trial, which is designed to assess long-term safety and durability
of response.
PEDFIC 2 includes another cohort for an expanded
range of PFIC patients. Patients are given high-dose odevixibat,
120 (µg/kg/day) and will be studied for 18 months. The first site
has been activated.
Odevixibat is being developed for multiple
pediatric cholestatic liver diseases, and Albireo announced biliary
atresia as the next indication for study. There is no approved
treatment for biliary atresia, and the disease is the number one
reason for pediatric liver transplants. Productive discussions on
the final protocol continue with regulatory agencies, and the
expected start of a biliary atresia pivotal trial will be in 2020.
Albireo expects the final study design to have a different size,
length and endpoint compared to the PFIC program, and the company
will provide further updates once the trial protocol and overall
timeline for the trial is established.
Bile acid modulation through IBAT inhibition
holds promise as a therapeutic approach for the treatment of NASH
based on measures of efficacy, safety and convenience. In clinical
and nonclinical studies, IBAT inhibitors have demonstrated
decreases in serum bile acids, cholesterol, liver inflammation and
liver fibrosis, as well as an increase in GLP1— all effects that
are of potential benefit in the treatment of NAFLD/NASH. IBAT
inhibitors can be given orally once a day, and they have low
systemic exposure, reducing the potential for off-target effects
and raising the possibility they could be used concomitantly with
other NASH or cardiovascular risk medicines.
Albireo’s NASH program includes a Phase 2 trial
with elobixibat, as well as development of investigational
preclinical novel bile acid modulators. The first patients were
screened in the elobixibat Phase 2 trial, which will assess the
safety and efficacy of elobixibat 5 mg in 46 NASH/NAFLD patients
over 16 weeks, as measured by liver steatosis and various
biomarkers. Topline data are expected mid-2020.
About OdevixibatOdevixibat 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 has received fast
track, rare pediatric disease and orphan drug designation
in the United States. In addition, the 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 ElobixibatElobixibat is a
first-in-class, once-daily, orally-available ileal bile acid
transporter (IBAT) inhibitor currently being evaluated in a Phase 2
clinical trial in nonalcoholic fatty liver disease (NAFLD) and
nonalcoholic steatohepatitis (NASH). In clinical studies,
elobixibat demonstrated a decrease in LDL/H cholesterol, as well as
decreased insulin resistance through an increase in GLP-1. The
first IBAT inhibitor approved globally, elobixibat 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 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. Elobixibat is in Phase 2
development in NAFLD and NASH. Approved in Japan for the
treatment of chronic constipation, elobixibat 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, Mass., 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,
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 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 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 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.,
212-915-2568
Media Contact: Heather Anderson, 6 Degrees,
980-938-0260, handerson@6degreespr.com Source: Albireo Pharma,
Inc.
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