Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company
developing novel bile acid modulators, will be presenting five
posters on Bylvay (odevixibat) at the North American Society for
Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN)
meeting being held December 12–18. Data will be presented that show
evidence of correlations in serum bile acid reductions and
long-term improvements in pruritus and sleep in patients with
progressive familial intrahepatic cholestasis (PFIC); reductions in
serum bile acids and improvements in pruritus with and without
concomitant ursodeoxycholic acid (UDCA) and/or rifampicin use; and
the efficacy and safety of Bylvay in patients with PFIC and prior
partial external biliary diversion (PEBD). Data on disease burden
and natural history of PFIC will be presented to describe the
clinical characteristics of the disease. There will also be a
poster presentation on the Phase 3 double-blind, randomized,
placebo-controlled ASSERT study of Bylvay in Alagille syndrome
(ALGS). Bylvay is a potent, non-systemic ileal bile acid transport
inhibitor (IBATi) that is approved in the U.S. for the treatment of
pruritus in patients 3 months of age and older in all types of PFIC
and in Europe for the treatment of all types of PFIC in patients
aged 6 months or older.
“We are pleased to present additional data supporting Bylvay’s
potential to reduce disease burden and improve the standard of care
in the treatment of pruritus in PFIC,” said Jan Mattsson, Chief
Scientific Officer at Albireo. “As we continue to see sustained
correlations between the reduction of serum bile acids and
long-term improvements in pruritus and quality of life measures, as
well as certain results from the use of Bylvay with post-PEBD
patients, we are encouraged that data could demonstrate Bylvay’s
impact on disease modification over time.”
Bylvay PEDFIC 1 & 2 Treatment Data PEDFIC 1
was the first and largest, global, pivotal Phase 3 study conducted
in PFIC, which evaluated the efficacy and tolerability of Bylvay in
reducing pruritus and serum bile acids (sBAs) in a randomized,
double-blind, placebo-controlled trial, and PEDFIC 2 is a
long-term, open-label Phase 3 extension study.
Poster of Distinction Poster
#69: Relationships Between Decreases in Serum Bile Acids,
Pruritus, and Sleep Disturbance Scores With Up to 72 Weeks of
Odevixibat Treatment in Patients With Progressive Familial
Intrahepatic CholestasisPoster Session I: Monday,
December 13, 1:30-2:15pm ET
- Significant correlations were observed between reductions in
sBAs and reductions in pruritus and most sleep disturbance
scores.
- Percentage change in sBAs from baseline to weeks 49−72 was
significantly correlated with change in pruritus scores during that
interval (r=0.58; P<0.001).
- Moderate correlations were also observed between percentage
change in sBAs from baseline to weeks 49−72 and changes during that
interval in caregiver-reported percentage of days where patients
had bleeding associated with scratching, needed soothing or help
falling asleep, and were sleeping with caregivers.
- No serious drug-related treatment emergent adverse events
(TEAEs) or deaths occurred. The overall incidence of any event of
diarrhea was 21% and all instances were mild to moderate in
severity and resolved, most without intervention.
Poster of Distinction Poster
#78: Efficacy and Safety of Odevixibat Therapy With
Concomitant UDCA or Rifampicin in Children With Progressive
Familial Intrahepatic Cholestasis: Data From the PEDFIC 1 and
PEDFIC 2 Trials Poster Session I: Monday, December
13, 1:30-2:15pm ET
- Patients with PFIC receiving Bylvay treatment experienced
reductions in sBAs and improvements in pruritus with and without
concomitant ursodeoxycholic acid (UDCA) and/or rifampicin
use.
- After 48 weeks of Bylvay treatment, percentages of patients
meeting criteria for sBA response were similar among patients using
vs not using UDCA and/or rifampicin (using vs not using,
each 67%)
- 74% and 40% in patients using vs not using UDCA,
respectively.
- 54% and 82% in patients using vs not using rifampicin.
- Mean proportions of positive pruritus assessments (PPAs) were
similar in patients using vs not using UDCA (65% vs 72%,
respectively) and in patients using vs not using UDCA and/or
rifampicin (66% vs 69%); mean proportions of PPAs in patients using
vs not using rifampicin (56% and 81%).
- Safety and tolerability were comparable in patients using vs
not using UDCA and/or rifampicin with Bylvay. Incidence of TEAEs
was similar in patients using vs not using UDCA and/or rifampicin
(78% vs 90%, respectively), as well as in patients using vs not
using UDCA (77% vs 87%) and rifampicin (75% vs 86%).
Poster #303: Efficacy And Safety Of Odevixibat
In Children With Progressive Familial Intrahepatic Cholestasis With
Prior Partial External Biliary Diversion Poster Session
II: Wednesday, December 15, 1:30-2:15pm ET
- Improvements were observed in sBA levels, pruritus, quality of
life (QoL), and sleep parameters in some patients who had prior
partial external biliary diversion, or PEBD surgery. Improvements
were observed despite elevated sBAs and pruritus in these patients
at baseline, suggesting that some patients with poor response to
PEBD could potentially respond to Bylvay treatment.
- All of the 10 patients with prior PEBD had elevated sBAs and
pruritus scores prior to the first dose of Bylvay, indicating that
prior PEBD surgery was unsuccessful or only partially successful.
- 7 patients had reductions in pruritus score and 4 met criteria
for pruritus response (i.e., ≥1-point drop in pruritus score).
- 5 patients had reductions in sBA levels; 1 patient, who also
met pruritus response criteria, met criteria for sBA response
(i.e., sBAs reduced by ≥70% or levels ≤70 μmol/L) at last
assessment.
- Of the 9 patients with post-baseline QoL assessments, 6 had
improved observer-reported PedsQL total scores, including all 4
pruritus and/or sBAs responders.
- Pruritus and/or sBA responders also had reductions from
baseline to last assessment in percentage of days with bleeding
associated with scratching, needing soothing or help falling
asleep, and sleeping with caregivers.
- TEAEs, including mild abdominal pain, were observed in the
subgroup of patients with prior PEBD; all TEAEs were mild to
moderate in severity, and no patients discontinued due to a
TEAE.
Poster #292: Disease Burden and Natural History
of Progressive Familial Intrahepatic Cholestasis: Baseline Clinical
Characteristics Among Odevixibat-Treated Patients in the Phase 3
PEDFIC Studies Poster Session II: Wednesday,
December 15, 1:30-2:15pm ET
- Baseline characteristics of the patient population studied
reflect the broader population of patients with PFIC; specifically
this is a pediatric population with cholestasis, impaired hepatic
function, growth deficits, and impacted sleep parameters; and PFIC
had a considerable impact on the QoL of patients and their
caregivers.
- The complexity of PFIC was illustrated by the heterogeneity and
severity of the clinical signs and symptoms in these patients with
different types of PFIC.
Bylvay in ASSERT StudyPoster
#281: The ASSERT Study: A Phase 3 Double-blind,
Randomized, Placebo-controlled Study of the Safety and Efficacy of
Odevixibat In Patients With Alagille SyndromePoster Session
II: Wednesday, December 15, 1:30-2:15pm ET
- ASSERT is an on-going, global Phase 3 pivotal trial designed to
evaluate the safety and efficacy of Bylvay for 24 weeks in
relieving pruritus in patients with Alagille syndrome.
- ASSERT is expected
to enroll approximately 63 patients, including approximately
45 patients aged <18 years and an additional
exploratory cohort of up to 18 patients aged ≥18
years.
Full scientific abstracts are available and can be viewed here:
Journal of Pediatric Gastroenterology and Nutrition.
About Bylvay
(odevixibat)Bylvay is the first drug approved in
the U.S. for the treatment of pruritus in patients 3 months of age
and older in all types of progressive familial intrahepatic
cholestasis (PFIC). The European Commission (EC) and UK Medicines
and Healthcare Products Regulatory Agency (MHRA) have also granted
marketing authorization of Bylvay for the treatment of PFIC in
patients aged 6 months or older. Bylvay is available for sale in
Germany and will be available for sale in other European countries
following pricing and reimbursement approval. A potent, once-daily,
non-systemic ileal bile acid transport inhibitor, Bylvay acts
locally in the small intestine. Bylvay can be taken as a capsule
for patients that are able to swallow capsules, or opened and
sprinkled onto food, which is a factor of key importance for
adherence in a pediatric patient population. The medicine can only
be obtained with a prescription. For more information about using
Bylvay, see the package leaflet or contact your doctor or
pharmacist. For full prescribing information, visit
www.bylvay.com.
In the U.S. and Europe, Bylvay has orphan exclusivity for its
approved PFIC indications, and orphan designations for the
treatment of Alagille syndrome, biliary atresia and primary biliary
cholangitis. Bylvay is being evaluated in the ongoing PEDFIC 2
open-label trial in patients with PFIC, in the BOLD Phase 3 study
for patients with biliary atresia and the ASSERT Phase 3 study for
Alagille syndrome.
About AlbireoAlbireo Pharma is a rare disease
company focused on the development of novel bile acid modulators to
treat rare pediatric and adult liver diseases. Albireo’s lead
product, Bylvay, was approved by the U.S. FDA as the first drug for
the treatment of pruritus in all types of progressive familial
intrahepatic cholestasis (PFIC), and it is also being developed to
treat other rare pediatric cholestatic liver diseases with Phase 3
trials in Alagille syndrome and biliary atresia, as well as an
Open-label Extension (OLE) study for PFIC. In Europe, Bylvay has
been approved for the treatment of PFIC and has been submitted for
pricing and reimbursement approval. The Company has also initiated
a Phase 1 clinical trial for A3907 to advance development in adult
cholestatic liver disease, with IND-enabling studies moving ahead
with A2342 for viral and cholestatic liver disease. Albireo was
spun out from AstraZeneca in 2008 and is headquartered in Boston,
Massachusetts, with its key operating subsidiary in Gothenburg,
Sweden. The Boston Business Journal named Albireo one of the 2019
and 2020 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: Albireo’s commercialization
plans and expectations for commercializing Bylvay in the U.S. and
Europe; the plans for, or progress, scope, cost, initiation,
duration, enrollment, results or timing for availability of results
of, development of Bylvay, A3907, A2342 or any other Albireo
product candidate or program; the pivotal trial for Bylvay in
biliary atresia (BOLD); the pivotal trial for Bylvay in Alagille
syndrome (ASSERT); the Phase 1 trial for A3907; the IND-enabling
studies for A2342; 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
long-term open-label extension study for Bylvay in PFIC, the BOLD
and ASSERT trials, Phase 1 trial for A3907 and the IND-enabling
studies for A2342; potential regulatory approval by and discussions
with the FDA or EMA regarding our programs; the potential benefits
or competitive position of Bylvay or any other Albireo product
candidate or program or the commercial opportunity in any target
indication; the potential effects of Bylvay of the treatment of
PFIC patients and its potential to improve the current standard of
care; 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,” or the negative of these terms or other 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: results achieved in Bylvay in the treatment of
patients with PFIC may be different than observed in clinical
trials, and may vary among patients; whether favorable findings
from clinical trials of Bylvay to date, including findings in
indications other than PFIC, will be predictive of results from
other clinical trials of Bylvay; there is no guarantee that Bylvay
will be approved in jurisdictions or for indications beyond the
jurisdictions in which or indications for which Bylvay is currently
approved; there is no guarantee that our other products candidates
will be approved; estimates of the addressable patient population
for target indications may prove to be incorrect; 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 Bylvay, including BOLD and ASSERT, and the
Phase 1 clinical trial of A3907, and the outcomes of such trials;
Albireo’s ability to obtain coverage, pricing or reimbursement for
approved products in the United States or Europe; delays or other
challenges in the recruitment of patients for, or the conduct of,
the Company’s clinical trials; and the Company’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.
Media Contact:Colleen Alabiso,
857-356-3905, colleen.alabiso@albireopharma.com
Investor Contact:Hans Vitzthum, LifeSci
Advisors, LLC., 617-430-7578
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