Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company
developing novel bile acid modulators, today announced new data
from the Phase 3 PEDFIC 1 study and PEDFIC 2 long-term extension
study of Albireo’s product, Bylvay (odevixibat), and first data
from preclinical studies of cholestatic and viral disease product
candidate, A2342. The data demonstrate evidence of long-term
treatment benefits of Bylvay based on improved liver health and
function across progressive familial intrahepatic cholestasis
(PFIC) types that reduce the disease burden in the treatment of
pruritus in PFIC. In addition, the company will showcase the first
data from A2342 showing potential of sodium-taurocholate
co-transporting peptide (NTCP) inhibition in viral and cholestatic
liver diseases. The data will be presented at the American
Association for the Study of Liver Diseases (AASLD) The Liver
Meeting® 2021, which is being held virtually November 12 – 15.
“The large body of evidence we have from our Phase 3 PEDFIC
studies demonstrate the long-term clinical benefit of Bylvay across
PFIC types, with some patients being tracked up to 128 weeks,” said
Patrick Horn, M.D., Ph.D., Chief Medical Officer at Albireo. “We
also see evidence of sustained improvements in hepatic health,
quality of sleep and growth, which support the potential of Bylvay
to reduce the disease burden and improve the standard of care.”
Bylvay PEDFIC 1 & 2 Treatment DataThe
Company will present data on Bylvay in one oral presentation and
four posters. 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. 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 in a randomized, double-blind,
placebo-controlled trial, and PEDFIC 2 is a long-term, open-label
Phase 3 extension study. Key findings include:
Data Demonstrate Long-Term Clinical Benefits of
Bylvay
- Long-Term Clinical Benefit Across PFIC Types with Large
and Sustained Improvements in Serum Bile Acids and Pruritus (poster
#1950): Results of pooled analysis in patients with PFIC
from two Phase 3 studies show that Bylvay was associated with
sustained improvements in mean serum bile acids (sBA) and pruritus
scores over time.
- Overall, 84 patients received Bylvay during the pooled analysis
period, and the overall median (range) exposure from the first dose
of Bylvay was 53 (3−128) weeks.
- sBA responders had larger improvements in pruritus than sBA
non-responders or partial responders as defined by those who did
not meet sBA response criteria reflecting either perceived
treatment effects or patients with a partial sBA response (i.e., a
sub-threshold reduction in sBAs).
- Approximately 40% of patients met criteria for sBA response
(≥70% reduction in sBAs or sBAs ≤70 μmol/L) during Bylvay
treatment. When treatment response was defined by sBA and/or
pruritus criteria (≥70% reduction in sBAs or sBAs ≤70 μmol/L),
approximately 60% of patients treated with Bylvay achieved a
treatment response. In addition, there are a number of patients
that are partial responders.
- Bylvay Reduced Autotaxin, Pruritus and sBAs (oral
presentation; session ID 2271): Autotaxin can be a marker
of liver injury in cholestatic patients and elevated levels have
been also associated with increased pruritus. Bylvay treatment
reduced autotaxin, pruritus and sBAs with change or percent change
from baseline to weeks 25 – 48 in patients with PFIC.
- Results of this pooled analysis in children with PFIC from
PEDFIC 1 and PEDFIC 2 studies show that Bylvay reduced autotaxin
(-50%), pruritus (-1.4) and sBAs (-49%).
- Significant correlations were observed between reductions in
each pair of these parameters.
Evidence of Reducing Disease Burden, Improving Quality
of Life
- Improvement in Hepatic Health, Sleep and Growth (poster
#1951): Bylvay treatment for up to 72 weeks in patients
with PFIC was associated with effects on hepatic parameters, growth
and sleep.
- Overall, 84 patients received Bylvay during the pooled analysis
period, and the overall median (range) exposure from the first dose
of Bylvay was 53 (3−128) weeks.
- From baseline to week 72, responders had mean improvement
in hepatic health parameters
in transaminases and total bilirubin levels, quality
of sleep and growth, with greater improvement observed in
responders compared with non-responders.
- sBA responders had large decreases in
caregiver-reported percentage of days patients
had scratching associated with
bleeding, needed soothing and needed help
falling asleep (–47%, –76%, –75%,
respectively); increases or smaller changes were
observed in sBA non-responders (3%, –24%, −35%,
respectively).
- Patients who were non-responders had more pronounced
growth deficits at baseline; however, with
treatment, mean height and weight Z
scores increased in both responders and non-responders.
- Natural Variation in
Clinical Signs and Symptoms (poster #1969): Prior to the
start of PEDFIC 1, patients with PFIC1 and PFIC2 had considerable
variations in sBAs and had variations in pruritus scores.
- These data highlight that patients
with PFIC experience natural fluctuations in clinical signs and
symptoms because of their underlying disease. Before study start in
the overall population, the median (range) of per-patient standard
deviations across all pre-treatment measurements was 42 (5–183)
μmol/L for sBAs and 0.6 (0–1.2) for pruritus scores.
Clinical Benefits Across PFIC Types
- Clinical Benefits of Bylvay in PFIC3 and PFIC6 (poster
#1951): During up to 54 weeks of Bylvay treatment,
patients with PFIC3 and PFIC6 experienced clinical benefits,
including reductions in sBAs and improvement in pruritus symptoms,
growth and sleep parameters.
- Patients with PFIC3 and PFIC6 experienced mean reductions vs.
baseline in sBAs (-91 mmol/L and -78 mmol/L, respectively)
improvement in pruritus symptoms (-1.6 and -1.8, respectively),
height (0.2 and 0.1, respectively), weight (0.1 and 0.5,
respectively) and sleep parameters (-29 and -21, respectively),
through week 36.
The observed safety and tolerability profile of Bylvay was
consistent across studies, treatment groups and doses, regardless
of PFIC classification or BSEP subtype. No drug-related serious
adverse events were reported in either PEDFIC 1 or PEDFIC 2.
Treatment-related diarrhea/frequent bowel movements reported in
9.5% of Bylvay treated patients in PEDFIC 1 and 5% of
placebo-treated patients. Individual abstracts present data for
specific safety and tolerability.
A2342 Preclinical Data in Viral DiseaseThe
Company will also present data from its preclinical studies of
A2342, the first oral NTCP inhibitor in development for viral and
cholestatic liver diseases. IND-enabling studies for A2342 are
currently being completed. Data to be presented is from studies on
the impact of A2342 in models of in vitro and in vivo hepatitis B
virus (HBV) infection and in animal models demonstrating NTCP
target engagement. The first study (poster #837) aimed to
investigate the antiviral efficacy of A2342 in vitro and in vivo,
with the second study (poster #848) assessing the contribution of
OATPs and identifying preclinical species to model NTCP inhibition
in humans. Key findings from the A2342 preclinical studies
include:
- Prevents HBV Entry In Vitro and Attenuates HBV
Replication In Vivo: In vitro experiments demonstrated
that A2342 displayed long-lasting inhibition of human NTCP bile
acid transport activity and prevented HBV infection of human
hepatocytes without affecting cell viability. A2342 also
demonstrates good oral pharmacokinetics and attenuates HBV
replication in humanized uPA/SCID mice in a dose-dependent
manner.
- Induced Dose-Dependent Elevation of sBAs in Organic
Anion Transporting Polypeptide (OATP) Knock Out (KO) Mice and
Cynomolgus Monkeys: A2342 increased sBA levels in a
dose-dependent manner in cynomolgus monkeys (3−30 mg/kg, n=2).
Similar transient increases in sBAs have been observed in humans
with SubQ NTCP inhibitors. This data thus provides excellent
translational predictability to humans.
“It’s exciting to show the first data from our preclinical
studies of A2342 that demonstrate activity in diseases like HBV. In
addition, the NTCP engagement in non-human primates provides
increased confidence in the translation of A2342 to humans,” said
Ron Cooper, President and Chief Executive Officer of Albireo. “To
be able to progress our entire pipeline in both the pediatric and
adult liver programs demonstrates our ability to execute and create
future value.”
Post-AASLD Conference CallAlbireo will host a
post-AASLD conference call and live audio webcast on November 16 at
10:00am EST. Presenters will include Ron Cooper, President and
Chief Executive Officer; Patrick Horn, M.D., Ph.D., Chief Medical
Officer and Jan Mattsson, Ph.D., Chief Scientific Officer and
Co-Founder. To access the live conference call by phone, please
dial 877-407-0792 (domestic) or 201-689-8263 (international) and
provide the access code 13724001. The virtual event will also be
accessible from the Albireo Media and Investors page. To ensure a
timely connection to the webcast, it is recommended that
participants register at least 15 minutes prior to the scheduled
start time. An archived version of the webcast will be available
for replay in the Events & Presentations section of the Media
& Investors page of Albireo’s website for two weeks following
the event.
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; expectations about Bylvay’s acceptance by healthcare
practitioners to treat PFIC patients; 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), and the pivotal trial
for Bylvay in Alagille syndrome (ASSERT); the Phase 1 trial for
A3907; 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, and the BOLD and
ASSERT trials; 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 the potential benefits of
an orphan drug designation. 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:
there are no guarantees that Bylvay will be commercially
successful; we may encounter issues, delays or other challenges in
launching or commercializing Bylvay; whether Bylvay receives
adequate reimbursement from third-party payors; the degree to which
Bylvay receives acceptance from patients and physicians for its
approved indication; challenges associated with execution of our
sales activities, which in each case could limit the potential of
our product; results achieved in Bylvay in the treatment of
patients with PFIC once we have launched the product may be
different than observed in clinical trials, and may vary among
patients; other potential 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 Bylvay to date,
including findings in indications other than PFIC, will be
predictive of results from other clinical trials of Bylvay; 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, Company’s clinical 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.
Media Contact:Colleen Alabiso,
857-356-3905, colleen.alabiso@albireopharma.comLauren Sneider,
857-300-1737, lauren.sneider@albireopharma.com
Investor Contact:Hans Vitzthum, LifeSci
Advisors, LLC., 617-430-7578
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