Albireo Pharma, Inc. (Nasdaq: ALBO), a rare liver disease company
developing novel bile acid modulators, today announced positive
topline results from its Phase 1 clinical trial of A3907, the first
oral systemic apical sodium-dependent bile acid transporter (ASBT)
inhibitor. The study achieved both primary and secondary
objectives. Phase 1 study is a first-in-human, double-blind, single
and multiple ascending dose study in healthy adult subjects to
investigate the safety, tolerability, pharmacokinetics of orally
administered A3907. A3907 was safe and well tolerated in this study
at systemic exposures that demonstrated therapeutic benefits in
preclinical models. With the potential to inhibit ileal, renal and
hepatic ASBT, A3907 could provide the optimal balance of efficacy
and tolerability in patients in multiple liver diseases.
The study met its primary objectives with data showing A3907
overall to be safe and well tolerated, with no serious adverse
events (SAEs) or discontinuations due to treatment emergent adverse
events (TEAEs). All TEAEs were mild, the most common were mainly
abdominal symptoms such as loose stools, and all participants
finished the trial protocol. No clinically significant effects in
clinical chemistry, hematology, physical examinations or ECG. The
study also met its secondary objectives with dose-related plasma
exposure up to 81 mg dose, with no accumulation. Exposure levels
were within the range that show effects in animal disease models.
Single doses of A3907 showed dose proportional increases in plasma
exposure up to 81 mg with no further increase at a dose of 162 mg.
The study showed target engagement with
7alpha-hydroxy-4-cholesten-3-one (C4) increases and low-density
lipoprotein cholesterol (LDL-C) reductions, with C4 increases
indicating elevation in bile acid excretion, while LDL-C reduction
indicating elevated synthesis of bile acids from cholesterol.
“The results from our Phase 1 study of A3907 show that the drug
candidate is safe and well tolerated with favorable
pharmacokinetics and high systemic exposure, giving us the
greenlight to advance to a Phase 2 study,” said Jan Mattsson, Chief
Scientific Officer of Albireo. “These results represent an exciting
milestone in the development of this next generation bile acid
modulators as we could potentially be offering physicians new
treatments to treat liver diseases that could increase efficacy
without sacrificing tolerability for patients.”
The Phase 1 trial was a randomized, double-blind,
placebo-controlled, single and seven day multiple-dose study was
conducted in 54 and 22, respectively, healthy subjects. Doses
evaluated were 1-162 mg in the single ascending dose portion, and
9-67.5 mg in the multiple ascending dose portion. Primary
objectives were safety and tolerability, secondary objectives were
evaluation of pharmacokinetics, and there were multiple exploratory
endpoints.
About A3907A3907 is the first oral systemic
apical sodium-dependent bile acid transporter (ASBT) inhibitor with
high oral bioavailability that has potential to inhibit intestinal
and renal bile acid reuptake as well as ASBT expressed by
cholangiocytes. Due to high oral bioavailability, A3907 can inhibit
ASBT in the intestine and kidney, with the potential to increase
elimination of bile acids by both fecal and urinary excretion. By
using dual pathway diversion of bile acids, next generation
modulators like A3907 seek to increase efficacy without the dose
limiting diarrhea seen with bile acid transport inhibitors today.
A3907 is being developed for adult cholestatic liver diseases such
as primary sclerosing cholangitis (PSC) and primary biliary
cholangitis (PBC).
The completed Phase 1 study of A3907 is a first-in-human,
double-blind, single and multiple ascending dose study in healthy
adult subjects to investigate the safety, tolerability,
pharmacokinetics and pharmacodynamics of an A3907 oral formulation.
In pre-clinical studies, A3907 showed high systemic exposure and
increased level of urinary bile acid secretion in mice, and in a
mice model of cholestasis and sclerosing cholangitis, A3907
decreased serum bile acids, and reduced plasma levels of
transaminases as well as markers for cell damage and fibrosis.
About Cholestatic Adult Liver DiseasesAdult
cholestatic diseases are a diverse group of disorders known for the
appearance of jaundice, fatigue, pruritus and/or complications of
cirrhosis. The most common adult cholestatic liver diseases are
primary biliary cholangitis and primary sclerosing cholangitis.
Primary biliary cholangitis is a chronic disease in which the bile
ducts in the liver are slowly destroyed. When the bile ducts are
damaged, bile can back up in the liver and sometimes lead to
irreversible scarring of liver tissue (cirrhosis). Primary
sclerosing cholangitis is a disease of the bile ducts where
inflammation causes scars within the bile ducts. These scars make
the ducts hard and narrow, gradually causing serious liver damage
that leads to liver failure, repeated infections and tumors of the
bile duct or liver.
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 completed
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: 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; or the
potential benefits or competitive position of Bylvay or any other
Albireo product candidate or program or the commercial opportunity
in any target indication; 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:
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
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
Albireo Pharma (NASDAQ:ALBO)
Historical Stock Chart
Von Mär 2024 bis Apr 2024
Albireo Pharma (NASDAQ:ALBO)
Historical Stock Chart
Von Apr 2023 bis Apr 2024