Amylyx Pharmaceuticals Announces Completion of Enrollment in HELIOS, a Phase 2 Study of AMX0035 for the Treatment of Wolfram Syndrome
08 Fevereiro 2024 - 11:00AM
Business Wire
Preliminary results from HELIOS anticipated in
the second half of 2024
Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the
“Company”) today announced it has completed enrollment of its Phase
2 HELIOS trial of AMX0035 (sodium phenylbutyrate [PB] and
taurursodiol [TURSO]) for the treatment of Wolfram syndrome (WS), a
rare genetic disease that typically results in neurodegeneration
and premature death, which has no known cure. The trial has
enrolled 12 adult participants living with WS, and preliminary
results are anticipated in the second half of 2024.
“The unmet need for people living with WS remains significant.
WS is progressive, often fatal, and difficult to diagnose, with
current treatments focused mostly on helping to address symptoms,”
said Fumihiko Urano, MD, PhD, Principal Investigator of the HELIOS
clinical trial and Professor of Medicine in the Division of
Endocrinology, Metabolism & Lipid Research at Washington
University School of Medicine. “Our preclinical findings showed
that AMX0035 may impact relevant WS disease pathways and
manifestations, and we look forward to the preliminary results from
HELIOS anticipated later this year.”
HELIOS is an open-label proof of biology trial designed to study
the effect of AMX0035 on safety and tolerability, and various
measures of endocrinological, neurological, and ophthalmologic
function in adult participants living with WS. Data from this
initial study will inform potential future Amylyx trials for
individuals with WS.
More information on the HELIOS clinical study can be found at
www.clinicaltrials.gov, NCT05676034.
In September 2022, researchers from Washington University School
of Medicine in St. Louis, in collaboration with Amylyx, published
preclinical data in the peer-reviewed Journal of Clinical
Investigation Insight. The data explored the potential of AMX0035
as a novel therapeutic approach for WS and provided initial
proof-of-concept for its therapeutic development in WS.
“Little progress has been made in the treatment of WS, and we
are excited by the potential we have been seeing in the preclinical
work with AMX0035,” said Camille L. Bedrosian, MD, Chief Medical
Officer of Amylyx. “These preclinical findings suggest the
possibilities of AMX0035 for individuals with WS that we continue
to explore, including in the HELIOS trial. We are grateful to those
who partnered to complete this important clinical trial enrollment
milestone, in particular, the WS community, their families and
caregivers, experts, clinicians, researchers, and advocacy
leaders.”
Amylyx announced that the FDA granted orphan drug designation to
AMX0035 for the treatment of WS in November 2020. The FDA may grant
this designation to drugs and biologics intended to treat a rare
disease or condition affecting fewer than 200,000 persons in the
U.S. Orphan designation qualifies a company for certain benefits,
including financial incentives to support clinical development and
the potential for seven years of market exclusivity in the U.S.
upon regulatory approval.
About Wolfram Syndrome
Wolfram syndrome (WS) is an autosomal recessive
neurodegenerative disease characterized by childhood-onset
diabetes, optic nerve atrophy, and neurodegeneration. Common
manifestations of WS include diabetes mellitus, optic nerve
atrophy, central diabetes insipidus, sensorineural deafness,
neurogenic bladder, and progressive neurologic difficulties.
Genetic and experimental evidence suggest that endoplasmic
reticulum (ER) dysfunction is a critical pathogenic component of
WS. The prognosis of WS is poor, and many people with the disease
die prematurely with severe neurological disabilities.
About AMX0035 / RELYVRIO®/ ALBRIOZA™ / ALBRIOZA®
AMX0035 is an oral, fixed-dose combination of sodium
phenylbutyrate and taurursodiol (known as ursodoxicoltaurine
outside of the U.S.). It is approved as RELYVRIO® to treat
amyotrophic lateral sclerosis (ALS) in adults in the U.S. and
approved with conditions as ALBRIOZA™ for the treatment of ALS in
Canada. AMX0035 is being studied for the potential treatment of
other neurodegenerative diseases, and Amylyx is exploring its
treatment in other populations and regions. The formulation of
RELYVRIO, ALBRIOZA, and AMX0035 is identical.
About Amylyx Pharmaceuticals
Amylyx Pharmaceuticals, Inc. is committed to supporting and
creating more moments for the neurodegenerative community through
the discovery and development of innovative new treatments. Amylyx
is headquartered in Cambridge, Massachusetts and has operations in
Canada, EMEA, and Japan. For more information, visit amylyx.com and
follow us on LinkedIn and X, formerly known as Twitter. For
investors, please visit investors.amylyx.com.
Forward-Looking Statements
Statements contained in this press release regarding matters
that are not historical facts are “forward-looking statements”
within the meaning of the Private Securities Litigation Reform Act
of 1995, as amended. Because such statements are subject to risks
and uncertainties, actual results may differ materially from those
expressed or implied by such forward-looking statements. Such
statements include, but are not limited to, the potential of
AMX0035 as a treatment for ALS and the Company’s plans to explore
the use of AMX0035 for other neurodegenerative diseases including
Wolfram Syndrome, the timelines for the HELIOS study in WS, and
expectations regarding our longer-term strategy. Any
forward-looking statements in this press release are based on
management’s current expectations of future events and are subject
to a number of risks and uncertainties that could cause actual
results to differ materially and adversely from those set forth in
or implied by such forward-looking statements. Risks that
contribute to the uncertain nature of the forward-looking
statements include: Amylyx’ ability to fund operations, the
success, cost, and timing of Amylyx’ program development
activities, Amylyx’ ability to execute on its commercial and
regulatory strategy, regulatory developments, expectations
regarding the timing and outcome of EMA’s review of AMX0035 for the
treatment of ALS, Amylyx’ reliance on third parties, including to
conduct clinical trials and manufacture products, and the effect of
global economic uncertainty and financial market volatility caused
by economic effects of rising inflation and interest rates, the
COVID-19 pandemic, geopolitical instability, changes in
international trade relationships and military conflicts, as well
as the risks and uncertainties set forth in Amylyx’ United States
Securities and Exchange Commission (SEC) filings, including Amylyx’
Quarterly Report on Form 10-Q for the quarter ended September 30,
2023, and subsequent filings with the SEC. All forward-looking
statements contained in this press release speak only as of the
date on which they were made. Subject to any obligations under
applicable law, Amylyx undertakes no obligation to update such
statements to reflect events that occur or circumstances that exist
after the date on which they were made.
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Media Amylyx Media Team (857) 799-7274
amylyxmediateam@amylyx.com Investors Lindsey Allen (857)
320-6244 Investors@amylyx.com
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