- Largest ever PSP clinical trial will evaluate
the efficacy and safety of AMX0035 in adults with PSP compared to
placebo
- Trial to enroll approximately 600
participants across the U.S., Canada, Europe, and Japan
Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the
“Company”) today announced that the first participant has been
dosed in ORION, a randomized, double-blind, placebo-controlled
Phase 3 clinical trial of AMX0035 (sodium phenylbutyrate [PB] and
taurursodiol [TURSO]) for the treatment of progressive supranuclear
palsy (PSP). The Phase 3 trial will enroll approximately 600
participants in approximately 100 sites across the United States,
Canada, the European Union, the United Kingdom, and Japan, making
this the largest PSP clinical trial to date.
More information on the ORION clinical trial can be found at
www.clinicaltrials.gov, NCT06122662.
“There is a pressing unmet need within the PSP community for new
and effective treatment options, as no disease-modifying therapies
have been approved for the treatment of the disease,” said Dr.
Kristophe Diaz, Executive Director and Chief Science Officer at
CurePSP. “We’re looking forward to continuing our collaboration on
ORION, recognizing that a united global effort is essential to
usher in promising advancements and raise awareness for people
living with PSP and their families.”
The primary efficacy endpoint will evaluate change in disease
progression from baseline to Week 52 as measured by total score on
the 28-item Progressive Supranuclear Palsy Rating Scale (PSPRS), an
established and validated endpoint in PSP clinical trials.
Secondary efficacy endpoints are disease progression as measured
by a modified 10-item PSPRS score and motor aspects of activities
of daily life as measured by the Movement Disorder Society-Unified
Parkinson's Disease Rating Scale Part 2 (MDS-UPDRS Part II).
Exploratory outcomes include changes in activities of daily living,
cognitive function, quality of life, overall survival, brain
regional volumes, fluid biomarkers of neuronal injury/inflammation,
and caregiver burden.
Safety and tolerability will be evaluated by assessing the
frequency of treatment emergent adverse events (TEAEs) and serious
adverse events (SAEs). Participants completing the 52-week
randomized, placebo-controlled phase of the trial will have the
option to enroll in an Open Label Extension where all participants
will receive AMX0035 for up to an additional year. Topline results
are anticipated in 2-3 years.
“AMX0035 has a strong scientific rationale in PSP, targeting key
upstream pathways in disease pathophysiology including the unfolded
protein response and mitochondrial dysfunction. Furthermore, PSP is
a tauopathy associated with tau dysfunction, tau aggregation, and
widespread neurodegeneration, and AMX0035 has been shown to reduce
p-tau in a previous Alzheimer’s disease trial. These components are
essential to the pathophysiology of PSP, and we are excited about
the strong scientific rationale supporting AMX0035 in PSP,” said
Prof. Dr. Günter H�glinger, Director of the Department of Neurology
at Ludwig-Maximilians-University (LMU) Hospital, Munich, Germany,
and Primary Investigator of the Phase 3 ORION clinical trial.
“Given PSP is a relentlessly progressive disease, it is
imperative that we respond to the unmet needs of the community with
a sense of urgency. Our current focus is on activating clinical
trial sites in all participating regions in order to complete the
trial as quickly and efficiently as possible, gearing toward what
could potentially be the first therapy for the PSP community,” said
Lahar Mehta, MD, Head of Global Clinical Development at Amylyx.
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 the ORION Trial
The Phase 3 ORION trial (NCT06122662) is a global, randomized,
double-blind, placebo-controlled Phase 3 clinical trial designed to
assess the efficacy, safety, and tolerability of AMX0035 compared
to placebo in people living with progressive supranuclear palsy
(PSP). Approximately 600 participants will be enrolled across the
United States, Canada, the European Union, the United Kingdom, and
Japan. The ORION Phase 3 trial was designed and planned in
collaboration with key global academic leaders, people living with
PSP and their caregivers, and industry advocacy organizations.
About PSP
Progressive supranuclear palsy (PSP) is a sporadic, rare and
adult-onset neurodegenerative disorder that affects walking and
balance, eye movement, swallowing, and speech.
People living with PSP have a life expectancy of six to eight
years after initial diagnosis, and its epidemiology is similar to
that of amyotrophic lateral sclerosis (ALS). PSP typically begins
in late-middle age and rapidly progresses over time. The disease
affects approximately seven in 100,000 people worldwide, and there
are currently no disease-modifying therapies approved for the
treatment of PSP.
PSP is characterized by abnormal tau inclusions and is
consequently also known as a tauopathy. Similar to other
neurodegenerative diseases, pathophysiologic changes underlying PSP
are multifactorial with several genetic and environmental factors
likely contributing to tau dysfunction and aggregation.
Multiple pathways, including genetic mutations, endoplasmic
reticulum (ER) stress and the activation of unfolded protein
response, mitochondrial dysfunction, and neuroinflammation have
been implicated as contributors to tau dysfunction and
aggregation.
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
PSP, the timelines for the ORION study in PSP, 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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version on businesswire.com: https://www.businesswire.com/news/home/20231222281941/en/
Media Amylyx Media Team (857) 799-7274
amylyxmediateam@amylyx.com
Investors Lindsey Allen Amylyx Pharmaceuticals, Inc.
(857) 320-6244 Investors@amylyx.com
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