- ORION is a randomized, double-blind,
placebo-controlled Phase 3 clinical trial evaluating the safety,
efficacy, and tolerability of AMX0035 in adults with PSP
- Study anticipates enrolling approximately 600
participants globally, with trial initiation anticipated by the end
of 2023
Amylyx Pharmaceuticals, Inc. (Nasdaq: AMLX) (“Amylyx” or the
“Company”) today announced an upcoming presentation at Neuro2023 on
the design of ORION, a planned global, Phase 3 clinical trial of
AMX0035 (sodium phenylbutyrate and taurursodiol [PB&TURSO]) in
progressive supranuclear palsy (PSP). Neuro2023: The PSP and CBD
International Research Symposium will take place on October 19-20,
2023, at the Pan Pacific Hotel in London, UK.
“We designed ORION in collaboration with the broader PSP
community, including people living with the disease, advocacy
leaders, clinicians, researchers, and other experts in the field,
and we are excited to launch what will potentially be the largest
PSP trial to date,” said Lahar Mehta, MD, Head of Global Clinical
Development at Amylyx. “There is robust scientific rationale
supporting the evaluation of AMX0035 in PSP, and we look forward to
the initiation of our Phase 3 trial which is anticipated by the end
of this year.”
Details of the poster presentation at Neuro2023 are as
follows:
Thursday, October 19, 2023, 5:00 pm –
7:00 pm BST
- Title: Design of a Global Phase 3, Randomized,
Double-blind, Placebo-Controlled Trial of AMX0035 in Progressive
Supranuclear Palsy (A35-009 ORION)
For conference information, visit:
https://www.psp.org/event/neuro2023-the-psp-and-cbd-international-research-symposium/
The presentation is available on the “Publications” tab of the
Amylyx website.
“There are currently no disease-modifying therapies approved for
the treatment of PSP, a rapidly progressive and fatal tauopathy and
movement disorder,” 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. “Based on preclinical evidence, AMX0035 is
proposed to directly target cell organelle function and mitigate
both the unfolded protein response and mitochondrial dysfunction,
two pathways that are associated with several neurodegenerative
diseases, including PSP. We look forward to investigating AMX0035’s
potential in targeting these two pathways that are implicated in
tau dysfunction and aggregation in PSP, which then lead to
neurodegeneration.”
ORION Trial Design
ORION 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. Approximately 600
participants will be enrolled across North America, Europe, and
Japan, with study initiation anticipated by the end of 2023
starting in the United States.
The primary efficacy endpoint evaluates 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).
The ORION Phase 3 trial was designed and planned in
collaboration with key global academic leaders, people living with
PSP, and industry advocacy groups.
Additional key elements of the study design:
- The study will enroll ambulant adults, 40-80 years of age, with
probable or possible PSP (also known as Richardson’s syndrome)
according to International Parkinson and Movement Disorder Society
2017 criteria and less than 5 years since developing PSP symptoms.
Key eligibility criteria include:
- A score of <40 on the 28-item PSPRS
- A score of ≥24 on the Mini Mental State Examination
- Study partner required
- No feeding tube use
- On stable dose of antiparkinsonian drugs for 60 days before
enrollment
- After a screening period, participants will be randomized in a
3 to 2 manner to receive AMX0035 or matching placebo for 52 weeks
(randomized phase).
- For all participants who complete the randomized phase, a
52-week open label extension phase will be available for continued
access to AMX0035 to further characterize the long-term efficacy
and safety of AMX0035 in this population.
Additional details regarding trial enrollment and eligibility
criteria will be shared upon initiation of the trial, which is
anticipated to start by the end of 2023.
About AMX0035
AMX0035 is an oral, fixed-dose combination of sodium
phenylbutyrate and taurursodiol (known as ursodoxicoltaurine
outside of the U.S.), which was granted full approval 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 explored in other
populations and regions, as well as for the potential treatment of
other neurodegenerative diseases. The formulation of RELYVRIO,
ALBRIOZA and AMX0035 are identical.
About PSP
Progressive supranuclear palsy (PSP) is a rare and adult-onset
neurological disorder that affects body movements, 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 ALS. PSP
typically begins in late-middle age and rapidly progresses over
time.
Multiple pathways likely contribute to the pathophysiology of
PSP, which is characterized by tau protein deposition in
subcortical regions resulting in widespread neurodegeneration. The
disease affects approximately seven in 100,000 people worldwide,
and there are currently no disease-modifying therapies approved for
the treatment of PSP.
About Amylyx Pharmaceuticals
Amylyx Pharmaceuticals, Inc. is committed to supporting and
creating more moments for the neurodegenerative disease community
through the discovery and development of innovative new treatments.
Amylyx is headquartered in Cambridge, Massachusetts and has
operations in Canada and EMEA. 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, our plans to make
AMX0035 available in Europe; the ongoing commercialization of
RELYVRIO and ALBRIOZA; the potential continued market acceptance
and market opportunity for RELYVRIO and ALBRIOZA; 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 June 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/20231019056075/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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