Amylyx Pharmaceuticals to Present Clinical Trial Design of ORION, a Phase 3 Global Study of AMX0035 in Progressive Supranuclear Palsy (PSP), at the Neuro2023 PSP and CBD International Research Symposium
- 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
“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
Details of the poster presentation at Neuro2023 are as follows:
- 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.
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
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 Society2017 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.
AMX0035 is an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol (known as ursodoxicoltaurine outside of the
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.
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
Amylyx Media Team