Amylyx Pharmaceuticals hopes to launch largest PSP trial to date by the end of 2023

By Liza Laws

- Last updated on GMT

© Getty Images
© Getty Images

Related tags progressive supranuclear palsy Clinical trials Data management Patient centricity Patient recruitment neurodegenerative diseases Rare disease

The treatment of rare brain disease, progressive supranuclear palsy (PSP), that has no disease modifying therapies, has been given a glimmer of hope by Amylyx Pharmaceuticals.

The company announced on Thursday (October 19) that it has an upcoming presentation on the design of a phase 3 clinical trial of a drug called AMX0035 (sodium phenylbutyrate and taurursodiol [PB&TURSO]) in PSP.

The presentation will be given at Neuro 2023 and CBD International Research Symposium.

“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, 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.”

“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 Höglinger, 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 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 2)​.

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. 

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