Amylyx Pharmaceuticals has dosed the first subject in its Phase III ORION clinical trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO]) to treat patients with progressive supranuclear palsy (PSP), a rare neurodegenerative ailment.

The randomised, international, double-blind, placebo-controlled trial will evaluate the safety, efficacy, and tolerability of AMX0035 versus placebo in PSP patients. 

It aims to enrol nearly 600 subjects across Canada, US, UK, Japan and Europe.

The trial’s primary efficacy goal is to measure variation in disease progression using the 28-item Progressive Supranuclear Palsy Rating Scale (PSPRS) from baseline to week 52.

Assessing the progression of the disease through a modified 10-item PSPRS score and daily life motor activities using the Movement Disorder Society-Unified Parkinson’s Disease Rating Scale Part 2 (MDS-UPDRS Part II) will comprise secondary efficacy goals. 

Exploratory outcomes will assess variations in daily living activities, quality of life, cognitive function, brain regional volumes, overall survival, fluid biomarkers and caregiver burden.

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The trial will monitor the treatment’s safety and tolerability by tracking treatment emergent adverse events (TEAEs) and serious adverse events (SAEs) in participants.

Amylyx anticipates reporting the topline trial data within two to three years.

AMX0035 is approved in the US under the brand name RELYVRIO to treat adult amyotrophic lateral sclerosis (ALS) patients and received conditional approval in Canada for ALS.

Amylyx Pharmaceuticals Global Clinical Development head Lahar Mehta said: “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.”

In April this year, the company dosed the first participant in its Phase ll HELIOS clinical trial of AMX0035 to treat Wolfram syndrome.