Neurizon Therapeutics (ASX:NUZ) commenced dosing for NUZ-001 within the HEALEY ALS platform trial, marking a milestone in the clinical development of this investigational therapy.
As part of "Regimen I," the study aims to evaluate the safety and efficacy of NUZ-001 in individuals living with Amyotrophic Lateral Sclerosis through a streamlined, multi-centre trial design.
The clinical program is structured into two primary phases.
The first is a 36-week double-blind, placebo-controlled period involving 160 patients. Using a 3:1 randomisation ratio, the majority of participants will receive a 10 mg/kg dose of NUZ-001, while the remainder receive a placebo.
Following this initial stage, all participants transition into a 36-week active treatment extension period, where they will receive the study drug to monitor long-term outcomes.
The trial’s primary endpoint focuses on changes in disease severity from baseline through Week 36, as measured by the ALSFRS-R total score and overall survival.
Secondary objectives include monitoring respiratory function via slow vital capacity and survival through the 36-week mark.
By utilising the HEALEY platform’s shared infrastructure, Neurizon expects to accelerate the evaluation of NUZ-001, potentially reducing the time required to bring a new treatment option to the ALS community.
At the time of reporting, Neurizon Therapeutics's share price was $0.10.