Neurizon’s ALS Trial Entry Raises Stakes Amid Urgent Need for New Therapies

Neurizon Advances ALS Treatment Development

Neurizon Therapeutics Limited (ASX: NUZ) has taken a significant stride forward in the fight against amyotrophic lateral sclerosis (ALS) with the initiation of dosing in Regimen I of the HEALEY ALS Platform Trial. This adaptive Phase 2/3 clinical trial in the United States is designed to rigorously evaluate the efficacy and safety of Neurizon’s lead candidate, NUZ-001, in a randomised, placebo-controlled setting.

The HEALEY ALS Platform Trial is a pioneering multicentre study that leverages a master protocol to simultaneously test multiple investigational therapies for ALS. By streamlining trial infrastructure and processes across more than 70 leading clinical sites, the platform aims to accelerate the development of new treatments for this rapidly progressive neurodegenerative disease.

Trial Design and Expectations

Regimen I will enrol approximately 160 participants living with ALS, randomising them in a 3:1 ratio to receive either daily NUZ-001 at the recommended Phase 2 dose of 10 mg/kg or placebo over a 36-week period. The study includes both a randomised controlled treatment phase and an active treatment extension phase, allowing for comprehensive assessment of disease progression and safety outcomes.

Neurizon’s decision to enter this trial follows encouraging results from a Phase 1 study involving 12 participants, which demonstrated that NUZ-001 was well tolerated and showed preliminary signals of efficacy. The current trial is expected to complete enrolment in the second half of 2026, with data anticipated to provide critical insights into NUZ-001’s potential as a disease-modifying therapy.

Collaborative Effort and Regulatory Engagement

The HEALEY ALS Platform Trial is conducted by the Sean M. Healey & AMG Center for ALS at Massachusetts General Brigham, in partnership with the Network of Excellence for ALS (NEALS). This collaboration brings together some of the world’s foremost ALS experts and clinical centres, ensuring high-quality data generation and regulatory alignment, including ongoing engagement with the U.S. Food and Drug Administration.

Professor Merit Cudkowicz, Principal Investigator of the trial, emphasised the importance of patient participation and the trial’s innovative design in accelerating ALS therapy development. Neurizon’s Managing Director, Dr Michael Thurn, highlighted the milestone as a defining moment for the company and its commitment to delivering meaningful progress for people living with ALS.

Looking Ahead

While the trial is still in its early stages, the initiation of dosing marks a critical juncture for Neurizon and the broader ALS community. The adaptive platform approach not only expedites clinical development but also offers hope for more rapid identification of effective treatments in a field where therapeutic options remain limited.