26-02-2026

The first participant has been dosed in Regimen I of the HEALEY ALS Platform Trial evaluating Neurizon Therapeutics’ lead candidate NUZ-001 to treat ALS.

• First participant enrolled and dosed with Neurizon’s NUZ-001 in Regimen I of the HEALEY ALS Platform Trial for treatment of ALS
• Around 160 people living with ALS to be recruited into trial across leading ALS centres in the US
• Study builds on a successful phase I trial in 12 participants with ALS, where NUZ-001 showed encouraging early signals of efficacy

The study will enrol ~160 people living with ALS, the most common form of motor neurone disease (MND) in a 36-week, randomised, double-blinded, placebo-controlled adaptive trial across leading treatment clinical centres in the US.  Enrolment is expected to be completed in the second half of calendar 2026.

The HEALEY study represents Neurizon Therapeutics’ (ASX:NUZ) registrational trial in ALS and has been designed to generate the clinical data required to support potential regulatory submissions, including to the US Food and Drug Administration  (FDA).

The trial is being conducted under the HEALEY ALS Platform Trial, a master protocol led by the Sean M. Healey & AMG Center for ALS at Mass General Brigham in partnership with the Network of Excellence for ALS (NEALS). 

The platform evaluates multiple investigational drugs concurrently under a shared infrastructure spanning more than 70 clinical sites, streamlining recruitment to deliver results more efficiently and reduce development timelines.

Entry into the landmark HEALEY platform is competitive, with candidates selected by expert committees based on scientific merit and evidence of potential benefit in ALS. 

Validation of NUZ-001

For Neurizon, inclusion validates the strength of its preclinical and early clinical data. NUZ-001 previously completed a small phase I study in 12 people living with ALS, where it was reported to be safe and well-tolerated, with encouraging preliminary signals of efficacy.

Regimen I of the study will randomise participants in a 3:1 ratio to receive either NUZ-001 at 10 mg/kg daily or placebo. 

The primary endpoint will assess efficacy on disease progression over 36 weeks, with additional measures of safety and secondary disease markers also evaluated. 

Regimen I study design. Source: Neurizon Therapeutics

‘Significant step for the regimen’ 

Professor Merit Cudkowicz, principal investigator, HEALEY ALS Platform Trial and Director, Sean M. Healey & AMG Center for ALS, Mass General Brigham said she looked forward to working with Neurizon on the new regimen and implementing its updated master protocol. 

“Beginning enrolment is a significant step for the regimen and would not be possible without the dedication of people living with ALS and their families, collaborators and our top trial sites,” he said. 

Managing Director and CEO Dr Michael Thurn said the dosing of the first participant in Regimen I of the HEALEY ALS Platform Trial marked a defining milestone for Neurizon and for NUZ-001. 

“This study represents our registrational trial in ALS – a rigorous, adaptive Phase 2/3 program designed to generate the clinical evidence required to support potential regulatory submissions,” he said. 

He said entry into the HEALEY ALS Platform Trial reflected the company’s scientific data package and the favourable safety and tolerability profile observed in its phase I and Open Label Extension studies. 

“The master protocol structure enables efficient study execution across leading ALS centres in the US, while maintaining the scientific rigour required at this stage of development.

“We are deeply grateful to the participants and their families who commit their time and energy to clinical research, often while navigating the significant challenges associated with living with ALS. 

“For people living with ALS, urgency matters. 

“Through this pivotal study, we are advancing NUZ-001 with scientific discipline, operational focus and deep respect for the ALS community as we work toward delivering meaningful progress in this devastating disease.”

Source: Stockhead

This article was developed in collaboration with Neurizon Therapeutics, a Stockhead advertiser at the time of publishing. me of publishing. on.