Neurizon Therapeutics (ASX: NUZ; OTCQB: NUZTF) has advised that the first participant in Regimen I of the Phase 2/3 HEALEY ALS Platform Trial has completed the 36-week placebo-controlled Randomised Controlled Trial (RCT) phase and moved into the Active Treatment Extension (ATE). The announcement was dated 13 November 2026.
As eligible participants reach Week 36, they may voluntarily continue into the extension, where all participants receive investigational NUZ-001 under the trial protocol. The milestone follows the completion of enrolment of 250 participants in July 2026.
Dr Chris Bremer, Chief Executive Officer
“The first participant entering the Active Treatment Extension is an important milestone for Regimen I and reflects the continued progress of our clinical development program…”
Key facts from the update:
- 250 participants enrolled in Regimen I
- 78 US clinical sites
- Enrolment completed in July 2026
- Topline RCT results anticipated in late Q2 CY2027
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Regimen I enrolment and FDA feedback
Enrolment completed at pace
All 250 participants were enrolled across 78 activated clinical sites throughout the US in around five months, which the company described as the fastest in ALS Trial history.
The earlier trial expansion lifted Regimen I from 160 participants and was funded without a change to company funding requirements, using a philanthropic contribution and the Australian R&D Tax Incentive.
Merit Cudkowicz, MD, MSc, Executive Director of the Mass General Brigham Neuroscience Institute and Director of the Healey & AMG Center for ALS
“…After enrolling 250 participants in 5 months, we are excited to now have taken this next step in Regimen I of the HEALEY ALS Platform Trial…”
FDA feedback
The milestone also follows Neurizon’s recent receipt of written feedback from the US Food and Drug Administration (FDA). The feedback noted that positive results from the HEALEY ALS Platform Trial may support a traditional approval pathway.
It also noted that Regimen I may serve as a single registrational study for NUZ-001, supporting a New Drug Application (NDA). This is subject to the results and confirmatory evidence meeting the applicable regulatory requirements.
What the Active Treatment Extension adds
An active treatment extension follows the placebo-controlled phase of a trial. In the RCT phase, some participants receive a placebo, while in the extension all participants receive the investigational therapy, irrespective of their original treatment allocation.
The ATE is designed to generate additional longer-term safety, tolerability, functional and biomarker data, beyond the evidence collected during the RCT phase. Participants will continue to undergo regular clinical and safety assessments, including:
- Functional decline, measured using the ALS Functional Rating Scale-Revised (ALSFRS-R)
- Respiratory function
- Quality of life
- Blood and urine biomarker assessments, including neurofilament light chain (NfL)
Treatment assignments remain blinded through the extension period.
These additional observations are expected to complement the RCT dataset and contribute to the broader clinical evidence package supporting the continued development of NUZ-001. The extension is not a separate controlled assessment of efficacy, and the contribution of ATE data to any future regulatory submission remains subject to applicable requirements.
The table below summarises the Regimen I design.
| Feature | Detail |
|---|---|
| Participants | 250 across 78 US sites |
| Randomisation | 3:1 NUZ-001 to placebo |
| RCT phase | 36 weeks |
| Optional ATE | 36 weeks |
| Primary evaluation | Functional decline (ALSFRS-R) combined with survival |
| Topline RCT results | Anticipated late Q2 CY2027 |
What happens next for Neurizon
- Neurizon will continue to work with the Healey & AMG Center for ALS at Mass General Brigham as Regimen I participants progress through their respective treatment periods and eligible participants transition into the ATE.
- The company remains focused on completing the RCT phase and progressing towards database lock.
- Topline results from the RCT phase are anticipated in late Q2 CY2027, and will inform subsequent regulatory engagement with the FDA and other relevant regulatory authorities regarding the potential approval pathway for NUZ-001.
Alongside the US trial, Neurizon is advancing an oral liquid formulation of NUZ-001 in a Phase 1 study in Australia, aimed at patients who develop swallowing difficulties as ALS progresses.
NUZ-001 is an investigational product and is not approved for commercial use in any jurisdiction.
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