Neurizon Therapeutics Limited had advised that Regimen I of the HEALEY ALS Platform Trial will increase its total patient cohort from 160 to 240 participants. The expansion is driven by the enrolment rate exceeding original expectations and the absence of a concurrent regimen during the expected recruitment period. Expanded design maintains statistical power for the primary endpoint aligned with the original assumptions underpinning the study and allows more robust analysis of subgroups and biomarkers.
The current recruitment momentum is expected to accelerate the overall trial timeline relative to previous expectations, despite the increased recruitment target. Based on current trends, the Company now anticipates last participant dosing in Second Quarter 2027, with topline results expected in early Third Quarter 2027. Following an extensive review of the scientific, statistical, financial and regulatory considerations, as well as ongoing engagement with the Sean M. Healey & AMG Center, Regimen I will now comprise 180 NUZ-001 participants and 60 Regimen I placebo controls.
As of 22 May 2026, 113 participants have been assigned to Regimen I and 74 participants have been dosed. There are 64 clinical trial sites activated across the United States and 193 participants have been screened to date under the master protocol. While current recruitment momentum continues to track ahead of prior expectations, future recruitment rates may vary over time.
Based on the expanded 240-patient design, accelerated enrolment timelines and a meaningful financial contribution of philanthropic funds from the Sean M. Healey & AMG Center for ALS at Mass General Brigham, Neurizon expects the total funding requirements through to study completion to remain unchanged. Neurizon remains focused on efficiently delivering a scientifically meaningful outcome from the HEALEY ALS Platform Trial. The expansion of the trial has no impact on Neurizon's expected costs through to topline results, with only a modest overall increase in the total cost of completing the full trial.
This increase is expected to be further offset through eligibility under the Australian Federal Government's R&D Tax Incentive Program, together with the financial contribution of philanthropic funds from the Sean M. Healey & AMG Center for ALS at Mass General Brigham. Neurizon believes expanding the regimen is a fiscally disciplined and strategically attractive decision to generate a larger, more statistically robust, and internally consistent ALS dataset, which eliminates reliance on placebo groups from other regimens and maintains the original statistical assumptions underpinning the study at topline results. The HEALEY ALS Platform Trial (ClinicalTrials.gov identifier: NCT04297683) is a multicentre, double-blind, placebo controlled adaptive Phase 2/3 clinical trial conducted by the Sean M. Healey & AMG Center for ALS at Mass General Brigham in the United States, created in partnership with the Network of Excellence for ALS (NEALS).
Entry into the HEALEY ALS Platform Trial is competitive, with drug candidates reviewed and selected by expert committees based on scientific merit and evidence of potential benefit in ALS. The goal of the HEALEY ALS Platform Trial is to accelerate the development of potential new ALS therapies. Neurizon is developing its lead drug candidate, NUZ-001, for the treatment of ALS, which is the most common form of motor neurone disease.
NUZ-001 is an investigational product and is not approved for commercial use in any jurisdiction.
