How Neurizon’s NUZ-001 Extends ALS Survival by 16 Months

Long-Term Safety Confirmed

Neurizon Therapeutics Limited (ASX – NUZ) has unveiled positive topline results from its Phase 1 Open-Label Extension (OLE) study evaluating NUZ-001, its lead candidate for amyotrophic lateral sclerosis (ALS). The study confirms that long-term treatment with NUZ-001 at the recommended Phase 2 dose is safe and well-tolerated, with no treatment-related deaths and only mild to moderate adverse events reported. Notably, five patients continue to receive NUZ-001 under Australia’s Therapeutic Goods Administration Special Access Scheme, with some on treatment for over 2.5 years.

Encouraging Efficacy Signals

Beyond safety, the OLE study revealed promising preliminary efficacy signals. Patients treated with NUZ-001 experienced a durable functional benefit, with the ALS Functional Rating Scale-Revised (ALSFRS-R) decline slowed to -0.88 points per month. Respiratory function, measured by Slow Vital Capacity (SVC), remained stable, indicating a potential disease-modifying effect. Biomarker analyses showed stable plasma neurofilament light chain (NfL) levels and a 17% reduction in urinary p75, both supportive of slowed disease progression.

Survival Advantage Compared to Historical Controls

Crucially, when compared to matched untreated historical controls from the extensive PRO-ACT database, NUZ-001 treatment was associated with a significant survival benefit. Statistical analysis demonstrated a 76.7% reduction in the risk of death (hazard ratio 0.233, p=0.0013) and an estimated median survival extension of approximately 16 months. Additionally, the rate of functional decline slowed by 31%, and respiratory decline by 43%, compared to controls, although these latter findings did not reach conventional statistical significance thresholds.

Next Steps – Pivotal HEALEY ALS Platform Trial

These encouraging results have reinforced Neurizon’s confidence in NUZ-001’s potential as a disease-modifying therapy for ALS. The company plans to advance NUZ-001 into the pivotal Phase 2/3 HEALEY ALS Platform Trial, anticipated to commence in the fourth quarter of 2025, pending FDA clearance. This innovative trial design will allow for a larger, placebo-controlled evaluation across multiple regimens, accelerating the path to potential regulatory approval.

Collaborative Efforts and Patient Impact

Neurizon’s progress reflects a collaborative effort involving clinical teams at Calvary Health Care Bethlehem and Macquarie University, alongside dedicated patients and caregivers. Managing Director Dr. Michael Thurn emphasized the significance of these findings for the ALS community, highlighting the urgent need for effective treatments in a field historically marked by limited options and rapid disease progression.