• The OLE study met its primary endpoint, confirming long-term treatment with NUZ-001 was safe and well-tolerated
• Treatment with NUZ-001 showed a durable functional benefit, stable respiration, and supportive biomarker trends
• NUZ-001 significantly increased survival, reduced the risk of death by 76.7%, and showed an estimated median survival extension of ~16 months

Neurizon Therapeutics Limited reported positive topline results from the Open-Label Extension (OLE) study of its lead candidate NUZ-001 for the treatment of amyotrophic lateral sclerosis (ALS), the major form of motor neurone disease (MND). Long-term treatment with NUZ-001 at the recommended Phase 2 dose was safe and well-tolerated, with no treatment-related deaths observed. Preliminary efficacy findings demonstrated that treatment with NUZ-001, compared to matched historical controls from the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database, was associated with a significant survival advantage, sustained slowing of global functional decline, a reduced rate of respiratory decline, and a stable or downward trend in key disease biomarkers. The OLE study met its primary endpoint, confirming long-term treatment with NUZ-001 was safe and well-tolerated. These encouraging results support the potential of NUZ-001 as a disease-modifying therapy for ALS and provide strong justification for advancing the program into further clinical development, including the planned pivotal HEALEY ALS Platform Trial expected to commence in Q4 CY2025.

Neurizon will advance NUZ-001 to the pivotal Phase 2/3 HEALEY ALS Platform Trial in Q4 CY2025, pending FDA clinical hold clearance, to confirm the observed benefits in a larger, placebo-controlled setting.