• Last Open-Label Extension (OLE) study patient successfully completes 12-month treatment period
• Treatment with NUZ-001 continues to be well-tolerated and shows promising results in extending the life expectancy of patients with MND/ALS
• No serious adverse events related to NUZ-001 have been reported during treatment

Neurizon Therapeutics Limited (ASX: NUZ & NUZOA) has announced that the last patient enrolled on to the 12-month Open Label Extension (OLE) Study of NUZ-001 for amyotrophic lateral sclerosis (ALS) has successfully completed treatment. Treatment with NUZ-001 daily at 10 mg/kg was well-tolerated by all patients, consistent with the planned dose for the upcoming Phase 2/3 HEALEY ALS Platform Trial. The release of top-line results from the OLE study remains on track for Q3 CY2025. An updated survival analysis shows that treatment with NUZ-001 significantly prolonged survival compared to historical controls from the PRO-ACT database, with the risk of death reduced by 78.5%. The Kaplan-Meier survival curves suggest that treatment with NUZ-001 may provide substantial clinical benefits, potentially extending the median survival by approximately 11 months. These results are particularly notable when compared to existing approved treatments, which typically extend life expectancy by 3 to 6 months. The study has been conducted at two clinical sites in Australia, and 6 of the 10 patients who enrolled on the OLE study are continuing treatment with NUZ-001 under a compassionate use program.

Once the FDA lifts the clinical hold on NUZ-001, anticipated in Q3 CY2025, Neurizon Therapeutics will proceed with the initiation of the HEALEY ALS Platform Trial, expected to commence in Q4 CY2025.