Neurizon results point to potential of NUZ-001 as disease-modifying therapy for ALS

21 August 2025

Australia’s Neurizon Therapeutics (ASX: NUZ & NUZOA), a biotech advancing innovative treatments for neurodegenerative diseases, has reported positive top-line 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 II dose was safe and well-tolerated. Importantly, preliminary efficacy findings showed that the use of NUZ-001, compared to matched historical controls from the Pooled Resource Open-Access ALS Clinical Trials 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.

According to Neurizon, these 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.

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