Neurizon Therapeutics: Advancing Targeted ALS Treatment with NUZ-001

Neurizon Therapeutics Limited (ASX: NUZ), an Australian clinical-stage biotech company, is pioneering treatments for neurodegenerative diseases with its lead candidate, NUZ-001. Recent preclinical studies confirmed that both NUZ-001 and its active metabolite, NUZ-001 Sulfone, significantly penetrate the blood-brain barrier (BBB) in rodents, achieving CNS concentrations aligned with levels needed to reverse TDP-43 protein aggregation—a key driver of ALS and other TDP-43-related diseases. These findings reinforce earlier Phase 1 efficacy results and indicate strong therapeutic potential for conditions like ALS, frontotemporal dementia, and Alzheimer’s disease. In vitro models demonstrated that both compounds effectively prevented TDP-43 aggregation at all tested doses. Importantly, these results were obtained with peripheral administration, highlighting the drug's translational potential. Neurizon aims to expedite clinical development and broaden NUZ-001’s application across neurodegenerative disorders, driven by a mission to deliver impactful, disease-modifying therapies.

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