Neurizon Files IND Application to Support HEALEY ALS Trial

Key points

• IND application submitted to FDA for Phase 2/3 clinical study of NUZ-001
• NUZ-001 targets TDP-43 protein aggregation, a hallmark of ALS pathology
• FDA has 30 days to review the IND application

Full summary

Neurizon Therapeutics Limited (ASX: NUZ & NUZOA), a clinical-stage biotech company advancing treatments for neurodegenerative diseases, has filed an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for its lead candidate, NUZ-001. This milestone is a pivotal step in enabling the commencement of a Phase 2/3 clinical trial within the HEALEY ALS Platform Trial framework. The IND application contains a comprehensive dossier of information, including animal and human studies, pharmacokinetic analyses, toxicology studies, and manufacturing information for NUZ-001. The FDA has a period of 30 days to review the IND application. NUZ-001 targets TDP-43 protein aggregation, a hallmark of ALS pathology, and has demonstrated a favorable safety and preliminary efficacy profile in earlier clinical studies. Pending FDA clearance of the IND application, Neurizon anticipates Massachusetts General Hospital (MGH) filing a protocol amendment to their IND for the HEALEY ALS Platform Trial to incorporate Neurizon's regimen-specific appendix in Q1 CY2025. Neurizon expects to initiate patient enrollment in the HEALEY ALS Platform Trial in H1 CY2025.

Outlook

Neurizon remains committed to advancing the NUZ-001 program and delivering hope to patients worldwide who are in urgent need of effective ALS treatments.