Biotechnology company Neurizon (ASX: NUZ & NUZOA) announced on Wednesday that it has submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for its lead candidate, NUZ-001.

This marks a critical step toward initiating a Phase 2/3 clinical study under the HEALEY ALS Platform Trial framework. The FDA will have 30 days to review the application.

The IND submission includes comprehensive data, such as animal and human studies, pharmacokinetic and toxicology analyses, and manufacturing details, to demonstrate the safety of NUZ-001. The drug targets TDP-43 protein aggregation, a key feature of ALS pathology, and has shown promising safety and efficacy in prior studies.

Upon FDA approval, Neurizon expects Massachusetts General Hospital (MGH) to amend their IND for the HEALEY ALS Platform Trial by Q1 2025, with patient enrollment anticipated to begin in H1 2025.

Neurizon is focused on advancing treatments for neurodegenerative diseases, particularly ALS, and exploring broader applications for NUZ-001.