Key Points:

• The IND application, submitted to the U.S. Food and Drug Administration (FDA), is a crucial step towards a Phase 2/3 clinical trial for NUZ-001.
• This comprehensive submission includes data from animal and human studies, pharmacokinetic and toxicology analyses, and manufacturing details for NUZ-001.
• The FDA will review the application within 30 days.
• NUZ-001 targets TDP-43 protein aggregation, a key feature of ALS. Its safety and initial effectiveness have been shown in previous studies.

MELBOURNE, Australia, Dec. 18, 2024 — (ASX: NUZ & NUZOA) Neurizon, a clinical-stage biotech company focused on neurodegenerative disease treatments, announced the filing of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for its lead product, NUZ-001. This is a major step towards initiating a Phase 2/3 clinical trial within the HEALEY ALS Platform Trial. The FDA has a 30-day review period for the IND application.

Dr. Michael Thurn, Neurizon’s Managing Director and CEO, stated:
“Submitting our IND application to the FDA marks a significant achievement in Neurizon’s mission to combat ALS. This application, representing years of research and development, includes many components needed for our New Drug Application (NDA) submission. Our dedicated team has worked diligently to create this foundational regulatory document to enable our participation in the HEALEY ALS Platform Trial. This trial offers an exceptional chance to assess NUZ-001’s therapeutic potential in collaboration with leading ALS neurologists. We’re committed to advancing this program and providing hope to patients in urgent need of effective ALS treatments.”

About the IND Process

An IND allows sponsors to provide the FDA with crucial information to obtain authorization for safe human administration of an investigational drug (or an approved drug for a new indication or patient population). The FDA requires comprehensive data—animal and human studies, pharmacokinetic and toxicology data, and manufacturing information—to evaluate the safety of the proposed human research.

Next Steps

Following FDA approval of the IND, Neurizon anticipates Massachusetts General Hospital (MGH) will submit a protocol amendment to their IND for the HEALEY ALS Platform Trial, including our regimen-specific appendix, in Q1 CY2025. Patient enrollment in the HEALEY ALS Platform Trial is expected to begin in H1 CY2025.

Neurizon Therapeutics Limited’s Board of Directors authorized this announcement.

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About Neurizon Therapeutics Limited

Neurizon Therapeutics Limited (ASX: NUZ) is a clinical-stage biotechnology company focused on developing treatments for neurodegenerative diseases. Neurizon is developing NUZ-001 for ALS, the most common form of motor neuron disease. Neurizon aims to expedite access to effective ALS treatments while also exploring NUZ-001’s potential in other neurodegenerative disorders. Through international collaborations and rigorous clinical trials, Neurizon is committed to improving outcomes for patients and families affected by complex neurological conditions.

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