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Neurizon Files IND Application for NUZ-001 in HEALEY ALS Platform Trial

• Neurizon has submitted an Investigational New Drug (IND) application to the FDA for NUZ-001, a drug targeting TDP-43 protein aggregation in ALS. • The IND submission includes comprehensive data from animal and human studies, crucial for initiating Phase 2/3 trials. • Pending FDA clearance, Neurizon anticipates commencing patient enrollment in the HEALEY ALS Platform Trial in the first half of 2025. • NUZ-001 has shown a preliminary safety and efficacy profile in earlier clinical studies, offering hope for ALS treatment.

Neurizon Therapeutics has announced the filing of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for its lead drug candidate, NUZ-001, marking a significant step toward initiating a Phase 2/3 clinical trial within the HEALEY ALS Platform Trial. The submission, a culmination of several years of research and development, includes extensive data intended to support the safe administration of NUZ-001 to humans.

IND Application Details

The IND application comprises a comprehensive dossier of information, including data from animal and human studies, pharmacokinetic analyses, toxicology studies, and manufacturing information related to NUZ-001. The FDA will review the application over a 30-day period to assess the safety of the proposed human research.

NUZ-001 and ALS

NUZ-001 is designed to target TDP-43 protein aggregation, a key characteristic of Amyotrophic Lateral Sclerosis (ALS) pathology. The drug has demonstrated a preliminary safety and efficacy profile in earlier clinical studies, providing a foundation for its further evaluation in the HEALEY ALS Platform Trial.

HEALEY ALS Platform Trial

The HEALEY ALS Platform Trial offers a streamlined and collaborative approach to evaluating potential ALS treatments. Pending FDA clearance of the IND application, Massachusetts General Hospital (MGH) is expected to file a protocol amendment to their IND for the HEALEY ALS Platform Trial to incorporate Neurizon's regimen-specific appendix in Q1 CY2025. Patient enrollment is anticipated to begin in the first half of 2025.

Management Commentary

"The submission of our IND application to the FDA represents a critical milestone in Neurizon's mission to address the devastating impacts of ALS," said Dr. Michael Thurn, Managing Director and Chief Executive Officer of Neurizon. "Our team has worked tirelessly around the clock to produce this foundation regulatory document to realise our goal of commencing the HEALEY ALS Platform Trial. This trial provides an unparalleled opportunity to evaluate the therapeutic potential of NUZ-001 in a collaborative, streamlined manner with the world's leading ALS neurologists. Our team remains committed to advancing this program and delivering hope to patients worldwide who are in urgent need of effective ALS treatments."
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Reference News

[1]
Neurizon Files IND Application to Support HEALEY ALS Platform Trial - Yahoo Finance
finance.yahoo.com · Dec 18, 2024

Neurizon Therapeutics submitted an IND application to the FDA for NUZ-001, targeting TDP-43 protein aggregation in ALS, ...

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