FDA Type C Feedback, HEALEY trial potentially registrational

• FDA Type C Meeting feedback received for NUZ-001
• HEALEY ALS Platform Trial may serve as single registrational study
• Positive results could support traditional approval pathway
• Topline results for Regimen I expected in late Q2 CY2027

Neurizon Therapeutics Limited, a late-stage clinical biotechnology company, has received written responses from the US Food and Drug Administration (FDA) regarding its Type C meeting request for NUZ-001, an investigational treatment for amyotrophic lateral sclerosis (ALS). The FDA feedback provides guidance on the clinical development plan, potential regulatory pathways, and use of biomarkers, which will inform the preparation of a New Drug Application (NDA). The HEALEY ALS Platform Trial, in which NUZ-001 is being evaluated, is a placebo-controlled study designed to assess clinically meaningful endpoints after 36 weeks of treatment. The FDA noted that positive results could potentially support a traditional approval pathway, and the trial may serve as the single registrational study for an NDA, subject to meeting the requirement for substantial evidence of effectiveness.

Topline results for Regimen I expected in late Q2 CY2027

Neurizon is preparing the evidence package for the NDA submission, with a focus on completing the trial and building the clinical and supporting evidence needed.