Melbourne, Australia – Neurizon Therapeutics Limited (ASX: NUZ), a late clinical-stage biotechnology company dedicated to advancing innovative treatments for neurodegenerative diseases, announced today that the US National Institutes of Health (NIH) has awarded a multimillion-dollar, multi-year grant to fund an Expanded Access Protocol (EAP) for its lead investigational therapy, NUZ-001, in people living with amyotrophic lateral sclerosis (ALS). Neurizon Therapeutics Limited is developing NUZ-001 for the treatment of ALS, which is the most common form of motor neurone disease. The Sean M. Healey & AMG Center for ALS will serve as the prime recipient of the NIH grant, with Neurizon receiving funding as a subawardee, while also acting as the regulatory sponsor and IND holder and supplying NUZ-001.
The NIH-funded EAP is designed to offer NUZ-001 access for up to 96 weeks to approximately 200 individuals with ALS who are not eligible for clinical trials. This program operates entirely through the NIH award, which covers Neurizon’s drug supply, manufacturing, and specified regulatory and operational costs. The EAP will run concurrently with Regimen I of the Phase 2/3 HEALEY ALS Platform Trial, where NUZ-001 is currently being evaluated. Its continuation beyond the topline results, anticipated in late Q2 CY2027, is contingent upon a positive outcome from that trial.
The program aims to broaden clinical exposure to NUZ-001 and generate complementary long-term safety, biomarker, survival, and clinical data from a wider ALS patient population. Data collection will include safety and tolerability, alongside survival, functional and respiratory measures, patient-reported outcomes, and exploratory biomarkers. Access will be provided across approximately 20 physical clinical sites already participating in the HEALEY ALS Platform Trial, complemented by a national virtual study site designed to reach patients in rural and underserved areas across 50 US states. Start-up activities for the EAP are in progress, with participant enrolment targeted to begin by Q1 CY2027.
