NIH funds Expanded Access Program for NUZ-001 in ALS

Key points

• NIH awards multimillion-dollar grant for NUZ-001 Expanded Access Protocol
• Program aims to provide access to NUZ-001 for up to 200 ALS patients
• EAP to run parallel with Phase 2/3 HEALEY ALS Platform Trial

Full summary

Neurizon Therapeutics Limited (ASX: NUZ) announced that the Sean M. Healey & AMG Center for ALS at Mass General Brigham has been awarded a multimillion-dollar, multi-year grant from the US National Institutes of Health (NIH) to conduct an Expanded Access Protocol (EAP) with Neurizon's lead investigational therapy, NUZ-001, in people living with amyotrophic lateral sclerosis (ALS). The NIH-funded EAP is expected to provide up to 200 people living with ALS who are otherwise ineligible to participate in clinical trials with access to NUZ-001 for up to 96 weeks. The program will run in parallel with Regimen I of the Phase 2/3 HEALEY ALS Platform Trial, where NUZ-001 is currently being evaluated in approximately 250 participants. The EAP will continue beyond the topline results, expected in late Q2 CY2027, contingent on a positive trial outcome.

Outlook

The program is expected to significantly expand the long-term safety database for NUZ-001 and provide additional survival, biomarker and clinical data to inform its ongoing clinical development.