By: IPP Bureau
Last updated : September 21, 2026 8:08 am
The NIH-funded programme is expected to provide the NUZ-001 for patients who are otherwise ineligible for clinical trials access for up to 96 weeks
Sydney based biotech company Neurizon Therapeutics has informed that Sean M. Healey & AMG Center for ALS at Mass General Brigham has received a multimillion-dollar, multi-year grant from the US National Institutes of Health (NIH).
The grant has been provided to conduct an Expanded Access Protocol (EAP) for Neurizon’s investigational therapy NUZ-001 in people living with amyotrophic lateral sclerosis (ALS).
The NIH-funded programme is expected to provide up to 200 people with ALS who are otherwise ineligible for clinical trials access to NUZ-001 for up to 96 weeks. The EAP will run alongside Regimen I of the Phase 2/3 HEALEY ALS Platform Trial, in which NUZ-001 is currently being evaluated in approximately 250 participants.
The EAP is expected to continue beyond the topline results of the Phase 2/3 trial, which Neurizon expects in late Q2 2027, subject to a positive trial outcome.
Expanded access, also known as compassionate use, provides a pathway for patients with serious or life-threatening diseases to receive investigational therapies that have not yet been approved by the US Food and Drug Administration when participation in an appropriate clinical trial is not available.
The programme is designed to generate long-term real-world data from a broader ALS population than is typically represented in randomised clinical trials. Data collection will include safety and tolerability, survival, ALS-specific functional and respiratory measures, including ALSFRS-R and slow vital capacity, patient-reported outcomes and exploratory biomarkers.
Neurizon said the EAP data will complement controlled efficacy and safety data from the HEALEY ALS Platform Trial and could expand the long-term safety database for NUZ-001 while providing additional survival, biomarker and clinical data for its development.
The programme is expected to operate through approximately 20 physical ALS clinical trial sites and a national virtual study site, potentially providing access across all 50 US states.
Sergio Duchini, Interim Executive Chairman, Neurizon, said the programme would provide access to NUZ-001 for ALS patients who are otherwise ineligible for clinical trials while expanding the therapy's long-term safety database and generating additional biomarker, survival and real-world clinical data.