A multimillion-dollar, multiyear NIH grant will fund an expanded access program for Neurizon’s investigational NUZ-001, potentially giving up to 200 US patients access to the therapy for as long as 96 weeks. The program is designed to add long-term safety, survival and biomarker data alongside the pivotal HEALEY ALS trial.
- NIH-funded program targeting up to 200 trial-ineligible ALS patients
- Access to NUZ-001 planned for up to 96 weeks
- Grant covers drug supply, manufacturing and specified regulatory costs
- Enrolment targeted to begin by Q1 CY2027
- Continuation after late Q2 CY2027 trial results depends on a positive outcome
Neurizon Therapeutics Limited (ASX:NUZ; OTCQB: NUZTF) has secured external funding for a major expansion of its NUZ-001 clinical program, with the US National Institutes of Health awarding a multimillion-dollar, multiyear grant for an expanded access program in amyotrophic lateral sclerosis.
The initiative could provide up to 200 people living with ALS who are not eligible for clinical trials with access to the investigational therapy for up to 96 weeks. It gives NUZ-001 a broader clinical footprint before the company has reported topline results from its controlled Phase 2/3 trial, although it does not amount to FDA approval or commercial availability.
NIH Grant Funds 200-Patient Access Program
The Sean M. Healey & AMG Center for ALS at Mass General Brigham will receive the NIH award and lead the program’s clinical and operational execution. Neurizon will act as regulatory sponsor and IND holder, supplying NUZ-001 under the program.
The grant will fund the program’s drug supply, manufacturing, shipment and specified regulatory, safety monitoring and operational costs associated with Neurizon’s participation. The announcement does not disclose the grant’s dollar value beyond describing it as multimillion-dollar, so the precise financial offset for Neurizon remains unclear.
Real-World Data to Complement HEALEY Trial
The expanded access program will run alongside Regimen I of the HEALEY ALS Platform Trial, where NUZ-001 is being evaluated in approximately 250 participants. Its data collection will cover safety and tolerability, survival, ALS-specific functional and respiratory measures, patient-reported outcomes and exploratory biomarkers.
That evidence will come from a wider ALS population than is typically represented in a randomised trial, but it will not replace the controlled efficacy and safety evidence from HEALEY. The program’s continuation beyond the trial’s topline results, expected in late Q2 CY2027, is explicitly conditional on a positive outcome.
US Sites and Virtual Access Planned
The program is expected to use about 20 physical ALS clinical sites and a national virtual site, potentially reaching eligible participants across all 50 US states. The virtual model is intended to make participation easier for people in rural and underserved areas, subject to the required regulatory, site activation and operational steps.
Start-up work is already underway, with enrolment targeted to begin by Q1 CY2027. Neurizon said its existing supply arrangements, including a long-term agreement with Elanco Animal Health for scalable active pharmaceutical ingredient supplies, are positioned to support the additional demand.
Bottom Line?
The NIH award removes a stated funding burden from the expanded access effort, but the key value inflection remains the HEALEY trial result expected in late Q2 CY2027.
Questions in the middle?
- Will the expanded access program begin enrolment on schedule in Q1 CY2027?
- How will real-world safety and survival data compare with the controlled HEALEY evidence?
- Will a positive HEALEY outcome support continuation of the program beyond topline results?