A US National Institutes of Health grant will fund an expanded access programme giving up to 200 people with ALS access to investigational therapy NUZ-001.

NIH grant will support access outside clinical trials

The Sean M Healey & AMG Center for ALS at Mass General Brigham has received an NIH grant to conduct an Expanded Access Protocol (EAP) for Neurizon Therapeutics’ investigational ALS therapy NUZ-001.

The programme is intended for people living with amyotrophic lateral sclerosis (ALS) who are otherwise ineligible to participate in clinical trials. It is expected to provide up to 200 people with access to NUZ-001 for up to 96 weeks.

The NIH-funded programme will run alongside Regimen I of the Phase 2/3 HEALEY ALS Platform Trial, which is evaluating NUZ-001 in people with ALS. The trial completed enrolment of 250 participants in July 2026, with topline safety and efficacy results expected in late Q2 2027.

Expanded access, sometimes referred to as compassionate use, can provide a route for people with serious or life-threatening diseases to receive an investigational therapy when participation in an appropriate clinical trial is not possible.

The EAP is currently in start-up, with enrolment expected to begin following the required regulatory, site activation and operational processes.

The federal award record lists the cooperative agreement at approximately $8.3m, with the General Hospital Corporation, which operates Mass General Brigham, as the recipient. The award runs from September 2026 through July 2030.

Programme will collect longer-term ALS data

Alongside providing access to NUZ-001, the programme is intended to generate additional clinical data from a broader ALS population than is typically represented in randomised trials.

Data collection is expected to include safety and tolerability, survival, ALS-specific functional and respiratory measures, patient-reported outcomes and exploratory biomarkers.

Sabrina Paganoni of the Healey & AMG Center said: “Expanded Access Programs provide an important opportunity for people living with ALS who are not otherwise eligible for clinical trials to access investigational products.”

She added that the programme would also support research into ALS by collecting additional safety, biomarker and clinical data.

The EAP will be led clinically and operationally by the Healey & AMG Center, while Neurizon will act as regulatory sponsor and IND holder and supply NUZ-001.

The programme is expected to use around 20 physical ALS clinical trial sites alongside a national virtual site, with the aim of providing potential access across the US.

EAP will complement Phase 2/3 trial

The expanded access programme is designed to complement the controlled evidence being generated in the HEALEY ALS Platform Trial rather than replace it.

Neurizon’s NUZ-001 is an oral investigational therapy being evaluated as Regimen I of the platform trial. The treatment is designed to target mechanisms associated with ALS, including TDP-43 protein aggregation and impaired autophagy. Mass General Brigham describes the EAP as an opportunity to provide access to people who cannot enter clinical trials while collecting additional safety, disease progression and biomarker information.

Continuation of the EAP beyond the initial programme is linked to the outcome of the Phase 2/3 trial.

Neurizon’s current clinical information states that NUZ-001 remains investigational and that authorised clinical trials are currently the available route to access the therapy, underlining that the newly funded EAP is still being established rather than already open to patients.

For people living with ALS who are unable to participate in clinical research because of eligibility restrictions, the programme could therefore provide an additional route to an investigational therapy while expanding the clinical dataset around NUZ-001.