Neurizon® Therapeutics Limited has announced that the Sean M. Healey & AMG Center for ALS at Mass General Brigham (Healey & AMG Center) has been awarded a multimillion-dollar, multi-year grant by the US National Institutes of Health (NIH). The funding will support an expanded access protocol (EAP) for NUZ-001, the Melbourne, Australia-based company’s lead investigational therapy for amyotrophic lateral sclerosis (ALS). The award carries NIH project number 1U01NS153995-01.

Access for patients outside clinical trials

The EAP is expected to give up to 200 people with ALS who are ineligible for clinical trials access to NUZ-001 for up to 96 weeks. Expanded access, also known as compassionate use, allows people with serious or life-threatening conditions to receive an investigational therapy not yet approved by the US Food and Drug Administration (FDA).

The programme will run alongside Regimen I of the Phase 2/3 HEALEY ALS Platform Trial, in which NUZ-001 is being evaluated in approximately 250 participants. Topline results from that trial are expected in late Q2 of calendar 2027. The EAP will continue beyond that point only if the trial outcome is positive.

Data collection and biomarker work

The EAP is designed to generate long-term real-world data from a broader ALS population than is usually represented in randomised clinical trials. Investigators will collect safety and tolerability data, together with survival, ALS-specific functional and respiratory measures, including the ALS Functional Rating Scale-Revised (ALSFRS-R) and slow vital capacity. Patient-reported outcomes and exploratory biomarkers will also be gathered.

Neurizon expects these findings to complement the controlled efficacy and safety data from the HEALEY ALS Platform Trial. It also expects them to enlarge the long-term safety database for NUZ-001 and to inform further clinical development.

“The EAP will also significantly expand our long-term safety database and generate valuable biomarker, survival and real-world clinical data from a broader ALS population,” said Sergio Duchini, Interim Executive Chairman.

Sites, roles and supply

Around 20 physical sites are planned, including some already taking part in the HEALEY ALS Platform Trial, along with a national virtual site. The company says this model could provide access across all 50 US states. The virtual site is intended to help people who may find it difficult to reach clinical research, including those in rural and underserved areas. The Healey & AMG Center is the prime recipient of the grant and will lead clinical and operational execution, including site activation, participant recruitment and data collection. Neurizon is a subawardee. It will act as regulatory sponsor, with the EAP conducted under its Investigational New Drug (IND) application, and will supply NUZ-001.

The NIH award fully funds the programme. It covers drug supply, manufacturing and specified regulatory, safety monitoring and operational costs linked to Neurizon’s participation. The company said its existing long-term supply agreement with
Elanco Animal Health Incorporated gives it access to scalable supplies of active pharmaceutical ingredient. The grant budget provides for API supply, drug product manufacturing, shipment, and regulatory and pharmacovigilance activities.

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

Timeline

Start-up activities are under way. Subject to regulatory, site activation and operational requirements, enrolment is targeted to begin by Q1 of calendar 2027, and the programme will be open to eligible people with ALS in the US. The Healey & AMG Center will publish further details on participating sites, eligibility criteria and enrolment as the programme progresses.