The United States National Institutes of Health has agreed to fund a multimillion-dollar program that will give up to 200 Americans living with motor neurone disease access to an experimental treatment developed by the Melbourne-based biotechnology company Neurizon Therapeutics.
The grant, announced by Neurizon, will finance an Expanded Access Protocol for NUZ-001, the company's lead drug candidate for amyotrophic lateral sclerosis, commonly known as ALS or motor neurone disease.
The program is designed for patients who are otherwise ineligible to join formal clinical trials, offering access to the drug for up to 96 weeks.
The Sean M Healey and AMG Center for ALS at Mass General Brigham in Boston will be the prime recipient of the NIH award, with Neurizon receiving funding as a subawardee.
Under the arrangement, Neurizon will serve as the regulatory sponsor and holder of the Investigational New Drug application and will supply NUZ-001, while the Healey and AMG Center will run the program day-to-day, including recruiting patients and collecting data.
The expanded access initiative is fully funded through the NIH award. That funding covers the company's drug supply and manufacturing costs as well as specified regulatory, safety monitoring and operational expenses, easing the financial burden typically associated with broadening access to an unapproved therapy.
Expanded access, sometimes called compassionate use, is a regulatory pathway that lets people with serious or life-threatening illnesses receive an investigational therapy that the US Food and Drug Administration has not yet approved, when no suitable clinical trial is available to them.
The new program will run alongside Regimen I of the Phase 2/3 HEALEY ALS Platform Trial, in which NUZ-001 is currently being tested in roughly 250 participants. Neurizon said the expanded access effort would continue beyond the trial's topline results, expected in late Q2 of calendar year 2027, provided that the trial produces a positive outcome.
The company said the program would generate valuable long-term data on safety, tolerability and survival, along with functional and respiratory measures such as the ALSFRS-R scale and slow vital capacity, patient-reported outcomes and exploratory biomarkers. This information, gathered from a broader and more diverse ALS population than is typically captured in randomised trials, is intended to complement the controlled efficacy and safety data emerging from the Healey ALS Platform Trial itself.
Around 20 physical clinical sites already participating in that platform trial are expected to take part, supplemented by a national virtual site intended to give patients across all 50 US states a chance to enrol, including those in rural or underserved communities who might otherwise struggle to access clinical research.
Neurizon's interim executive chairman Sergio Duchini said the company was proud to support the initiative. He said the expanded access program would significantly grow the long-term safety database for NUZ-001 while generating biomarker, survival and real-world clinical data from a wider group of patients. He thanked the NIH and the Healey and AMG Center team for their leadership, and expressed gratitude to people living with ALS and their families whose participation continues to drive research into more effective treatments.
Dr Sabrina Paganoni of the Healey and AMG Center said expanded access programs offered an important opportunity for people with ALS who cannot otherwise take part in clinical trials to gain access to investigational products. She said the centre was grateful to the NIH for supporting the effort and helping advance opportunities for ALS research.
Start-up activities for the program are already underway. Subject to regulatory approvals, site activation, and other operational steps, patient enrolment is targeted to begin in the first quarter of calendar year 2027.
