Neurizon Therapeutics (ASX:NUZ) has announced that Regimen I of the HEALEY ALS Platform Trial will grow from 160 participants to 240, a decision the company attributed to faster-than-expected enrolment and the absence of a concurrent regimen during the recruitment window.
The expanded cohort will include 180 participants receiving NUZ 001 and 60 placebo controls and is designed to preserve the original statistical power for the primary endpoint while enabling stronger subgroup and biomarker analyses.
The company reported that, as of 22 May, 113 participants had been assigned and 74 had been dosed, with 64 clinical trial sites activated across the United States and 193 participants screened under the master protocol. Because recruitment momentum remains ahead of prior expectations, Neurizon now anticipates last participant dosing in the second quarter of calendar year 2027 and topline results in early the third quarter of that year. A philanthropic contribution from the Sean M. Healey and AMG Centre for ALS at Mass General Brigham will materially offset the additional participant cost, and Neurizon expects no change to total funding requirements through to study completion.
Interim Executive Chairman Sergio Duchini framed the expansion as a vote of confidence in the program and in the trial network. He said, “The expansion of Regimen I to 240 participants reflects our considerable confidence in NUZ 001 and is underpinned by strong momentum being delivered across the HEALEY ALS Platform Trial study. Importantly, this decision maintains the statistical power for the analysis of the primary endpoint and the potential for topline results ahead of our previously stated schedule.”
He added, “The strength of recruitment and engagement across the HEALEY ALS Platform Trial network has positioned Neurizon to proactively optimise the study design at an important point in the trial. We believe the expanded dataset will strengthen the statistical robustness, interpretability and strategic value of the program as we advance toward topline results.”
He added, “Importantly, the larger and more internally consistent dataset is expected to enhance future regulatory, partnering and commercial opportunities for NUZ 001, while also strengthening our ability to generate valuable biomarker and translational insights relevant to the broader neurodegenerative disease landscape.”
Healey and AMG Center's Merit Cudkowicz said, "This partnership with Neurizon reflects a shared commitment to advancing meaningful science while keeping people living with ALS at the center of everything we do. We thank our patient advisory committee members and the entire ALS community for active participation in this important clinical trial. By working collaboratively, we can accelerate progress, strengthen the research ecosystem, and ultimately deliver solutions that better serve the ALS community."
Co-Principal Investigator Sabrina Paganoni said, "I want to sincerely thank the Network of Excellence for ALS NEALS investigators and site teams for achieving our most rapid enrollment to date — an extraordinary milestone that reflects their dedication and collaboration. I also want to recognize the coordination center at the Healey and AMG Center and key vendors for outstanding implementation and execution of the study, which made this success possible, as well as participants and their families for their dedication and support."