A major step forward in ALS drug development has arrived for Neurizon Therapeutics (ASX:NUZ), with the US Food and Drug Administration clearing the company’s lead candidate, NUZ-001, to enter the HEALEY ALS Platform Trial.
The decision follows the FDA’s completion of its 30-day review, officially designating NUZ-001 as Regimen I, a key milestone for a therapy designed to address the devastating neurodegenerative disease.
The HEALEY Trial, run by the Sean M. Healey and AMG Center for ALS at Mass General Brigham, is known for accelerating ALS research by testing multiple investigational therapies simultaneously under a shared clinical infrastructure. With the FDA’s protocol acceptance, NUZ-001 now moves into the trial’s operational phase, including IRB submissions, site activations, and clinical start-up activities. Patient enrollment is expected to begin in early 2026.
NUZ-001 targets two central ALS disease mechanisms, TDP-43 protein aggregation and impaired autophagy, and early data suggest favourable oral bioavailability, strong CNS penetration, and an encouraging safety profile.
Neurizon CEO Dr Michael Thurn called the FDA clearance “a major milestone” and praised the HEALEY Trial as the gold standard for collaborative ALS development. Trial principal investigator Professor Merit Cudkowicz similarly welcomed NUZ-001, noting the importance of advancing new potential treatments for patients.
For Neurizon, the milestone strengthens its strategy of leveraging global clinical partnerships to advance disease-modifying therapies for ALS, an area where new treatment options remain urgently needed.