FDA clears Neurizon’s NUZ-001 to join HEALEY ALS trial

Australian biotech Neurizon Therapeutics (ASX: NUZ) said the US regulator has lifted the clinical hold on its lead drug NUZ-001 (monepantel) for amyotrophic lateral sclerosis, clearing the way for Phase II/III evaluation within the HEALEY ALS platform trial. Patient enrolment is expected to begin in the fourth quarter.

The clinical hold, imposed in January, required more animal exposure data on dosing risks. Neurizon submitted its response in July, strengthened by pre-clinical safety work and manufacturing support from its licensing agreement with US firm Elanco, which holds rights to monepantel.

Massachusetts General Hospital, which oversees HEALEY, will file a protocol amendment to add NUZ-001’s regimen in the coming weeks. The drug showed encouraging survival outcomes in an open-label extension, while preclinical studies suggested it could address TDP-43 protein dysfunction. Chief executive Michael Thurn described the clearance as “a significant milestone” for the ALS community.