Biotech giant Neurizon Therapeutics has secured a major regulatory win, with the Sean M Healey & AMG Center for ALS at Mass General Brigham receiving FDA clearance to advance the company’s NUZ-001 regimen into the Healey ALS Platform Trial.

 

The green light follows the FDA’s 30-day review and formally designates NUZ-001 as Regimen I in the multi-arm study, marking what Neurizon called a pivotal step toward bringing a new therapeutic option to people living with ALS.

 

The approval of the protocol amendment allows the Healey Center to begin clinical activities under its Investigational New Drug (IND) application. Operational work now accelerates: IRB submissions, site activations, and study start-up are next in line, with first patient enrollment expected in early 2026.

 

Designed to speed the evaluation of promising ALS treatments, the Healey ALS Platform Trial is a multicenter, double-blind, placebo-controlled, adaptive study run in partnership with the Network of Excellence for ALS (NEALS). By testing multiple therapies simultaneously and sharing trial infrastructure, the platform has become a global model for rapid, efficient ALS drug development.

 

“Receiving the FDA’s clearance is a major milestone for Neurizon and a critical step in advancing NUZ-001 as a potential new treatment option for people living with ALS,” said Dr Michael Thurn, Managing Director & CEO. 

 

“The HEALEY ALS Platform Trial represents the gold standard for efficient and collaborative clinical development in this field, and we are proud to progress into the next phase of activation. We look forward to working closely with the HEALEY team as we move toward site initiations and study start-up, bringing us one step closer to addressing this significant unmet medical need.”

 

Professor Merit Cudkowicz, the trial’s Principal Investigator and Director of the Healey & AMG Center, welcomed the addition. 

 

“We are pleased to welcome Neurizon’s NUZ-001 regimen into the HEALEY ALS Platform Trial. The Healey ALS Platform Trial Therapy Evaluation Committee previously selected NUZ-001 for inclusion as a new regimen. The FDA’s clearance to proceed with the NUZ-001 regimen is an important next step. We look forward to initiating clinical sites and beginning enrollment so we can evaluate the potential of NUZ-001 to benefit people with ALS.”