Novel therapy gains FDA clearance for ALS clinical trial

Neurizon Therapeutics Ltd has received FDA clearance for its lead candidate, NUZ-001, to enter the HEALEY ALS Platform Trial as Regimen I. This approval allows the company to begin site activations, obtain single IRB approvals, and prepare for patient enrolment, expected to start in early 2026.

The HEALEY ALS Platform Trial is a multicentre, adaptive study designed to accelerate the evaluation of potential ALS therapies. By testing multiple investigational drugs simultaneously, the trial streamlines operations and improves efficiency across sites.

NUZ-001 targets key pathological features of ALS, including TDP-43 aggregation and impaired autophagy, and has shown promising oral bioavailability, CNS penetration, and a favourable safety profile in preclinical studies. FDA clearance of the protocol represents a critical milestone for Neurizon as it advances its development program toward potentially disease-modifying therapies for ALS.

Dr Michael Thurn, CEO of Neurizon, said: “This FDA clearance is an important step forward in bringing NUZ-001 to patients living with ALS. The HEALEY ALS Platform Trial provides an efficient, collaborative framework to test new treatments, and we look forward to beginning patient enrolment in 2026.”

Professor Merit Cudkowicz, Principal Investigator of the HEALEY ALS Platform Trial, added: “We are excited to welcome NUZ-001 to the platform trial. The FDA’s clearance allows us to move swiftly to start sites and begin enrolment, bringing us closer to identifying new therapies for ALS.”

More information online