FDA Lifts Clinical Hold on NUZ-001

The U.S. Food and Drug Administration (FDA) has lifted the clinical hold on Neurizon Therapeutics Limited’s lead investigational drug, NUZ‑001. This decision marks a pivotal regulatory milestone for Neurizon and the ALS community, clearing the way for Phase 2/3 development of NUZ-001 as part of the HEALEY ALS Platform Trial, expected to commence Q4 CY2025.

With IND now active, Neurizon anticipates Mass General Hospital (MGH) filing a protocol amendment to their IND for the HEALEY ALS Platform Trial to incorporate our specific protocol regimen early in the coming weeks. Neurizon expects to initiate patient enrollment in the HEALEY ALS Platform Trial in Q4 CY2025.

Opening an IND for a platform molecule establishes a regulatory foundation that accelerates the development of the first candidate and streamlines future programs. By creating a validated framework for safety, manufacturing, and clinical design, it reduces regulatory risk, shortens timelines, and enables efficient expansion into new indications.

In this case, the IND is further strengthened by the preclinical safety data and the manufacturing and quality information secured through the company’s licensing agreement with Elanco. 

These resources enhance confidence in the platform’s readiness for clinical development and reinforce its potential as a therapeutic platform with broad applicability, offering both strategic flexibility and long-term commercial value.

Dr Michael Thurn, Managing Director and CEO, commented: “The FDA’s clearance of our IND application marks a significant milestone in our mission to bring NUZ-001 closer to patients living with ALS… We are now partnering with the HEALEY ALS Platform Trial team to take the next critical steps in advancing NUZ-001. Supported by our compelling TDP-43 preclinical data and encouraging survival outcomes from the Open-Label Extension (OLE) study in ALS patients, we believe NUZ-001 has the potential to meaningfully slow the progression of this devastating disease.”