The U.S. Food and Drug Administration (FDA) has lifted the clinical hold on Neurizon Therapeutics Limited’s lead investigational therapy, NUZ-001, which is aimed at treating Amyotrophic Lateral Sclerosis (ALS). This decision will allow the company to advance into the HEALEY ALS Platform Trial, anticipated to begin in the fourth quarter of 2025, marking a significant step forward for both Neurizon and the ALS community.
Dr. Michael Thurn, Managing Director and CEO of Neurizon, expressed his enthusiasm regarding the FDA’s decision, stating, “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.” He emphasized the company’s commitment to rigorous scientific development and acknowledged the vital support from patient advocacy groups and key opinion leaders in advancing this program.
The FDA’s approval is supported by robust preclinical safety data and comprehensive manufacturing information obtained through a licensing agreement with Elanco. This IND (Investigational New Drug) approval establishes a regulatory framework that not only facilitates the development of NUZ-001 but also opens opportunities for future programs and indications.
Next Steps for NUZ-001
With the IND now active, Neurizon expects Mass General Hospital to file a protocol amendment to include their specific regimen in the HEALEY ALS Platform Trial. Patient enrollment is projected to begin in the fourth quarter of 2025, aligning with the trial’s timeline. These developments are pivotal for Neurizon’s goal of expediting patient access to innovative therapies while creating long-term value for shareholders.
The encouraging TDP-43 preclinical data and positive survival outcomes from the Open-Label Extension study in ALS patients further reinforce the potential of NUZ-001. The company believes this investigational product could significantly slow the progression of ALS, a disease that severely impacts the lives of many individuals and their families.
Neurizon Therapeutics Limited, based in Melbourne, Australia, is dedicated to advancing treatments for neurodegenerative diseases. Their strategy includes exploring the broader applications of NUZ-001 while focusing on effective ALS treatments. As the company continues its clinical programs, it aims to pave new avenues for patients affected by complex neural disorders.
This announcement has been approved for release by the Board of Neurizon Therapeutics Limited. NUZ-001 remains an investigational product and is not approved for commercial use in any jurisdiction.