Neurizon Therapeutics (ASX:NUZ) has announced that the US Food and Drug Administration (FDA) has lifted the clinical hold on its lead investigational therapy, NUZ-001, a significant regulatory milestone that clears the path for the company’s entry into the prestigious HEALEY ALS Platform Trial later this year.
The company said the decision marks a pivotal moment for it and the amyotrophic lateral sclerosis (ALS) community, enabling the company to progress to Phase 2/3 clinical development of NUZ-001 in collaboration with Massachusetts General Hospital, which oversees the multi-arm platform trial.
Neurizon’s Managing Director and CEO, Dr Michael Thurn, described the FDA’s clearance as “a defining moment” for the company’s mission to accelerate access to innovative therapies for people living with ALS.
“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,” Dr Thurn said. “This achievement reflects both our unwavering commitment to rigorous scientific and clinical development and the tireless dedication of our team. We’re deeply grateful to the key opinion leaders and patient advocacy groups whose support was instrumental in advancing this program.”
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NUZ-001 is supported by a robust preclinical data package targeting TDP-43, a pathological protein central to the progression of ALS. Encouraging survival outcomes from Neurizon’s earlier Open-Label Extension study also underscore its therapeutic potential. The company believes NUZ-001 could play a meaningful role in slowing the progression of this devastating neurodegenerative disease.
The lifting of the clinical hold activates Neurizon’s Investigational New Drug (IND) application, which establishes a regulatory foundation for NUZ-001 as a platform molecule, a strategy designed to streamline future development programs and expand potential indications. The IND is supported by comprehensive preclinical safety data and detailed manufacturing information secured under Neurizon’s licensing agreement with Elanco, which bolsters the company’s regulatory readiness and commercial flexibility.
With the IND now active, Mass General Hospital is expected to file an amendment to incorporate Neurizon’s protocol into the HEALEY ALS Platform Trial in the coming weeks, with patient enrolment scheduled to begin in Q4 2025.
Dr Thurn said the decision positions Neurizon to move rapidly into the next stage of clinical development. “We are now partnering with the HEALEY ALS Platform Trial team to advance NUZ-001,” he said. “These milestones reflect our determination to bring forward a treatment that can truly make a difference for patients and families affected by ALS.”
Neurizon’s broader strategy is to leverage NUZ-001 as a platform for innovation in neurodegenerative diseases, enabling accelerated regulatory pathways and future partnering opportunities. The company remains committed to its goal of expanding therapeutic options for patients with ALS while exploring the molecule’s potential across a wider spectrum of neurological disorders.