Neurizon Therapeutics (ASX: NUZ & OTCQB: NUZTF) has completed manufacturing three Good Manufacturing Practice (GMP) registration batches of its NUZ-001 tablet, providing essential material for its next stage of U.S. regulatory work. The tablets were produced ahead of schedule at Catalent’s certified facility using full-scale commercial processes. The batches support Chemistry, Manufacturing and Controls requirements for an upcoming FDA New Drug Application.
Regulatory Pathway… Moving Forward
The registration batches enable Neurizon to begin a Rolling Review of its NDA, allowing the FDA to assess Chemistry, Manufacturing and Controls data before the full dossier is submitted. This pathway can shorten review timelines under Fast Track or Breakthrough Therapy programs.
The batches have entered long-term and accelerated stability studies, which will determine shelf-life and generate data for the Quality module. Manufacturing at one-tenth of the intended commercial scale ensures processes closely mirror future market production.
Manufacturing with Catalent
Catalent produced the tablets under U.S. and International Council for Harmonisation standards. Its experience in orphan-drug manufacturing aligns with NUZ-001’s clinical focus and reduces scale-up risk. Neurizon’s licensing agreement with Elanco Animal Health adds supply-chain support for eventual commercial operations.
Chief executive Dr Michael Thurn said, “Working with Catalent, one of the world’s most respected contract manufacturers, ensures that NUZ-001 has been produced to the highest global standards for quality and compliance.”
Clinical Pipeline Progress
NUZ-001 is being developed for amyotrophic lateral sclerosis and targets mechanisms including TDP-43 protein aggregation and impaired autophagy. Early studies show favourable oral bioavailability, central nervous system penetration and a strong safety profile.
Neurizon plans to begin patient enrolment in the HEALEY ALS Platform Trial in Q1 2026. The HEALEY trial is a large U.S. platform study run by Massachusetts General Hospital, designed to evaluate multiple ALS drugs simultaneously under one master protocol. It uses shared placebos, fast recruitment and adaptive trial structures, allowing new therapies to be added without starting separate studies. This framework accelerates Phase 2/3 testing and has become a key global pathway for ALS drug evaluation. For NUZ-001, inclusion provides rapid trial execution and high-quality clinical oversight.
The completed GMP batches ensure supply for the HEALEY trial and other pivotal work. NUZ-001’s progression into NDA-enabling activities reflects Neurizon’s preparedness for accelerated regulatory routes in neurodegenerative disease.
ALS remains a field with limited treatment options and high unmet need. Reliable manufacturing and validated regulatory data are essential for advancing candidates like NUZ-001 toward potential market access.
What Is ALS?
Amyotrophic lateral sclerosis, or ALS, is a progressive neurodegenerative disease that damages the motor neurons that control voluntary muscles. As these neurons deteriorate, patients experience increasing weakness, difficulty speaking and swallowing, and loss of mobility. Breathing muscles also weaken, often requiring ventilatory support. Most cases occur without a known genetic cause, though a minority arise from inherited mutations. Thinking and sensory function usually remain intact. With few effective therapies and no cure, drug candidates targeting mechanisms such as TDP-43 protein aggregation and impaired autophagy remain a key focus in neurological development. Robust manufacturing and regulatory readiness are critical steps for advancing potential ALS treatments.
