Neurizon Therapeutics Announces FDA Clearance to Advance NUZ-001 in ALS Platform Trial

Neurizon Therapeutics Announces FDA Clearance to Advance NUZ-001 in ALS Platform Trial

By, Admin 8 October 2025

Neurizon Therapeutics Limited, a clinical-stage biotech focused on neurodegenerative diseases, announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on its lead investigational therapy, NUZ-001. This milestone clears the way for Phase 2/3 development of NUZ-001 as part of the HEALEY ALS Platform Trial, expected to begin Q4 2025.

Key Highlights

  • Regulatory Milestone: FDA clearance of the IND application for NUZ-001 enables the initiation of pivotal clinical studies for ALS.
  • Platform Strategy: The IND establishes a regulatory framework for safety, manufacturing, and clinical design, reducing regulatory risk, shortening timelines, and enabling expansion into future indications.
  • Strong Preclinical and OLE Data: NUZ-001 is supported by robust TDP-43 preclinical data and encouraging survival outcomes from the Open-Label Extension (OLE) study in ALS patients.
  • Collaboration with HEALEY ALS Platform Trial: Neurizon is working with Mass General Hospital (MGH) to file a protocol amendment to incorporate NUZ-001’s regimen, with patient enrollment anticipated in Q4 2025.

CEO Comment

"The FDA’s clearance marks a significant step in our mission to bring NUZ-001 closer to ALS patients. This milestone reflects our commitment to rigorous scientific and clinical development, as well as the support of KOLs and patient advocacy groups. We believe NUZ-001 has the potential to meaningfully slow the progression of this devastating disease."

  • Dr. Michael Thurn, MD, CEO of Neurizon.

About NUZ-001

NUZ-001 is Neurizon’s lead investigational therapy for amyotrophic lateral sclerosis (ALS), the most common form of motor neuron disease. As a platform molecule, NUZ-001’s regulatory clearance supports future development programs while providing a validated framework for clinical design, safety, and manufacturing.

About Neurizon Therapeutics

Neurizon Therapeutics Limited is a clinical-stage biotechnology company dedicated to advancing treatments for neurodegenerative diseases. The company’s mission is to accelerate patient access to innovative therapies and develop potential disease-modifying treatments for ALS and related conditions.

Optimize Your trial insights with Clival Database.

Are you exhausted from the uncertainty of trial insights pricing? Clival Database ensures the clarity in the midst of the global scenario for clinical trials to you.

Clival Database is one of the best databases that offers an outstanding number of clinical trial data in terms of 50,000+ molecules and from primary regulatory markets as well as new entrants like Indian and Chinese markets.

With Clival, you get accurate positioning of historical sales data, patent database, company profiling, safety & efficacy, and prediction of launch of new innovative molecules helping you to align your research and driving down the cost.

To add value, we further break down our analytics for you so that improving your operational effectiveness; optimizing your clinical trials; and offering you accurate and high-quality data at lowest possible prices becomes possible.

Elevate your trial success rate with the cutting-edge insights from Clival database.

Check it out today and make more informed sourcing decisions! Learn More!