FDA's Decision to Lift Clinical Hold on NUZ-001 Marks Major Progress for Neurizon in ALS Treatment

FDA Lifts Clinical Hold on NUZ-001

Melbourne, Australia — Neurizon Therapeutics Limited has received significant news as the U.S. Food and Drug Administration (FDA) has officially lifted the clinical hold on its lead investigational therapy, NUZ-001, for Amyotrophic Lateral Sclerosis (ALS). This key development clears the path for Neurizon to advance to the next phase of clinical trials and participate in the HEALEY ALS Platform Trial, which is expected to begin in the fourth quarter of 2025.

Dr. Michael Thurn, the Managing Director and CEO of Neurizon, has characterized this FDA decision as a major milestone. He expressed that this landmark approval is a reflection of Neurizon's commitment to scientific rigor and clinical excellence in working towards effective treatment options for ALS patients. This confirmation follows considerable groundwork laid over the years involving extensive preclinical safety studies and a comprehensive set of data from its recent licensing agreement with Elanco, reinforcing the credibility of NUZ-001’s development journey.

The HEALEY ALS Platform Trial aims to evaluate multiple therapies simultaneously within a unified framework, allowing for efficiency in assessing potential options for patients dealing with this devastating disease. Neurizon’s NUZ-001 is now poised to become a pivotal player within this platform, benefitting from the strong foundation established by the IND application and prior positive outcomes observed during the Open-Label Extension (OLE) studies.

NUZ-001’s advancement into the HEALEY ALS Platform Trial will not only facilitate immediate access to treatment for ALS patients but also serve to expand Neurizon's portfolio of future clinical trials. With the IND approval now active, Neurizon is working closely with the trial team at Mass General Hospital (MGH) to finalize protocol amendments and initiate patient enrollment into the trial.

As a platform molecule, NUZ-001 is recognized for its broader application potential beyond ALS, with aspirations for further neurodegenerative applications. This prepares Neurizon for a future that includes strategic partnerships and an expedited regulatory pathway for multiple drug candidates aimed at improving the quality of life for individuals suffering from neurodegenerative diseases.

Next Steps

In the near term, Neurizon expects MGH to file updates to their IND which will include specific protocols related to NUZ-001 as part of the HEALEY ALS study. Once these amendments are approved, patient recruitment is anticipated to commence in Q4 CY2025, marking a consequential step in accelerating access to cutting-edge ALS therapies for patients in urgent need of effective treatment.

Thus far, Neurizon has navigated the complexities of drug development with a clear vision and a committed team, aligning scientific innovation with the urgent requirements of the ALS community. As they look forward, the company remains focused on delivering meaningful outcomes for patients and advancing their mission of transforming neurologic health through innovative therapies.

About Neurizon Therapeutics Limited

Founded to combat neurodegenerative diseases, Neurizon Therapeutics Limited is a clinical-stage biotech company specializing in developing effective ALS treatments. The company aims to offer fast access to transformative therapies while exploring comprehensive applications of NUZ-001 for other neurodegenerative conditions. Through dynamic international collaborations and stringent research methodologies, Neurizon is committed to reshaping the treatment landscape for patients and their families affected by complex neurological disorders.