FDA Lifts Clinical Hold on Investigational ALS Drug
The Food and Drug Administration (FDA) has lifted the clinical hold on NUZ-001 (Neurizon Therapeutics, Melbourne, Victoria, Australia) an investigational treatment for people with amyotrophic lateral sclerosis (ALS), allowing the company to proceed with clinical development. The decision enables NUZ-001’s entry into the HEALEY ALS Platform Trial led by Merit Cudkowicz, MD, Director of the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital. Enrollment is expected to begin in the fourth quarter of 2025.
NUZ-001, a common deworming drug used in veterinary medicine, is under investigation for the treatment of people with ALS because of its inhibitory activity on the mTOR signaling pathway. Last year, Neurizon Therapeutics submitted an Investigational New Drug (IND) application for NUZ-001 to initiate a phase 2/3 clinical study within the HEALEY ALS Platform Trial. In January 2025, the FDA placed a clinical hold on the IND application for NUZ-001, requesting clarification about human risks and proposed dosing.
“We are now partnering with the HEALEY ALS Platform Trial team to take the next critical steps in advancing NUZ-001,” said Michael Thurn, PhD, Managing Director and CEO of Neurizon Therapeutics. “Supported by our compelling TDP-43 preclinical data and encouraging survival outcomes from the Open-Label Extension (OLE) study in ALS patients, we believe NUZ-001 has the potential to meaningfully slow the progression of this devastating disease.”
According to a statement from Neurizon Therapeutics, the HEALEY ALS Platform Trial is expected to file a protocol amendment to their IND to initiate investigation of NUZ-001.
Sources: Neurizon Therapeutics. FDA lifts Clinical Hold on NUZ-001. Biotech Newswire. Published October 6, 2025. Accessed October 13, 2025. https://www.biotechnewswire.ai/202510062692/fda-lifts-clinical-hold-on-nuz-001.html
Neurizon Therapeutics. Neurizon files IND application to support HEALEY ALS Platform Trial. PR Newswire. Published December 18, 2025. Accessed October 13, 2025. https://www.prnewswire.com/news-releases/neurizon-files-ind-application-to-support-healey-als-platform-trial-302334461.html?utm_source=chatgpt.com
Neurizon Therapeutics. Neurizon updates on NUZ-001 IND application. Neurizon Investor Hub. Published January 16, 2025. Accessed October 13, 2025. https://investorhub.neurizon.com/announcements/6753351