Neurizon on track to lift FDA clinical hold on ALS drug, eyes Healey trial in Q4 CY25

Neurizon Therapeutics has announced progress to lift a US Food and Drug Administration (FDA) clinical hold on its lead drug NUZ-001.

Neurizon is focused on entering the HEALEY ALS platform trial in Q4 CY25. Pic via Getty

Special Report: Neurizon Therapeutics has announced it has received positive feedback from the US Food and Drug Administration (FDA) on its strategy to lift the clinical hold on its lead drug NUZ-001. The Company expects to enter the HEALEY amyotrophic lateral sclerosis (ALS) platform trial in Q4 CY25. 

• Neurizon on the right track to lifting the US FDA clinical hold on lead drug NUZ-001
• Regulator provides positive feedback on the strategy of conducting two preclinical pharmacokinetic studies 
• Neurizon aims to enter the HEALEY amyotrophic lateral sclerosis (ALS) platform trial in Q4 CY25

Following recent formal interactions, the FDA has provided positive written feedback to Neurizon Therapeutics (ASX:NUZ) on its strategy of conducting two preclinical pharmacokinetic (PK) studies to lift the clinical hold on NUZ-001. 

Neurizon has already completed the two PK studies ahead of schedule, enabling continued momentum towards initiating the HEALEY ALS platform trial, a significant ongoing trial in the US aimed at accelerating ALS treatment development.

NUZ is targeting the treatment of the rare but devastating neurodegenerative disease ALS, the most common form of motor neurone disease.

The PK studies were conducted by a global contract research organisation (CRO) specialising in preclinical PK studies. 

The treatment phase of both studies has been successfully completed, along with the analysis of blood samples, and the study reports are being finalised. 

The studies were completed within budget and may qualify for a rebate under the Australian government’s Research and Development (R&D) Tax Incentive Scheme.

Neurizon anticipates submitting the complete response containing data from these two PK studies to the FDA in the coming weeks as part of the formal hold resolution process. 

The company said it remained focused on activating participation in the HEALEY ALS Platform Trial in Q4 CY2025.

Listen: Michael Thurn chats with Tim Boreham

In a previous instalment of Health Kick, Tim speaks with Neurizon managing director Dr Michael Thurn about the company’s research on motor neurone disease and its drug development of NUZ-001 to treat ALS.

‘Commitment to advancing NUZ-001’

Managing director and CEO Dr Michael Thurn said the company was delighted to receive written confirmation from the FDA supporting its strategy to resolve the clinical hold for NUZ-001. 

“By proactively progressing and completing the required PK studies ahead of schedule, we’ve maintained strong momentum and demonstrated our commitment to advancing NUZ-001 with urgency and scientific precision,” he said. 

“We are well-positioned to submit the complete response in the coming weeks and anticipate that the clinical hold will be lifted in August 2025. 

“We are focused on achieving key milestones that will enable Neurizon’s active participation in the HEALEY ALS Platform Trial later this year.”

This article was developed in collaboration with Neurizon Therapeutics, a Stockhead advertiser at the time of publishing. 

This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.