FDA clinical hold lift closer for Neurizon
Neurizon submits formal response to US FDA to resolve NUZ-001 clinical hold
Submission includes new bridging PK data to demonstrate comprehensive exposure data in rats and dogs
FDA review underway with Neurizon targeting participation in HEALEY trial in Q4 CY25
Neurizon Therapeutics (ASX:NUZ) a clinical-stage biotech company dedicated to advancing treatments for neurodegenerative diseases, said the clinical hold complete response (CHCR) includes new bridging pharmacokinetic (PK) data from 28-day studies in rats and dogs.
The PK studies were completed ahead of schedule and designed to address the FDA's request for more comprehensive animal exposure data to support safety margins of NUZ-001, the company's lead therapy for amyotrophic lateral sclerosis (ALS), and its primary sulfone metabolite.
The response was submitted following constructive FDA engagement with the new PK results demonstrating:
Greater than 10-fold safety margins based on projected human plasma exposure levels for both NUZ-001 and its active sulfone metabolite
Enhanced confidence in dose selection and systemic tolerability to support progression to Phase 2/3 clinical evaluation
FDA review of the CHCR is now underway and Neurizon expects formal feedback within 30 calendar days, in line with the regulator's standard statutory review period for hold responses.
Focus on advancing into HEALEY ALS Platform trial
The formal response to the FDA marks a significant step toward lifting the clinical hold on NUZ-001 and initiating enrolment in the HEALEY ALS Platform Trial, anticipated in Q4 CY25.
ALS is the most common form of motor neurone disease (MND) and the HEALEY ALS Platform Trial is a significant ongoing program in the US aimed at accelerating ALS treatment development.
'With continued momentum and scientific clarity, we are advancing NUZ-001 toward a potentially first-in-class therapy for ALS, aligned with our commitment to create meaningful treatment options for patients,' managing director and CEO Dr Michael Thurn said.
'This submission underscores our disciplined execution and ability to deliver critical development milestones ahead of schedule.
'By proactively completing the required PK studies, we have significantly de-risked the regulatory path moving forward.
'We anticipate resolution of the clinical hold in August 2025, positioning Neurizon to initiate participation in the HEALEY ALS Platform Trial before year-end."
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.
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