Neurizon Therapeutics has achieved a significant regulatory milestone after receiving written confirmation from the US FDA that its strategy to lift the clinical hold on NUZ-001, an investigational treatment for amyotrophic lateral sclerosis (ALS), has been accepted. The FDA's positive feedback represents a crucial step forward for the Australian biotech company's lead program targeting this devastating neurodegenerative disease.
FDA Confirms Procedural Alignment
Following recent formal interactions with the regulatory agency, the FDA provided written feedback confirming procedural alignment with Neurizon's approach to resolve the clinical hold. The regulatory feedback specifically outlines the FDA's acceptance of conducting two preclinical pharmacokinetic (PK) studies to address the hold, which was initially imposed in February due to requests for additional animal exposure data to assess systemic exposure adequacy during clinical studies.
The FDA had requested this additional data to evaluate whether systemic exposure to NUZ-001 during clinical studies would be adequate, prompting Neurizon to develop a comprehensive response strategy in consultation with scientific and regulatory advisors.
Preclinical Studies Completed Ahead of Schedule
Demonstrating its commitment to advancing the program with urgency, Neurizon has already completed both necessary PK studies ahead of the anticipated timeline. The company reported that the treatment phase of both studies has been successfully completed, along with the analysis of blood samples, and study reports are currently being finalized.
These studies, which were expected to take approximately four months from commencement to completion including study start-up, the 28-day treatment period, data analysis, and reporting, were completed at a cost between $400,000 to $600,000. The proactive approach allowed Neurizon to maintain momentum while addressing the FDA's straightforward request for additional animal exposure data.
Timeline for Hold Resolution
Neurizon anticipates submitting a complete response containing data from the PK studies to the FDA in the coming weeks as part of the formal hold resolution process. The company expects the clinical hold to be lifted by August 2025, positioning NUZ-001 for entry into the HEALEY ALS Platform Trial during the second half of 2025.
"We are delighted to receive written confirmation from the FDA affirming our strategy to resolve the clinical hold for NUZ-001," said Dr. Michael Thurn, Neurizon's Managing Director and CEO. "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."
Strategic Positioning for Platform Trial
The regulatory progress enables Neurizon's continued momentum toward initiating participation in the HEALEY ALS Platform Trial, a significant opportunity for advancing NUZ-001 as a potential treatment for ALS and other neurodegenerative diseases. Dr. Thurn emphasized that the company is "well-positioned to submit the Complete Response in the coming weeks" and remains "focused on achieving key milestones that will enable Neurizon's active participation in the HEALEY ALS Platform Trial later this year."
The proactive decision to undertake the additional studies prior to receiving the formal FDA response highlights Neurizon's strategic approach to regulatory engagement and its commitment to advancing potential treatments for patients with ALS, a condition with significant unmet medical needs.
