Neurizon Therapeutics Edges Closer to ALS Trial Milestone as FDA Review Gets Underway

Key Highlights:

• Neurizon submits response to FDA to lift clinical hold on NUZ-001
• Bridging PK data shows >10x safety margin, supports dose selection
• FDA feedback expected by August; Phase 2/3 trial targeted for Q4 2025
• CEO says milestone “significantly de-risks” regulatory path
• Shares of NUZ rise 19.36% to $0.185 amid investor optimism

Clinical-stage biotech company Neurizon Therapeutics Ltd (ASX: NUZ) surged nearly 20% on the ASX today after announcing the submission of its Complete Response to the U.S. FDA, marking a pivotal regulatory step for its lead candidate NUZ-001, an investigational therapy for amyotrophic lateral sclerosis (ALS).

The company’s formal submission addresses the clinical hold that had been placed on its Investigational New Drug (IND) application, and comes backed by new pharmacokinetic (PK) data designed to assure the U.S. Food and Drug Administration of NUZ-001’s safety profile. The regulatory update places Neurizon back on track for potential inclusion in the HEALEY ALS Platform Trial in Q4 CY2025, a prestigious multi-center adaptive trial platform evaluating promising ALS therapies.

Bridging the Gap with New Data

At the core of Neurizon’s response are 28-day animal studies in rats and dogs, conducted to provide comprehensive exposure data for NUZ-001 and its active sulfone metabolite. The new PK results showed greater than 10-fold safety margins based on projected human plasma exposure levels.

“This data enhances our confidence in dose selection and systemic tolerability, supporting our transition into Phase 2/3 clinical evaluation,” the company stated in its announcement .

The FDA is now reviewing the Clinical Hold Complete Response (CHCR), with formal feedback expected within 30 calendar days, adhering to the agency’s standard review timeframe. If successful, this review could clear the way for patient enrollment in late 2025.

CEO Confident in Regulatory Momentum

Dr. Michael Thurn, Neurizon’s Managing Director and CEO, emphasized the significance of this milestone in the company’s journey:

“With continued momentum and scientific clarity, we are advancing NUZ-001 toward a potentially first-in-class therapy for ALS. This submission underscores our disciplined execution and ability to deliver critical development milestones ahead of schedule,” said Dr. Thurn.

“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 strategic push aligns with Neurizon’s broader mission to expedite therapies for neurodegenerative diseases through international partnerships and robust clinical development pathways.

A Promising Entry into the ALS Landscape

ALS, or Lou Gehrig’s disease, remains a condition with few treatment options and a high unmet need. NUZ-001’s potential first-in-class status stems from its novel therapeutic mechanism and tolerability profile.

If approved, Neurizon’s entry into the HEALEY ALS Platform Trial will place it alongside some of the most promising drug candidates in the neurodegenerative research field. The platform is designed to streamline trial timelines and reduce redundancy, offering faster insights into efficacy and safety across multiple therapies.

According to the company, the Phase 2/3 trial participation remains on track for Q4 2025, pending regulatory clearance, with the platform trial offering both flexibility and global validation.

What Comes Next?

Pending FDA feedback, Neurizon is now in a wait-and-see period. Investors and analysts alike will be watching closely for August’s response from the FDA. A green light would not only validate the company’s approach to regulatory engagement but also reinforce NUZ-001’s prospects in the ALS treatment landscape.

Meanwhile, the company has encouraged shareholders to stay engaged through its newly launched Neurizon Investor Hub, where video updates and detailed responses to investor questions will be shared.

Final Thoughts

Neurizon Therapeutics’ proactive strategy in addressing regulatory hurdles highlights its commitment to scientific rigor and patient outcomes. With clear milestones ahead and an ambitious roadmap for NUZ-001, the coming months could be pivotal for this emerging biotech player.

As of 1:00 PM AEST on July 25, 2025, NUZ shares were trading at $0.185, up 19.36% on the day.