Neurizon Raises $5.2m, Starts GMP Manufacturing Ahead of ALS Platform Trial

Regulatory Breakthrough Unlocks Clinical Trial Momentum

Neurizon Therapeutics Limited has achieved a pivotal regulatory milestone with the U.S. Food and Drug Administration lifting the clinical hold on its Investigational New Drug application for NUZ-001, its lead candidate targeting Amyotrophic Lateral Sclerosis (ALS). This clearance, announced shortly after the quarter ended, clears the path for Neurizon to enter the HEALEY ALS Platform Trial, a significant step toward potential regulatory approval.

The FDA’s decision follows Neurizon’s comprehensive response addressing safety concerns through bridging pharmacokinetic studies in animals, underscoring the company’s rigorous approach to clinical development. This regulatory green light not only advances NUZ-001’s ALS program but also strengthens its positioning as a platform molecule with potential applications across other neurodegenerative diseases.

Strategic Licensing and Manufacturing Milestones

Complementing the regulatory progress, Neurizon secured an exclusive global licensing agreement with Elanco Animal Health, a major player in veterinary pharmaceuticals. This deal grants access to a robust package of non-clinical animal safety data and Good Manufacturing Practice (GMP) manufacturing information, critical for future regulatory submissions and commercialisation efforts.

In parallel, the company commenced GMP manufacturing of NUZ-001 tablet registration batches at Catalent, a leading contract development and manufacturing organisation. These batches, produced using full-scale commercial processes, will support stability studies and regulatory filings, marking a key step in scaling production capabilities.

Clinical Data Signals Promise Amidst Ongoing Development

Neurizon reported encouraging topline results from a 12-month Open-Label Extension study involving ALS/MND patients. The study met its primary safety endpoint, confirming long-term tolerability of NUZ-001 at the recommended Phase 2 dose. Importantly, preliminary efficacy signals emerged, including a significant survival benefit compared to matched historical controls, suggesting a potential median survival extension of 16 months.

These findings, while preliminary and derived from a small patient cohort, provide a compelling rationale for advancing NUZ-001 into larger, controlled clinical trials. The company is also developing a multi-omics biomarker strategy to enhance clinical trial insights and regulatory engagement.

Financial Strengthening and Market Engagement

On the financial front, Neurizon bolstered its balance sheet by raising $5.2 million through a share placement supported by institutional investors, management, and the Board. Additionally, it secured a $1.5 million loan against its anticipated R&D tax rebate, with a total rebate of approximately $5.6 million expected in the coming quarter. This non-dilutive funding provides crucial runway to support ongoing clinical and manufacturing activities.

To enhance investor visibility and liquidity, Neurizon’s shares were approved for trading on the U.S. OTCQB Venture Market under the ticker NUZTF. The company also appointed Integrous Communications to strengthen North American investor relations ahead of the HEALEY ALS Platform Trial commencement.

Community Commitment and Forward Outlook

Neurizon continues to engage actively with the ALS/MND community through fundraising, awareness initiatives, and collaboration with patient advocacy groups globally. This patient-centric approach remains central to its mission of delivering transformative therapies.

Looking ahead, Neurizon is focused on submitting protocol amendments, completing GMP manufacturing batches, and initiating patient recruitment for the HEALEY ALS Platform Trial. The company also aims to expand its pipeline into other neurodegenerative diseases and explore strategic partnerships to accelerate development.