Neurizon Advances ALS Drug NUZ-001 Despite $7.28M Half-Year Loss

Financial Overview

Neurizon Therapeutics Limited (ASX: NUZ), formerly PharmAust Limited, revealed a substantial increase in its net loss for the half-year ended 31 December 2024, reporting a loss after tax of $7.28 million, up from $389,000 in the previous period. This 1770% increase reflects intensified investment in research and development as the company advances its neurodegenerative disease drug pipeline.

Despite the widening losses, Neurizon strengthened its balance sheet, ending the period with net assets of $11.9 million and cash reserves of $14 million, supported by recent capital raises and government R&D tax incentives totaling $1.54 million. These funds provide a runway to support ongoing clinical programs and regulatory activities.

Clinical and Regulatory Milestones

The company’s lead candidate, NUZ-001, achieved a significant milestone by being accepted into the prestigious HEALEY ALS Platform Trial, a multi-center clinical study designed to accelerate the evaluation of promising ALS treatments. This inclusion not only validates NUZ-001’s therapeutic potential but also positions Neurizon at the forefront of ALS drug development.

Interim data from the ongoing Open Label Extension (OLE) study demonstrated encouraging clinical outcomes. Patients treated with NUZ-001 showed a 43.2% reduction in the rate of ALS functional decline at four months post-treatment and sustained benefits at eight months, including significantly increased survival rates and reduced risk of death compared to historical controls. Importantly, NUZ-001 was well tolerated with no serious adverse events reported at the recommended dosing.

On the regulatory front, Neurizon secured Small and Medium-Sized Enterprise (SME) status from the European Medicines Agency (EMA), unlocking fee reductions and enhanced regulatory support. What's more, NUZ-001 was granted Orphan Medicinal Product Designation by the EMA, complementing prior U.S. FDA orphan drug status, which affords market exclusivity and strengthens intellectual property protections in key markets.

Challenges and Next Steps

While the company made strides, it faced a setback when the U.S. FDA placed the Investigational New Drug (IND) application for NUZ-001 on clinical hold pending additional animal exposure data. Neurizon’s management expressed confidence in addressing the FDA’s requests promptly to resume clinical progress.

Neurizon also completed a strategic rebranding to align its corporate identity with its focus on neurodegenerative diseases, signaling a renewed commitment to pioneering therapies for ALS, Alzheimer’s, Parkinson’s, and Huntington’s diseases.

Financially, the company raised $8.7 million through share placements, including a $0.19 per share tranche to related parties, and continues to invest heavily in R&D activities. The board’s focus remains on advancing NUZ-001 through pivotal clinical trials while maintaining financial flexibility.

Outlook

Neurizon’s half-year results underscore the high-risk, high-reward nature of biotech development. The company’s clinical and regulatory achievements provide a solid foundation, but the path to commercialisation hinges on overcoming regulatory hurdles and demonstrating definitive clinical efficacy in larger trials. Investors will be watching closely as Neurizon navigates these critical next phases.