Neurizon Therapeutics Limited (ASX: NUZ & NUZOA), a trailblazer in the development of treatments for neurodegenerative diseases, is excited to share positive preclinical findings for its leading drug candidate, NUZ-001, at the 7th Annual ALS Research Symposium organized by ALS ONE. The symposium is a key event that unites global experts, patients, and advocates to discuss progress in research and therapeutic strategies for Amyotrophic Lateral Sclerosis (ALS).

Insights from Dr. Michael Thurn’s Presentation

Dr. Michael Thurn, Managing Director and CEO of Neurizon, delivered a compelling presentation titled "Exploring the Benefits of mTOR Inhibition in Patients with Amyotrophic Lateral Sclerosis." His report detailed promising results from recent preclinical studies conducted on human in vitro iPSC Motor Neuron models of ALS, demonstrating that NUZ-001 effectively inhibits the mTOR pathway, which is critical for neuronal health and disease progression.

Key Preclinical Findings

The studies focused on the capability of NUZ-001 and its major active metabolite to significantly reduce the aggregation of TAR DNA-binding protein 43 (TDP-43) — a protein often found in deteriorated forms in ALS patients. Findings revealed:

• A 50% reduction in TDP-43 aggregation by NUZ-001 and a 65% reduction by its major metabolite in M337V Motor Neurons under stress conditions.
• Significant improvements in the electrophysiological functionality of neurons treated with NUZ-001 and its metabolite.

These results position NUZ-001 as a potentially transformative treatment for ALS, offering new hope for managing and possibly slowing the progression of this debilitating disease.

About the ALS ONE Symposium

The ALS ONE Symposium is renowned for its commitment to fostering collaboration and innovation in ALS research. It plays a pivotal role in bringing together diverse stakeholders to accelerate the development of new treatments and share vital knowledge to combat ALS.

About Neurizon Therapeutics Limited

Based in South Melbourne, Neurizon Therapeutics Limited is at the forefront of developing innovative treatments for neurodegenerative diseases, particularly ALS, the most common form of motor neuron disease. The company’s strategy focuses on accelerating the availability of effective treatments for ALS patients and exploring the broader potential of NUZ-001 for other neurodegenerative conditions. Through its rigorous clinical programs and international partnerships, Neurizon is dedicated to improving outcomes for patients and families affected by these complex disorders.

Further Information and Investor Relations

For additional details regarding Neurizon’s research and developments, stakeholders are encouraged to contact Dr. Michael Thurn directly or Catherine Strong at Sodali & Co. Investors and interested parties can also access more information through the Neurizon Investor Hub, which offers resources such as video summaries of key announcements and a platform for submitting questions and comments.

Conclusion

Neurizon Therapeutics’ presentation at the ALS Research Symposium marks a significant milestone in the fight against ALS. By harnessing the potential of mTOR inhibition, Neurizon continues to push the boundaries of scientific research, bringing hope and potential new solutions to the forefront of neurodegenerative disease treatment.