Neurizon Therapeutics Achieves Milestone with NUZ-001 Approval

FDA Lifts Clinical Hold on NUZ-001

Highlights:

• U.S. FDA lifts the Clinical Hold on NUZ?001, Neurizon's lead investigational therapy for ALS.
• Clears the way for entry into the HEALEY ALS Platform Trial expected to commence in Q4 CY2025.
• IND supported by robust preclinical safety data and comprehensive manufacturing and quality information from the previously signed Elanco licensing agreement.
• Expedites NUZ-001 as a platform molecule with pipeline expansion potential.
• Positions Neurizon for accelerated regulatory pathways and future partnering opportunities.

Neurizon Therapeutics Limited (ASX: NUZ & NUZOA), a pioneering clinical-stage biotech firm focused on advancing treatments for neurodegenerative diseases, announces significant progress with the U.S. Food and Drug Administration's (FDA) recent decision to lift the clinical hold on their flagship investigational drug, NUZ?001. This essential step forward not only signifies a critical regulatory milestone for Neurizon but also offers new hope for the ALS community, setting the stage for Phase 2/3 development of NUZ-001 as part of the HEALEY ALS Platform Trial, slated to start in the fourth quarter.

Dr. Michael Thurn, Managing Director and CEO, expressed his enthusiasm, stating, "The FDA's clearance of our IND application represents a noteworthy achievement in our journey to provide NUZ-001 to ALS patients. This progress is a testament to our team's dedication and commitment to thorough scientific and clinical research."

He further highlighted the invaluable assistance from key opinion leaders (KOLs) and patient advocacy groups, whose support was instrumental in driving this program forward. Their ongoing efforts to raise awareness and advocate for the ALS community’s pressing needs have played a crucial role in reaching this momentous achievement.

Neurizon is now collaborating with the HEALEY ALS Platform Trial team to proceed with essential next steps in advancing NUZ-001. Backed by convincing data regarding TDP-43 and promising survival outcomes from an Open-Label Extension study, there is strong belief in NUZ-001's ability to significantly alter the course of this challenging disease.

Establishing an IND for a platform molecule like NUZ-001 lays the groundwork not just for the development of the primary candidate but also facilitates future programs. This framework ensures a validated approach for safety, manufacturing, and clinical design, thus minimizing regulatory risks and enabling expedited movement into new indications.

The strength of the IND is further bolstered by the detailed packaging of preclinical safety data and thorough manufacturing information obtained through a licensing agreement with Elanco. Such assets enhance confidence surrounding the platform's readiness for advanced clinical development, ensuring it maintains broad applicability and significant long-term commercial potential.

Next Steps:

With the IND now in effect, Neurizon expects Mass General Hospital (MGH) to submit a protocol amendment to their IND for including Neurizon’s specific regimen in the HEALEY ALS Platform Trial shortly. Patient enrollment is anticipated to begin in Q4 CY2025.

These milestones contribute to Neurizon’s mission of facilitating patient access to innovative therapies, creating sustainable value for shareholders, and securing NUZ-001’s position as a potentially effective treatment for ALS.

This announcement has been officially authorized for release by the Board of Neurizon Therapeutics Limited.

About Neurizon Therapeutics Limited

Neurizon Therapeutics Limited (ASX: NUZ) is a clinical-stage biotechnology company dedicated to innovating treatments for neurodegenerative diseases. The company is actively advancing its lead drug candidate, NUZ-001, aimed at treating ALS, the most prevalent form of motor neuron disease. Neurizon's strategic focus includes accelerating patient access to effective therapies for ALS while investigating the broader applicability of NUZ-001 across various neurodegenerative conditions. Through robust international collaborations and stringent clinical programs, Neurizon strives to create new opportunities for those affected by complex neurological disorders. NUZ-001 remains an investigational product and is not yet approved for commercial use in any region.

Frequently Asked Questions

What is NUZ-001?

NUZ-001 is Neurizon Therapeutics' investigational drug aimed at treating ALS, currently undergoing clinical trials to establish its efficacy.

What does the FDA's approval mean for Neurizon?

The FDA lifting the clinical hold allows Neurizon to proceed with clinical trials for NUZ-001, moving closer to making potential therapies available to patients.

When will the HEALEY ALS Platform Trial begin?

Enrollment for the HEALEY ALS Platform Trial is expected to start in Q4 CY2025 as part of the study of NUZ-001.

What is the significance of the IND for NUZ-001?

The IND approval is crucial as it streamlines the regulatory process, establishing a foundation for both the current drug and any future candidates in development.

How does Neurizon support the ALS community?

Neurizon collaborates with KOLs and advocacy groups, raising awareness and working towards better treatment options for patients living with ALS.

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