Neurizon Therapeutics (ASX: NUZ & NUZOA) has taken a significant step forward in its fight against ALS, unveiling a promising respiratory outcome from its lead drug candidate, NUZ-001, and sharing updates on its upcoming participation in the HEALEY ALS Platform Trial.
NUZ-001 Shows 48% Slowing in Respiratory Decline
A fresh analysis from the Phase 1 MEND study, using data matched from the widely respected PRO-ACT database, found that NUZ-001 slowed respiratory decline by 48% compared to untreated patients. The measure, Slow Vital Capacity (SVC), is a key respiratory function and survival metric in ALS – a disease where respiratory muscle weakness is often a primary cause of death.
This promising result mirrors a previously announced 39% slowing in functional decline, measured by ALSFRS-R, and reinforces NUZ-001’s potential as a meaningful treatment for ALS patients.
Platform Trial Protocol Updated for Better Patient Outcomes
The HEALEY ALS Platform Trial, considered a game-changer for ALS drug development, has updated its Master Protocol following insights from the first five investigational therapies. Key changes include:
- Longer treatment period: The trial’s core treatment window has been extended from 24 to 36 weeks, offering more time for potential effects to show.
- Refined patient selection: Inclusion criteria now target patients within 24 months of symptom onset, focusing on faster-progressing individuals.
- Increased biological sampling: Collection of peripheral blood mononuclear cells will aid future research and development.
- More flexible participation: Streamlined visit schedules and improved access to remote appointments will support patient engagement.
Neurizon on Track for HEALEY Trial Entry in H2 CY2025
Neurizon is now finalising its Regimen Specific Appendix (RSA), which will outline the company’s protocol under the broader trial framework. Submission to the FDA is expected once the current clinical hold on NUZ-001 is lifted.
Managing Director and CEO, Dr. Michael Thurn, said:
“We’re encouraged by the positive SVC data, which strongly supports our belief that NUZ-001 has the potential to deliver a meaningful benefit to ALS patients. Combined with the refined HEALEY protocol, we’re well positioned to make the most of this opportunity.”
In parallel, Neurizon will complete two short-term pharmacokinetic studies to satisfy FDA requirements. Trial participation is anticipated in H2 CY2025, marking a pivotal milestone for the company’s ALS program.
