Highlights:
Updates to HEALEY ALS Platform Trial Master Protocol have been made to enhance the potential of establishing a clinically meaningful outcome for patients, including:
Treatment period extended from 24 to 36 weeks to allow longer evaluation of treatment effects
Inclusion Criteria modified for time since symptom onset from 36 to 24 months to enrich the study population for fast progressors
peripheral blood mononuclear cell (PBMC) collection has been added to aid future research and therapy development
New PRO-ACT data analysis undertaken by Berry Consultants confirms Slow Vital Capacity (SVC), a measure of respiratory function, as a key secondary endpoint for NUZ-001
Compared to untreated matched controls, treatment with NUZ-001 resulted in a 48% slowing in the loss of respiratory function for all 12 patients in the Phase 1 MEND Study
Neurizon to finalise its Regimen Specific Appendix (RSA) under the Master Protocol for submission to the FDA as soon as the clinical hold on NUZ-001 has been lifted
Neurizon remains well placed for trial entry in H2 CY2025
Neurizon Therapeutics Limited (ASX: NUZ & NUZOA) (“Neurizon” or “the Company”), a clinical-stage biotech company dedicated to advancing treatments for neurodegenerative diseases, is pleased to provide the following update on preparations for entry into the HEALEY ALS Platform Trial, along with encouraging new data showing that NUZ-001 slows the decline in slow vital capacity (SVC), a key respiratory function and survival metric in Amyotrophic Lateral Sclerosis (ALS).
Updates to HEALEY ALS Platform Trial Master Protocol:
The HEALEY ALS Platform Trial is the first of its kind clinical trial for ALS. The trial is a multicenter, double-blind, placebo-controlled, perpetual and adaptive study evaluating the safety and efficacy of multiple investigational products for ALS treatment. As previously advised, Neurizon expects entry into the trial in H2 CY2025 and advises of the following changes to the Master Protocol from the Healey & AMG Center.
The platform trial features a perpetual and adaptive design facilitating integration of new information from experience, data analysis, and participant feedback. Initial regimens tested in the trial have subsequently undergone thorough testing and analysis by a suite of project experts including lead medical investigators, biostatisticians, the United States (US) Food and Drug Administration (FDA) and industry partners.
Following the latest round of testing, updates to the Master Protocol have been included to enhance and optimise future learning within the trial based on data collected from the first 5 regimens. The changes include:
● The Randomized Controlled Trial (RCT) period has been extended from 24 to 36 weeks to allow for a longer evaluation of treatment effects and increase the power to detect positive treatment outcomes, especially for therapies that take longer to show effects
● The Inclusion Criteria for the trial have also been modified, setting the time since symptom onset from 36 to 24 months to enrich the study population for fast progressors
● Peripheral blood mononuclear cell (PBMC) collection has been added to be banked for future generation of induced pluripotent stem cells (iPSCs), aiding research and therapy development
● In addition, patient-centric aspects of the trial have been refined, with a more streamlined and flexible visit schedule.
In connection with efforts to improve the patient experience, increased opportunities for remote visits are now available in the Active Treatment Extension (ATE) program.
The Company believes that these changes further reduce the risks associated with NUZ-001’s clinical development program and enhance the potential to establish a clinically meaningful outcome for patients with ALS. These modifications allow Neurizon to finalise its Regimen Specific Appendix (RSA) under the Master Protocol for submission to the FDA as soon as the clinical hold on NUZ-001 has been lifted. Further information on the RSA for NUZ-001 will be provided following FDA acceptance in H2 CY2025.
New data analysis confirms SVC as a key secondary endpoint for NUZ-001:
As part of finalising the RSA for NUZ-001’s new data analysis of SVC results captured from the completed Phase 1 MEND study last year, the Company has confirmed the selection of SVC as a k...
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