Neurizon Therapeutics Limited provided 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. 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 key primary endpoint in the HEALEY ALS Platform trial. SVC is a pulmonary function test that measures the maximum volume of air a person can exhale slowly after a full inhalation. Research has shown a strong correlation in the rate of decline between percentage predicted SVC and ALSFRS-R scores.
To aid confirmation of secondary endpoints for the HEALEY ALS Platform trials, a comparative analysis was performed by Berry Consultants against 35 matched controls from the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database for SVC decline against the 12 patients treated with NUZ-001 in the Phase 1 MEND Study. The estimated rate of decline for all 12 patients treated with NU Z-001 was -1.51 VC% predicted (PP) points per month compared to -2.93 VC PP points per month (difference 1.42; p=0.058) for untreated matched controls. SVC has been selected as a key secondary endpoint for the trial, alongside the targeted primary endpoint of overall function (ALSFRS-R) and survival in the patient population.
The PRO-ACT data analysis was performed by Berry Consultant against the Healey & AMG center.
