Neurizon®? Therapeutics Limited announced that the first participant has been dosed in Regimen I of the HEALEY ALS Platform Trial evaluating Neurizon's lead candidate, NUZ-001, for the treatment of amyotrophic lateral sclerosis (ALS). The HEALEY ALS Platform Trial (ClinicalTrials.gov identifier: NCT04297683) is a multicentre, double-blind, placebo-controlled adaptive Phase 2/3 clinical trial conducted by the Sean M. Healey & AMG Center for ALS at Mass General Hospital Brigham in the United States (US), created in partnership with the Network of Excellence for ALS (NEALS).
Entry into the HEALEY ALS Platform trial is competitive, with drug candidates reviewed and selected by expert committees based on scientific merit and evidence of potential benefit in ALS. The goal of the HEALEy ALS Platform Trial is to accelerate the development of potential new ALS therapies. The trial evaluate multiple investigational drugs (Regimens) concurrently under a single framework or master protocol, leveraging shared infrastructure across over 70 participating clinical sites.
By streamlining start-up and enrollment processes, it accelerates study execution and delivers results more efficiently. Regimen I (NUZ-001) includes a randomised, placebo-controlled treatment (RCT) phase followed by an active treatment extension (ATE) phase, both with a 36-week treatment period. Approximately 160 participants with ALS will be randomised to receive either daily NUZ-001 at the recommended Phase 2 dose of 10 mg/kg or placebo at a 3:1 ratio.
The primary objective is to evaluate the efficacy of NUZ-001 compared with placebo on ALS disease progression, with secondary objectives including additional measures of disease progression and safety. Participation in the HEALEY ALS platform Trial provides Neurizon with access to an established clinical development framework supported by the world's most highly regarded ALS investigators and leading clinical centres across the US. This infrastructure improves trial efficiency, supports consistent data generation and facilitates ongoing engagement with regulatory authorities, including the U.S. Food and Drug Administration (FDA), as the trial progresses.
