Neurizon Therapeutics (ASX:NUZ) has reported positive topline results from the Open-Label Extension (OLE) study of its lead candidate NUZ-001 for the treatment of amyotrophic lateral sclerosis (ALS), which is the primary form of motor neurone disease (MND).
The company said that long-term treatment with NUZ-001 at the recommended Phase 2 dose was safe and well-tolerated. Preliminary efficacy findings demonstrated that treatment with NUZ-001, compared to matched historical controls from the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database, was associated with a significant survival advantage, sustained slowing of global functional decline, a reduced rate of respiratory decline, and a stable or downward trend in key disease biomarkers.
Neurizon Managing Director and CEO Dr Michael Thurn, said, “We are very encouraged by these long-term treatment results, which reinforce the potential of NUZ-001 to deliver meaningful clinical benefits for people living with ALS. Importantly, the therapy has been shown to be safe and well-tolerated, even with extended use.
"For too long, patients and families have faced this devastating disease with very few treatment options, and the field has struggled to find truly viable new options. To see sustained functional and respiratory benefits, a clear survival advantage, and supportive biomarker trends after nearly three years of treatment gives us additional confidence as we finalise preparations for entry into the HEALEY ALS Platform Trial.
"I would like to thank Associate Professor Susan Mathers at Calvary Health Care Bethlehem and Professor Dominic Rowe at Macquarie University, as well as their clinical teams and the incredible patients, families, and caregivers who made this study possible."
Associate Professor Susan Mathers, Principal Investigator at Calvary Health Care Bethlehem, added, “It has been a pleasure to be part of the development of NUZ-001 as a potential drug treatment for people living with ALS, and to work with Prof Rowe’s team at Macquarie University, Neurizon Therapeutics and especially our participants, their families and the research team at Calvary Health Care Bethlehem. NUZ-001 has been a well-tolerated therapy and we hope that the future phase 2/3 study will confirm its potential to benefit the wider ALS community.”