Highlights:
The OLE study met its primary endpoint, confirming long-term treatment with NUZ-001 at the recommended Phase 2 dose was safe and well-tolerated
Treatment during the OLE was associated with a durable functional benefit (ALSFRS-R decline –0.88 points/month), minimal respiratory decline (stable SVC), and supportive biomarker trends (stable plasma NfL and decreased (–17%) urinary p75ECD)
Preliminary efficacy was assessed by comparing NUZ-001-treated patients to matched, untreated historical controls from the PRO-ACT database from the start of the Phase 1 MEND Study:
NUZ-001 significantly increased survival (χ2=13.75, p=0.00021)
NUZ-001 significantly reduced the risk of death by 76.7% (HR=0.233, p=0.0013)
Treatment with NUZ-001 showed an estimated median survival extension of ~16 months
NUZ-001 slowed the rate of functional decline by 31% (–0.83 vs –1.21 ALSFRS-R points/month; p = 0.145)
NUZ-001 reduced the rate of respiratory decline by 43% (–1.65 vs -2.93 VC PP points/month; p=0.078)
NUZ-001 has been safely used for more than 2.5 years, with five patients still receiving treatment under the TGA’s Special Access Scheme
These encouraging results reinforce the rationale for the planned pivotal HEALEY ALS Platform Trial, expected to commence in Q4 CY2025
Neurizon® Therapeutics Limited (ASX: NUZ & NUZOA) (“Neurizon” or “the Company”), a clinical-stage biotech company dedicated to advancing innovative treatments for neurodegenerative diseases, is pleased to report positive topline results from the Open-Label Extension (OLE) study of its lead candidate NUZ-001, for the treatment of amyotrophic lateral sclerosis (ALS), the major form of motor neurone disease (MND).
Long-term treatment with NUZ-001 at the recommended Phase 2 dose was safe and well-tolerated. Importantly, 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.
These encouraging results support the potential of NUZ-001 as a disease-modifying therapy for ALS and provide strong justification for advancing the program into further clinical development.
Managing Director and Chief Executive Officer, Dr. Michael Thurn, commented: “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.”
Study Overview and Results
The OLE study was an open-label, safety, tolerability, and preliminary efficacy study of NUZ-001 in 10 patients with ALS who completed the Phase 1 MEND Study, at the recommended Phase 2 dose of 10 mg/kg administered orally daily for 12-months.
Daily treatment with NUZ-001 was safe and well-tolerated, with no treatment-related deaths observed. Eight patients completed the study, while two patients died during the OLE due to respir...
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