FDA Hold Looms as Neurizon Reports Major Survival Gains in ALS Trial
Neurizon Therapeutics has announced the successful completion of its Open-Label Extension study for NUZ-001 in ALS, revealing significant survival benefits and strong tolerability. The company is on track to release top-line results in Q3 2025 and advance to a pivotal Phase 2/3 trial.
- Last patient completes 12-month treatment in NUZ-001 OLE study
- NUZ-001 reduces risk of death by 78.5% compared to historical controls
- Median survival extended by approximately 11 months
- No serious adverse events related to NUZ-001 reported
- Phase 2/3 HEALEY ALS Platform Trial planned for Q4 2025 pending FDA clearance
Completion of OLE Study Marks Key Milestone
Neurizon Therapeutics Limited (ASX: NUZ) has reached a pivotal moment in its clinical development of NUZ-001, its lead drug candidate for amyotrophic lateral sclerosis (ALS). The last patient enrolled in the 12-month Open-Label Extension (OLE) study has successfully completed treatment, marking the end of this critical phase of long-term safety and efficacy evaluation. Treatment was well-tolerated across all patients, consistent with the dosing planned for the upcoming Phase 2/3 HEALEY ALS Platform Trial.
Significant Survival Benefits Compared to Historical Controls
Updated survival analyses conducted by Berry Consultants compared NUZ-001 treated patients to untreated matched controls from the PRO-ACT historical database. The results are compelling: NUZ-001 treatment reduced the risk of death by 78.5% (hazard ratio 0.215, p=0.0015) and significantly increased survival (χ2=14.1, p=0.00017). Patients treated with NUZ-001 have experienced a median survival extension of approximately 11 months, a notable improvement over existing approved ALS therapies that typically extend life expectancy by 3 to 6 months.
Long-Term Treatment and Safety Profile
Patients have now been on continuous NUZ-001 treatment for up to 31 months, with no serious adverse events attributed to the drug. Of the original 12 patients from the Phase 1 MEND Study, seven remain alive, and six continue treatment under a compassionate use program. This sustained exposure and tolerability profile bolster confidence in NUZ-001’s potential as a transformative therapy for ALS.
Looking Ahead: Regulatory and Clinical Milestones
Neurizon anticipates releasing the top-line results from the OLE study in Q3 2025. The company is also preparing to initiate the HEALEY ALS Platform Trial, a pivotal Phase 2/3 study expected to commence in Q4 2025, contingent on the FDA lifting its current clinical hold on NUZ-001. This next trial phase will be critical in confirming NUZ-001’s efficacy and safety in a larger patient population and advancing regulatory approval efforts.
Implications for the ALS Treatment Landscape
The promising survival data from Neurizon’s OLE study could represent a meaningful breakthrough in ALS therapeutics, a field historically challenged by limited treatment options and modest survival benefits. If these results are replicated in larger, controlled trials, NUZ-001 may set a new benchmark for extending life expectancy and improving quality of life for patients with this devastating neurodegenerative disease.
Bottom Line?
Neurizon’s NUZ-001 is poised to reshape ALS treatment, but upcoming trial results and regulatory decisions will be decisive.
Questions in the middle?
- Will the FDA lift the clinical hold on NUZ-001 in time to start the HEALEY ALS Platform Trial as planned?
- How will NUZ-001’s survival benefits compare in a randomized controlled trial versus historical controls?
- What are the long-term safety implications of continuous NUZ-001 treatment beyond 31 months?
