Neurizon confirms positive results for prospective ALS treatment
Ella Day | June 23, 2025 | News story | Research and Development | Neurizon Therapeutics, Neurology, amyotrophic lateral sclerosis, blood-brain barrier, clinical data, neurodegenerative diseases
Neurizon Therapeutics has announced new preclinical data confirming that NUZ-001 and its active metabolite, NUZ-001 Sulfone, effectively cross the blood-brain barrier (BBB) and reach concentrations in the brain sufficient to reverse toxic TDP-43 protein aggregation – a key driver of neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS).
In a rodent pharmacokinetic study, both compounds achieved brain concentrations that closely matched the levels shown to significantly reduce TDP-43 aggregation in patient-derived neurons in vitro. The data provides critical translational validation for NUZ-001’s mechanism of action, reinforcing its therapeutic potential not only in ALS, but also in other TDP-43 proteinopathies such as frontotemporal dementia, Alzheimer’s disease, and limbic predominant age-related TDP-43 encephalopathy.
“These excellent results provide compelling evidence that NUZ-001 and its Sulfone metabolite not only effectively penetrate the BBB but do so at concentrations that are proven to reverse pathological TDP-43 aggregation,” said Michael Thurn, CEO of Neurizon. “We’re more determined than ever to deliver a disease-modifying therapy for ALS.”
TDP-43 protein misfolding and accumulation is characteristic of ALS and related neurodegenerative diseases, disrupting neuronal function and leading to cell death. In a repeated TDP-43 aggregation assay, NUZ-001 and its metabolite significantly inhibited aggregation at all tested concentrations.
Following promising phase 1 data and this new pharmacokinetic confirmation, NUZ-001 is now positioned to progress through clinical development as a targeted therapy for ALS and other neurodegenerative diseases.
Ella Day
23/6/25
Related Content
FDA decision on Neurizon’s ALS therapy delayed until October
Neurizon Therapeutics has announced that the US Food and Drug Administration (FDA) will delay its …
PLL Therapeutics to present ALS research strategy at Bioshares Biotech Summit
French biotech, PLL Therapeutics, will present its approach to early detection and treatment of Amyotrophic …
Sanofi completes acquisition of Vigil Neuroscience to early neurology pipeline
Sanofi has announced that it has finalised its acquisition of Vigil Neuroscience, a US-based biotechnology …
