Neurizon’s NUZ-001 Achieves Blood-Brain Breakthrough, Boosting ALS Therapy Hopes

Key Highlights:

• NUZ-001 and its metabolite shown to significantly penetrate the blood-brain barrier (BBB)
• In vitro studies confirm efficacy in reversing ALS-linked TDP-43 aggregation
• Results validate therapeutic potential for ALS and other neurodegenerative diseases
• CEO Michael Thurn hails findings as “transformative” for disease modification
• Stock last traded at $0.175, up 2.94% on the ASX
  
   

Neurizon Therapeutics Ltd (ASX: NUZ) surged into scientific prominence on Friday with the release of compelling preclinical data that demonstrates its lead drug candidate, NUZ-001, effectively crosses the blood-brain barrier and directly targets pathological protein aggregation linked to ALS.

The study, conducted in rodent models, revealed that both NUZ-001 and its active metabolite NUZ-001 Sulfone reached therapeutic levels in the brain—levels shown in parallel in vitro studies to significantly reverse aggregation of TAR DNA-binding protein 43 (TDP-43), a known pathological hallmark of ALS and other TDP-43-linked neurodegenerative diseases like frontotemporal dementia (FTD), Alzheimer’s disease (AD), and LATE.

In the pharmacokinetic (PK) study, brain concentrations of NUZ-001 ranged from 285 nM to 1,300 nM, while NUZ-001 Sulfone reached 177 nM to 1,231 nM. These concentrations aligned with dose levels shown to reverse TDP-43 aggregation in a human patient-derived iPSC neuronal model of ALS.

Therapeutic Implications

Crossing the blood-brain barrier (BBB) has long posed a formidable challenge in neurodegenerative drug development. Neurizon’s latest results mark a critical milestone by demonstrating CNS exposure sufficient to deliver disease-modifying effects—without requiring invasive administration routes.

The company highlighted that this BBB permeability strengthens the case for NUZ-001 not just in ALS but also in broader TDP-43 proteinopathies. The results also support the efficacy previously seen in the Phase 1 MEND study, which laid the clinical groundwork for advancing NUZ-001 in ALS treatment.

CEO Comments

“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 in vitro,” said Dr. Michael Thurn, Managing Director and CEO of Neurizon Therapeutics.

“This new data provides strong translational validation for our mechanism and reinforces the potential of NUZ-001 as a transformative disease-modifying therapy for ALS. With these findings, we are more determined than ever to deliver a therapy that precisely targets the underlying pathology driving this devastating disease,” Dr. Thurn added.

Scientific Rigor and Reproducibility

Importantly, the TDP-43 aggregation assay results published in this announcement build upon Neurizon’s previous findings from November 2024, with the updated study extending the dose-response range and reaffirming efficacy across all tested concentrations (270–4,400 nM).

NUZ-001’s ability to reduce TDP-43 aggregation in M337V motor neurons under stress (induced by proteasomal inhibitor MG-132) was statistically significant at all doses—underlining the robustness of the compound’s therapeutic action.

Investor Outlook

Neurizon’s update marks a substantial value-inflection point for the company, validating its development strategy and opening potential for expanded indications. The company has also rolled out an Investor Hub platform for real-time updates, Q&A, and announcement digests.

At the time of writing this article, Neurizon shares traded at $0.175, up 2.94%, with a 1-year return of -22.22% and a 52-week trading range of $0.096–$0.245.