Neurizon Therapeutics Limited (ASX:NUZ) Reports New Preclinical Data Supporting ALS Treatment
Preclinical Pharmacokinetic Results
Neurizon Therapeutics has revealed new rodent preclinical pharmacokinetic data showing that NUZ-001 and its major metabolite, NUZ-001 Sulfone, effectively cross the blood-brain barrier, achieving brain concentrations consistent with reversing pathological TDP-43 aggregation in ALS models.
TDP-43 Aggregation Assay
In a repeat TDP-43 aggregation assay using a patient-derived iPSC neuronal model of ALS, both compounds significantly prevented TDP-43 aggregation at all tested concentrations, aligning with CNS exposure achieved in the rodent PK study.
Executive Comments
Dr Michael Thurn, Managing Director and Chief Executive Officer, stated, “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. 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, supporting the accelerated advancement of NUZ-001.”
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