Neurizon Therapeutics (ASX: NUZ) said high levels of its drug candidate NUZ-001 and its major active Sulfone metabolite crossed the blood-brain barrier in a rodent preclinical pharmacokinetic study, according to a Friday Australian bourse filing.
They achieved brain concentrations consistent with those shown to reverse pathological transactive response DNA-binding protein 43 aggregation in patient-derived induced pluripotent stem cells.
The ability of therapeutics to access the central nervous system remains a major barrier in the treatment of neurodegenerative diseases such as amyotrophic lateral sclerosis. Transactive response DNA-binding protein 43 is a known driver of amyotrophic lateral sclerosis pathology.
NUZ-001 and its sulfone metabolite significantly prevented the aggregation of transactive response DNA-binding protein 43 in M337V motor neurons treated simultaneously with the proteasomal stressor at all concentrations tested.
The firm's shares fell 3% on market close.
Disclaimer: Investing carries risk. This is not financial advice. The above content should not be regarded as an offer, recommendation, or solicitation on acquiring or disposing of any financial products, any associated discussions, comments, or posts by author or other users should not be considered as such either. It is solely for general information purpose only, which does not consider your own investment objectives, financial situations or needs. TTM assumes no responsibility or warranty for the accuracy and completeness of the information, investors should do their own research and may seek professional advice before investing.