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Neurizon Therapeutics confirms potential of NUZ-001 as platform therapy for ALS and Huntington’s disease

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By Colin Hay - 
Neurizon Therapeutics ASX NUZ Sulfone blood barrier ALS
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Clinical-stage biotechnology company Neurizon Therapeutics (ASX: NUZ) has continued its success in proving the versatility of lead drug candidate NUZ-001.

New studies have confirmed NUZ-001’s potential to provide significant blood–brain barrier penetration.

Rodent preclinical pharmacokinetic (PK) data have found that NUZ-001 and its major active metabolite NUZ-001 sulfone effectively cross the blood–brain barrier (BBB) and achieve brain concentrations similar to those shown to reverse pathological TAR DNA-binding protein 43 (TDP-43) aggregation in patient-derived induced pluripotent stem cells.

Major barrier

Chief executive officer Dr Michael Thurn said the ability of therapeutics to access the central nervous system remains the major barrier in treating neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS).

“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,” Dr Thurn said.

“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.”

The company believes the results support broader applications including for frontotemporal dementia, Alzheimer’s disease and limbic predominant age-related TDP-43 encephalopathy.

HD trial progress

Neurizon reported earlier this month that new preclinical data had demonstrated significant neuroprotective effects of NUZ-001 sulfone in a zebrafish model of Huntington’s disease (HD).

HD is a rare, inherited neurodegenerative disorder that causes progressive degeneration of motor function, cognition and mental health affecting between 2.7 and 4.8 per 100,000 people globally.

“There is no cure [for HD] and no disease-modifying treatments, only treatments that manage symptoms,” Dr Thurn said.

“These exciting results demonstrate NUZ-001 has consistent neuroprotective effects beyond ALS, strengthening our conviction in its potential as a disease-modifying platform therapy across a range of neurodegenerative conditions.”