Positive preclinical data supports potential of Neurizon’s NUZ-001 in Huntington’s disease

Neurizon Therapeutics, which is dedicated to advancing treatments for neurodegenerative diseases, has rolled out new preclinical data demonstrating significant neuroprotective effects of NUZ-001 and its active metabolite NUZ-001 Sulfone, in a zebrafish model of Huntington’s disease.

Clinical-stage biotech Neurizon Therapeutics has reached a milestone in development of its lead drug candidate NUZ-001 to treat Huntington’s disease. 

Huntington’s disease is a rare, inherited neurodegenerative disorder that causes progressive degeneration of motor function, cognition and mental health. The disease affects between 2.7 and 4.8 per 100,000 people globally with no cure and no disease-modifying treatment. The treatments available only manage symptoms.

Neurizon plans to initiate additional validation studies in mammalian models of Huntington’s.

In the Huntington’s disease model, targeted mRNA knockdown of the Htt (huntingtin) protein triggered hallmark disease characteristics, including:

·      Increased cell death

·      Morphological malformations (smaller eyes and swollen hindbrains)

·      Impaired haemoglobin production and

·      Reduced expression of brain-derived neurotrophic factor (BDNF), a critical biomarker of neuronal function and survival.

mRNA knockdown is a lab technique that reduces the activity of a specific gene, in this case the HTT gene, which produces the protein involved in Huntington’s disease.

Treatment with either NUZ-001 or NUZ-001 Sulfone after Htt knockdown:

·      Prevented developmental and morphological abnormalities

·      Attenuated neuronal cell death

·      Restored the delayed production of haemoglobin; and

·      Rescued BDNF expression.

Neurizon said the results provided evidence of NUZ-001 and NUZ-001 Sulfone’s potential to counteract early neurodegenerative damage.