Neurizon Therapeutics (ASX:NUZ) said its treatment candidate, NUZ-001, and major active metabolite NUZ-001 sulfone showed in-vivo biological activity, based on data from ongoing pre-clinical studies, according to a Thursday Australian bourse filing.

The company is evaluating NUZ-001 in various Huntington's disease models and showed disease-dependent restoration of brain-derived neurotrophic factor levels and enhanced autophagic markers, among others, the filing said.

Huntington's disease is a fatal neurodegenerative disorder caused by a mutation in the HTT gene, which produces a toxic form of the huntingtin protein that damages neurons.

Drug exposure studies confirmed that both NUZ-001 and NUZ-001 sulfone were readily taken up in zebrafish, with both compounds demonstrating biological activity in vivo, per the filing. The restorative effects of NUZ-001 and NUZ-001 sulfone on brain-derived neurotrophic factor levels were shown to be selective to the HTT knockdown condition and were not observed in wild-type animals.