NUZ-001 sulfone

NUZ-001 sulfone is the major active metabolite of the small molecule drug candidate NUZ-001, developed by Neurizon Therapeutics for neurodegenerative diseases. It has demonstrated significant neuroprotective effects in preclinical models of amyotrophic lateral sclerosis (ALS) and Huntington’s disease. Mechanistically, both NUZ-001 and its sulfone metabolite reduce aggregation of TAR DNA-binding protein 43 (TDP-43), a key driver in ALS pathology, and restore normal electrophysiological function in TDP-43 mutant motor neurons. The compound also protects against ferroptosis—a form of programmed cell death implicated in several neurodegenerative disorders—and promotes neural branching and plasticity. Preclinical pharmacokinetic studies show that both compounds effectively cross the blood–brain barrier at concentrations sufficient to reverse pathological TDP-43 aggregation. In zebrafish models of Huntington’s disease, treatment with NUZ-001 sulfone prevented hallmark developmental abnormalities, protected against neuronal cell death, restored delayed haemoglobin production, and rescued BDNF expression[1][4][5][7].

Other names

major active metabolite of NUZ-001