ASN120290

ASN120290 is an orally bioavailable, small molecule drug developed by Asceneuron for the treatment of progressive supranuclear palsy (PSP) and other tauopathies. It acts as a potent and selective inhibitor of the O-linked N-acetylglucosaminidase enzyme, which leads to increased O-GlcNAcylation of tau protein, thereby reducing the formation of toxic tau aggregates and neurofibrillary tangles in the brain. These tangles are key drivers in PSP and other neurodegenerative diseases. Preclinical studies have shown that ASN120290 can significantly reduce pathological tau accumulation, while Phase 1 clinical trials demonstrated good safety and tolerability in healthy volunteers. The drug has received orphan drug designation from the FDA for PSP[1][2][5][6].

Other names

(s)-n-(5-(4-(1-(benzo[d][1,3]dioxol-5-yl)ethyl)piperazin-1-yl)-1,3,4-thiadiazol-2-yl)acetamide hydrochloride(S)-N-(5-(4-(1-(benzo[d][1,3]dioxol-5-yl)ethyl)piperazin-1-yl)-1,3,4-thiadiazol-2-yl)acetamide