ceperognastat

Ceperognastat is an orally available, highly potent small molecule inhibitor of the enzyme O‑GlcNAcase (OGA), designed to be central nervous system (CNS) penetrant. Its mechanism of action is inhibition of OGA, which increases O‑GlcNAc modification on tau protein and other proteins in the brain. This modification is hypothesized to reduce the formation of pathological tau tangles associated with Alzheimer's disease and other tauopathies. Ceperognastat demonstrated high (>80–95%) brain enzyme occupancy at low doses in both preclinical and clinical studies. It was developed by Eli Lilly for the treatment of Alzheimer's disease but failed to meet its primary endpoint in a Phase 2 trial and development has been discontinued[1][2][5][6][7].

Other names

N-[4-fluoro-5-[[2-methyl-4-[(5-methyl-1,2,4-oxadiazol-3-yl)methoxy]piperidin-1-yl]methyl]-1,3-thiazol-2-yl]acetamide