verubecestat

Verubecestat is an investigational small molecule drug developed as an oral inhibitor of β-site amyloid precursor protein cleaving enzyme 1 (BACE1, also known as β-secretase). By inhibiting BACE1, verubecestat was designed to reduce the production of amyloid-beta (Aβ) peptides, which are implicated in the pathogenesis of Alzheimer's disease. The drug showed dose-dependent reductions in Aβ levels in cerebrospinal fluid during early clinical trials. Despite initial promise and progression to phase 3 clinical trials for Alzheimer's disease and prodromal Alzheimer's disease (amnestic mild cognitive impairment), verubecestat failed to demonstrate efficacy in slowing cognitive decline or functional deterioration compared with placebo. Moreover, it was associated with increased adverse events such as falls, injuries, suicidal ideation, weight loss, sleep disturbances, rash, and hair color changes. All late-stage development for Alzheimer’s disease has been terminated due to lack of efficacy and safety concerns[1][2][3][4][6][7][8].

Other names

1286770-55-5