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AZD3839 is a potent and selective small molecule inhibitor of β-site amyloid precursor protein cleaving enzyme 1 (BACE1), with approximately 14-fold selectivity over BACE2. BACE1 is a key enzyme involved in the processing of amyloid precursor protein (APP) and the formation of amyloid β peptide (Aβ), which accumulates in the brains of patients with Alzheimer's disease. By inhibiting BACE1, AZD3839 reduces Aβ production in vitro and lowers Aβ levels in plasma, brain, and cerebrospinal fluid across several preclinical species. The drug was developed using fragment-based screening and structure-based design approaches. It advanced to Phase 1 clinical trials for Alzheimer's disease but further development has been discontinued[1][4][5][6].
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