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Elenbecestat is an orally available, small-molecule inhibitor of beta-site amyloid precursor protein cleaving enzyme 1 (BACE1), also known as β-secretase. It was developed to interfere with the amyloid cascade by inhibiting BACE1, thereby reducing the production of amyloid-beta (Aβ) peptides implicated in Alzheimer's disease pathology. Elenbecestat demonstrated selectivity for BACE1 over BACE2 and showed a reduction in Aβ levels in preclinical and clinical studies. The drug was primarily developed for the treatment of early Alzheimer's disease, including mild cognitive impairment due to Alzheimer's and early-stage mild Alzheimer’s dementia. Clinical development reached Phase III before being discontinued due to an unfavorable risk-benefit profile[2][4][5].
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