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Cleavage of amyloid precursor protein by beta-site amyloid precursor protein-cleaving enzyme 1 (BACE-1) leads to amyloid-beta (Aβ) production in Alzheimer disease (AD). To investigate the safety and efficacy of verubecestat, an investigational oral BACE-1 inhibitor, researchers conducted a randomized, double-blind, placebo-controlled, manufacturer-funded, phase III clinical trial. Participants were randomized to 12 mg verubecestat (n=653), 40 mg verubecestat (n=652), or placebo (n=653) daily for 78 weeks. Primary outcomes included scores on the ADAS-Cog and the ADCS-ADL scales. AD biomarker measurements included cerebrospinal fluid (CSF) tau and amyloid-beta levels and amyloid load based on positron emission tomography imaging. At baseline,…