Efficacy and Safety of Brexpiprazole for the Treatment of Agitation in Alzheimer's Dementia: Two 12-Week, Randomized, Double-Blind, Placebo-Controlled Trials.

•These two randomized controlled trials addressed the question, is brexpiprazole efficacious, safe, and well tolerated for the treatment of agitation in Alzheimer's dementia (AAD)?•In Study 1, brexpiprazole 2 mg/day (fixed dose) was superior to placebo in terms of improvement of agitated behaviors (measured using the Cohen-Mansfield Agitation Inventory), with good tolerability. In Study 2, brexpiprazole 0.5–2 mg/day (flexible dose) was numerically superior to placebo (a non-statistically significant difference); the subgroup that was titrated to brexpiprazole 2 mg/day achieved nominal superiority compared with similarly titrated placebo patients.•These studies provide evidence that brexpiprazole 2 mg/day has the potential to be an efficacious, safe, and well-tolerated treatment for AAD, a clinical condition with a high burden and no FDA-approved treatments.