AXS-05: an investigational treatment for Alzheimer’s disease-associated agitation

INTRODUCTION: Agitation is common in patients with Alzheimer's disease (AD). Although nonpharmacologic de-escalation strategies are recommended as first-line treatment, medication is often needed to treat agitation. Currently, there are no FDA-approved medications for this indication. Psychotropics used to treat agitation include antipsychotics, which are notable for their efficacy but also their potential to cause serious side effects. AXS-05, a combination of dextromethorphan and bupropion, is currently being investigated for this indication. AREAS COVERED: This review will discuss the pharmacology of AXS-05 and available clinical trial results from completed Phase I and Phase II/III studies assessing the potential for this compound to treat agitation in patients with AD. Ongoing research investigating AXS-05 for this indication will also be highlighted. Resources used for this review include PubMed, Embase, clinicaltrials.gov, and literature available on the manufacturer's website. EXPERT OPINION: Early released clinical trial data indicate that AXS-05 may be a useful option to treat agitation in patients with AD and that it appears to be generally well tolerated. AXS-05 may be especially helpful for patients with comorbid depression, when considering available data from separate phase III studies assessing the efficacy and safety of this compound in the treatment of depression.