AXS-05 is being developed as a novel oral treatment for multiple Central Nervous System (CNS) conditions, offering a differentiated approach through its multimodal pharmacological activity. The investigational drug combines a proprietary formulation and dose of dextromethorphan and bupropion to target key neurotransmitter pathways involved in CNS disorders.

The dextromethorphan component of AXS-05 acts as an antagonist of the N-methyl-D-aspartate (NMDA) receptor—an ionotropic glutamate receptor—and as an agonist of the sigma-1 receptor. Through these actions, dextromethorphan is believed to modulate glutamatergic neurotransmission, a pathway implicated in several CNS disorders.

Bupropion, the second component of AXS-05, primarily serves to increase the bioavailability of dextromethorphan by inhibiting its metabolism. In addition, bupropion functions as a norepinephrine and dopamine reuptake inhibitor, contributing further to the drug’s multimodal pharmacological profile.

AXS-05 is currently under clinical development for the treatment of agitation associated with Alzheimer’s disease and for smoking cessation. The programme has received Breakthrough Therapy Designation (BTD) from the US Food and Drug Administration (FDA) for Alzheimer’s disease agitation, underscoring the unmet medical need and the therapy’s potential clinical benefit.

The safety and effectiveness of AXS-05 in Alzheimer’s disease agitation and smoking cessation have not yet been established, and the drug is not currently approved by the FDA for these indications.