Vafidemstat (ORY-2001) is a selective, brain-penetrant inhibitor of lysine-specific demethylase 1 (LSD1), targeting epigenetic mechanisms to modulate gene expression in central nervous system (CNS) disorders. It is under clinical investigation primarily for Alzheimer's disease (to improve cognition and reduce agitation), aggression in psychiatric disorders such as borderline personality disorder, and potentially multiple sclerosis. By inhibiting LSD1, it aims to reduce neuroinflammation, enhance neuroplasticity, and alleviate cognitive, behavioral, and emotional symptoms, providing a novel therapeutic approach for complex neurological and psychiatric conditions.