GXV-001

GXV-001 is an investigational oral small molecule developed by GEXVal, a spinoff of Takeda Pharmaceutical. It acts as a modulator of G protein-coupled receptors (GPCRs) and is being developed for the treatment of neurodevelopmental disorders, including Fragile X syndrome (FXS), Rett syndrome, and Angelman syndrome. The compound has completed a Phase 1 randomized, double-blind, placebo-controlled ascending-dose study in healthy adult volunteers, which established its safety, tolerability, and pharmacokinetic profile. GXV-001 has received FDA Orphan Drug Designation for Fragile X syndrome and has completed the FRAXA Drug Validation Initiative (FRAXA-DVI) preclinical program. It is currently advancing toward Phase 2a clinical trials in Europe.