SPG601

SPG601 is an investigational, orally administered small molecule developed by Spinogenix for the treatment of Fragile X syndrome (FXS), a leading inherited cause of intellectual disability and autism. The drug acts as an activator of large-conductance, calcium-activated potassium channels (BK channels), aiming to restore synaptic function by increasing BK channel activation. This mechanism targets a well-established molecular dysfunction in FXS, with the goal of improving core symptoms such as severe anxiety, social aversion, hyperactivity and attention deficit, sensory hypersensitivity, aggression, and developmental seizures. In Phase 2 clinical trials in adult men with FXS, SPG601 demonstrated a strong efficacy signal by significantly reducing high-frequency gamma band activity on EEG—a marker associated with learning and memory deficits in FXS. The FDA has granted both Orphan Drug and Fast Track designations to SPG601 for this indication[1][2][4][5][6][7][8][10].

Other names

(R) 3-(5-Dimethylcarbamoyl-pent-1-enyl)-N-(2-hydroxy-1-methylethyl) benzamide