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Drug intelligence / Profile preview
OV350 (also known as Cmp2) is a first-in-class, brain-penetrant small molecule direct activator of the potassium-chloride cotransporter 2 (KCC2; SLC12A5). Developed originally by AstraZeneca and licensed to Ovid Therapeutics, OV350 directly binds to and enhances the activity of KCC2, a neuron-specific chloride transporter that maintains inhibitory balance in the brain by enabling GABA to exert its hyperpolarizing effect. Preclinical studies demonstrated that OV350 reduces neuronal chloride accumulation, limits hyperexcitability, and restores the efficacy of benzodiazepines in models of refractory status epilepticus, Rett syndrome, and CDKL5 deficiency disorder. Although Ovid successfully completed a Phase 1 clinical trial of intravenous OV350 in healthy volunteers, the company decided in December 2025 to discontinue further development of this intravenous tool program to focus resources on its more potent oral KCC2 direct activator, OV4071.
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