Ovid Therapeutics Reports Phase 1 Results for the First-Ever Direct Activator of Potassium-Chloride Cotransporter 2 (KCC2), OV350 Intravenous (IV)

Core Viewpoint - Ovid Therapeutics Inc. announced positive results from its Phase 1 study of OV350, the first KCC2 direct activator tested in humans, which supports the advancement of its portfolio of oral KCC2 direct activators into clinical development [1][2]. Study Design and Key Results - The Phase 1 study was a randomized, placebo-controlled, single-ascending dose trial involving 16 healthy participants, with doses of 50 mg and 100 mg administered via IV infusion [3]. - Key findings indicated that OV350 demonstrated a good safety profile, with no treatment-related laboratory findings or serious adverse events [6][7]. - Pharmacokinetics for OV350 were as predicted, informing future dosing strategies for KCC2 development programs [6][7]. Future Development Plans - Ovid plans to submit a regulatory application for a Phase 1/1b study of OV4071, the first oral KCC2 direct activator, in Q1 2026, with clinical studies expected to begin in Q2 2026 [2][9]. - OV4071 is noted to be twenty-fold more potent than OV350 in pharmacodynamic models, targeting conditions such as psychosis associated with Parkinson's disease and Lewy body dementia [9]. KCC2 Direct Activators Portfolio - Ovid is advancing a portfolio of KCC2 direct activators, with OV4071 being the most advanced candidate, aimed at restoring excitatory/inhibitory balance in the brain [11][12]. - The company is also developing next-generation KCC2 activators from its proprietary library, designed for both oral and injectable formulations [10].