Core Insights - Ovid Therapeutics' shares increased by 39.2% following positive top-line data from an early-stage study of OV329, a next-generation GABA-AT inhibitor aimed at treating rare and drug-resistant epilepsies [1][6] - The therapy's mechanism involves blocking GABA-AT to enhance brain GABA levels, potentially positioning it as a best-in-class treatment for seizures [1][6] Study Details - The phase I study of OV329 included 68 healthy volunteers and assessed safety, tolerability, pharmacokinetics, and pharmacodynamics with doses ranging from 1 to 5 mg [2] - Participants received once-daily oral doses, with comprehensive safety and ophthalmic evaluations conducted throughout the study [2] Efficacy Results - OV329 demonstrated strong GABAergic activity, increasing inhibition by 53% on the abductor pollicis brevis (APB) muscle and 44% on the first dorsal interosseous (FDI) muscle at the 5 mg dose, compared to no significant change with placebo [7] - The therapy also significantly prolonged the cortical silent period (CSP) by up to 13 milliseconds at the 5 mg dose, indicating enhanced neural inhibition [8] Biomarker Analysis - Magnetic resonance spectroscopy (MRS) indicated a 7.13% increase in brain GABA levels after seven days of treatment, while placebo showed negligible change [9] - Exploratory EEG data suggested increased inhibitory brainwave activity, supporting OV329's GABA-enhancing mechanism [9] Safety Profile - The phase I study reported that OV329 had a favorable safety and tolerability profile, with treatment-related adverse events primarily mild to moderate [10] Future Plans - Following the encouraging phase I results, Ovid Therapeutics plans to initiate a phase IIa study of OV329 in adults with drug-resistant focal onset seizures in Q2 2026, with completion expected by mid-2027 [11] - The company aims to further define OV329's anti-convulsant profile through an open-label seizure study [11] Additional Pipeline - Ovid Therapeutics is also developing another investigational candidate, OV350, in a separate early-stage study for brain diseases [12]