Ovid Therapeutics (US:OVID) Globenewswireยท2025-10-03 11:00Core Insights - Ovid Therapeutics Inc. announced positive topline results from its Phase 1 study of OV329, a next-generation GABA-aminotransferase inhibitor aimed at treating drug-resistant epilepsies, demonstrating a favorable safety and tolerability profile [1][2][6] Trial Design - The Phase 1 trial involved 68 healthy volunteers, with 51 receiving active treatment and 17 receiving placebo, testing doses from 1 mg to 5 mg [3] - Safety, tolerability, pharmacokinetic, and pharmacodynamic activities were assessed, including rigorous ophthalmic evaluations and extensive exploratory biomarker analyses [3][4] Biomarker and Efficacy Results - OV329 showed significant GABAergic inhibition, with a 53% increase in inhibition at the 5 mg dose as measured on the abductor pollicis brevis muscle [8] - The study confirmed OV329's ability to penetrate the brain and engage its target, achieving biological modulation consistent with elevated GABA levels [4][8] - After 7 days of dosing at 5 mg, mean GABA levels increased by 7.13% in the medial parietal lobe compared to 0.24% in placebo [8] Safety and Tolerability - The safety profile of OV329 was favorable, with all treatment-related adverse events reported as mild and transient, and no evidence of ophthalmic or retinal changes observed [6][9] - Extensive ophthalmic tests showed no adverse effects, contrasting with existing treatments like vigabatrin, which can accumulate in the retina [6] Future Development Plans - Ovid plans to advance OV329 into a Phase 2a study for drug-resistant focal onset seizures, expected to start in Q2 2026 [10] - The company is also progressing its KCC2 direct activator portfolio, with several regulatory and clinical milestones anticipated in the next 12 months [10][11]