Ovid Therapeutics’ OV329 Shows Promising Safety and Biomarker Activity in Phase I Trial for Drug-Resistant Epilepsy

Ovid Therapeutics announced positive topline results from its Phase I trial of OV329, a next-generation GABA-aminotransferase (GABA-AT) inhibitor aimed at drug-resistant epilepsy. The study evaluated pharmacokinetics, pharmacodynamics, safety, and tolerability in 68 healthy volunteers, with 51 receiving OV329 and 17 on placebo. Doses ranged from 1 mg to 5 mg in both single and multiple ascending dose cohorts.

Extensive biomarker analyses were conducted in the 3 mg and 5 mg multiple-dose groups using transcranial magnetic stimulation, electromyography, EEG, and magnetic resonance spectroscopy to assess inhibitory activity and target engagement. OV329 demonstrated favourable tolerability, a clean ocular and overall safety profile, and all treatment-related adverse events were mild and transient.

Building on these results, Ovid plans a Phase IIa randomized placebo-controlled trial in adults with drug-resistant focal onset seizures, expected to start in Q2 2026 and complete by mid-2027, aiming to confirm efficacy and safety in the target patient population.

06-10-2025