Ovid Therapeutics (OVID) Study Result summary

Pipeline Overview and Unmet Need

• OV329 is a next-generation GABA aminotransferase inhibitor targeting drug-resistant epilepsies, aiming to address limitations of current GABA-modulating drugs, such as vigabatrin's ocular toxicity and high dosing requirements.

• Focus on novel CNS targets for neural hyperexcitability, including KCC2 activators for other neuropsychiatric conditions.

• Pipeline includes IV and oral KCC2 activators, with multiple clinical milestones expected through 2027.

• Significant unmet need remains in epilepsy, with many patients experiencing uncontrolled seizures despite polypharmacy.

Study Design and Objectives

• Phase 1 trial evaluated OV329 in 68–69 healthy volunteers across single and multiple ascending dose cohorts, with doses ranging from 1 mg to 5 mg.

• Safety, tolerability, pharmacokinetics, and pharmacodynamics were assessed, including rigorous ophthalmic evaluations.

• Extensive biomarker analyses used TMS, MRS, and EEG to measure GABAergic inhibition and brain activity.

• OV329 was compared to vigabatrin, a first-generation GABA-AT inhibitor, using established TMS biomarkers.

Safety and Tolerability Results

• OV329 demonstrated a strong safety profile with no treatment-related serious adverse events and only mild, transient AEs such as headache, drowsiness, and metallic taste.

• No ophthalmic safety findings or retinal changes were observed, supported by extensive ophthalmic testing.

• Most frequent AE was cannula site reaction, unrelated to the drug.

• Preclinical studies showed no accumulation in the eye or retina, differentiating OV329 from vigabatrin.