Ovid Therapeutics (OVID) Status Update summary

Strategic focus and pipeline overview

• OV329 is a next-generation GABA aminotransferase inhibitor designed to address limitations of first-generation GABA-AT inhibitors, offering improved potency, safety, and suitability for polypharmacy regimens in drug-resistant epilepsy.

• The pipeline includes additional programs such as OV350, OV4071, and OV4041, targeting various neurological indications.

• OV329's mechanism is validated, with potential applications beyond seizures, including pain, substance abuse, and developmental epileptic encephalopathies.

• Emphasis on targeting neural hyperexcitation and excess neural excitation with highly selective small molecules and differentiated mechanisms of action.

• The initial indication is drug-resistant epilepsy, a significant unmet need affecting over 1.2 million adults in the US and 30%-40% of patients who fail existing therapies.

Biomarker-driven clinical development

• The phase I study incorporates a comprehensive biomarker strategy using MRS, TMS, and EEG to assess target engagement, pharmacodynamic effects, and GABAergic activity.

• Biomarkers are selected to differentiate OV329 from other anti-seizure drugs and to benchmark effects against vigabatrin.

• Multiple metrics across these tools provide robust evidence of drug action, reducing reliance on single measures and enhancing confidence in results.

• Targeted outcomes include increased GABA (MRS), enhanced cortical inhibition (TMS), and increased beta/gamma EEG power, mirroring effects seen with vigabatrin but with improved safety.

Safety and trial progress

• Phase I results, expected in Q3 2025, will include safety, tolerability, pharmacokinetics, and biomarker data.

• Extensive ophthalmologic monitoring is in place to address historical concerns with visual side effects from GABA-AT inhibitors; no visual changes or ocular findings have been observed to date.

• The most common adverse event is mild headache, with no serious adverse events or retinal accumulation reported.

• Phase II-A in drug-resistant focal onset seizures is set to begin in Q1 2026, with results anticipated in early 2027.