Summary of Ovid Therapeutics Conference Call Company Overview - **Company**: Ovid Therapeutics (NasdaqGS:OVID) - **Focus**: Development of neurotherapeutics targeting conditions driven by neural hyperexcitability, particularly in epilepsy Key Points and Arguments Pipeline and Product Development - Ovid is advancing a pipeline that includes: - **OV329**: A next-generation GABA aminotransferase (GABA AT) inhibitor for treatment-resistant epilepsies and focal onset seizures - **KCC2 Direct Activators**: Including OV350 (IV) and OV4071 (oral), targeting potassium chloride cotransporter 2 - **Phase 1 Results**: OV329 has completed Phase 1 safety and biomarker studies, with plans to initiate Phase 2a in Q2 2026 and report top-line data in mid-2027 [8][10][42] Market Need and Competitive Landscape - There is a significant unmet need in epilepsy treatment, with only two novel mechanisms approved in the last 30 years despite 30 anti-seizure medications being available [10][11] - Approximately 40% of epilepsy patients are treatment-resistant, representing a market opportunity exceeding $1 billion [44] Safety and Efficacy of OV329 - **Safety Profile**: OV329 demonstrated a favorable safety profile with no serious adverse events and only mild, transient side effects [24][41] - **Efficacy Metrics**: Positive biomarker data indicates OV329 effectively inhibits GABA AT, with a potential for superior efficacy compared to existing treatments like vigabatrin [14][20][41] - **Dosing**: Expected dosing for OV329 is significantly lower than vigabatrin, with estimates of 5-7 mg compared to 2-3 grams for vigabatrin [20][21] Mechanism of Action - OV329 provides both phasic and tonic inhibition of GABA, which may lead to better tolerability and efficacy compared to previous GABAergic medications [15][16][64] - The drug's design aims to avoid the ocular safety issues associated with vigabatrin, which has been linked to vision loss [19][21] Phase 2a Trial Design - The upcoming Phase 2a trial will evaluate a 7 mg dose of OV329 against placebo over eight weeks, focusing on treatment-resistant focal onset seizures [43][44] - The trial will measure various efficacy outcomes, including seizure frequency and responder rates [43] Future Development Plans - Ovid plans to continue developing its KCC2 portfolio, with expected readouts for OV350 by the end of 2025 and initiation of Phase 1 studies for OV4071 in 2026 [45][46] - The company has sufficient resources to fund its development programs through 2028 [45] Additional Important Information - The call emphasized the importance of differentiating OV329 from existing treatments through its unique mechanism and safety profile [54][64] - The company is committed to building a robust safety database to support the development of OV329 and mitigate monitoring requirements for patients [27][41] - The anticipated market dynamics and reimbursement landscape are favorable for OV329, despite the competitive nature of the epilepsy treatment market [44]