Ovid Therapeutics (OVID) 24th Annual Needham Virtual Healthcare Conference summary

Strategic focus and differentiation

• Focused on precision small molecules targeting neural hyperexcitability in CNS disorders, aiming for differentiated mechanisms of action to avoid crowded therapeutic areas.

• Pipeline includes programs targeting neurotransmitters and ion channels, with a strategy to avoid direct competition in future markets.

• Cash runway extends into the second half of 2026, supporting multiple upcoming milestones.

OV329 program highlights

• OV329 is a next-generation GABA-aminotransferase inhibitor for treatment-resistant epilepsies, designed to avoid the ocular toxicity seen in prior drugs like vigabatrin.

• Phase I readout expected in Q3 2024, with phase II trials planned for early 2026 targeting both developmental and adult epilepsies.

• OV329 demonstrates high potency, durable inhibition, and a favorable safety profile with no observed ocular adverse events to date.

• Biomarker-driven approach using TMS and MRS to assess pharmacodynamic effects and GABAergic activity.

• Broad dosing flexibility and robust therapeutic index, with additional high-dose cohorts added due to clean safety data.

KCC2 direct activator portfolio

• First company to dose direct activators of KCC2 in humans, with a portfolio exceeding 100 unique compounds.

• OV350 (IV) and OV4071 (oral) are advancing, targeting psychosis in Parkinson’s disease and Lewy body dementia, with phase I data for OV350 expected by end of 2025.

• KCC2 activators show antipsychotic, anticonvulsant, and anxiolytic properties, with rapid onset and dose-dependent effects in preclinical models.

• Market opportunity for psychosis in Parkinson’s and Lewy body dementia exceeds $2.5 billion with significant unmet need.