Ovid Therapeutics (OVID) investor relations material

Strategic focus and pipeline overview

• Developing small molecule medicines targeting neural hyperexcitability, aiming for broad therapeutic utility in neurological disorders.

• Pipeline includes OV329 (GABA-AT inhibitor) and OV4071 (KCC2 direct activator), with multiple clinical milestones expected through 2026–2027.

• Differentiated mechanisms of action designed to address unmet needs in drug-resistant seizures, psychosis, and neurodevelopmental disorders.

OV329: Next-generation GABA-AT inhibitor

• Precision inhibition for drug-resistant epilepsies and developmental epileptic encephalopathies, with a favorable safety and tolerability profile.

• Demonstrated dose-dependent GABA elevation and cortical inhibition, exceeding effects of first-generation vigabatrin without retinal accumulation.

• No treatment-related serious adverse events or ophthalmic changes observed in clinical studies.

• Targeting adult focal onset seizures, tuberous sclerosis complex, and infantile spasms, with significant commercial potential and anticipated earlier-line use.

OV4071: KCC2 direct activator for neuropsychiatric disorders

• Targets the KCC2 transporter to restore excitatory/inhibitory balance in the CNS, with broad applications in psychosis, neurodegeneration, and neurodevelopmental conditions.

• Demonstrated robust, dose-dependent antipsychotic effects in preclinical models, restoring social interaction and normalizing EEG biomarkers.

• Phase 1 studies in healthy volunteers and elderly, with parallel ketamine challenge to assess target engagement and electrophysiology biomarkers.

• Phase 2 proof-of-concept studies planned for schizophrenia, Parkinson’s disease psychosis, and Lewy body dementia psychosis.