Ovid Therapeutics (OVID) investor relations material

Strategic focus and pipeline overview

• Focuses on small molecules targeting universal biological mechanisms in the brain to address neural hyperexcitability across multiple neurological and neuropsychiatric disorders.

• Prioritizes differentiated mechanisms of action to avoid competition and offer novel solutions for conditions like epilepsy and psychosis.

• By year-end, expects five clinical programs with four to five milestones in the next 12–18 months.

OV329: Epilepsy program highlights

• OV329, a next-generation GABA aminotransferase inhibitor, aims to improve safety and efficacy over vigabatrin, with a much wider therapeutic index and better tolerability.

• Phase II trial underway, with additional biomarker and photosensitivity studies to de-risk development; key readouts expected within 6–12 months.

• Targeting focal onset seizures, tuberous sclerosis complex, and infantile spasms, with potential for broader application in neurodevelopmental disorders and pain.

• Market opportunity estimated at $1.75–2 billion, with potential to double historical sales in pediatric epilepsies.

• Phase I data confirmed brain target engagement, elevated GABA levels, and strong biomarker correlation, supporting dose selection for later trials.

KCC2 direct activators: Neuropsychiatric innovation

• KCC2 direct activators, including OV4071, represent a novel approach with broad potential in neurodegenerative and neuropsychiatric diseases.

• OV4071 shows robust preclinical efficacy in models of schizophrenia, psychosis, and cognitive impairment, with phase I and electrophysiology studies ongoing.

• Plans to initiate phase II proof-of-concept study in schizophrenia by mid-2025, with potential expansion to Alzheimer's psychosis, Lewy body dementia, and Parkinson's disease psychosis.