Ovid Therapeutics (OVID) investor relations material

Strategic vision and opportunity

• Advancing a novel class of CNS medicines targeting KCC2, a master regulator of neural excitation/inhibition balance, with a focus on broad syndromic psychosis and neural hyperexcitability conditions.

• Lead oral molecule OV4071 is positioned for development in schizophrenia, Parkinson’s disease psychosis, Lewy body dementia, and other large indications.

• Multiple proof-of-concept and clinical studies are planned, including phase II in schizophrenia and exploratory studies in neurodegenerative psychoses, with milestones anticipated through 2026–2027.

• Commercial opportunity is estimated at over $6 billion for initial indications, with peak revenue projected at $6.8B in 2038.

• The company aims to introduce at least one new development candidate annually from its discovery engine.

Pipeline and clinical development

• OV4071 is advancing through Phase 1, with proof-of-mechanism ketamine studies and Phase 1b/2 PoC studies planned for 2026–2027.

• OV329 targets drug-resistant focal onset seizures, tuberous sclerosis complex, and infantile spasms, with Phase 2 trials and topline results expected by mid-2027.

• Additional undisclosed KCC2 activators are in preclinical development.

• OV4071 is being developed as a monotherapy, but combination therapy remains a future option.

• Strong IP protection extends to 2046, with a pipeline of next-generation KCC2 activators in development.

Scientific rationale and preclinical evidence

• KCC2 is uniquely expressed in the CNS, regulates chloride extrusion, restores GABAergic inhibition, and neural network stability.

• Direct KCC2 activation cannot be over-modulated, offering a favorable safety profile compared to neurotransmitter-based drugs.

• Preclinical models show OV4071 restores E/I balance, improves cognition, and outperforms existing antipsychotics in working memory and task switching.

• OV4071 demonstrates rapid, dose-dependent antipsychotic effects, restoring social interaction and cognitive function in preclinical models.

• Efficacy demonstrated across >25 pharmacodynamic models, including genetic and stimulant-induced psychosis, with consistent results.