Ovid Therapeutics (OVID) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Pipeline and clinical development updates

• Advancing a GABA aminotransferase inhibitor and a KCC2 direct activator, with four to six proof-of-concept and signal readouts expected over the next 6–24 months across epilepsy and KCC2 programs.

• Phase I study underway for the first-ever KCC2 activator, with a concurrent ketamine challenge to assess translational biomarkers and neuroactivity, aiming for early validation in humans.

• OV4071, the lead KCC2 activator, is in phase I with data expected early next year, including results from elderly cohorts and ketamine challenge studies.

• OV329, a next-generation GABA-AT inhibitor, is being evaluated in focal-onset seizures and rare pediatric epilepsies, with phase II and photosensitivity studies ongoing.

• Pediatric epilepsy programs are expanding into tuberous sclerosis complex and infantile spasms, with a granule and sachet formulation launching in a TSC study before year-end.

Scientific rationale and differentiation

• KCC2 is a neuron-specific ion transporter critical for GABAergic inhibition; dysfunction is implicated in multiple CNS diseases, including psychoses and epilepsy.

• Direct activation of KCC2 restores inhibitory function, with animal and postmortem human data showing rescue of disease-relevant brain activity and dopamine regulation.

• OV329 offers a validated mechanism for seizure control without the toxicity of first-generation drugs, aiming for better safety, tolerability, and ease of use in polypharmacy.

• Pediatric formulations are tailored for developmental needs, enabling rare orphan pricing and earlier intervention in treatment paradigms.

Market opportunity and investor perspective

• Current valuation is seen as undervaluing the KCC2 program, with significant upside expected as clinical data emerges.

• OV329 targets a >$1B opportunity in focal-onset seizures, with additional potential in developmental and epileptic encephalopathies.

• The company is positioned to unlock broad therapeutic utility for KCC2, with a discovery engine supporting multiple molecules for diverse CNS indications.

• Recent transaction with a Perceptive-backed entity for soticlestat provides potential royalty upside, with no current financial downside.