Ovid Therapeutics (OVID) investor relations material

Pipeline overview and unmet need

• OV329 is a next-generation GABA aminotransferase inhibitor targeting neural hyperexcitability disorders, including drug-resistant epilepsy and CNS diseases, with a differentiated mechanism from existing therapies.

• Pipeline includes OV329 for treatment-resistant epilepsies and KCC2 direct activators for CNS indications.

• Significant unmet need remains in epilepsy, with many patients experiencing uncontrolled seizures despite multiple approved drugs.

• OV329 aims to address safety and tolerability issues seen with first-generation GABA modulators like vigabatrin.

• Market opportunity exceeds $1 billion annually, with a target population of 65,000 adults in the U.S.

Study design and objectives

• Phase 1 trial evaluated OV329 in 68–69 healthy volunteers, assessing safety, tolerability, pharmacokinetics, and pharmacodynamics across single and multiple ascending dose cohorts (1–5 mg).

• Extensive biomarker analyses included TMS, MRS, and EEG to measure GABAergic inhibition and brain engagement.

• OV329 was compared to placebo and to published data for vigabatrin, a first-generation GABA-AT inhibitor.

• The Phase 1 study evaluated safety, pharmacokinetics, pharmacodynamics, and biomarker effects in healthy volunteers to inform Phase 2 dosing.

• OV329 is designed for once-daily oral dosing, with lower anticipated human doses compared to existing therapies.

Safety and tolerability results

• OV329 demonstrated a strong safety profile with only mild, transient adverse events and no serious treatment-related or ophthalmic events.

• No ocular or retinal changes were observed, supported by rigorous ophthalmic safety monitoring and preclinical studies.

• No evidence of drug accumulation in the eye, differentiating OV329 from vigabatrin and reducing risk of vision loss.

• No titration is anticipated for future dosing due to the clean safety profile.

• All potentially treatment-related adverse events were mild and transient, including headache, drowsiness, and metallic taste.