Ovid Therapeutics (OVID) Stifel 2026 Healthcare Conference 2026 summary

Pipeline strategy

• Clinical-stage pipeline targets broad brain biology with differentiated mechanisms and small-molecule medicines designed for easier use and potential step-change patient benefit.

• Near-term programs are advancing through proof-of-concept and proof-of-signal studies, with milestones expected across 2027.

OV329 epilepsy program

• OV329 inhibits GABA-aminotransferase, increasing GABA and supporting the brain’s inhibitory signaling; the mechanism may apply across adult and pediatric seizure types.

• Preclinical work differentiated OV329 from first-generation vigabatrin’s retinal toxicity; Phase I included 100-plus patients but was short, so long-term ophthalmic safety is being assessed in Phase II and extensions.

• The 12-week Phase II is described as registrational quality and is planned to read out in H2 2027; open-label extensions will build long-term patient safety data, including rigorous ophthalmic monitoring.

• Photosensitivity study tests 5 mg, the planned 7 mg dose and 9 mg; readout is expected in Q1 2027 to assess anticonvulsant activity and help de-risk dose selection, not replace Phase II efficacy testing.

• Phase I TMS biomarkers showed OV329 matched or outperformed vigabatrin in several comparisons; management sees at least 30% seizure reduction as a competitive efficacy threshold and expects favorable tolerability, no titration and no drug-drug interactions.

Pediatric and rare epilepsy opportunities

• Pediatric formulation development is on track; separate, non-interchangeable formulations are intended to suit children’s developmental needs and support appropriate positioning in rare populations.

• A first tuberous sclerosis complex study is planned to start before year-end 2026; seizures affect 85% of this population, and endpoints include seizure reduction, responder rates and seizure freedom, with exploratory behavior and sleep measures.

• Infantile spasms have two established drug approaches, including GABA-aminotransferase inhibition; OV329’s hoped-for improved safety and tolerability could support earlier and longer use, potentially reducing later epilepsy and developmental impacts.