Ovid Therapeutics (OVID) 3rd Annual Guggenheim Healthcare Innovation Conference summary

Pipeline strategy

• Pipeline focuses on differentiated small-molecule mechanisms for conditions involving excessive neuronal excitation.

• Programs span OV329, a GABA aminotransferase inhibitor for epilepsy, and OV4071, an oral direct KCC2 activator.

KCC2 and OV4071

• Phase I has completed several SAD cohorts and moved into MAD; the study was described as progressing well.

• More than 100 pharmacology and pharmacodynamic studies, including over 40 psychosis studies, showed consistent preclinical signals across symptom domains.

• Animal studies showed reduced hyperlocomotion without comparable levels of sedation, plus improvements in negative-symptom models, working memory and task switching.

• A ketamine challenge is planned to assess brain exposure, electrophysiological and other biomarkers, and whether human results align with animal findings.

• Acute schizophrenia proof-of-concept is planned around mid-2027; positive signals could support expansion into broader psychosis indications.

OV329 epilepsy program

• OV329 targets adult and pediatric epilepsies, including focal epilepsy and developmental and epileptic encephalopathies; its design aims to avoid vigabatrin's retinal partitioning and OAT-related liabilities.

• After testing in about 100 people, OV329 was described as clean and well tolerated, with potentially therapeutic levels of brain enzyme inhibition.

• A 12-week, registrational-quality Phase II focal epilepsy study is underway globally; open-label extensions are planned through registration to build longer-term safety data.

• A photosensitivity study is expected to read out and inform focal epilepsy dose selection; a tuberous sclerosis complex open-label signal-finding and safety study is starting in 2026.

• The focal epilepsy Phase II study is expected to read out in H2 2027.