Ovid Therapeutics (NASDAQ:OVID) expects several clinical milestones over the next 12 months as it advances experimental treatments for schizophrenia and epilepsy, President and CEO Meg Alexander said at a company presentation.
Alexander said the neurotherapeutics company has cash to fund operations into 2029. Its programs include OV4071, an oral drug targeting KCC2, and OV329, a potential epilepsy treatment.
Schizophrenia Program Advances
OV4071 is in a Phase 1 study, with the company having completed several single-ascending-dose cohorts and moved into the multiple-ascending-dose portion, Alexander said. Ovid expects to report Phase 1 results in the first quarter of next year and plans a ketamine challenge study to gather additional information about the drug’s activity in humans.
KCC2 helps regulate chloride levels in neurons, enabling the neurotransmitter GABA to perform its inhibitory function, according to Alexander. She said animal studies of OV4071 have shown activity across models related to psychosis, including measures of negative symptoms and cognition. Those findings have not yet established clinical efficacy in humans.
The planned ketamine challenge is intended to assess whether OV4071 reaches the brain, produces activity consistent with its proposed mechanism and affects biomarkers associated with altered neurotransmission. Alexander said the results could help guide decisions about indications and clinical trial endpoints.
Ovid plans an initial proof-of-concept study in acute schizophrenia around the middle of next year. Alexander said the company chose schizophrenia first because of the consistency of its preclinical findings and sees potential to explore other forms of psychosis if the drug succeeds.
Epilepsy Studies and Safety Follow-Up
Ovid’s lead clinical program, OV329, inhibits GABA aminotransferase, an enzyme involved in breaking down GABA. Alexander said the company designed it to address safety limitations associated with the older drug vigabatrin, including retinal degeneration and irreversible vision loss.
OV329 has been tested in about 100 people, Alexander said. She acknowledged that the Phase 1 study was not designed to definitively establish long-term safety. Ovid is running a 12-week Phase 2 study in focal epilepsy and plans open-label extensions to build a longer-term safety database.
The company expects the focal epilepsy study to read out in the second half of next year. A separate photosensitivity study is also due to report results, which Alexander said could provide further validation for the dose being used in focal epilepsy.
Ovid also plans to start an open-label study in tuberous sclerosis complex, a pediatric epilepsy indication, this year. Alexander said the study is intended to identify safety and efficacy signals and that the company could share findings as the trial progresses if results are encouraging.
About Ovid Therapeutics (NASDAQ:OVID)
Ovid Therapeutics Inc. is a biopharmaceutical company focused on developing medicines for epilepsies and other neurological disorders. Its work targets conditions with significant unmet treatment needs, including rare and difficult-to-treat seizure disorders. The company’s lead development program, OV329, is an investigational GABA aminotransferase inhibitor being studied as a potential treatment for seizures, including infantile spasms and focal-onset seizures. Ovid’s pipeline and research programs may change as clinical development progresses.
