Ovid Therapeutics (NASDAQ:OVID) is advancing multiple clinical studies of its experimental brain medicines, with data from two programs expected in 2027, CEO Meg Alexander said during a discussion following the company’s third-quarter release.
The company is developing OV329 for epilepsy and OV4071, an experimental drug targeting a protein known as KCC2, for potential use in psychiatric and neurological conditions. Alexander said both programs are progressing, while emphasizing that upcoming studies will address questions about efficacy and safety.
Epilepsy Drug Faces Key Tests
OV329 inhibits GABA-aminotransferase, an enzyme that breaks down GABA, an inhibitory neurotransmitter in the brain. Alexander said the approach could have applications across adult and pediatric seizure disorders.
The drug targets the same enzyme as the older medicine vigabatrin, which Alexander said can accumulate in the retina and cause irreversible vision loss. She said Ovid’s preclinical work supports the view that the toxicity is specific to the older compound rather than the underlying mechanism.
Alexander acknowledged that Ovid’s Phase 1 study, which included more than 100 participants, was too short to establish long-term eye safety in humans. The company’s 12-week Phase 2 epilepsy study will be followed by open-label extension studies to collect longer-term safety data, including measures of eye health, she said.
Ovid expects Phase 2 data in the second half of 2027. Alexander said she considers at least a 30% seizure reduction necessary to be competitive, alongside safety, tolerability and ease of use.
A separate photosensitivity study is expected to read out in the first quarter of 2027. It is testing 5-, 7- and 9-milligram doses in people whose epilepsy is triggered by light, with the aim of assessing anticonvulsant activity and informing dose selection.
Alexander cautioned that the small study is not equivalent to a randomized Phase 2 trial and may not provide a clear dose-response relationship. She said a partial or complete reduction in the subclinical seizure activity measured in such studies has historically been a useful sign of anticonvulsant effect.
Pediatric Plans and KCC2 Program
Ovid is on track to initiate its first study in a developmental and epileptic encephalopathy, focused on tuberous sclerosis complex, before year-end, Alexander said. The study is expected to examine measures including seizure reduction, responder rates and seizure freedom, with potential exploratory assessments of behavior and sleep.
Alexander said Ovid has developed different formulations of OV329 for adult and pediatric use, reflecting the needs of younger patients. She added that the formulations could allow the company to price and position them differently if they reach the market.
Ovid’s OV4071, an oral direct activator of KCC2, is in a Phase 1 study. Alexander said an additional translational study is expected to produce results in the first quarter of 2027 and that both studies include electrophysiological biomarkers.
The company is considering an initial proof-of-concept study in schizophrenia. Alexander said preclinical work suggests potential applications in other conditions, including psychosis associated with Alzheimer’s disease, Parkinson’s disease and Lewy body dementia, though those applications remain to be established.
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.
