Ovid Therapeutics (NASDAQ:OVID) outlined new pipeline and financing updates alongside its fourth-quarter and full-year 2025 results, highlighting regulatory clearance in Australia for its KCC2 direct activator OV4071, new safety and tolerability data for its seizure candidate OV329, and a $60 million PIPE financing led by Point72.
PIPE financing, warrant trigger, and cash runway
Management said the company ended 2025 with $90.4 million in cash, cash equivalents, and marketable securities. Ovid also announced a $60 million PIPE financing (gross proceeds before fees and expenses) led by Point72, with participation from existing shareholders including Janus, RA, Bellevue, Affinity, Coastland, Eventide, Adage, and ADAR1, according to CEO Meg Alexander.
Alexander noted that Australia’s clearance for OV4071 triggered a 30-day period for the Series A warrants, and if exercised could generate additional proceeds of more than $53 million.
OV329: 7 mg safety update and dose strategy
Ovid’s main clinical update focused on OV329, its next-generation GABA aminotransferase inhibitor, which the company is developing for focal onset seizures and is now expanding into pediatric epilepsies. Alexander reported that Ovid now has safety and tolerability data for the 7 mg dose and said there were no serious adverse events and no adverse events associated with the 7 mg dose.
Across the broader single-ascending dose (SAD) and multiple-ascending dose (MAD) cohorts, Alexander said there were adverse events in the overall cohort but that they were not associated with treatment (citing examples such as cannula site reactions). She added that adverse events considered potentially associated with OV329 across doses included headache, drowsiness, and metallic taste, described as low frequency, mild, and resolving.
In response to a question about exposure targets, Alexander said all subjects in the 5 mg and 7 mg cohorts were within the targeted exposure range. She also addressed why Ovid is not pursuing doses above 7 mg, arguing there is a ceiling effect for central nervous system GABA aminotransferase inhibition of roughly 60%–65%, and that higher doses may not provide additional benefit relative to potential tolerability tradeoffs.
Ophthalmic monitoring and differentiation from vigabatrin
A recurring theme of the call was differentiating OV329 from vigabatrin (Sabril), a first-generation GABA aminotransferase inhibitor with known ophthalmic safety risks. Alexander emphasized that Ovid is conducting extensive visual testing and structural eye imaging and said the company has seen no ophthalmic safety concerns with OV329 to date.
In the Q&A, she said Ovid will continue optic and retinal monitoring in the phase II focal onset seizure study and in pivotal studies thereafter, not because the company expects to see issues, but to build a robust patient dataset for regulators. The stated goal is to demonstrate no structural or visual changes and avoid the type of monitoring and risk mitigation requirements that limited vigabatrin’s use.
Discussing the commercial history of vigabatrin, Alexander said the drug was initially expected to be a “multi-blockbuster” due to anticonvulsant efficacy but later faced post-market findings related to retinal partitioning that, in some patients, led to blindness. She said Sabril’s U.S. sales peaked at more than $320 million in 2018 across infantile spasms and tuberous sclerosis complex (TSC) seizures, while noting the company could not provide a sales breakdown by indication because Lundbeck did not disclose it.
OV329 clinical plans: focal onset seizures and new pediatric programs
Ovid said OV329 remains on track for proof-of-concept work in focal onset seizures, with additional studies designed to inform dosing and de-risk later development.
- Phase II focal onset seizures: Ovid plans to initiate a randomized, placebo-controlled phase II trial in Q2 2026 in adults with treatment-resistant focal onset seizures. Alexander described endpoints consistent with “traditional” seizure trials, including percent reduction in seizures and seizure frequency measures, along with CGI-type endpoints.
- Photosensitivity study: Later in 2026, Ovid expects to initiate an open-label photoparoxysmal response (PPR) study in adults with photosensitive epilepsy at a specialized site in the Netherlands. Alexander said the goal is to show anticonvulsant effect and provide dose confidence ahead of the full phase II readout. She described key measures as reductions in the number of intermittent photic stimulation frequencies inducing PPR, changes in the threshold frequency, and complete suppression of PPR during and post-dose.
Using proceeds from the PIPE, Ovid said it is expanding OV329 into infantile spasms and TSC-associated seizures. Alexander characterized infantile spasms as a medical emergency occurring typically at four to seven or eight months of age, with risks including developmental disabilities, increased mortality, and lifelong epilepsies. She cited current standards of care including Acthar Gel or high-dose steroids first line and vigabatrin second line.
For TSC, Alexander said seizures occur in about 80% of patients and described current standards of care including Afinitor and Sabril, with limitations related to safety. She said Ovid believes a safe, well-tolerated GABA aminotransferase inhibitor could be used earlier and for longer in both conditions, and suggested the company sees a potential path to efficient registration through signal-finding and pediatric dose confirmation.
On development sequencing, Alexander said Ovid is designing the TSC program as open-label and will enroll older patients first, then step down to younger pediatric patients to establish safety, observe signal, and confirm dose modeling before initiating the infantile spasms study. She said Ovid intends to provide updates as enrollment grows to a sufficient size to support decisions.
OV4071: regulatory clearance and next steps for KCC2
Ovid also highlighted progress for OV4071, which Alexander called the “first-ever” oral KCC2 direct activator to receive regulatory clearance (in Australia). She said the company is on track to initiate a phase I study in Q2, followed by a ketamine challenge study later in 2026, designed to help characterize on-mechanism GABAergic activity using quantitative EEG and other measures.
Alexander said the ketamine challenge approach has been used in other antipsychotic development programs and is intended to demonstrate brain penetration and mechanism-consistent effects, potentially correlating EEG readouts with clinical symptom changes. She also said Ovid plans to discuss additional details, including biomarker strategy and broader portfolio efforts beyond OV4071, at a KCC2-focused event on April 14.
In closing remarks, management thanked investors participating in the PIPE and said the company does not plan to host regular quarterly earnings calls going forward, tying this call to the business and pipeline updates released alongside year-end results.
About Ovid Therapeutics (NASDAQ:OVID)
Ovid Therapeutics is a clinical-stage biopharmaceutical company focused on the development of therapies for rare neurological disorders. Founded in 2014 and headquartered in New York, the company applies a precision medicine approach to target underlying mechanisms of disease in patients with genetic conditions affecting the central nervous system. Its research platform centers on small-molecule modulators of neurotransmitter pathways to restore neural network function in disorders with high unmet medical need.
The company’s lead development candidate, OV101 (gaboxadol), is a selective extrasynaptic GABAA receptor agonist being investigated for the treatment of Angelman syndrome and Fragile X syndrome.
