Financial Data and Key Metrics Changes - As of December 31, 2025, Ovid Therapeutics reported $90.4 million in cash equivalents and marketable securities [42] - The company announced a PIPE financing with gross proceeds totaling $60 million, which is expected to extend the cash runway into the second half of 2028 [42] - If the Series A warrants are fully exercised, the pro forma cash runway could extend well into 2029 [42] Business Line Data and Key Metrics Changes - The company received regulatory clearance for OV4071, the first-ever oral KCC2 direct activator, a quarter earlier than expected [5] - OV329, a next-generation GABA aminotransferase inhibitor, has shown no serious adverse events at the 7 mg dose, indicating a strong safety and tolerability profile [5][13] - The company is launching additional studies for OV329 in infantile spasms and seizures associated with tuberous sclerosis complex, supported by new capital [7][8] Market Data and Key Metrics Changes - The market opportunity for OV329 is significant, especially in treating conditions like infantile spasms and tuberous sclerosis complex, where current treatment options are limited [24][27] - The peak sales of vigabatrin (Sabril) reached over $320 million, indicating a substantial market despite safety concerns that limited its use [79] Company Strategy and Development Direction - Ovid Therapeutics aims to develop safer, more effective treatments for neural hyperexcitability, focusing on small molecule programs [9] - The company plans to initiate a phase II randomized placebo-controlled trial for OV329 in focal onset seizures in Q2 2026, with a focus on traditional efficacy endpoints [59] - The strategy includes developing differentiated formulations for adult and pediatric populations, allowing for differential pricing based on specific needs [70] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of OV329 to be used earlier and longer in treatment regimens, which could change disease trajectories for patients [78] - The company is optimistic about the upcoming trials and the potential for OV4071 to address a broad range of psychosis applications [34][36] - Management emphasized the importance of robust safety data to support regulatory submissions and avoid the limitations faced by previous treatments [50] Other Important Information - The company does not plan to host regular quarterly earnings calls moving forward, indicating a shift in communication strategy [4] - Ovid Therapeutics is preparing for a KCC2 day event to provide further insights into their KCC2 program and future candidates [33] Q&A Session Summary Question: Can you provide more detail on OV329 and the visual monitoring? - Management confirmed that optic and retinal monitoring will continue through phase II and pivotal studies to ensure robust safety data [50] Question: What are the key endpoints for the planned phase II design for OV329? - The phase II study will target adults with treatment-resistant focal onset seizures, focusing on traditional efficacy endpoints such as percent reduction in seizures [59] Question: What type of patients will be enrolled in the open-label photosensitive epilepsy study? - The study will enroll adults with diagnosed epilepsy and documented photosensitivity, using a specialized site in the Netherlands [67] Question: How does the company view the long-term clinical development for OV329? - Management believes OV329 has broad therapeutic utility and will use evidence from ongoing studies to inform future development directions [91]

Financial Data and Key Metrics Changes - As of December 31, 2025, Ovid Therapeutics reported $90.4 million in cash equivalents and marketable securities [42] - The company announced a PIPE financing with gross proceeds totaling $60 million, which is expected to extend the cash runway into the second half of 2028 [42] - If the Series A warrants are fully exercised, the pro forma cash runway could extend well into 2029 [42] Business Line Data and Key Metrics Changes - The company received regulatory clearance for OV4071, the first-ever oral KCC2 direct activator, a quarter earlier than expected [5] - OV329, a next-generation GABA aminotransferase inhibitor, has shown no serious adverse events at the 7-milligram dose, indicating a strong safety and tolerability profile [5][13] - The company is launching additional studies for OV329 in infantile spasms and seizures associated with tuberous sclerosis complex, supported by new capital [7][8] Market Data and Key Metrics Changes - The market opportunity for OV329 is significant, especially in treating conditions like infantile spasms and tuberous sclerosis complex, where current treatment options are limited [24][27] - The peak sales of Sabril (Vigabatrin) reached over $320 million, indicating a substantial market potential for a safer alternative like OV329 [79] Company Strategy and Development Direction - Ovid Therapeutics aims to develop gentler, better medicines for the brain, focusing on controlling neural hyperexcitability through small molecule programs [9] - The company plans to initiate a phase 1 study for OV4071 and a ketamine challenge study to demonstrate proof of mechanism [33][40] - The strategy includes developing differentiated formulations for adult and pediatric populations, allowing for differential pricing based on the specific needs of each community [70] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of OV329 to be used earlier and longer in treatment, which could change the trajectory of diseases for children with severe conditions [78] - The company is optimistic about the upcoming milestones and catalysts, indicating a busy couple of years ahead [37] Other Important Information - Ovid Therapeutics does not plan to host regular quarterly earnings calls moving forward, indicating a shift in communication strategy [4] - The company is preparing for a KCC2 day to provide more insights into the KCC2 portfolio and its therapeutic potential [36] Q&A Session Summary Question: Can you provide more detail on OV329 and the visual monitoring? - Management confirmed that optic and retinal monitoring will continue through phase 2 and pivotal studies to ensure robust safety data [50] Question: What are the key endpoints for the planned phase two design for OV329? - The phase 2 study will target adults with treatment-resistant focal onset seizures, focusing on traditional endpoints like percent reduction in seizures and CGI endpoints [59] Question: What type of patients will be enrolled in the open-label photosensitive epilepsy study? - The study will enroll adults with diagnosed epilepsy and documented photosensitivity, using intermittent photic stimulation for screening [67] Question: How does the company plan to address long-term clinical development for OV329? - Management indicated that OV329 has broad therapeutic utility and that evidence will inform potential expansions into other indications [91]

Financial Data and Key Metrics Changes - As of December 31, 2025, Ovid Therapeutics reported $90.4 million in cash equivalents and marketable securities [40] - The company announced PIPE financing with gross proceeds totaling $60 million, which is expected to extend the cash runway into the second half of 2028 [40] - Pro forma cash runway is anticipated to extend well into 2029, assuming full exercise of Series A warrants triggered by regulatory clearance [40] Business Line Data and Key Metrics Changes - The company received regulatory clearance for OV4071, the first-ever oral KCC2 direct activator, a quarter earlier than expected [4] - OV329, a next-generation GABA aminotransferase inhibitor, has shown no serious adverse events at the 7-milligram dose, indicating a strong safety and tolerability profile [5][11] - The company is launching additional studies for OV329 in infantile spasms and tuberous sclerosis complex, expanding its clinical pipeline [5][19] Market Data and Key Metrics Changes - The market opportunity for OV329 is significant, especially in treating conditions like infantile spasms and tuberous sclerosis complex, where current treatment options are limited [20][25] - The peak sales of Sabril (Vigabatrin) reached over $320 million, indicating a substantial market despite safety concerns that limited its use [76] Company Strategy and Development Direction - Ovid Therapeutics aims to develop safer and more effective treatments for neural hyperexcitability, focusing on small molecule programs [6][7] - The company plans to initiate a phase 2 randomized placebo-controlled trial for OV329 in focal onset seizures and is also exploring pediatric formulations for underserved populations [17][68] - The strategic focus includes expanding the use of OV329 beyond focal onset seizures to potentially include status epilepticus in the future [92] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the safety and efficacy of OV329, emphasizing the importance of robust safety data to support regulatory submissions [48] - The company is optimistic about the potential of OV4071 in treating various psychosis-related conditions, with plans for further data releases in the coming months [32][34] - Management highlighted the importance of addressing unmet needs in epilepsy treatment, particularly for pediatric patients [19][21] Other Important Information - The company does not plan to host regular quarterly earnings calls moving forward, indicating a shift in communication strategy [3] - The clearance of OV4071 triggers a 30-day period for Series A warrants, which could bring in additional capital exceeding $53 million [6] Q&A Session Summary Question: Can you elaborate on the visual monitoring for OV329? - Management confirmed that optic and retinal monitoring will continue through phase 2 and pivotal studies to ensure robust safety data [47] Question: What are the key endpoints for the phase 2 design of OV329? - The phase 2 study will target adults with treatment-resistant focal onset seizures, focusing on traditional endpoints like percent reduction in seizures and CGI endpoints [58] Question: What is the rationale for the ketamine challenge study for OV4071? - The ketamine challenge aims to demonstrate that OV4071 is effectively entering the brain and exhibiting GABAergic activity, with plans to correlate this with clinical symptom improvement [60] Question: What patient population will be enrolled in the open-label photosensitive epilepsy study? - The study will enroll adults with diagnosed epilepsy and documented photosensitivity, using specialized sites for the research [65] Question: How does the company plan to address differential pricing for different populations? - The company plans to develop differentiated formulations for adult and pediatric populations, allowing for appropriate and responsible differential pricing [68]

Summary of Ovid Therapeutics Conference Call Company Overview - **Company**: Ovid Therapeutics (NasdaqGS:OVID) - **Focus**: Development of neurotherapeutics targeting conditions driven by neural hyperexcitability, particularly in epilepsy Key Points and Arguments Pipeline and Product Development - Ovid is advancing a pipeline that includes: - **OV329**: A next-generation GABA aminotransferase (GABA AT) inhibitor for treatment-resistant epilepsies and focal onset seizures - **KCC2 Direct Activators**: Including OV350 (IV) and OV4071 (oral), targeting potassium chloride cotransporter 2 - **Phase 1 Results**: OV329 has completed Phase 1 safety and biomarker studies, with plans to initiate Phase 2a in Q2 2026 and report top-line data in mid-2027 [8][10][42] Market Need and Competitive Landscape - There is a significant unmet need in epilepsy treatment, with only two novel mechanisms approved in the last 30 years despite 30 anti-seizure medications being available [10][11] - Approximately 40% of epilepsy patients are treatment-resistant, representing a market opportunity exceeding $1 billion [44] Safety and Efficacy of OV329 - **Safety Profile**: OV329 demonstrated a favorable safety profile with no serious adverse events and only mild, transient side effects [24][41] - **Efficacy Metrics**: Positive biomarker data indicates OV329 effectively inhibits GABA AT, with a potential for superior efficacy compared to existing treatments like vigabatrin [14][20][41] - **Dosing**: Expected dosing for OV329 is significantly lower than vigabatrin, with estimates of 5-7 mg compared to 2-3 grams for vigabatrin [20][21] Mechanism of Action - OV329 provides both phasic and tonic inhibition of GABA, which may lead to better tolerability and efficacy compared to previous GABAergic medications [15][16][64] - The drug's design aims to avoid the ocular safety issues associated with vigabatrin, which has been linked to vision loss [19][21] Phase 2a Trial Design - The upcoming Phase 2a trial will evaluate a 7 mg dose of OV329 against placebo over eight weeks, focusing on treatment-resistant focal onset seizures [43][44] - The trial will measure various efficacy outcomes, including seizure frequency and responder rates [43] Future Development Plans - Ovid plans to continue developing its KCC2 portfolio, with expected readouts for OV350 by the end of 2025 and initiation of Phase 1 studies for OV4071 in 2026 [45][46] - The company has sufficient resources to fund its development programs through 2028 [45] Additional Important Information - The call emphasized the importance of differentiating OV329 from existing treatments through its unique mechanism and safety profile [54][64] - The company is committed to building a robust safety database to support the development of OV329 and mitigate monitoring requirements for patients [27][41] - The anticipated market dynamics and reimbursement landscape are favorable for OV329, despite the competitive nature of the epilepsy treatment market [44]