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]

Business and pipeline update March 18, 2026 © Copyright 2026 Ovid Therapeutics Forward-looking statements This presentation includes certain disclosures by Ovid that contain "forward-looking statements" including, without limitation statements regarding: the reproducibility and durability of any favorable results initially seen to date in clinical trials; the potential therapeutic opportunity of OV329, OV4071 and other compounds from Ovid's library of direct activators of KCC2; the expected timing of initia ...

Core Insights - Ovid Therapeutics Inc. reported progress in its pipeline and business updates, highlighting favorable results for OV329, a GABA-AT inhibitor, and plans for additional development programs [1][3][4] Pipeline and Business Updates - OV329 demonstrated a favorable safety and tolerability profile in a 7 mg dose cohort, leading to plans for patient studies [4][5] - The company is expanding OV329's development to include tuberous sclerosis complex (TSC) seizures and infantile spasms (IS), supported by a $60 million private placement [1][6][10] - A Phase 1 trial for OV4071, a first-in-class oral KCC2 direct activator, has been approved and is set to begin [2][14] - Ovid plans to host a KCC2-focused R&D Day on April 14, 2026, to discuss its KCC2 portfolio [6][14] Financial Results - As of December 31, 2025, Ovid reported $90.4 million in cash, cash equivalents, and marketable securities, which is expected to fund operations into late 2028 [6][23] - The company reported a net income of $9.7 million for Q4 2025, compared to a net loss of $9.3 million in Q4 2024, primarily due to a gain from a long-term equity investment [27][30] - Total revenue for 2025 was $7.3 million, an increase from $0.6 million in 2024, driven by royalties and a one-time payment [23][30] Clinical Development Plans - Ovid plans to initiate a Phase 2 trial for OV329 in focal onset seizures and an open-label proof-of-concept study in Q2 2026 [9][20] - The company is also advancing a pediatric-specific formulation of OV329 for use in infants and children [12][11] - A ketamine challenge study for OV4071 is planned for mid-2026 to explore its pharmacodynamic effects [16][20] Business Strategy - The approval of OV4071 triggers a 30-day period for the exercise of Series A Warrants, potentially raising an additional $53.9 million [17][18] - Proceeds from the recent private placement will support the expansion of OV329 into additional indications [19][18]

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]