OV350 showed a good safety profile, supporting the advancement of the Company’s KCC2 portfolio, including the first oral direct activator, OV4071There were no treatment-related laboratory findings, no safety findings, and no treatment-related serious adverse events (SAEs)Exploratory quantitative electrophysiology results suggest OV350 had central activity and spectral power consistent with expected physiological effects of KCC2 modulation; aligned with expected drug exposure in the brainPharmacokinetics for ...

Core Insights - Ovid Therapeutics Inc. has appointed Dr. Petra Kaufmann as Chief Medical Officer to lead clinical, medical, and regulatory strategy for its pipeline of brain disorder therapeutics [1][2][3] - Dr. Kaufmann has extensive experience in developing CNS therapeutics, including leadership roles in biopharmaceutical companies and the National Institutes of Health [2][3] - The company is focused on advancing its next-generation GABA-AT inhibitor, OV329, and a portfolio of KCC2 direct activators for various CNS disorders [4] Company Overview - Ovid Therapeutics is a biopharmaceutical company based in New York, dedicated to developing small molecule medicines for brain conditions with significant unmet needs [4] - The company is advancing a pipeline that includes OV329 for treatment-resistant seizures and other undisclosed indications, as well as additional compounds targeting the KCC2 transporter [4] Leadership and Expertise - Dr. Kaufmann's background includes significant contributions to the development and global approval of Zolgensma, the first gene therapy for spinal muscular atrophy [2][3] - Her expertise in patient-focused trial design and biomarker strategies is expected to enhance Ovid's R&D capabilities [2][3] - The appointment is seen as a strategic move to strengthen the company's research and development organization [3]

Core Insights - Ovid Therapeutics Inc. announced a CEO succession plan with Meg Alexander set to become CEO on January 1, 2026, while Dr. Jeremy M. Levin transitions to Executive Chairman [1][2][3] - The company reported third-quarter financial results and provided updates on its clinical pipeline, highlighting progress in its drug development programs [1][4][11] Leadership Transition - Meg Alexander has been with Ovid since 2021 and has played a key role in developing the company's pipeline and overseeing operations [2] - Dr. Levin expressed confidence in Alexander's leadership and emphasized the importance of continuity during the transition [3] Pipeline and Clinical Updates - Ovid's OV329, a next-generation GABA-aminotransferase inhibitor, is advancing to Phase 2 patient studies following positive Phase 1 results, demonstrating strong inhibitory activity [4][5][8] - The KCC2 direct activator portfolio is progressing, with OV350 expected to report first-in-human data in Q4 2025 and OV4071 anticipated to enter clinical trials in Q2 2026 [5][9][10] Financial Performance - For Q3 2025, Ovid reported total revenue of $132,000, a decrease from $173,000 in Q3 2024 [15] - Research and development expenses were $5.9 million, down from $7.9 million in the same period last year, while general and administrative expenses increased to $6.8 million from $5.5 million [15] - The net loss for Q3 2025 was $12.2 million, compared to a net loss of $14.0 million in Q3 2024, with a basic and diluted net loss per share of $0.17 [15][19] Funding and Financial Strategy - Ovid completed a private placement of up to $175 million, with an initial closing of approximately $81 million, expected to extend its cash runway into the second half of 2028 [5][11] - As of September 30, 2025, Ovid had $25.6 million in cash, cash equivalents, and marketable securities [15][21]

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]

Core Viewpoint - Ovid Therapeutics Inc. has announced a private investment in public equity (PIPE) financing expected to generate gross proceeds of up to $175 million, with an initial closing of approximately $81 million to support its operating plan and clinical pipeline through 2028 [1][6]. Financing Details - The PIPE financing is anticipated to close around October 6, 2025, pending customary closing conditions [1]. - New investors participating in the PIPE include Janus Henderson Investors, RA Capital Management, and others, alongside existing investors [2]. - The company is selling 57,722 shares of Series B non-voting convertible preferred stock, Series A warrants for 38,481,325 shares of common stock, and Series B warrants for 28,861,000 shares of common stock [3]. Securities Structure - Each share of Series B Preferred Stock is convertible into 1,000 shares of common stock, subject to stockholder approval and ownership limitations [4]. - Series A and Series B Warrants have an exercise price of $1.40 per share and will be exercisable following stockholder approval [5]. Use of Proceeds - The net proceeds from the PIPE financing will be used for research and development, general corporate expenses, and working capital needs, with expectations to fund operations into 2028 [6]. Company Overview - Ovid Therapeutics is focused on developing small molecule medicines for brain conditions with significant unmet needs, advancing a pipeline that includes OV329 for treatment-resistant seizures and other compounds targeting CNS disorders [10].

Core Insights - Ovid Therapeutics Inc. announced positive topline results from its Phase 1 study of OV329, a next-generation GABA-aminotransferase inhibitor aimed at treating drug-resistant epilepsies, demonstrating a favorable safety and tolerability profile [1][2][6] Trial Design - The Phase 1 trial involved 68 healthy volunteers, with 51 receiving active treatment and 17 receiving placebo, testing doses from 1 mg to 5 mg [3] - Safety, tolerability, pharmacokinetic, and pharmacodynamic activities were assessed, including rigorous ophthalmic evaluations and extensive exploratory biomarker analyses [3][4] Biomarker and Efficacy Results - OV329 showed significant GABAergic inhibition, with a 53% increase in inhibition at the 5 mg dose as measured on the abductor pollicis brevis muscle [8] - The study confirmed OV329's ability to penetrate the brain and engage its target, achieving biological modulation consistent with elevated GABA levels [4][8] - After 7 days of dosing at 5 mg, mean GABA levels increased by 7.13% in the medial parietal lobe compared to 0.24% in placebo [8] Safety and Tolerability - The safety profile of OV329 was favorable, with all treatment-related adverse events reported as mild and transient, and no evidence of ophthalmic or retinal changes observed [6][9] - Extensive ophthalmic tests showed no adverse effects, contrasting with existing treatments like vigabatrin, which can accumulate in the retina [6] Future Development Plans - Ovid plans to advance OV329 into a Phase 2a study for drug-resistant focal onset seizures, expected to start in Q2 2026 [10] - The company is also progressing its KCC2 direct activator portfolio, with several regulatory and clinical milestones anticipated in the next 12 months [10][11]

Core Insights - Ovid Therapeutics Inc. reported business updates and financial results for Q2 2025, highlighting advancements in its drug pipeline and financial performance [1][19]. Pipeline and Business Updates - The company is advancing its pipeline with key readouts expected soon, including Phase 1 data for OV329, a potential treatment for conditions driven by excess neuronal excitation [2][3]. - OV329 is designed to be a safer GABA-aminotransferase inhibitor, aiming to replace vigabatrin, which has serious safety concerns [4][5]. - The Phase 1 study for OV329 is on track for topline results in late Q3 2025, with plans for a Phase 2a trial in adults with treatment-resistant focal onset seizures [9][16]. - Ovid is also progressing its KCC2 direct activator programs, with OV350's safety data expected in Q4 2025 and OV4071's first-in-human studies anticipated in early 2026 [10][11][12]. Financial Performance - As of June 30, 2025, Ovid had cash, cash equivalents, and marketable securities totaling $38.3 million, expected to support operations into early H2 2026 [7][22]. - Revenue from royalty agreements increased to $6.3 million in Q2 2025, compared to $169,000 in the same period of 2024 [22]. - Research and development expenses decreased to $6.5 million in Q2 2025 from $12.6 million in Q2 2024, reflecting organizational restructuring [22]. - The company reported a net loss of $4.7 million for Q2 2025, compared to a net income of $8.5 million in Q2 2024 [22]. Strategic Developments - Ovid entered a $7.0 million agreement with Immedica Pharma AB to monetize ganaxolone royalties, strengthening its balance sheet and extending its operational runway [14][15]. - The company plans to explore strategic options to accelerate development and offset costs, including partnerships and monetizing intellectual property [17][18].

Company Overview - Ovid Therapeutics Inc. is a biopharmaceutical company based in New York, focused on developing small molecule medicines for brain conditions with significant unmet needs [2] - The company is advancing a pipeline of novel, targeted small molecule candidates that address neuronal hyperexcitability associated with various neurological and neuropsychiatric disorders [2] Product Pipeline - Ovid is developing OV329, a next-generation GABA-aminotransferase inhibitor, aimed at treating treatment-resistant seizures and other undisclosed indications [2] - The company is also working on OV350, OV4071, and additional compounds that activate the KCC2 transporter for multiple central nervous system disorders [2] Upcoming Events - Company management will participate in the BTIG Virtual Biotechnology Conference scheduled for July 29-30, 2025 [1]

Core Viewpoint - Ovid Therapeutics has entered into a definitive agreement with Immedica Pharma for the sale of future royalties related to ganaxolone sales outside of China, providing Ovid with a non-dilutive capital infusion of $7 million to support its ongoing operations [1][2]. Group 1: Transaction Details - Immedica will pay $7 million in cash to acquire 100% of the royalty rights held by Ovid, enhancing Immedica's focus on ganaxolone by gaining control of additional intellectual property rights [1]. - The transaction includes royalties associated with a previous agreement Ovid had with Marinus Pharmaceuticals regarding ganaxolone for CDKL5 deficiency disorder [4]. - Ovid recorded approximately $566,000 in ganaxolone royalty revenues in 2024, indicating the current revenue scale from this asset [2]. Group 2: Company Backgrounds - Ovid Therapeutics is a biopharmaceutical company focused on developing small molecule medicines for brain conditions with significant unmet needs, advancing a pipeline of novel candidates targeting neurological disorders [7]. - Immedica is a pharmaceutical company dedicated to the commercialization of medicines for rare diseases, with a global distribution network serving patients in over 50 countries [6].

Core Insights - Ovid Therapeutics Inc. is hosting an event on June 12, 2025, to discuss the application of biomarkers in epilepsy and the pharmacodynamic activity of its OV329 program [1][2] - The event will feature Dr. Alexander Rotenberg, a leading expert in neurology, who will discuss the emerging use of biomarkers in anti-seizure medicine development [1][3] Company Overview - Ovid Therapeutics is focused on developing small molecule medicines for brain conditions with significant unmet needs, including treatment-resistant epilepsy [5] - The company is advancing a pipeline that includes OV329, a next-generation GABA-aminotransferase inhibitor designed to provide a novel approach for patients with treatment-resistant seizures [3][5] Clinical Development - OV329 is currently completing a Phase 1 study that evaluates its effects on pharmacodynamic biomarkers, safety, tolerability, and pharmacokinetics [3] - The program is expected to have a topline readout in Q3 2025, indicating progress in its clinical development [2]