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]

Core Insights - Ovid Therapeutics Inc. has been identified as a promising investment opportunity with significant upside potential, particularly following a price target increase by B. Riley from $3 to $5, maintaining a Buy rating on the shares [1][2] Company Overview - Ovid Therapeutics Inc. is a biopharmaceutical company focused on developing effective treatments for patients with epilepsies and seizure-related neurological disorders in the United States [3] Recent Developments - The increase in price target is attributed to strong Phase 1 biomarker results for OV329, which demonstrated superior efficacy compared to vigabatrin without the associated ocular toxicity [1][2] - The favorable profile of OV329 supports its use in earlier lines of treatment, with the potential to expand its market to over $700 million [2] - The company has also secured a $175 million PIPE financing, which enhances its balance sheet and extends its cash runway into 2028 [2]

Core Insights - Ovid Therapeutics' shares increased by 39.2% following positive top-line data from an early-stage study of OV329, a next-generation GABA-AT inhibitor aimed at treating rare and drug-resistant epilepsies [1][6] - The therapy's mechanism involves blocking GABA-AT to enhance brain GABA levels, potentially positioning it as a best-in-class treatment for seizures [1][6] Study Details - The phase I study of OV329 included 68 healthy volunteers and assessed safety, tolerability, pharmacokinetics, and pharmacodynamics with doses ranging from 1 to 5 mg [2] - Participants received once-daily oral doses, with comprehensive safety and ophthalmic evaluations conducted throughout the study [2] Efficacy Results - OV329 demonstrated strong GABAergic activity, increasing inhibition by 53% on the abductor pollicis brevis (APB) muscle and 44% on the first dorsal interosseous (FDI) muscle at the 5 mg dose, compared to no significant change with placebo [7] - The therapy also significantly prolonged the cortical silent period (CSP) by up to 13 milliseconds at the 5 mg dose, indicating enhanced neural inhibition [8] Biomarker Analysis - Magnetic resonance spectroscopy (MRS) indicated a 7.13% increase in brain GABA levels after seven days of treatment, while placebo showed negligible change [9] - Exploratory EEG data suggested increased inhibitory brainwave activity, supporting OV329's GABA-enhancing mechanism [9] Safety Profile - The phase I study reported that OV329 had a favorable safety and tolerability profile, with treatment-related adverse events primarily mild to moderate [10] Future Plans - Following the encouraging phase I results, Ovid Therapeutics plans to initiate a phase IIa study of OV329 in adults with drug-resistant focal onset seizures in Q2 2026, with completion expected by mid-2027 [11] - The company aims to further define OV329's anti-convulsant profile through an open-label seizure study [11] Additional Pipeline - Ovid Therapeutics is also developing another investigational candidate, OV350, in a separate early-stage study for brain diseases [12]

Group 1 - The call is hosted by Victoria Fort, Senior Vice President of Corporate Affairs and Corporate Strategy, to discuss the Top Line OV329 Phase I readout [1][2] - Key executives participating in the call include Dr. Jeremy Levin (Chairman and CEO), Meg Alexander (President and COO), Jeff Rona (Chief Business and Financial Officer), and Zhong Zhong (Chief Scientific Officer) [2] - The company will be making forward-looking statements regarding future expectations, plans, and prospects, which are subject to risks and uncertainties [3]

Core Viewpoint - Ovid Therapeutics is conducting a data call to discuss the Top Line OV329 Phase I readout, indicating progress in their clinical development efforts [2]. Group 1: Company Overview - The call is hosted by Victoria Fort, Senior Vice President of Corporate Affairs and Corporate Strategy, along with key executives including Dr. Jeremy Levin, Meg Alexander, Jeff Rona, and Zhong Zhong [2]. Group 2: Forward-Looking Statements - The company will be making forward-looking statements during the call, which are subject to risks and uncertainties that could lead to actual results differing from expectations [3].

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]