Epilepsies, Myoclonic

− Pre-Specified Interim Analysis Shows Positive Safety and Efficacy Results; Mezagitamab Safe and Well Tolerated − Takeda Intends to Initiate a Global Phase 3 Trial of Mezagitamab in Patients with Primary Immune Thrombocytopenia in Fiscal Year 2024 − Late-Stage Pipeline Continues to Gain Momentum With Five New Molecular Entities in Phase 3 Development in Fiscal Year 2024 OSAKA, Japan & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Takeda (TSE:4502/NYSE:TAK) today announced positive topline results from a Phase 2, randomized, double-blind, placebo-controlled study evaluating the safety, tolerability and efficacy of mezagitamab (TAK-079) in patients with persistent or chronic primary immune thrombocytopenia (ITP). Mezagitamab is a fully human immunoglobulin IgG1 monoclonal antibody (mAb) with high affinity for CD38 expressing cells (including plasmablasts, plasma cells, natural killer cells) resulting in their depletion. The TAK-079-1004 trial (NCT04278924) evaluated three different doses of subcutaneous mezagitamab vs placebo, given once weekly for eight weeks in patients with chronic (more than one year in duration) or persistent (3-12 months in duration) primary ITP. An interim analysis of the ongoing Phase 2 study demonstrated positive safety and efficacy results. Mezagitamab has been generally safe and well tolerated across all three cohorts. All mezagitamab doses tested demonstrated a higher platelet response rate than placebo. The increases in platelet count were dose-dependent with the greatest platelet response observed at the highest dose tested. Platelet response in mezagitamab treated patients occurred rapidly and was maintained post-therapy. Based on these positive results, and following consultation with global health authorities, Takeda plans to initiate a global Phase 3 trial of mezagitamab in ITP in fiscal year 2024. When mezagitamab advances to Phase 3 in ITP Takeda will have five new molecular entities in Phase 3 development in fiscal year 2024 including TAK-279 for the treatment of psoriasis and psoriatic arthritis, TAK-861 for the treatment of narcolepsy type 1, soticlestat for the treatment of Lennox-Gastaut syndrome and Dravet syndrome, and fazirsiran for the treatment of α1-antitrypsin associated liver disease. ITP is a rare, IgG mediated autoimmune disease caused, in part, by the development of autoantibodies to platelets (and/or megakaryocytes), which are blood cells responsible for preventing or stopping bleeding. ITP is characterized by the accelerated destruction of platelets (with or without impaired production), resulting in a decreased platelet count and an increased risk of bleeding, which can be debilitating (including fatigue and impaired quality of life), and in severe cases may be life-threatening. The precedent for approval of new drugs in this indication requires that platelet counts be maintained at 50,000/uL or more for a sustained period. Approximately 20 percent of patients with ITP do not achieve a platelet count above 50,000/uL following treatment with available first- and second-line therapies creating significant patient burden and unmet need for a disease modifying treatment that is also tolerable.1,2 “These Phase 2 results demonstrate mezagitamab’s compelling disease modifying mechanism of action, which has the potential to achieve disease remission for people with ITP. There remains considerable unmet need among ITP patients who may not respond or have inadequate response to prior treatment. In addition, some patients who do respond to available therapies struggle to manage side effects or relapse despite treatment,” said Chinwe Ukomadu, Head of the Gastrointestinal & Inflammation Therapeutic Area Unit at Takeda. “We look forward to initiating the Phase 3 trial and presenting these data at an upcoming scientific congress.” Therapy with mezagitamab is designed to deliver rapid and sustained improvement in platelet response and generally rapidly restores platelet counts to functional levels. Mezagitamab previously received Orphan Drug Designation for the treatment of ITP from the U.S. Food and Drug Administration and the program recently received Fast Track Designation. Results from the Phase 2 trial have no impact on the full year consolidated reported forecast for the fiscal year ending March 31, 2024 (Fiscal Year 2023). About Takeda Takeda is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience, and vaccines. Together with our partners, we aim to improve the patient experience and advance a new frontier of treatment options through our dynamic and diverse pipeline. As a leading values-based, R&D-driven biopharmaceutical company headquartered in Japan, we are guided by our commitment to patients, our people and the planet. Our employees in approximately 80 countries and regions are driven by our purpose and are grounded in the values that have defined us for more than two centuries. For more information, visit . 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These forward-looking statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those expressed or implied by the forward-looking statements: the economic circumstances surrounding Takeda’s global business, including general economic conditions in Japan and the United States; competitive pressures and developments; changes to applicable laws and regulations, including global health care reforms; challenges inherent in new product development, including uncertainty of clinical success and decisions of regulatory authorities and the timing thereof; uncertainty of commercial success for new and existing products; manufacturing difficulties or delays; fluctuations in interest and currency exchange rates; claims or concerns regarding the safety or efficacy of marketed products or product candidates; the impact of health crises, like the novel coronavirus pandemic, on Takeda and its customers and suppliers, including foreign governments in countries in which Takeda operates, or on other facets of its business; the timing and impact of post-merger integration efforts with acquired companies; the ability to divest assets that are not core to Takeda’s operations and the timing of any such divestment(s); and other factors identified in Takeda’s most recent Annual Report on Form 20-F and Takeda’s other reports filed with the U.S. Securities and Exchange Commission, available on Takeda’s website at: or at . Takeda does not undertake to update any of the forward-looking statements contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule. Past performance is not an indicator of future results and the results or statements of Takeda in this press release may not be indicative of, and are not an estimate, forecast, guarantee or projection of Takeda’s future results. Medical Information This press release contains information about products that may not be available in all countries, or may be available under different trademarks, for different indications, in different dosages, or in different strengths. Nothing contained herein should be considered a solicitation, promotion or advertisement for any prescription drugs including the ones under development. References Provan D, Donald A, et al. Blood Advances. 2019;26;3(22):3780-3817. Rodeghiero F. International Journal of Hematology. 2023;117:316–33.

Bexicaserin (LP352) Phase 1b/2a PACIFIC Study positive topline data in participants with Developmental and Epileptic Encephalopathies (DEEs) was reported in January Announcing an update to the primary efficacy endpoint data previously reported in January, which show further improvement in seizure reductions and no change in the reported safety results – bexicaserin achieved a median seizure reduction of 59.8% in countable motor seizures compared to 17.4% in the placebo group across the DEE study population. A median seizure reduction of 74.6% in Dravet Syndrome (DS), 50.8% in Lennox-Gastaut Syndrome (LGS) and 65.5% in DEE Other was achieved PACIFIC data to be presented at medical meetings in Q2 2024 Preparing for End of Phase 2 Meeting with regulators and conducting start-up activities for bexicaserin’s global Phase 3 program; expect to initiate the Phase 3 program by YE 2024 LP659 first-in-human Phase 1 single-ascending dose (SAD) study topline data expected Q2 2024 Completed public offering of common stock with gross proceeds of $241.5 million LA JOLLA, Calif.--(BUSINESS WIRE)-- Longboard Pharmaceuticals, Inc. (Nasdaq: LBPH), a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases, today provided a corporate update and reported full year 2023 financial results. “I am extremely proud of what our team has accomplished starting with the immense effort that went into the PACIFIC Study in participants with DEEs. We are impressed with the data in this study showing evidence of a potentially clinically meaningful benefit for both overall median seizure reduction and seizure reduction across all subgroups for Dravet, LGS and DEE Other. We are motivated by the enthusiasm and anticipation from the DEE community for our Phase 3 program and look forward to providing additional details later in the year. With the completion of our recent financing, we believe we are well positioned to deliver on key milestones later this year, including presenting additional topline and open-label extension data from PACIFIC, conducting our End of Phase 2 Meeting with the FDA, and initiating our global Phase 3 program. We appreciate the tremendous support from our existing and new shareholders who play an integral part in the continued success of Longboard. “We also look forward to Phase 1 topline SAD data next quarter from our second clinical-stage asset, LP659, an oral, centrally acting, highly selective S1P receptor modulator,” stated Kevin R. Lind, Longboard’s President and Chief Executive Officer. PACIFIC STUDY UPDATE In January 2024, we announced positive topline data from the Phase 1b/2a PACIFIC Study evaluating bexicaserin (LP352) in 52 participants with a broad range of DEEs, including DS (4), LGS (29) and other DEEs (19). Of the 52 participants enrolled in the study, 43 participants were randomized to bexicaserin (DS=4, LGS=24, DEE Other=15) and 9 to placebo (DS=0, LGS=5, DEE Other=4). Of note, results were on top of current standard of care; participants were typically on 3-4 other anti-seizure medications. Following our review of the full data set, we are announcing an update to the previously reported primary efficacy endpoint data. The updated data, which show even further improvements in seizure reductions and do not change the reported safety results, reflect corrections made by the study’s contract research organization to their statistical programming errors. The following table outlines the revisions: Revised Previously Reported Median percent change from baseline in countable motor seizure frequency: Bexicaserin Placebo Delta Bexicaserin Placebo Delta Overall 59.8% 17.4% 42.4% 53.3% 20.8% 32.5% DS 74.6% N/A N/A 72.1% N/A N/A LGS 50.8% 17.4% 33.4% 48.1% 20.8% 27.3% DEE Other 65.5% 32.2% 33.3% 61.2% 32.6% 28.6% UPCOMING MILESTONES: Bexicaserin (LP352), an oral, centrally acting, 5-HT2C superagonist in development for the potential treatment of seizures associated with DEEs PACIFIC data to be presented at medical meetings in Q2 2024 PACIFIC open-label extension (OLE) data expected in H2 2024 100% of PACIFIC completers entered into the OLE study Preparing for our End of Phase 2 Meeting with U.S. Food and Drug Administration (FDA) and aligning with other regulatory agencies Planning for Phase 3 initiation before YE 2024 LP659, an oral, centrally acting, S1P receptor subtypes 1 and 5 (S1P1,5) modulator in development for rare neuroinflammatory conditions Phase 1 SAD topline data expected in Q2 2024 FULL YEAR 2023 FINANCIAL RESULTS: Balance Sheet Highlights At December 31, 2023, Longboard’s cash, cash equivalents and short-term investments were approximately $48.5 million. On January 8, 2024, we completed a public offering of 11,500,000 shares of common stock and received gross proceeds of $241.5 million before deducting underwriting discounts and commissions of $14.5 million and offering expenses of $0.5 million. As of January 31, 2024, Longboard’s cash, cash equivalents and short-term investments were approximately $272.4 million. Operating Results Research and development expenses were $43.8 million for the year ended December 31, 2023, an increase of $9.2 million or 26.3%, compared to $34.6 million for the year ended December 31, 2022. The net increase of $9.2 million is primarily related to increases of $6.0 million in preclinical and clinical trial expenses related to bexicaserin, $2.7 million in personnel-related expenses, $0.5 million in other preclinical programs and early stage research expenses and $0.2 million of other miscellaneous expenses, offset by a decrease of $0.3 million in preclinical and clinical trial expenses related to LP659. General and administrative expenses were $13.0 million for the year ended December 31, 2023, an increase of $2.8 million or 28.0%, compared to $10.2 million for the year ended December 31, 2022. The net increase of $2.8 million is primarily related to increases of $1.7 million in personnel-related costs, $1.2 million of professional services and consulting expenses, and $0.4 million of other miscellaneous expenses, offset by a decrease of $0.5 million in insurance expense. ABOUT LONGBOARD PHARMACEUTICALS Longboard Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on developing novel, transformative medicines for neurological diseases. Longboard is working to advance a portfolio of centrally acting product candidates designed to be highly selective for specific G protein-coupled receptors (GPCRs). Longboard’s small molecule product candidates are based on more than 20 years of GPCR research. Longboard plans to advance bexicaserin (LP352), an oral, centrally acting 5-hydroxytryptamine 2C (5-HT2C) receptor superagonist, with no observed impact on 5-HT2B and 5-HT2A receptor subtypes, into a global Phase 3 program. Longboard recently reported positive topline data from a Phase 1b/2a clinical trial (the PACIFIC Study) evaluating bexicaserin in participants ages 12 to 65 years old with Developmental and Epileptic Encephalopathies (DEEs), including Lennox-Gastaut syndrome, Dravet syndrome and other DEEs. Longboard is also evaluating LP659, an oral, centrally acting, sphingosine-1-phosphate (S1P) receptor subtypes 1 and 5 modulator, which is in development for the potential treatment of rare neuroinflammatory conditions. Longboard is conducting a Phase 1 single-ascending dose (SAD) clinical trial for LP659 in healthy volunteers, with topline data expected in the second quarter of 2024. FORWARD-LOOKING STATEMENTS Certain statements in this press release are forward-looking statements that involve a number of risks and uncertainties. In some cases, you can identify forward-looking statements by words such as “to be”, “expect”, “focused on”, “anticipation”, “look forward”, “well positioned”, “plan”, “working to”, “designed to”, the negative, plural or other tenses of these words, references to specific future dates or time periods, or other comparable language, and they may include, without limitation, statements about the following: Longboard’s clinical and preclinical product candidates and programs, including their advancement (including plans for an End of Phase 2 Meeting and for alignment with other regulatory agencies and plans for a global Phase 3 program for bexicaserin), timing of study initiation (including for a global Phase 3 program for bexicaserin), timing of topline data (including for the PACIFIC OLE study for bexicaserin and the Phase 1 SAD study for LP659), their potential (including to be transformative, best-in-class, clinically meaningful or highly selective, the number and type of conditions they may address and their commercial opportunity), and their design and characteristics; upcoming presentations (including of additional PACIFIC topline data); Longboard’s cash position, expenses and runway to support operations; and Longboard’s focus and work. For such statements, Longboard claims the protection of the Private Securities Litigation Reform Act of 1995. Actual events or results may differ materially from Longboard’s expectations. Factors that could cause actual results to differ materially from those stated or implied by Longboard’s forward-looking statements include, but are not limited to, the following: risks related to Longboard’s limited operating history, financial position and need for additional capital; Longboard will need additional managerial and financial resources to advance all of its programs, and you and others may not agree with the manner Longboard allocates its resources; risks related to the development and commercialization of Longboard’s product candidates; Longboard’s product candidates are in the early phase of a lengthy research and development process, the timing, manner and outcome of research, development and regulatory review is uncertain, and Longboard’s product candidates may not advance in research or development or be approved for marketing; enrolling participants in Longboard’s ongoing and intended clinical trials is competitive and challenging; PACIFIC Study participants’ diagnoses are as of time of screening and are subject to change; risks related to unexpected or unfavorable new data; nonclinical and clinical data is voluminous and detailed, and regulatory agencies may interpret or weigh the importance of data differently and reach different conclusions than Longboard or others, request additional information, have additional recommendations or change their guidance or requirements before or after approval; results of clinical trials and other studies are subject to different interpretations and may not be predictive of future results; topline data may not accurately reflect the complete results of a particular study or trial; risks related to relying on licenses or collaborative arrangements; other risks related to Longboard’s dependence on third parties; competition; product liability or other litigation or disagreements with others; government and third-party payor actions, including relating to reimbursement and pricing; risks related to regulatory compliance; and risks related to Longboard’s and third parties’ intellectual property rights. Additional factors that could cause actual results to differ materially from those stated or implied by Longboard’s forward-looking statements are disclosed in Longboard’s filings with the Securities and Exchange Commission (SEC). These forward-looking statements represent Longboard’s judgment as of the time of this release. Longboard disclaims any intent or obligation to update these forward-looking statements, other than as may be required under applicable law. LONGBOARD PHARMACEUTICALS, INC. BALANCE SHEETS December 31, December 31, (in thousands, except share and per share data) 2023 2022 ASSETS Current assets: Cash and cash equivalents $ 14,331 $ 10,775 Short-term investments 34,167 56,814 Prepaid expenses and other current assets 1,723 2,249 Total current assets 50,221 69,838 Right-of-use assets 472 736 Property and equipment 4 9 Other long-term assets — 33 Total assets $ 50,697 $ 70,616 LIABILITIES AND EQUITY Current liabilities: Accounts payable $ 1,001 $ 1,310 Accrued research and development expenses 4,556 4,168 Accrued compensation and related expenses 3,374 2,438 Accrued other expenses 368 490 Right-of-use liabilities, current portion 475 358 Total current liabilities 9,774 8,764 Right-of-use liabilities, net of current portion — 382 Commitments and contingencies Stockholders' equity: Preferred stock, $0.0001 par value; authorized shares - 10,000,000 at December 31, 2023 and 2022, respectively; issued and outstanding shares - none at December 31, 2023 and 2022 — — Voting common stock, $0.0001 par value; authorized shares - 300,000,000 at December 31, 2023 and 2022, respectively; issued and outstanding shares - 22,096,494 and 13,585,950 at December 31, 2023 and 2022, respectively 2 1 Non-voting common stock, $0.0001 par value; authorized shares - 10,000,000 at December 31, 2023 and 2022, respectively; issued and outstanding shares - 2,420,755 and 3,629,400 at December 31, 2023 and 2022, respectively — — Additional paid-in capital 181,563 148,303 Accumulated other comprehensive loss (78 ) (692 ) Accumulated deficit (140,564 ) (86,142 ) Total stockholders' equity 40,923 61,470 Total liabilities and stockholders' equity $ 50,697 $ 70,616 LONGBOARD PHARMACEUTICALS, INC. STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS Year Ended December 31, (in thousands, except share and per share data) 2023 2022 Operating expenses: Research and development $ 43,752 $ 34,638 General and administrative 13,007 10,160 Total operating expenses 56,759 44,798 Loss from operations (56,759 ) (44,798 ) Interest income, net 2,405 837 Other income (expense) (68 ) 16 Net loss $ (54,422 ) $ (43,945 ) Net loss per share, basic and diluted $ (2.39 ) $ (2.56 ) Weighted-average shares outstanding, basic and diluted 22,726,325 17,150,907 Comprehensive loss: Net loss $ (54,422 ) $ (43,945 ) Unrealized gain (loss) on short-term investments 614 (528 ) Comprehensive loss $ (53,808 ) $ (44,473 )

Two pivotal Phase 3 clinical trials studying soticlestat as a treatment for Lennox-Gastaut syndrome (LGS) and Dravet syndrome (DS) completed enrollment; Takeda anticipates topline data readout by or before September 2024OV888 (GV101) is progressing on-track in a Phase 1, double-blind, multiple-ascending dose trial; a higher dose cohort has been added and no serious adverse events have been observed Feedback from a pre-IND session with the FDA supports potential initiation of a Phase 2 study of OV888 (GV101) in the second half of 2024Phase 1 study with OV329 continues to progress; data expected in the second half of 2024Cash, cash equivalents and marketable securities of $105.8 million as of December 31, 2023; expected to support runway into first half of 2026 NEW YORK, March 08, 2024 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc. (NASDAQ: OVID), a biopharmaceutical company dedicated to meaningfully improving the lives of people affected by rare epilepsies and brain conditions, today reported business updates and financial results for the fourth quarter and year ended December 31, 2023. “Ovid is progressing well and we are excited. This year we anticipate five clinical and regulatory milestones from our pipeline of novel compounds. Our ROCK2 collaboration with Graviton Bioscience is producing results faster than expected. We look forward to initiating a Phase 2 study of OV888 (GV101) in cerebral cavernous malformations (CCM) in the second half of this year. By year end 2024, we expect to deliver biomarker data from our oral OV329 program and investigational new drug submissions for intravenous formulations of both OV329 and OV350,” said Dr. Jeremy Levin, D. Phil, MB Chir, Chairman and CEO of Ovid. “Importantly, the first potential rare epilepsy medicine that Ovid helped shape, soticlestat, is expected to have two pivotal Phase 3 study topline readouts by Takeda. If the trials are successful, we believe a new and important medicine will be available to LGS and DS patients in 2025. If approved and successfully commercialized, Ovid will be eligible to receive regulatory and sales milestone payments as well as royalties on all future sales of soticlestat,” Dr. Levin added. Pipeline Updates Ovid anticipates five clinical and regulatory milestones this year from its own pipeline. In addition, Takeda is expected to read out results from two pivotal Phase 3 studies evaluating soticlestat. Soticlestat: Enrollment is complete for two pivotal Phase 3 trials studying soticlestat for Lennox-Gastaut syndrome and Dravet syndrome. Takeda reported that it anticipates Phase 3 data readouts for both trials by or before September 2024 and regulatory filings for soticlestat by or before March 31, 2025. Ovid co-developed soticlestat with Takeda through the completion of Phase 2 studies and subsequently sold its rights back to Takeda. Ovid retains significant financial interest in soticlestat. If soticlestat is approved and commercialized, Ovid is eligible to receive regulatory and commercial milestone payments of up to $660 million, as well as tiered low double-digit royalties up to 20% on global net sales from Takeda. Of these potential future payments, Ovid sold a 13% interest to Ligand Pharmaceuticals for $30 million in October 2023. Ovid retains 87% of its interest in any milestone payments and royalties and has no ongoing obligations or costs associated with the development of soticlestat. OV888 (GV101): Ovid and its collaborator Graviton Bioscience are currently progressing the study of OV888 (GV101) in a Phase 1 multiple-ascending dose study, which is expected to complete in the first half of 2024. No serious adverse events have been observed and a higher dose cohort has been added. OV888 (GV101) is a highly selective ROCK2 inhibitor for the potential treatment of CCM and other rare neurological conditions. Ovid and Graviton Bioscience expect to initiate a Phase 2 efficacy and safety study of OV888 (GV101) in patients living with CCM in the second half of 2024. Additionally, Ovid and Graviton Bioscience amended their collaboration agreement. Ovid paid $2.2 million to obtain an advanced gelcap formulation of OV888 (GV101), thereby enabling the acceleration of the development timeline toward Phase 2 trials. OV329: Ovid continues to advance two development programs for OV329, a potential next generation GABA-aminotransferase (GABA-AT) inhibitor. Both programs are anticipated to have milestones this year. Oral formulation An oral formulation of OV329 for the potential treatment of chronic seizures is being actively evaluated in a Phase 1 safety study, which includes surrogate biomarkers to measure target engagement and clinical efficacy. No safety signals were seen in single-ascending dose cohorts. The multiple-ascending dose study is designed to use magnetic resonance spectrometry and transcranial magnetic stimulation as biomarkers. This biomarker design is reinforced by preclinical data that Ovid presented at the American Epilepsy Society 2023 annual meeting. This preclinical data demonstrates that OV329 elicits an electroencephalogram response which is a potential pharmaco-dynamic marker of anti-convulsant activity. Data is expected from the Phase 1 study in late 2024, as is further preclinical data characterizing OV329's safety profile relative to vigabatrin.Intravenous (IV) formulationAn IV formulation of OV329 for potential treatment of acute seizures is on track for an anticipated IND application or an equivalent regulatory application by late 2024. The IV formulation is supported by emerging evidence that GABA-AT inhibition may be effective in the treatment of status epilepticus. Potassium chloride co-transporter 2 (KCC2) direct activator portfolio and OV350: Findings from several preclinical disease models indicate that Ovid's portfolio of KCC2 direct activator compounds hold therapeutic promise in multiple indications, including in seizures and psychosis. The initial program from the portfolio, which is an IV formulation of OV350, is anticipated to submit an IND by year-end 2024. In addition to OV350, Ovid is conducting non-clinical studies to characterize multiple compounds from its library of direct activators of KCC2 to assess them for therapeutic and formulation potential.Non-epilepsy indications for KCC2 direct activators may represent future collaborative development opportunities for Ovid. General Corporate & Business Updates Financial runway: Ovid anticipates its cash runway will support operations and clinical development programs into the first half of 2026. Ovid also anticipates several events for its current pipeline programs and clinical results for soticlestat from Takeda in 2024. To manage its capital resources, the Company leadership has executed several steps. For example, Ovid received $30 million of non-dilutive capital from Ligand in exchange for 13% of Ovid's interest in soticlestat’s potential future milestone payments and royalties. This capital is being directed toward anticipated high-value research and development activities.Ovid retains 87% of its interest in soticlestat’s future milestones and royalties, which is not reflected in its current runway guidance. If successfully commercialized, this may provide significant non-dilutive capital to further fund Ovid and its pipeline. Scientific strategy: Ovid is focused on the development of novel, targeted small molecules that have the potential to be first-in-class or best-in-class neurotherapeutics. The mechanisms of action in Ovid’s pipeline modulate extrinsic or intrinsic causes of neuronal hyperexcitability and function by acting on neurotransmitters, vascular structure causes of seizures or physiological properties within neurons. Hyperexcitability is implicated in many neurological indications, including seizures, psychiatric conditions, neurodevelopmental and neurodegenerative diseases, thus underscoring a broader therapeutic opportunity for Ovid’s pipeline assets.Business development activities: Ovid’s future business development efforts are focused on: exploring out-licensing opportunities for its genetic programs and elements of its patent estate that are not needed for Ovid's internal programs; potential collaborations to advance development opportunities for its KCC2 portfolio in non-core indications; and the exploration of commercial opportunities for some of its programs in territories outside the United States. Fourth Quarter and Annual 2023 Financial Results Cash, cash equivalents and marketable securities as of December 31, 2023 totaled $105.8 million.Revenue was $0.4 million for the year ended December 31, 2023, as compared to $1.5 million in the same period in 2022. Revenues were the result of royalty and licensing agreements.Research and development expenses were $10.6 million and $28.6 million for the fourth quarter and full year ended December 31, 2023, compared to $5.5 million and $24.6 million in the same periods in 2022. The increase is related to advancement of Ovid's clinical pipeline as described above, including the addition of the OV888 (GV101) Phase 1 MAD program and OV329 clinical development activities.General and administrative expenses were $7.7 million and $31.1 million for the fourth quarter and year ended December 31, 2023, as compared to $6.7 million and $32.4 million for the same periods in 2022.Total operating expenses were $18.3 million and $59.7 million for the fourth quarter and year ended December 31, 2023, as compared to $12.2 million and $57.1 million for the same periods last year.Ovid reported a net loss of $15.3 million, or basic and diluted net loss per share attributable to common stockholders of $0.22, for the fourth quarter of 2023, as compared to a net loss of $11.5 million, or basic and diluted net loss per share attributable to common stockholders of $0.16, for the same period in 2022. Ovid reported a net loss of $52.3 million, or basic and diluted net loss per share attributable to common stockholders of $0.74 for the year ended December 31, 2023, compared to a net loss of $54.2 million, or basic and diluted net loss per share attributable to common stockholders of $0.77, for the same period in 2022. About Ovid Therapeutics Ovid Therapeutics Inc. is a New York-based biopharmaceutical company that is dedicated to meaningfully improving the lives of people affected by certain epilepsies and brain conditions with seizure symptoms. The Company is advancing a pipeline of novel, targeted small molecule candidates that modulate the intrinsic and extrinsic factors involved in neuronal hyperexcitability causative of seizures and other neurological symptoms. Ovid is developing: OV888 (GV101), a potent and highly selective ROCK2 inhibitor, for the potential treatment of lesions associated with cerebral cavernous malformations and other brain disorders; OV329, a GABA-aminotransferase inhibitor, a potential therapy for treatment-resistant seizures; and OV350, a direct activator of the KCC2 transporter, for the potential treatment of epilepsies and other psychiatric conditions. Ovid also maintains a significant financial interest in the future regulatory development and potential commercialization of soticlestat, which Takeda is responsible for advancing globally. Soticlestat is a cholesterol 24-hydroxylase inhibitor, which is currently in Phase 3 trials for Dravet and Lennox-Gastaut syndromes. For more information about these and other Ovid research programs, please visit www.ovidrx.com. Forward-Looking Statements This press release includes certain disclosures by Ovid that contain “forward-looking statements,” including, without limitation: statements regarding the potential use and development of OV888 (GV101), OV329, OV350 and compounds from Ovid's library of direct activators of KCC2; the potential therapeutic opportunity of OV888 (GV101), OV329 and compounds from Ovid's library of direct activators of KCC2; the potential opportunity for soticlestat; the timing and data readout of Takeda’s two pivotal Phase 3 trials evaluating soticlestat for Dravet syndrome and Lennox-Gastaut syndrome; Ovid’s expectations regarding the duration of its cash runway and the expectation that it will support the advancement of Ovid’s pipeline and Ovid's potential future business development opportunities. You can identify forward-looking statements because they contain words such as “anticipates,” “expected,” “intends,” “may,” “potentially,” “seek,” and “will,” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances). Forward-looking statements are based on Ovid’s current expectations and assumptions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that may differ materially from those contemplated by the forward-looking statements, which are neither statements of historical fact nor guarantees or assurances of future performance. Important factors that could cause actual results to differ materially from those in the forward-looking statements include, without limitation, uncertainties inherent in the preclinical and clinical development and regulatory approval processes, risks related to Ovid’s ability to achieve its financial objectives, the risk that Ovid may not be able to realize the intended benefits of its technology or its business strategy, or risks related to Ovid’s ability to identify business development targets or strategic partners, to enter into strategic transactions on favorable terms, or to consummate and realize the benefits of any business development transactions. Additional risks that could cause actual results to differ materially from those in the forward-looking statements are set forth under the caption “Risk Factors” in Ovid’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (“SEC”) on November 3, 2023, and in future filings Ovid makes with the SEC. Any forward-looking statements contained in this press release speak only as of the date hereof, and Ovid assumes no obligation to update any forward-looking statements contained herein, whether because of any new information, future events, changed circumstances or otherwise, except as otherwise required by law. Condensed Consolidated Statements of OperationsUnaudited For the ThreeMonths EndedDecember 31,2023 For the ThreeMonths EndedDecember 31,2022 For the YearEndedDecember 31,2023 For the YearEndedDecember 31,2022Revenue: License and other revenue$141,562 $46,280 $391,695 $1,502,748 Total revenue 141,562 46,280 391,695 1,502,748 Operating expenses: Research and development 10,641,957 5,545,269 28,587,884 24,618,399 General and administrative 7,687,951 6,673,710 31,085,274 32,432,510 Total operating expenses 18,329,908 12,218,979 59,673,158 57,050,909 Loss from operations (18,188,346) (12,172,699) (59,281,464) (55,548,161)Other income (expense), net 2,866,312 668,124 6,942,505 1,379,132 Loss before provision for income taxes (15,322,035) (11,504,575) (52,338,959) (54,169,029)Provision for income taxes — — — — Net loss$(15,322,035) $(11,504,575) $(52,338,959) $(54,169,029)Net loss per share, basic$(0.22) $(0.16) $(0.74) $(0.77)Net loss per share, diluted$(0.22) $(0.16) $(0.74) $(0.77)Weighted-average common shares outstanding, basic 70,687,307 70,472,598 70,580,604 70,424,819 Weighted-average common shares outstanding, diluted 70,687,307 70,472,598 70,580,604 70,424,819 Select Condensed Balance Sheet DataUnaudited December 31, 2023 December 31, 2022Cash, cash equivalents and marketable securities$105,833,375 $129,001,411Working capital(1) 98,123,417 124,389,166Total assets 144,026,783 155,265,814Total stockholder's equity 87,796,887 132,272,564(1)Working capital defined as current assets less current liabilities Contacts Investors & Media:Garret BonneyIR@ovidrx.com617-735-6093