Ovid Therapeutics Reports Business Updates and Fourth Quarter and Full Year 2024 Financial Results

   -- Stelios Papadopolous, Ph.D., a pioneering leader in biotech, appointed to 
      Board of Directors; two industry veterans joined management team as Ovid 
      prepares to take OV329 into patient trials and move its first KCC2 direct 
      activator into the clinic 
 
   -- Topline results from Phase 1 study of OV329 expected in Q3 2025, which 
      will include biomarkers that measure clinical effect and target 
      engagement, safety, and tolerability 
 
   -- OV350, Ovid's first program in its KCC2 direct activator library, 
      initiated a first-in-human study in Q1 2025 
 
   -- Cash, cash equivalents and marketable securities of $53.1 million as of 
      December 31, 2024, are expected to support operations and development 
      programs into the second half of 2026 

NEW YORK, March 11, 2025 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc. (Nasdaq: OVID), a biopharmaceutical company dedicated to developing small molecule medicines for brain conditions with significant unmet need, today reported business updates and financial results for the fourth quarter and year ended December 31, 2024.

"Ovid is at an exciting inflection point. Our pipeline programs are moving into the next stage of clinical development and I believe our team is the strongest that it has been since the founding of the Company, " said Dr. Jeremy Levin, D.Phil., MB BChir., Chairman and CEO of Ovid Therapeutics. "To support us in capturing the substantial potential therapeutic and financial value associated with OV329, our program for treatment-resistant epilepsies, and our portfolio of first-in-class KCC2 activators, Dr. Stelios Papadopoulos has joined our Board and two industry leaders, Dr. Manal Morsy and Victoria "Tori" Fort joined our management team. Stelios's extensive industry expertise and strategic vision will be invaluable to Ovid through our next stage of growth. Manal and Tori will support our regulatory and investor strategy and engagement," stated Levin.

Business Strategy & Updates

Ovid expects its cash runway to support operations and clinical development programs into the second half of 2026, during which time multiple pipeline and regulatory milestones are anticipated. These anticipated milestones include: results for OV329 biomarker and safety data (Q3 2025); initiation of a Phase 2a patient study for OV329 in drug resistant epilepsies (Q1 2026); results from a first-in-human safety and exploratory biomarker study with OV350, our first KCC2 direct activator (Q4 2025); and initiation of human trials for the first oral KCC2 direct activator, OV4071 (Q2 2026). In the event the Company is unable to raise capital or enter into partnerships or co-development opportunities as and when needed, it will be required to delay, reduce the scope of or prioritize various research and development activities.

The Company will continue to manage its clinical development programs, operations and cash expenditures with fiscal discipline to support the potential achievement of key value-creating clinical milestones. Given the breadth and depth of its pipeline, and the broad therapeutic opportunity it may yield, Ovid will continue to explore partnerships and co-development opportunities for select programs and regions to accelerate development and offset costs. Additional pipeline updates follow below.

Organizational Updates

Ovid has strengthened its Board of Directors and leadership team with key appointments intended to support the Company's pipeline, business development and financial strategies. These include:

   -- Appointed Stelios Papadopolous, Ph.D., to serve on Board of Directors. 
      Dr. Papadopolous is a scientist, investor and entrepreneur who has played 
      a pivotal role in shaping the biotechnology industry for the last 30 
      years through bridging scientific discovery with financial strategy. His 
      leadership, deal-making creativity, and ability to catalyze growth will 
      be instrumental to Ovid as it seeks to capture the full potential value 
      of its pipeline programs. Dr. Papadopoulos has served in roles including: 
      the Vice Chairman of Cowen & Co., LLC; a leader in investment banking at 
      PaineWebber, Chairman of PaineWebber Development Corp., a subsidiary 
      focusing on biotechnology; and Vice President in the Equity Research 
      Department of Drexel Burnham Lambert, covering the biotechnology 
      industry. Dr. Papadopolous will serve on the Audit and Compensation 
      Committees of Ovid's Board of Directors. 
 
   -- Leadership appointments. In February 2025, Manal Morsy, M.D., Ph.D., MBA 
      and Victoria Fort joined Ovid as Chief Regulatory Officer and Head of 
      Corporate Affairs & Corporate Strategy, respectively. Dr. Morsy brings 
      deep expertise from successful global regulatory strategies and 
      submissions spanning three decades, including adult and pediatric 
      development drug candidates at J&J, Merck, PTC, and Athersys. She is a 
      trained scientist, clinician and geneticist whose work has been published 
      in many leading scientific and medical journals. Ms. Fort joins Ovid from 
      Frontier Medicines, where she led corporate strategy, guided the 
      company's transition to a clinical-stage organization and helped secure 
      critical financing. 

Pipeline Strategy & Updates

Ovid is advancing a differentiated pipeline of potential first-in-class and best-in-class small molecule medicines that are intended to bring hyperexcited neurons back to homeostasis. The Company seeks to become a leader in neurotherapeutics by addressing intractable brain disorders with significant unmet need. This includes treating conditions and symptoms that manifest from excessive neural excitation such as seizures and psychosis.

Central to the Company's strategy is identifying and developing differentiated mechanisms of action to interdict unaddressed biological targets in the central nervous system $(CNS)$ that are fundamental to disease pathology. This strategy includes developing a potential best-in-class, next-generation GABA aminotransferase (GABA-AT) inhibitor; translating the direct activation of the potassium chloride co-transporter 2 (KCC2) for a broad range of neurological and psychiatric conditions; and pioneering Rho-associated coiled-coil containing protein kinase 2 (ROCK2) inhibition for neurovascular and neuro-inflammatory conditions.

OV329

   -- A next-generation GABA-AT inhibitor: Ovid is developing OV329 as a 
      next-generation GABA-AT inhibitor for the potential treatment of 
      drug-resistant epilepsies (DREs) in adult and pediatric patients. OV329 
      seeks to endogenously deliver optimal levels of GABA to reduce seizures 
      and provide a preferable safety and tolerability profile relative to the 
      first-generation, GABA-AT inhibitor, vigabatrin. Ovid's preclinical 
      characterization suggests that OV329 is 100-fold more potent than 
      vigabatrin in animals, delivers synaptic and extra-synaptic inhibition, 
      and has a lasting pharmacodynamic effect despite rapid tissue clearance. 
      In animal models, OV329 has been shown to have a therapeutic index and 
      does not appear to induce sedation at therapeutic doses, whereas 
      vigabatrin has no therapeutic window. A therapeutic dose of vigabatrin 
      has been shown by independent researchers, and by Ovid, to preferentially 
      accumulate in the retina. 
 
   -- Human safety & tolerability: Ovid is completing a Phase 1 single- 
      ascending dose (SAD) and multiple ascending dose $(MAD.AU)$ study evaluating 
      OV329's safety, tolerability, pharmacokinetics $(PK)$ and biomarkers that 
      may serve as a measure of clinical effect and target engagement. In the 
      cohorts completed to-date, there have been no serious adverse events and 
      no adverse events reported have been associated with OV329. 
 
   -- Topline biomarker & safety data expected Q3 2025: Ovid added a higher 
      dose cohort to its Phase 1 study, to increase dosing opportunities for 
      future Phase 2 programs. Topline results from this cohort is expected in 
      Q3 2025, and will include findings regarding how OV329 performed in 
      specific parameters of transcranial magnetic stimulation $(TMS.AU)$ and 
      magnetic resonance spectroscopy (MRS). Collectively, Ovid believes TMS 
      and MRS have the potential to detect GABAergic activity, target 
      engagement and clinical effect. 
 
          -- TMS is a proven protocol for focal non-invasive electrical 
             cortical stimulation that can safely and non-invasively measure 
             target engagement. TMS has been previously used to correlate 
             anti-seizure activity with vigabatrin and other anti-seizure 
             medications ("ASMs"). Ovid is utilizing a range of TMS parameters 
             for OV329, which can measure cortical excitation, including 
             GABA-ergic, GABA $(A)$ and GABA (B) receptor activity1. 
 
          -- MRS technology has been utilized with other ASMs, including 
             vigabatrin, as a way to measure GABA levels in selected regions of 
             the brain. Utilizing MRS, Ovid will be able to evaluate and 
             quantify the change in GABA levels in the medial parietal lobe 
             relative to an untreated baseline. 
 
   -- Ocular safety profile: To evaluate OV329's safety compared to vigabatrin, 
      Ovid has conducted additional animal experiments to evaluate the risk of 
      drug accumulating in the eye and causing retinal cell dysregulation. At 
      the 2024 American Epilepsy Society meeting in December, Ovid presented 
      the full results from an animal study, which demonstrated that OV329 did 
      not accumulate in animal eyes, in contrast to vigabatrin, which was found 
      to accumulate in less than 48 hours. OV329 was present in the brain and 

      plasma of mice, then rapidly cleared the tissue and remained undetectable 
      in the retina, eye, and brain. In contrast, vigabatrin was demonstrated 
      to preferentially accumulate in the eye, retina and brain, which is 
      consistent with previously published independent research. 

OV350 and KCC2 library

   -- Initiated a Phase 1, first-in-human study for a KCC2 direct activator, 
      OV350: In Q4 2024, Ovid successfully submitted a regulatory application 
      for a Phase 1 trial of OV350 and initiated a first-in-human study for 
      this class of molecule in Q1 2025. OV350 is the first of multiple 
      anticipated programs from Ovid's library of KCC2 direct activators. The 
      Phase 1 trial intends to evaluate the safety, tolerability and 
      pharmacokinetic parameters of an intravenous formulation of OV350 in 
      healthy human volunteers. It will also include an exploratory biomarker 
      using quantitative EEG. In preclinical and animal disease models, OV350 
      has demonstrated antipsychotic and anticonvulsant effects, indicating 
      that it may have broad therapeutic utility. The initial indication 
      intended for OV350, and the oral program to follow, is psychosis 
      associated with neuronal-synuclein diseases (NSD), including Parkinson's 
      disease, and Lewy body dementia (LBD). These conditions have similar 
      underlying biology, significant unmet need and an established regulatory 
      pathway. 
 
   -- KCC2 direct activator library. Ovid has made significant progress with 
      its KCC2 direct activator library and is progressing four programs toward 
      clinical development, which are expected to yield successive regulatory 
      submissions annually for the next three to four years. These programs are 
      unique structures with discreet pharmacology and differentiated 
      therapeutic attributes as characterized in phenotypic and disease biology 
      models. Ovid believes these molecules may have relevance across a range 
      of conditions that have symptoms driven by neural hyperexcitability such 
      as seizures and psychoses. Based upon the bioavailability and potency 
      characteristics, Ovid believes different programs in the library have 
      amenability for oral and intramuscular or subcutaneous formulations to 
      address clinical indications across the care continuum. 
 
   -- OV4071, the first oral KCC2 direct activator: Ovid has initiated 
      IND-enabling studies of OV4071, the first oral direct activator of the 
      biological target, KCC2. It anticipates initiating human studies with 
      OV4071 in Q2 2026, with both healthy volunteer and patient cohorts. This 
      oral program behaves similarly to OV350 in phenotypic and disease model 
      screens and is similarly intended for the potential treatment of 
      psychoses in NSD and LBD. 

OV888/GV101 & ROCK2 inhibitor programs

   -- OV888/GV101 Capsule for CCM: In the third quarter of 2024, Ovid and 
      Graviton Bioscience announced the decision to pause the initiation of the 
      Phase 2 proof-of-concept study of OV888/ GV101 capsule, following the 
      recent completion of long-duration competitor and academic trials in CCM, 
      and after discussions with key stakeholders. Ovid and Graviton Bioscience 
      are evaluating clinical design learnings and regulatory feedback from 
      recently completed competitor Phase 2 programs. Ovid and Graviton 
      Bioscience are confident of the potential therapeutic effects of ROCK2 
      inhibition for CCM and will seek to optimize future development 
      approaches with the benefit of further insights on study duration, 
      enrichment strategies, endpoints, and time-to-event measurements. 

Fourth Quarter and Annual 2024 Financial Results

   -- Cash, cash equivalents and marketable securities as of December 31, 2024 
      totaled $53.1 million. 
 
   -- Revenues from royalty agreements were $566,000 for the year ended 
      December 31, 2024, as compared to $392,000 for the same period in 2023. 
 
   -- Research and development expenses were $5.9 million and $36.8 million for 
      the three months and full year ended December 31, 2024, compared to $10.6 
      million and $28.6 million for the same periods in 2023. The overall 
      increase is related to the advancement of Ovid's clinical and preclinical 
      pipeline programs as described above. The decrease between the 
      three-month periods is the result of the organizational restructuring in 
      the second quarter of 2024, which re-prioritized development programs. 
 
   -- General and administrative expenses were $4.9 million and $25.7 million 
      for the three months and full year ended December 31, 2024, as compared 
      to $7.7 million and $31.1 million for the same periods in 2023. The 
      decrease was driven by the previous organizational restructuring and 
      related cost-reduction efforts. 
 
   -- Total operating expenses were $10.8 million and $62.5 million for the 
      three months and full year ended December 31, 2024, as compared to $18.3 
      million and $59.7 million for the same periods in 2023. 
 
   -- Ovid reported a net loss of $9.3 million, or basic and diluted net loss 
      per share attributable to common stockholders of $0.13 for the three 
      months ended December 31, 2024, as compared to a net loss of $15.3 
      million, or basic and diluted net loss per share attributable to common 
      stockholders of $0.21 for the same period in 2023. Ovid reported a net 
      loss of $26.4 million, or basic and diluted net loss per share 
      attributable to common stockholders of $0.37 for the year ended 
      December 31, 2024, as compared to a net loss of $52.3 million, or basic 
      and diluted net loss per share attributable to common stockholders of 
      $0.73 for the same period in 2023. 

(1) Tsuboyama M, Lee Kaye H, Rotenberg A. Biomarkers Obtained by Transcranial Magnetic Stimulation of the Motor Cortex in Epilepsy.

Front Integr Neurosci. 2019 Oct 30;13:57. doi: 10.3389/fnint.2019.00057. PMID: 31736722; PMCID: PMC6837164.

About Ovid Therapeutics

Ovid Therapeutics Inc. is a New York-based biopharmaceutical company dedicated to developing small molecule medicines for brain conditions with significant unmet need. The Company is advancing a pipeline of novel, highly specific, small molecule candidates that modulate the intrinsic and extrinsic factors involved in neuronal hyperexcitability causative of multiple neurological and neuropsychiatric disorders. Ovid is developing: OV329, a next-generation GABA-aminotransferase inhibitor, as a potential therapy for treatment-resistant seizures and other undisclosed indications; OV350, and a library of compounds that directly activate the KCC2 transporter, for multiple neurological and psychiatric conditions; and OV888/GV101, a highly selective ROCK2 inhibitor for undisclosed neurovascular and neuro-inflammatory conditions. 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 expected timing of initiation, completion, and results and data of Ovid's clinical studies; Ovid's expectations regarding the duration of its cash runway and the expectation that it will support Ovid's operations and development programs; Ovid's ability to achieve expected benefits of cost-savings efforts; Ovid's ability to secure additional sources of funding; Ovid's potential future business development opportunities; the potential use and development of OV329, OV350 and other compounds from Ovid's library of direct activators of KCC2, and OV888/GV101; the potential therapeutic opportunity of OV329, OV350 and other compounds from Ovid's library of direct activators of KCC2, and OV888/GV101; Ovid's evaluation of the results of recently completed competitor trials to OV888/GV101 for CCM; and other statements that are not historical fact. You can identify forward-looking statements because they contain words such as "anticipates," "believes," "expects," "intends," "may," "plan," "potentially," 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, impediments to Ovid's ability to achieve expected benefits of cost-savings efforts, 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 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 most recently filed Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission ("SEC"), and in subsequent and 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 Operations 
                                    Unaudited 
                    For the Three    For the Three   For the Year 
 (in thousands,     Months Ended     Months Ended       Ended       For the Year 
except share and    December 31,     December 31,    December 31,  Ended December 
 per share data)        2024             2023            2024         31, 2023 
                   ---------------  ---------------  ------------  -------------- 
Revenue: 
  License and 
   other revenue   $        76      $       142      $       566   $       392 
      Total 
       revenue              76              142              566           392 
Operating 
expenses:                                                     --            -- 
  Research and 
   development           5,923           10,642           36,767        28,588 
  General and 
   administrative        4,878            7,688           25,684        31,085 
                    ----------       ----------       ----------    ---------- 
      Total 
       operating 
       expenses         10,801           18,330           62,451        59,673 
Loss from 
 operations            (10,725)         (18,188)         (61,885)      (59,281) 
Other income 
 (expense), net          1,444            2,866           35,452         6,943 
                    ----------       ----------       ----------    ---------- 
Loss before 
 provision for 
 income taxes           (9,281)         (15,322)         (26,433)      (52,339) 
Provision for 
income taxes                --               --               --            -- 
                    ----------       ----------       ----------    ---------- 
Net loss           $    (9,281)     $   (15,322)     $   (26,433)  $   (52,339) 
                    ==========       ==========       ==========    ========== 
Net loss per 
 share of Series 
 A preferred 
 stock, basic and 
 diluted           $   (128.44)     $   (212.99)     $   (366.33)  $   (728.64) 
Weighted-average 
 Series A 
 preferred stock 
 shares 
 outstanding, 
 basic and 
 diluted                 1,250            1,250            1,250         1,250 
Net loss per 
 share of common 
 stock, basic and 
 diluted           $     (0.13)     $     (0.21)     $     (0.37)  $     (0.73) 
Weighted-average 
 common stock 
 shares 
 outstanding, 
 basic and 
 diluted            71,009,866       70,687,307       70,905,422    70,580,604 
 
 
              Select Condensed Consolidated Balance Sheet Data 
                                  Unaudited 
          (in thousands)             December 31, 2024    December 31, 2023 
                                    -------------------  ------------------- 
Cash, cash equivalents and 
 marketable securities                $          53,075    $         105,834 
Working capital(1)                               45,418               98,125 
Total assets                                     92,167              144,027 
Total stockholders' equity                       68,226               87,797 
  (1) Working capital defined as current assets less 
   current liabilities 
 

Contacts

Investor Relations & Media

Victoria Fort

VFort@ovidrx.com

(END) Dow Jones Newswires

March 11, 2025 08:00 ET (12:00 GMT)