Ovid Therapeutics (OVID) - 2025 Q2 - Quarterly Report

Special Note Regarding Forward-Looking Statements This section identifies forward-looking statements, outlines factors causing actual results to differ, and notes the company's non-obligation to update them Forward-Looking Statements This section identifies forward-looking statements, outlines factors causing actual results to differ, and notes the company's non-obligation to update them - Forward-looking statements are identified by terms such as 'aim,' 'anticipate,' 'believe,' 'expect,' 'intend,' 'may,' 'plan,' 'potential,' 'project,' 'should,' 'will,' 'would,' and similar expressions[9] - Key factors that may cause actual results to differ materially include the ability to acquire or in-license novel compounds, successful development and therapeutic potential of drug candidates, regulatory approval, timing of clinical trials, market size, funding, intellectual property disputes, and the impact of geopolitical tensions and economic conditions[10] - The company assumes no obligation to update or revise these forward-looking statements, even if new information becomes available[11] PART I. Financial Information Item 1. Financial Statements (Unaudited) This section presents Ovid Therapeutics Inc.'s unaudited condensed consolidated financial statements and detailed notes on accounting policies Condensed Consolidated Balance Sheets The condensed consolidated balance sheets provide a snapshot of the company's financial position as of June 30, 2025, and December 31, 2024, showing a decrease in total assets and stockholders' equity, primarily driven by a reduction in marketable securities and an increase in accumulated deficit Condensed Consolidated Balance Sheets (in thousands) | Item | June 30, 2025 | December 31, 2024 | | :-------------------------------- | :------------ | :---------------- | | Cash and cash equivalents | $28,415 | $26,301 | | Marketable securities | $9,932 | $26,774 | | Total current assets | $42,089 | $55,940 | | Total assets | $77,428 | $92,167 | | Total current liabilities | $8,924 | $10,522 | | Total liabilities | $21,639 | $23,941 | | Total stockholders' equity | $55,789 | $68,226 | | Accumulated deficit | $(319,218) | $(304,299) | Condensed Consolidated Statements of Operations Revenue significantly increased for the three and six months ended June 30, 2025, driven by a licensing agreement, resulting in a net loss for 2025 Condensed Consolidated Statements of Operations (in thousands) | Item | 3 Months Ended June 30, 2025 | 3 Months Ended June 30, 2024 | 6 Months Ended June 30, 2025 | 6 Months Ended June 30, 2024 | | :----------------------------------- | :--------------------------- | :--------------------------- | :--------------------------- | :--------------------------- | | Total revenue | $6,272 | $169 | $6,402 | $317 | | Research and development | $6,465 | $12,582 | $13,123 | $22,984 | | General and administrative | $4,880 | $8,104 | $10,902 | $15,267 | | Total operating expenses | $11,345 | $20,686 | $24,025 | $38,251 | | Loss from operations | $(5,073) | $(20,517) | $(17,623) | $(37,934) | | Other income (expense), net | $389 | $29,038 | $2,704 | $34,760 | | Net (loss) income | $(4,684) | $8,521 | $(14,919) | $(3,174) | | Net (loss) income per share of common stock, basic | $(0.06) | $0.12 | $(0.21) | $(0.04) | Condensed Consolidated Statements of Comprehensive Loss The condensed consolidated statements of comprehensive loss show a comprehensive loss for the three and six months ended June 30, 2025, primarily driven by the net loss, with minor impacts from cumulative translation adjustments and unrealized losses on marketable securities Condensed Consolidated Statements of Comprehensive (Loss) Income (in thousands) | Item | 3 Months Ended June 30, 2025 | 3 Months Ended June 30, 2024 | 6 Months Ended June 30, 2025 | 6 Months Ended June 30, 2024 | | :----------------------------------- | :--------------------------- | :--------------------------- | :--------------------------- | :--------------------------- | | Net (loss) income | $(4,684) | $8,521 | $(14,919) | $(3,174) | | Cumulative translation adjustment | $(11) | — | $(31) | — | | Unrealized (loss) gain on marketable securities | $(1) | $7 | $(3) | $(13) | | Comprehensive (loss) income | $(4,696) | $8,528 | $(14,953) | $(3,187) | Condensed Consolidated Statements of Stockholders' Equity The condensed consolidated statements of stockholders' equity detail changes in equity components, showing a decrease in total stockholders' equity from December 31, 2024, to June 30, 2025, primarily due to net losses and other comprehensive losses, partially offset by stock-based compensation expense Condensed Consolidated Statements of Stockholders' Equity (in thousands) | Item | Balance, Dec 31, 2024 | Balance, June 30, 2025 | | :----------------------------------- | :-------------------- | :--------------------- | | Common Stock (shares) | 71,009,866 | 71,109,514 | | Common Stock (amount) | $71 | $71 | | Additional Paid-In Capital | $372,489 | $375,005 | | Accumulated Other Comprehensive Loss | $(35) | $(69) | | Accumulated Deficit | $(304,299) | $(319,218) | | Total Stockholders' Equity | $68,226 | $55,789 | - Issuance of common stock from exercise of stock options and purchases from employee stock purchase plan contributed $13 thousand to additional paid-in capital for the six months ended June 30, 2025[22] - Stock-based compensation expense totaled $2,503 thousand for the six months ended June 30, 2025[22] Condensed Consolidated Statements of Cash Flows The condensed consolidated statements of cash flows indicate a net increase in cash, cash equivalents, and restricted cash for the six months ended June 30, 2025, primarily driven by cash provided by investing activities, which offset significant cash used in operating activities Condensed Consolidated Statements of Cash Flows (in thousands) | Item | 6 Months Ended June 30, 2025 | 6 Months Ended June 30, 2024 | | :----------------------------------- | :--------------------------- | :--------------------------- | | Net cash used in operating activities | $(15,054) | $(30,941) | | Net cash provided by investing activities | $17,155 | $33,010 | | Net cash provided by financing activities | $13 | $584 | | Net increase in cash, cash equivalents and restricted cash | $2,114 | $2,653 | | Cash, cash equivalents and restricted cash, at end of period | $30,346 | $31,625 | Notes to Unaudited Condensed Consolidated Financial Statements These notes explain the company's financial statements, covering operations, accounting policies, assets, liabilities, equity, and agreements NOTE 1 – Nature of Operations Ovid Therapeutics Inc. is a biopharmaceutical company focused on developing small molecule medicines for brain conditions. The company has historically incurred losses and negative operating cash flows, raising substantial doubt about its ability to continue as a going concern, necessitating additional funding or strategic alternatives - Ovid Therapeutics Inc. is a biopharmaceutical company developing small molecule medicines for brain conditions with significant unmet need[26] - The company has an accumulated deficit of $319.2 million as of June 30, 2025, and has incurred recurring losses and negative operating cash flows[29] - Management believes existing cash, cash equivalents, and marketable securities ($38.3 million as of June 30, 2025) will not be sufficient to fund operations for the next 12 months, raising substantial doubt about its ability to continue as a going concern[29][30] NOTE 2 – Summary of Significant Accounting Policies This note details the accounting principles for financial statements, covering interim nature, consolidation, estimates, and policies for various financial items - The interim condensed consolidated financial statements are unaudited and prepared in accordance with GAAP and SEC interim reporting requirements, with certain notes condensed or omitted[35] - Marketable securities, primarily U.S. treasury instruments, are classified as available-for-sale, with unrealized gains/losses reported in accumulated other comprehensive (loss) income[38] - The company expenses research and development costs as incurred, including clinical trial costs, manufacturing, contracted services, and license fees[47] - The company accounts for stock-based compensation using the Black-Scholes valuation model and recognizes expense over the vesting period[49] - Revenue is recognized when the customer obtains control of promised goods or services, reflecting the consideration expected, with upfront license fees deferred until performance obligations are satisfied[56][59] NOTE 3 – Cash, Cash Equivalents and Marketable Securities This note provides a detailed breakdown of the company's cash, cash equivalents, and marketable securities, categorized by fair value hierarchy levels, showing a decrease in total holdings from December 31, 2024, to June 30, 2025 Fair Value of Cash, Cash Equivalents and Marketable Securities (in thousands) | Item | June 30, 2025 Fair Value | December 31, 2024 Fair Value | | :----------------------------------- | :--------------------------- | :--------------------------- | | Cash | $7,679 | $522 | | Cash equivalents | $20,736 | $25,779 | | Marketable securities | $9,932 | $26,774 | | Total | $38,347 | $53,075 | - The company's Level 1 assets (unadjusted quoted prices in active markets) primarily consisted of investments in a U.S. treasury money market fund, totaling $20.7 million as of June 30, 2025[43] - Level 2 assets (observable inputs other than Level 1 quoted prices) consisted of U.S. treasury bills, totaling $9.9 million as of June 30, 2025[43] NOTE 4 – Property and Equipment and Intangible Assets This note details the company's property and equipment, net, and intangible assets, net, showing a decrease in both categories from December 31, 2024, to June 30, 2025, due to accumulated depreciation and amortization Property and Equipment, Net (in thousands) | Item | June 30, 2025 | December 31, 2024 | | :----------------------------------- | :------------ | :---------------- | | Furniture and equipment | $1,534 | $1,534 | | Leasehold improvements | $306 | $306 | | Less accumulated depreciation | $(1,555) | $(1,407) | | Total property and equipment, net | $285 | $433 | - Intangible assets, net, decreased from $219 thousand as of December 31, 2024, to $0 as of June 30, 2025[68] - Depreciation expense was $61 thousand for the three months ended June 30, 2025, and $148 thousand for the six months ended June 30, 2025[67] NOTE 5 – Leases This note outlines the company's operating lease for its corporate headquarters, detailing the ROU asset and lease liabilities, remaining lease term, and future minimum commitments. The lease has a 10-year term commencing March 2022, with an annual base rent of $2.3 million - The company has a 10-year operating lease for its corporate headquarters, with a remaining term of approximately seven years and an annual base rent of $2.3 million[69][70] ROU Asset and Lease Liabilities (in thousands) | Item | June 30, 2025 | December 31, 2024 | | :----------------------------------- | :------------ | :---------------- | | ROU asset, net | $12,215 | $12,797 | | Current lease liability | $1,384 | $1,336 | | Long-term lease liability | $12,715 | $13,419 | Future Minimum Commitments Under Non-Cancelable Operating Lease (in thousands) | Year | Amount | | :----------------------------------- | :----- | | 2025 | $1,158 | | 2026 | $2,316 | | 2027 | $2,316 | | 2028 | $2,469 | | 2029 | $2,469 | | Thereafter | $7,408 | | Total | $18,136 | NOTE 6 – Accrued Expenses This note provides a breakdown of accrued expenses, showing a significant decrease from December 31, 2024, to June 30, 2025, primarily driven by reductions in payroll and bonus accruals and research and development accruals Accrued Expenses (in thousands) | Item | June 30, 2025 | December 31, 2024 | | :----------------------------------- | :------------ | :---------------- | | Payroll and bonus accrual | $1,838 | $2,959 | | Research and development accrual | $485 | $2,779 | | Professional fees accrual | $265 | $168 | | Other | $284 | $88 | | Total | $2,872 | $5,994 | NOTE 7 – Stockholders' Equity This note details the company's capital structure, including common and Series A Preferred Stock, authorized shares, voting rights, and conversion features - The company is authorized to issue up to 125,000,000 shares of common stock and 10,000,000 shares of preferred stock[74] - As of June 30, 2025, 71,109,514 shares of common stock and 1,250 shares of Series A Preferred Stock were issued and outstanding[16][74][76] - Each share of Series A Preferred Stock is convertible into 1,000 shares of common stock, subject to beneficial ownership limitations[76] - No dividends have been declared through June 30, 2025[77] NOTE 8 – Stock-Based Compensation This note details the company's stock-based compensation plans, including options, RSUs, associated expenses, and valuation assumptions - The 2017 Equity Incentive Plan had 3,626,702 shares reserved and available for issuance as of June 30, 2025[78] - Total stock-based compensation expense was $1.2 million for the three months ended June 30, 2025, and $2.5 million for the six months ended June 30, 2025[83] Stock-Based Compensation Expense by Operating Expense (in thousands) | Item | 3 Months Ended June 30, 2025 | 3 Months Ended June 30, 2024 | 6 Months Ended June 30, 2025 | 6 Months Ended June 30, 2024 | | :----------------------------------- | :--------------------------- | :--------------------------- | :--------------------------- | :--------------------------- | | Research and development | $376 | $537 | $777 | $1,066 | | General and administrative | $843 | $1,203 | $1,726 | $2,642 | | Total | $1,219 | $1,740 | $2,503 | $3,708 | - As of June 30, 2025, there was $11.2 million of unrecognized stock-based compensation expense, expected to be recognized over an average vesting period of 2.42 years[84] NOTE 9 – Income Taxes This note explains income tax accounting, noting no tax benefit due to pre-tax losses and a full valuation allowance, and mentions the OBBBA's potential impact - The company did not record any tax benefit or expense for the three and six months ended June 30, 2025 and 2024, due to being in a pre-tax loss position[86] - A full valuation allowance is maintained against net deferred tax assets as of June 30, 2025, due to cumulative losses in recent years[87] - The One Big Beautiful Bill Act (OBBBA) was enacted on July 4, 2025, and the company is currently assessing its impact on its consolidated financial statements[88] NOTE 10 – Commitments and Contingencies This note details license agreements, outlining payments and obligations, and covers a recovered fraudulent transfer and general contingencies - Under the Northwestern Agreement, the company is developing OV329 and is obligated to pay up to $5.3 million in development and regulatory milestones, plus tiered royalties on net sales[89][91] - The AstraZeneca license agreement for the KCC2 transporter library includes potential milestone payments of up to $203.0 million[94][95] - The Gensaic Collaboration Agreement involves an equity investment of $5.1 million and potential tiered royalty payments (mid-single to low double-digit range) and milestone payments (up to $452.0 million) for PDP products[96][98] - A $1.8 million fraudulent transfer due to a business email compromise in Q3 2024 was fully recovered in January 2025, resulting in a gain[100][101] NOTE 11 – Collaboration and License Agreements This note updates key collaboration and license agreements, including Takeda, Healx, Marinus, and Graviton, detailing program statuses and amendments - Takeda discontinued the soticlestat program in January 2025, following missed primary endpoints in Phase 3 trials in June 2024. The company has no debt or obligations to Ligand related to the royalty monetization[108] - The Healx License and Option Agreement for gaboxadol was amended in June 2023, revising option exercise fees, milestone payments, and royalty structures, with Healx assuming development and commercialization responsibilities[109][112] - The Marinus License Agreement was amended in June 2025, replacing ongoing royalty obligations with a one-time payment of $7.0 million, of which $6.3 million was recognized as revenue[116] - The Graviton License Agreement for ROCK2 inhibitors (GV101/OV888) is currently paused, and all amounts due to Graviton have been paid in full as of June 30, 2025[117][118] NOTE 12 – Related Party Transactions This note refers to the Takeda Collaboration as a related party transaction, further detailed in Note 11 - The Takeda Collaboration, described in Note 11, is considered a related party transaction[119] NOTE 13 – Net (Loss) Income Per Share This note explains basic and diluted net (loss) income per share calculation using the two-class method and lists excluded dilutive securities - The Series A Preferred Stock is treated as in-substance common stock for net (loss) income per share calculation using the two-class method[120] Net (Loss) Income Per Share (in thousands, except share and per share data) | Item | 3 Months Ended June 30, 2025 | 3 Months Ended June 30, 2024 | 6 Months Ended June 30, 2025 | 6 Months Ended June 30, 2024 | | :----------------------------------- | :--------------------------- | :--------------------------- | :--------------------------- | :--------------------------- | | Net (loss) income per share, basic (Common Stock) | $(0.06) | $0.12 | $(0.21) | $(0.04) | | Net (loss) income per share, diluted (Common Stock) | $(0.06) | $0.12 | $(0.21) | $(0.04) | | Weighted average common stock shares outstanding, basic | 71,109,514 | 70,916,471 | 71,077,747 | 70,816,585 | - Potentially dilutive securities, including stock options (18.8 million) and common stock issuable upon conversion of Series A Preferred Stock (1.25 million), were excluded from diluted EPS calculations for the six months ended June 30, 2025, as they were anti-dilutive[123] NOTE 14 – Segment Reporting The company operates as a single segment focused on developing small molecule medicines for brain conditions, with the Chairman and CEO as CODM - The company operates as one segment, focused on developing small molecule medicines for brain conditions, with the Chairman and CEO as the CODM[124] Segment Information (in thousands) | Item | 3 Months Ended June 30, 2025 | 3 Months Ended June 30, 2024 | 6 Months Ended June 30, 2025 | 6 Months Ended June 30, 2024 | | :----------------------------------- | :--------------------------- | :--------------------------- | :--------------------------- | :--------------------------- | | Revenue | $6,272 | $169 | $6,402 | $317 | | Total research and development expenses | $6,465 | $12,582 | $13,123 | $22,984 | | Total general and administrative expenses | $4,880 | $8,104 | $10,902 | $15,267 | | Operating loss | $(5,073) | $(20,517) | $(17,623) | $(37,934) | | Net (loss) income | $(4,684) | $8,521 | $(14,919) | $(3,174) | - The negative program expense for OV888 (GV101) in 2025 is due to a contra-expense recognized upon settlement with a collaboration partner and reversal of an accrual estimate[125] Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations Management discusses financial condition and operations, covering biopharmaceutical focus, pipeline, risks, revenue, expenses, liquidity, and accounting policies Overview Ovid Therapeutics Inc. is a biopharmaceutical company focused on developing small molecule medicines for brain conditions, with a pipeline of three novel mechanisms of action targeting seizures. The company has incurred significant operating losses since inception and expects this trend to continue as it advances its drug candidates through development - Ovid Therapeutics is a biopharmaceutical company focused on developing small molecule medicines for brain conditions, with a pipeline of three novel mechanisms of action (MoAs) targeting seizures[128] - The company has generated $6.4 million in royalty and licensing revenue for the six months ended June 30, 2025, compared to $0.3 million for the same period in 2024[130] - As of June 30, 2025, the company had $38.3 million in cash, cash equivalents, and marketable securities, and an accumulated deficit of $319.2 million[130] - The company expects to incur significant expenses and operating losses for at least the next several years as it advances drug candidates through preclinical and clinical development[131] Significant Risks and Uncertainties This section highlights risks from global economic slowdown, inflation, geopolitical tensions, and challenges in pharmaceutical development and regulatory approval - The company faces risks from global economic slowdown, inflation, interest rate fluctuations, and geopolitical tensions (e.g., China-Taiwan, Ukraine, Israel wars) which may adversely affect its business and financial performance[133] - Other specific risks include challenges in identifying, acquiring, or in-licensing product candidates, obtaining regulatory approval, pharmaceutical product development uncertainty, protecting intellectual property, and complying with regulatory requirements[134] Going Concern Insufficient capital raises substantial doubt about the company's going concern ability, prompting evaluation of strategic alternatives like financing or partnerships - As of June 30, 2025, the company had $38.3 million in cash, cash equivalents, and marketable securities, which is not sufficient to fund projected operating expenses for at least 12 months[135] - The accumulated deficit was $319.2 million as of June 30, 2025, and the company expects to continue incurring operating losses[135] - Strategic alternatives being evaluated include additional financing, partnerships, collaborations, or licensing agreements, which may involve relinquishing rights to certain drug candidates or reducing R&D programs[135][136] Financial Operations Overview This section outlines financial operations, including revenue from licensing, R&D and G&A expenses, and other income/expense items - Revenue is primarily generated from licensing and collaboration agreements, with no commercial drug sales to date[137] - Research and development expenses are expensed as incurred and are expected to increase as drug candidates advance through preclinical and clinical trials[138][139] - General and administrative expenses consist mainly of employee-related costs, public company operating costs, and professional fees[143] - Other income (expense), net, includes interest income, unrealized gains/losses on long-term equity investments, and gains from fraudulent funds transfer recovery[144] Results of Operations This section compares financial performance for Q2 and H1 2025 versus 2024, focusing on revenue, R&D, G&A, and other income/expense Comparison of the Three Months Ended June 30, 2025 and 2024 Q2 2025 saw increased revenue from a licensing amendment and decreased operating expenses due to restructuring, resulting in a net loss Results of Operations (in thousands) | Item | 3 Months Ended June 30, 2025 | 3 Months Ended June 30, 2024 | Change $ | | :----------------------------------- | :--------------------------- | :--------------------------- | :------- | | License and other revenue | $6,272 | $169 | $6,103 | | Research and development | $6,465 | $12,582 | $(6,117) | | General and administrative | $4,880 | $8,104 | $(3,224) | | Total operating expenses | $11,345 | $20,686 | $(9,341) | | Net (loss) income | $(4,684) | $8,521 | $(13,205) | - Revenue increased by $6.1 million, primarily from the Marinus License Agreement amendment[146] - Research and development expenses decreased by $6.1 million, mainly due to the pause of the OV888 (GV101) program and reduced headcount from organizational restructuring[147] - General and administrative expenses decreased by $3.2 million, driven by organizational restructuring and cost reductions[148] - Other income (expense), net, decreased by $28.6 million, primarily due to a $29.0 million gain from royalty monetization liability adjustment in the prior year[149] Comparison of the Six Months Ended June 30, 2025 and 2024 H1 2025 revenue increased from a licensing amendment, operating expenses decreased due to program pause and restructuring, resulting in a net loss Results of Operations (in thousands) | Item | 6 Months Ended June 30, 2025 | 6 Months Ended June 30, 2024 | Change $ | | :----------------------------------- | :--------------------------- | :--------------------------- | :------- | | License and other revenue | $6,402 | $317 | $6,085 | | Research and development | $13,123 | $22,984 | $(9,861) | | General and administrative | $10,902 | $15,267 | $(4,365) | | Total operating expenses | $24,025 | $38,251 | $(14,226) | | Net loss | $(14,919) | $(3,174) | $(11,745) | - Revenue increased by $6.1 million, primarily from the Marinus License Agreement amendment[151] - Research and development expenses decreased by $9.9 million, mainly due to the pause of the OV888 (GV101) program and reduced headcount from organizational restructuring[152] - General and administrative expenses decreased by $4.4 million, due to organizational restructuring, reduced headcount, and cost reduction strategies[153] - Other income (expense), net, decreased by $32.1 million, primarily due to a $29.0 million gain from royalty monetization liability adjustment in the prior year[154] Liquidity and Capital Resources Liquidity is constrained, raising going concern doubts; significant future R&D funding requires additional capital, potentially leading to dilution or relinquished rights - As of June 30, 2025, total cash, cash equivalents, and marketable securities were $38.3 million, insufficient to fund operations for the next 12 months[155] - The company had an accumulated deficit of $319.2 million and working capital of $33.2 million as of June 30, 2025[156] - Future capital uses include compensation, R&D services, clinical costs, legal, regulatory, and general overhead expenses[157] - The company may need to raise additional capital through equity offerings, debt financings, or collaborations, which could result in dilution or relinquishing valuable rights[159][161] Cash Flows The company's cash flows for the six months ended June 30, 2025, show significant cash used in operating activities, largely offset by cash provided by investing activities, resulting in a net increase in cash, cash equivalents, and restricted cash Summary of Cash Flows (in thousands) | Item | 6 Months Ended June 30, 2025 | 6 Months Ended June 30, 2024 | | :----------------------------------- | :--------------------------- | :--------------------------- | | Net cash used in operating activities | $(15,054) | $(30,941) | | Net cash provided by investing activities | $17,155 | $33,010 | | Net cash provided by financing activities | $13 | $584 | | Net increase in cash, cash equivalents and restricted cash | $2,114 | $2,653 | - Net cash used in operating activities was $15.1 million for the six months ended June 30, 2025, primarily due to net loss and a reduction in accounts payable and accrued expenses, partially offset by stock-based compensation[166] - Net cash provided by investing activities was $17.2 million for the six months ended June 30, 2025, mainly from the maturity of marketable securities[167] Smaller Reporting Company Status and a Non-Accelerated Filer As a smaller reporting company and non-accelerated filer, the company benefits from scaled-back disclosure obligations in SEC filings - The company is a smaller reporting company, allowing for scaled-back disclosures, such as reduced executive compensation information and two years of audited financial statements[169][170] - As a non-accelerated filer, the company is exempt from auditor attestation requirements of Section 404 of the Sarbanes-Oxley Act[171] Critical Accounting Policies and Estimates No material changes to critical accounting policies and estimates occurred during the three and six months ended June 30, 2025 - No material changes to critical accounting policies and estimates occurred during the three and six months ended June 30, 2025[173] Item 3. Quantitative and Qualitative Disclosures About Market Risk The company's primary market risk is interest rate sensitivity, but short-term, low-risk investments mitigate material impact from rate changes - The company's primary market risk is interest rate sensitivity, with $38.3 million in cash, cash equivalents, and marketable securities as of June 30, 2025[174] - An immediate 100 basis point change in interest rates would not materially affect the fair market value of its cash equivalents and marketable securities due to their short-term maturities and low-risk profile[174] - The company intends to maintain its portfolio in institutional money market funds comprised of U.S. treasury and U.S. treasury-backed repurchase agreements, as well as treasury notes and high-quality short-term corporate bonds[174] Item 4. Controls and Procedures Management, including the principal executive and financial officers, evaluated the effectiveness of the company's disclosure controls and procedures as of June 30, 2025, concluding they were effective. No material changes in internal control over financial reporting occurred during the quarter - Management concluded that the company's disclosure controls and procedures were effective as of June 30, 2025[176] - No changes in internal control over financial reporting occurred during the quarter ended June 30, 2025, that materially affected, or are reasonably likely to materially affect, internal control over financial reporting[178] PART II. Other Information Item 1. Legal Proceedings The company is not currently subject to any material legal proceedings - The company is not currently involved in any material legal proceedings[180] Item 1A. Risk Factors This section details comprehensive risks impacting the company's business, financial condition, and growth, covering development, regulatory, IP, and operational aspects Summary of Select Risks Associated with Our Business Key risks include going concern doubts, need for capital, expected losses, macroeconomic impacts, capital raising limitations, and early-stage drug development challenges - There is substantial doubt about the company's ability to continue as a going concern, requiring additional capital that may not be available[182] - The company expects to incur substantial operating losses for the foreseeable future and may never achieve profitability[182] - Macroeconomic conditions, including economic downturns, inflation, interest rates, and geopolitical events, could adversely affect the business[182] - The company is early in its drug development efforts, and failure to successfully develop, receive regulatory approval for, and commercialize drug candidates would harm the business[182] Risks Related to Our Financial Position and Need for Additional Capital This section details the company's precarious financial position, emphasizing going concern doubts, expected losses, and macroeconomic impacts on financing - The company's existing cash, cash equivalents, and marketable securities ($38.3 million as of June 30, 2025) are insufficient to fund operations for the next 12 months, raising substantial doubt about its going concern ability[185] - The company expects to continue incurring substantial operating losses and may never achieve profitability, with an accumulated deficit of $319.2 million as of June 30, 2025[190] - Macroeconomic factors, including inflation, high interest rates, trade disputes, and geopolitical tensions, could adversely affect the company's business, increase operating costs, and limit access to capital[197][198][199] - The company's ability to raise capital using a Form S-3 registration statement is limited to one-third of its public float (currently less than $75.0 million), potentially increasing the cost and difficulty of future financings[201] Risks Related to the Development and Commercialization of Our Drug Candidates This section outlines risks in drug development and commercialization, including early-stage challenges, clinical trial unpredictability, side effects, market acceptance, and competition - The company is early in its drug development efforts and has no approved drugs for commercial sale, making its ability to generate revenue dependent on successful development, regulatory approval, and commercialization of current and future candidates[205][206] - Preclinical and early clinical trial results are not necessarily predictive of future outcomes; for example, soticlestat failed to meet primary endpoints in Phase 3 trials after promising early results[214] - Drug candidates may cause undesirable side effects, leading to delays or prevention of regulatory approval, limited commercial potential, or significant negative consequences post-approval[230][232] - Market acceptance of approved drug candidates is uncertain and depends on factors like efficacy, safety, cost, and marketing efforts, especially given the small and difficult-to-assess patient populations for rare neurological disorders[234][236][241] - The company faces substantial competition from major pharmaceutical and biotechnology companies with greater resources and experience, which could limit its ability to develop or commercialize drug candidates[237][238][239] Risks Related to Licensing and Collaboration Arrangements This section details risks from licensing and collaborations, including loss of control, increased capital needs, dilution, and uncertain outcomes - Collaborations may require the company to relinquish important rights and control over drug candidate development and commercialization, and partners may not commit adequate resources or may terminate agreements[250][252][257] - Future acquisitions or strategic partnerships could increase capital requirements, dilute stockholders, incur debt or contingent liabilities, and divert management attention[253] - The company's business strategy relies on acquiring or in-licensing compounds, but there is no assurance that suitable strategic collaborations will materialize or be successful[256] - The discontinuation of the soticlestat program by Takeda exemplifies the risk that collaborations may not result in successful development and commercialization[257] Risks Related to Regulatory Compliance This section addresses regulatory compliance risks, including fraud laws, privacy, non-compliance penalties, and the impact of healthcare reforms on drug pricing and orphan designations - The company's relationships with healthcare professionals and third-party payors are subject to federal and state healthcare fraud and abuse laws, false claims laws, and privacy laws (e.g., Anti-Kickback Statute, False Claims Act, HIPAA, Sunshine Act)[258] - Non-compliance with these laws could lead to significant civil, criminal, and administrative penalties, damages, fines, and exclusion from government healthcare programs[260] - Market acceptance and sales of approved drugs depend on adequate coverage and reimbursement from third-party payors, which are influenced by cost containment efforts and legislative reforms like the PPACA and IRA[262][264][266][267][271] - Failure to obtain or maintain orphan drug designations or exclusivity could limit the profitability of drug candidates, as exclusivity can be lost under certain conditions[274][276] - Even with regulatory approval, drug candidates remain subject to ongoing oversight, including manufacturing, labeling, promotion, and post-market requirements, with non-compliance leading to potential restrictions or withdrawal of approval[277][279] Risks Related to Our Intellectual Property This section highlights IP protection uncertainties, covering patent validity, infringement claims, trade secret risks, and impacts of changing patent laws - The company relies on patents, trade secrets, and confidentiality agreements to protect its intellectual property, but obtaining and maintaining broad patent protection is expensive, time-consuming, and uncertain[284] - Third parties may challenge the validity, enforceability, or scope of the company's patents, or allege infringement of their own intellectual property rights, leading to costly litigation and potential loss of commercialization rights[285][295][297] - Changes in U.S. or foreign patent laws, or their interpretation, could diminish the value of patents and weaken the company's ability to obtain or enforce patent protection[289][303] - Reliance on third parties requires sharing trade secrets, increasing the risk of misappropriation or unauthorized disclosure, which could harm the business[305] Risks Related to Our Dependence on Third Parties This section outlines risks from reliance on third parties for manufacturing and clinical trials, including delays, quality issues, and regulatory non-compliance - The company relies entirely on third parties for drug manufacturing, formulation, storage, distribution, and testing, introducing risks such as inability to meet specifications, manufacturing capacity issues, and non-compliance with cGMP[307][309] - Reliance on Contract Research Organizations (CROs) for preclinical studies and clinical trials means limited influence over their performance, and non-compliance with GLPs/GCPs could lead to unreliable data and delays in regulatory approval[310][311][313] - Termination of relationships with CROs or manufacturers could result in substantial costs, management time, and delays in clinical development timelines[316] Risks Related to Our Business Operations, Employee Matters and Managing Growth This section addresses operational risks, including management dependence, talent retention, growth management, employee misconduct, and cybersecurity and data privacy threats - The company is highly dependent on its senior management team, including the CEO, and the loss of key personnel or inability to attract and retain qualified staff could harm the business[317][318][319] - Expanding the organization poses challenges in identifying, hiring, integrating, and retaining new personnel, potentially diverting management attention and leading to operational inefficiencies[322] - The company is exposed to risks of employee misconduct, including non-compliance with regulatory standards and fraud, which could lead to significant fines, sanctions, and reputational harm[324] - Increasing dependence on IT systems and sensitive data makes the company vulnerable to cyberattacks, security incidents, and data breaches, which could disrupt operations, lead to regulatory actions, and harm reputation[325][329][330] - The company is subject to stringent and evolving data privacy and security laws (e.g., HIPAA, CCPA, GDPR), and non-compliance or perceived failure to comply could result in investigations, litigation, fines, and business disruptions[338][341][342][343][348] Risks Related to Being a Public Company This section addresses public company risks, including reduced investor appeal from smaller reporting status and challenges in maintaining effective internal controls - As a 'smaller reporting company,' the company's reduced disclosure requirements may make its common stock less attractive to investors, potentially leading to a less active trading market and increased stock price volatility[352][354] - The company previously identified and remediated material weaknesses in internal control over financial reporting related to fund transfers and vendor information updates, and failure to maintain effective controls in the future could adversely affect investor confidence and stock value[355][357][358][359] Risks Related to the Ownership of Our Common Stock and Other General Matters This section discusses common stock ownership risks, including Nasdaq delisting, price volatility, dilution, ownership concentration, and anti-takeover provisions - The company received notice of non-compliance with Nasdaq's minimum bid price requirement ($1.00 per share) and was granted an extension until February 9, 2026, to regain compliance, with a potential reverse stock split as a measure[360][361] - Failure to maintain Nasdaq listing requirements could reduce liquidity, lower the stock price, and negatively impact the ability to raise equity financing and provide employee incentives[362] - The market price of the common stock has been and is likely to remain volatile, influenced by clinical trial results, regulatory developments, competition, and general market conditions[363] - Stockholders will be diluted by the conversion of 1,250,000 shares of Series A Preferred Stock and the exercise of 18,752,703 outstanding stock options[366] - Concentration of ownership (approximately 41.6% by executive officers, directors, and principal stockholders) and anti-takeover provisions in organizational documents could prevent new investors from influencing significant corporate decisions or make an acquisition more difficult[367][373][374][378] Item 2. Unregistered Sales of Equity Securities and Use of Proceeds This section states that there were no unregistered sales of equity securities, no use of proceeds to report, and no purchases of equity securities by the issuer or affiliated purchasers during the period - No unregistered sales of equity securities occurred during the period[380] - No purchases of equity securities by the issuer or affiliated purchasers were made[381] Item 5. Other Information This section reports that no director or officer adopted or terminated a Rule 10b5-1 trading arrangement or non-Rule 10b5-1 trading arrangement during the three months ended June 30, 2025 - No director or officer adopted or terminated a Rule 10b5-1 or non-Rule 10b5-1 trading arrangement during the three months ended June 30, 2025[382] Item 6. Exhibits This section lists all exhibits filed with the Form 10-Q, including organizational documents, stock certificates, certifications of principal officers, and XBRL-related documents - Exhibits include the Amended and Restated Certificate of Incorporation, Bylaws, stock certificates, and certifications of principal executive and financial officers[386] - XBRL Instance Document, Taxonomy Extension Schema, and Cover Page are also furnished as exhibits[386] Signatures Signatures This section contains the duly authorized signatures of the company's Chief Executive Officer and Chief Business and Financial Officer, certifying the submission of the Quarterly Report on Form 10-Q - The report is signed by Jeremy M. Levin, Chief Executive Officer, and Jeffrey Rona, Chief Business and Financial Officer, on August 13, 2025[390]