Cartesian Therapeutics(RNAC) - 2025 Q1 - Quarterly Report

Forward-Looking Statements Forward-Looking Statements Overview This section outlines forward-looking statements in the report, subject to risks and uncertainties, with no commitment to public updates unless legally required - The report contains forward-looking statements covered by safe harbor provisions, indicating future results, financial position, business strategy, product approvals, R&D costs, and the sufficiency of cash resources[9] - Key risks and uncertainties include future CVR payouts, benefits from the Merger, market size, preclinical and clinical development, dependence on third parties, product candidate efficacy and safety, timing of trials and approvals, macroeconomic conditions, global events, and the ability to achieve or maintain profitability[10][13] - The company operates in an evolving environment with new risks and uncertainties emerging, and does not plan to publicly update or revise forward-looking statements unless required by applicable law[11][12] PART I. FINANCIAL INFORMATION Item 1. Financial Statements (unaudited) This section presents the unaudited consolidated financial statements for Cartesian Therapeutics, Inc. and its subsidiaries, along with detailed notes Consolidated Balance Sheets This section presents the unaudited consolidated balance sheets, detailing assets, liabilities, and stockholders' deficit as of March 31, 2025, and December 31, 2024 | Metric | March 31, 2025 (in thousands) | December 31, 2024 (in thousands) | | :--------------------------------- | :----------------------------- | :----------------------------- | | Cash and cash equivalents | $180,434 | $212,610 | | Total current assets | $185,120 | $216,626 | | Total assets | $409,130 | $435,023 | | Total current liabilities | $14,998 | $22,976 | | Total liabilities | $430,919 | $441,825 | | Total stockholders' deficit | $(21,789) | $(6,802) | - Cash and cash equivalents decreased by $32.176 million from December 31, 2024, to March 31, 2025[16] - Total assets decreased by $25.893 million, while total liabilities decreased by $10.906 million over the three-month period[16] - Stockholders' deficit significantly increased from $(6.802) million to $(21.789) million, indicating a worsening financial position[16] Consolidated Statements of Operations and Comprehensive Loss This section presents the unaudited consolidated statements of operations and comprehensive loss for the three months ended March 31, 2025 and 2024 | Metric | Three Months Ended March 31, 2025 (in thousands) | Three Months Ended March 31, 2024 (in thousands) | | :------------------------------------------ | :------------------------------------------ | :------------------------------------------ | | Total revenue | $1,100 | $5,840 | | Research and development | $14,674 | $9,738 | | General and administrative | $8,315 | $9,450 | | Total operating expenses | $22,989 | $19,188 | | Operating loss | $(21,889) | $(13,348) | | Net loss | $(17,710) | $(56,824) | | Basic and diluted net loss per share | $(0.68) | $(10.50) | - Total revenue decreased by 81% from $5.84 million in Q1 2024 to $1.1 million in Q1 2025, primarily due to a significant drop in collaboration and license revenue[19] - Research and development expenses increased by 51% to $14.674 million in Q1 2025, while general and administrative expenses decreased by 12% to $8.315 million[19] - Net loss significantly improved from $(56.824) million in Q1 2024 to $(17.710) million in Q1 2025, largely driven by a favorable change in the fair value of contingent value right liability[19] Consolidated Statements of Changes in Convertible Preferred Stock and Stockholders' Deficit This section presents the unaudited consolidated statements of changes in convertible preferred stock and stockholders' deficit for the three months ended March 31, 2025 | Metric | Balance at Dec 31, 2024 (in thousands) | Balance at Mar 31, 2025 (in thousands) | | :-------------------------------- | :----------------------------------- | :----------------------------------- | | Common stock | $3 | $3 | | Additional paid-in capital | $689,887 | $692,578 | | Accumulated deficit | $(692,071) | $(709,781) | | Total stockholders' deficit | $(6,802) | $(21,789) | - Additional paid-in capital increased by $2.691 million, primarily due to stock-based compensation expense and issuance of common stock upon option exercise[21] - Accumulated deficit increased by $17.710 million, reflecting the net loss for the three months ended March 31, 2025[21] - The company effected a 1-for-30 reverse stock split on April 4, 2024, adjusting all share and per-share figures retrospectively[21] Consolidated Statements of Cash Flows This section presents the unaudited consolidated statements of cash flows for the three months ended March 31, 2025 and 2024 | Metric | Three Months Ended March 31, 2025 (in thousands) | Three Months Ended March 31, 2024 (in thousands) | | :------------------------------------------ | :------------------------------------------ | :------------------------------------------ | | Net cash used in operating activities | $(23,108) | $(15,917) | | Net cash used in investing activities | $(1,075) | $(602) | | Net cash (used in) provided by financing activities | $(8,025) | $43,031 | | Net change in cash, cash equivalents, and restricted cash | $(32,176) | $26,507 | | Cash, cash equivalents, and restricted cash at end of period | $182,103 | $104,795 | - Net cash used in operating activities increased to $23.108 million in Q1 2025 from $15.917 million in Q1 2024[27] - Net cash used in investing activities increased to $1.075 million in Q1 2025 from $0.602 million in Q1 2024, primarily due to purchases of property and equipment[27] - Financing activities shifted from providing $43.031 million in Q1 2024 (due to Series A Preferred Stock issuance) to using $8.025 million in Q1 2025 (due to CVR distributions)[27] Notes to Consolidated Financial Statements This section provides detailed notes explaining the company's business, accounting policies, and specific financial line items Note 1. Description of the Business This note describes Cartesian Therapeutics, Inc.'s business as a clinical-stage biotechnology company, its merger, key financial events, and liquidity position - Cartesian Therapeutics, Inc. (formerly Selecta Biosciences, Inc.) is a clinical-stage biotechnology company focused on cell therapies for autoimmune diseases, leveraging proprietary technology for repeated dosing without pre-treatment chemotherapy[30] - The company acquired Old Cartesian in November 2023 through a stock-for-stock merger, issuing common stock and Series A Non-Voting Convertible Preferred Stock[31] - A Contingent Value Right (CVR) was distributed to legacy shareholders, entitling them to payments from the disposition or monetization of the company's legacy assets[33] - A 1-for-30 reverse stock split was effected on April 4, 2024, and a private placement in July 2024 generated approximately $130.0 million in gross proceeds from the issuance of common and Series B Preferred Stock[35][36] - As of March 31, 2025, cash, cash equivalents, and restricted cash totaled $182.1 million, projected to fund operations for at least the next 12 months[43] Note 2. Basis of Presentation This note outlines the principles of consolidation, use of estimates, and single operating segment information for the financial statements - The consolidated financial statements include Cartesian Therapeutics, Inc. and its wholly-owned subsidiaries, with all significant intercompany accounts and transactions eliminated[46] - Management uses significant estimates in preparing financial statements, particularly for fair value of acquired intangible assets, CVRs, deferred income taxes, revenue recognition, R&D expenses, stock-based compensation, and warrant liabilities[47] - The company operates as a single operating segment, focused on the research and development of cell therapy product candidates, with performance assessed by the CEO based on consolidated net loss[48] Note 3. Summary of Significant Accounting Policies This note confirms no material changes to accounting policies and discusses recent pronouncements not expected to significantly impact financial statements - No material changes to significant accounting policies occurred during the three months ended March 31, 2025[49] - ASU 2023-09 (Income Taxes) will be effective for annual periods beginning December 31, 2025, and is not expected to have a material impact[50] - ASU 2024-03 (Expense Disaggregation Disclosures) will be effective for annual periods beginning December 31, 2027, and its impact is currently being evaluated[51] Note 4. Goodwill and Intangible Assets This note details the goodwill and indefinite-lived intangible assets, primarily in-process R&D, resulting from the November 2023 merger - The Merger with Old Cartesian on November 13, 2023, resulted in goodwill of approximately $48.2 million, with no changes to its carrying value during the three months ended March 31, 2025[52][53] Indefinite-Lived Intangible Assets | Indefinite-Lived Intangible Assets (in thousands) | March 31, 2025 | December 31, 2024 | | :------------------------------------------------ | :------------- | :---------------- | | Descartes-08 for MG | $93,900 | $93,900 | | Descartes-08 for SLE | $56,700 | $56,700 | | Total in-process research and development assets | $150,600 | $150,600 | Note 5. Investments This note describes the company's $2.0 million cost-method investment in Cyrus Biotechnology, Inc., with no impairment indicators - The company holds a $2.0 million investment in Cyrus Biotechnology, Inc.'s Series B Preferred Stock, recognized at cost[55] - The company is not the primary beneficiary of Cyrus and therefore does not consolidate it[56] - As of March 31, 2025, and December 31, 2024, no impairment indicators were present, and the investment's carrying value remained $2.0 million[57] Note 6. Net Loss Per Share Allocable to Common Stockholders This note provides the computation of basic and diluted net loss per share, showing significant improvement in Q1 2025 Net Loss Per Share | Metric | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :------------------------------------------ | :-------------------------------- | :-------------------------------- | | Net loss (in thousands) | $(17,710) | $(56,824) | | Weighted-average common shares outstanding | 25,902,650 | 5,414,020 | | Net loss per share (Basic and diluted) | $(0.68) | $(10.50) | - The net loss per share improved significantly from $(10.50) in Q1 2024 to $(0.68) in Q1 2025[58] - Potential dilutive shares (totaling 8,204,424 in Q1 2025 and 21,065,841 in Q1 2024) were excluded from diluted EPS calculation as the company was in a net loss position[58] Note 7. Fair Value Measurements This note details fair value measurements for warrant and CVR liabilities (Level 3) and money market funds (Level 1), noting decreases in liabilities Fair Value Measurements | Fair Value Measurements (in thousands) | March 31, 2025 | December 31, 2024 | | :------------------------------------- | :------------- | :---------------- | | Money market funds (Level 1) | $39,509 | $39,088 | | Warrant liabilities (Level 3) | $2,018 | $3,836 | | Contingent value right liability (Level 3) | $387,400 | $395,500 | - Warrant liabilities decreased by $1.818 million in Q1 2025, primarily due to a decrease in the underlying common stock price and the expiration of 2019 warrants[68][182] - The CVR liability decreased by $8.1 million in Q1 2025, reflecting $7.754 million in distributions and a $0.346 million decrease in fair value, mainly due to changes in interest rates[73][183] - The CVRs entitle holders to 100% of milestone payments, royalties, and other proceeds from the Sobi License and other legacy asset dispositions, net of specified deductions[70][72] Note 8. Property and Equipment This note details the company's property and equipment, net, showing an increase driven by laboratory equipment and leasehold improvements Property and Equipment | Property and Equipment (in thousands) | March 31, 2025 | December 31, 2024 | | :------------------------------------ | :------------- | :---------------- | | Laboratory equipment | $7,690 | $7,295 | | Leasehold improvements | $4,076 | $3,427 | | Construction in process | $480 | $695 | | Total property and equipment | $13,102 | $12,269 | | Less: Accumulated depreciation | $(2,928) | $(2,357) | | Property and equipment, net | $10,174 | $9,912 | - Depreciation expense for Q1 2025 was $0.6 million, compared to $0.2 million for Q1 2024, indicating increased asset utilization or new asset additions[75] Note 9. Accrued Expenses This note provides a breakdown of accrued expenses, which decreased primarily due to lower payroll and professional service costs Accrued Expenses | Accrued Expenses (in thousands) | March 31, 2025 | December 31, 2024 | | :------------------------------ | :------------- | :---------------- | | Payroll and employee related expenses | $1,424 | $3,534 | | Accrued patent fees | $221 | $813 | | Accrued external R&D costs | $4,917 | $2,987 | | Accrued professional and consulting services | $1,668 | $3,674 | | Total accrued expenses | $9,197 | $12,076 | - Accrued external research and development costs increased by $1.93 million, while payroll and employee-related expenses decreased by $2.11 million[76] Note 10. Leases This note details operating lease agreements for manufacturing and office spaces, including expansions and an impairment charge for a ceased-use space - The company has expanded its Frederick, Maryland manufacturing and office space through multiple amendments to the Frederick Lease Agreement, with the latest amendment in March 2025 for an additional 6,439 square feet[77][80][82][85] - The Watertown Lease Agreement, including a sublease to Sobi that expired in November 2024, led to a $7.6 million impairment charge in 2024 for right-of-use assets and related property and equipment, as the company decided to cease use of the space[92][94] Lease Costs | Lease Costs (in thousands) | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :------------------------- | :-------------------------------- | :-------------------------------- | | Operating lease cost | $584 | $775 | | Variable lease cost | $406 | $397 | | Short-term lease cost | $11 | $3 | | Less: Sublease income | $0 | $(510) | | Total lease cost | $1,001 | $665 | Operating Lease Liabilities | Operating Lease Liabilities (in thousands) | March 31, 2025 | | :----------------------------------------- | :------------- | | Total future minimum lease payments | $18,122 | | Less: Imputed interest | $4,056 | | Total operating lease liabilities | $14,066 | - The weighted-average remaining lease term is 4.4 years, and the weighted-average discount rate is 11.8% as of March 31, 2025[96] Note 11. Equity This note details equity activities, including private placements, an ATM program, a reverse stock split, and outstanding preferred stock - The 2024 Private Placement, completed on July 2, 2024, generated approximately $130.0 million in gross proceeds from the issuance of common stock and Series B Preferred Stock[98] - An 'At-the-Market' offering program was established in December 2024 to sell up to $100.0 million of common stock, but no shares were sold under this program during Q1 2025[99][100] - A 1-for-30 reverse stock split became effective on April 4, 2024, adjusting all common stock figures[102] - As of March 31, 2025, 120,790.402 shares of Series A Preferred Stock and 437,927 shares of Series B Preferred Stock were outstanding, convertible into 4,464,273 common shares[103] Reserved Shares | Reserved Shares (March 31, 2025) | Number of Shares | | :------------------------------- | :--------------- | | Exercise of warrants | 692,523 | | Shares available for future stock incentive awards | 4,169,806 | | Unvested restricted stock units | 572,605 | | Outstanding common stock options | 2,475,023 | | Series A Preferred Stock | 4,026,346 | | Series B Preferred Stock | 437,927 | | Total | 12,374,230 | Note 12. Stock Incentive Plans This note details stock incentive plans, stock-based compensation expense, and unrecognized compensation for options and restricted stock units - The 2008 Stock Incentive Plan was terminated, and awards are now granted under the 2016 Incentive Award Plan and the 2018 Employment Inducement Incentive Award Plan, with additional shares reserved for issuance[105][106][107][108] - The Old Cartesian Plan was assumed in the merger, converting outstanding options into options for the company's common and Series A Preferred Stock (later common stock)[109][110] Stock-Based Compensation Expense | Stock-Based Compensation Expense (in thousands) | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :---------------------------------------------- | :-------------------------------- | :-------------------------------- | | Research and development | $1,275 | $712 | | General and administrative | $1,233 | $719 | | Total stock-based compensation expense | $2,508 | $1,431 | - As of March 31, 2025, unrecognized compensation expense for stock options was $17.9 million (weighted-average period of 3.2 years) and for restricted stock units was $8.0 million (weighted-average period of 3.0 years)[113][116] Note 13. Revenue Arrangements This note details revenue from collaboration and license agreements and grant funding, noting a significant decrease in collaboration revenue - Collaboration and license revenue decreased by $5.4 million (93%) to $0.4 million in Q1 2025, primarily due to the termination of the Astellas Agreement in June 2024[119][175] - Grant revenue increased to $0.7 million in Q1 2025, from zero in Q1 2024, due to funding approval from the National Institute of Neurological Disorders and Stroke (NINDS) for RNA-based CAR-T cell research[124][125][176] - The Sobi License, which includes potential milestone payments up to $630.0 million and tiered royalties, resulted in a $30.0 million milestone payment received in July 2024 for initiating a rolling BLA for SEL-212[120][121] - As of March 31, 2025, there were no unsatisfied performance obligations from contracts with customers[123] Note 14. Related-Party Transactions This note outlines related-party transactions in Q1 2024, including stock purchases and warrant exercises by a board member and affiliate Related-Party Transactions | Related-Party Transactions (Q1 2024) | Shares of Series A Preferred Stock purchased | Total aggregate purchase price (in thousands) | | :----------------------------------- | :------------------------------------------- | :-------------------------------------------- | | Timothy A. Springer, Ph.D. | 99,140.326 | $40,000 | - TAS Partners LLC, an affiliate of Dr. Timothy A. Springer, exercised 65,681 Amended 2019 Warrants for $2.9 million in cash in Q1 2024, receiving common stock and CVRs[128] - No related-party transactions occurred during the three months ended March 31, 2025[128] Note 15. Collaboration and License Agreements This note details various collaboration and license agreements, including terms for T-cell engineering, CAR-T cell products, and the ImmTOR platform - The Biogen Agreement (September 2023) grants a non-exclusive, worldwide, perpetual patent license for engineering T-cells modified with mRNA for autoimmune diseases, with no expenses, fees, or royalties payable to Biogen[129][130] - The NCI Agreement (September 2019) grants a non-exclusive license for anti-BCMA CAR-T cell products for myasthenia gravis, pemphigus vulgaris, and immune thrombocytopenic purpura, requiring a low five-digit annual royalty, low single-digit earned royalties on net sales, and up to $0.8 million in benchmark royalties[133][134][135] - The Genovis Agreement, which provided an exclusive license to the Xork enzyme technology, was terminated effective September 13, 2024[140][141] - The 3SBio License (May 2014) involves potential future payments up to $15.0 million contingent upon clinical and regulatory approval milestones for products containing the ImmTOR platform[142] Note 16. Income Taxes This note states that the company has not recorded income tax benefits for net losses or credits due to uncertainty of future realization - The company has not recorded U.S. federal or state income tax benefits for net losses or earned research and orphan drug credits due to uncertainty of future realization[143] Note 17. Commitments and Contingencies This note indicates no material litigation, outlines indemnification practices for officers and directors, and confirms no material losses - As of March 31, 2025, the company was not involved in any litigation with a material adverse effect on its business or financial results[144] - The company indemnifies its officers, directors, consultants, and employees, and has directors' insurance coverage to limit potential exposure[145][146] - No material losses have been experienced from indemnification obligations, and no material claims are outstanding[145][146][147] Note 18. Restructuring This note details the 2023 restructuring plan, including headcount reduction, with all payments completed by March 31, 2025 - In 2023, the company implemented a restructuring plan, including pausing product candidate development and an approximate 90% headcount reduction, to extend its cash runway[148] Severance Liability | Severance Liability (in thousands) | As of Dec 31, 2024 | Charges | Cash Payments | As of Mar 31, 2025 | | :--------------------------------- | :----------------- | :------ | :------------ | :----------------- | | Severance liability | $80 | $0 | $(80) | $0 | - Payments for the restructuring plan were completed by March 31, 2025, with no restructuring charges recognized in Q1 2025[149] Note 19. Segment Information This note presents selected financial information for the company's single operating segment, highlighting revenue, R&D, G&A, and net loss changes Selected Financial Information | Selected Financial Information (in thousands) | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :-------------------------------------------- | :-------------------------------- | :-------------------------------- | | Total revenue | $1,100 | $5,840 | | Research and development | $14,674 | $9,738 | | General and administrative | $8,315 | $9,450 | | Net loss | $(17,710) | $(56,824) | - Research and development expenses for Descartes-08 for MG increased significantly from $1.266 million in Q1 2024 to $7.036 million in Q1 2025, reflecting advancement into Phase 3 activities[150] - Other (income) expense, net, shifted from an expense of $43.476 million in Q1 2024 to an income of $(4.179) million in Q1 2025, primarily due to changes in fair value of CVR and forward contract liabilities[150] Note 20. Subsequent Events This note confirms that no subsequent events requiring disclosure occurred through the date of financial statement issuance - No subsequent events requiring disclosure occurred through the date of financial statement issuance[151] Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations This section provides management's perspective on the company's financial condition and results, including the merger's impact and future financing needs Overview This section provides an overview of Cartesian Therapeutics as a clinical-stage biotechnology company focused on cell therapies for autoimmune diseases - Cartesian Therapeutics is a clinical-stage biotechnology company developing cell therapies for autoimmune diseases, characterized by repeated dosing, outpatient administration, and no pre-treatment chemotherapy[153] - The lead product candidate, Descartes-08, showed deep and durable clinical benefit in a Phase 2b trial for myasthenia gravis (MG), with 83% of participants maintaining clinically meaningful improvements at six months and sustained improvements at 12 months[153] Merger This section details the November 2023 merger between the company (formerly Selecta Biosciences, Inc.) and Old Cartesian - On November 13, 2023, the company (formerly Selecta Biosciences, Inc.) merged with Old Cartesian, resulting in the company changing its corporate name to Cartesian Therapeutics, Inc[154] Financial Operations This section discusses the company's financing, recurring net losses, expected future expenses, and projected cash runway into mid-2027 - Operations have been financed primarily through public offerings, private placements, research grants, and collaboration/license arrangements, with no product sales to date[155] - The company incurred net losses of $17.7 million in Q1 2025 and $56.8 million in Q1 2024, with an accumulated deficit of $709.8 million as of March 31, 2025[156] - Significant expenses and operating losses are expected to continue due to advancing Descartes-08 into Phase 3, developing other product candidates, seeking regulatory approvals, protecting intellectual property, and hiring additional staff[156] - Existing cash, cash equivalents, and restricted cash of $182.1 million as of March 31, 2025, are expected to fund operations into mid-2027[160] Components of our Results of Operations This section outlines the primary components of the company's financial results, including revenue, R&D, G&A expenses, and other income/expense items - Revenue primarily consists of collaboration and license revenue (upfront and milestone payments) and grant revenue for specific R&D services[162][163] - Research and development expenses include fees to CROs, internal manufacturing, process development, employee compensation, and capital equipment, expensed as incurred[165][166] - General and administrative expenses cover salaries, benefits, stock-based compensation for executive and support functions, facility costs, travel, and professional fees[167] - Other income/expense items include interest income, changes in fair value of warrant liabilities, contingent value right liability, and forward contract liabilities[169][171][172][173] Results of Operations This section analyzes the company's financial performance for the three months ended March 31, 2025 and 2024, highlighting key revenue and expense changes Financial Performance | Metric | Three Months Ended March 31, 2025 (in thousands) | Three Months Ended March 31, 2024 (in thousands) | Increase (Decrease) (in thousands) | Change (%) | | :------------------------------------------ | :------------------------------------------ | :------------------------------------------ | :--------------------------------- | :--------- | | Collaboration and license revenue | $400 | $5,840 | $(5,440) | (93)% | | Grant revenue | $700 | $0 | $700 | 100% | | Total revenue | $1,100 | $5,840 | $(4,740) | (81)% | | Research and development | $14,674 | $9,738 | $4,936 | 51% | | General and administrative | $8,315 | $9,450 | $(1,135) | (12)% | | Operating loss | $(21,889) | $(13,348) | $(8,541) | 64% | | Interest income | $2,015 | $1,164 | $851 | 73% | | Change in fair value of warrant liabilities | $1,818 | $1,042 | $776 | 74% | | Change in fair value of CVR liability | $346 | $(39,300) | $39,646 | (101)% | | Net loss | $(17,710) | $(56,824) | $39,114 | (69)% | - Collaboration and license revenue decreased by $5.4 million due to the termination of the Astellas Agreement[175] - Research and development expenses increased by $5.0 million, driven by increased spending on Descartes-08 for MG (up $5.77 million) and early-stage programs (up $0.863 million), as well as headcount growth[177][179] - General and administrative expenses decreased by $1.2 million, primarily due to reductions in professional fees related to the Merger[180] - Net loss improved by $39.1 million, largely due to a $39.6 million favorable change in the fair value of the CVR liability and the settlement of forward contract liabilities[183][184][186] Liquidity and Capital Resources This section discusses the company's cash position, recurring losses, and the need for additional capital to fund future operations - The company has incurred recurring net losses and expects losses to increase, requiring additional capital through equity offerings, debt financings, and collaborations[187] - Cash, cash equivalents, and restricted cash totaled $182.1 million as of March 31, 2025, with $1.7 million restricted for lease commitments[188] - The CVR liability will be settled solely through cash flow from the Sobi License and other Gross Proceeds, with no contractual obligation for the company to fund it[189][190] - The 2023 Private Placement generated $60.25 million, and the 2024 Private Placement generated $130.0 million in gross proceeds[196][197] - Existing cash resources are projected to fund operating expenses and capital expenditure requirements into mid-2027, but additional funding will be needed for long-term operations[201] Cash Requirements due to Contractual Obligations and Other Commitments This section details the company's future cash requirements for lease payments and contingent obligations under license agreements - Remaining lease payments for Watertown, Massachusetts space total approximately $9.0 million through May 2028, with payments subject to potential reimbursement through CVR deductions[203] - Lease payments for Gaithersburg, Maryland space total approximately $0.6 million through January 2027[204] - Lease payments for Frederick, Maryland space, including multiple expansions, total $9.7 million through June 2031, with annualized base rent of approximately $1.2 million[205] - Future payments under license and collaboration agreements (Biogen, NCI, 3SBio) are contingent on milestones or royalties, with timing and likelihood currently unestimable[206] Summary of Cash Flows This section summarizes the company's cash flows from operating, investing, and financing activities for the three months ended March 31, 2025 and 2024 Cash Flows | Cash Flows (in thousands) | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :------------------------ | :-------------------------------- | :-------------------------------- | | Operating activities | $(23,108) | $(15,917) | | Investing activities | $(1,075) | $(602) | | Financing activities | $(8,025) | $43,031 | | Net change in cash | $(32,176) | $26,507 | - Net cash used in operating activities increased by $7.2 million, primarily due to higher net loss (adjusted for non-cash items) and increased cash used in changes in operating assets and liabilities[208] - Net cash used in investing activities increased by $0.5 million, mainly due to purchases of property and equipment[209] - Net cash from financing activities decreased by $51.0 million, shifting from a net cash provider in Q1 2024 (2023 Private Placement proceeds) to a net cash user in Q1 2025 (CVR distribution payments)[211] Recent Accounting Pronouncements This section refers to Note 3 for details on recently adopted or issued accounting pronouncements - Refer to Note 3 for a discussion of recently adopted or issued accounting pronouncements[212] Off-Balance Sheet Arrangements This section confirms that the company did not have any off-balance sheet arrangements as of March 31, 2025 - As of March 31, 2025, the company did not have any off-balance sheet arrangements[213] Critical Accounting Policies and Use of Estimates This section states that no material changes occurred to critical accounting policies during the three months ended March 31, 2025 - No material changes occurred to critical accounting policies during the three months ended March 31, 2025, from those described in the Annual Report on Form 10-K for 2024[214] Smaller Reporting Company This section explains the company's status as a 'smaller reporting company' and the associated scaled disclosure requirements - The company qualifies as a 'smaller reporting company,' allowing it to take advantage of scaled disclosure requirements[215] - It will remain a smaller reporting company until its public float exceeds $700 million or it has at least $100 million in revenue and $250 million in public float[215] Item 3. Quantitative and Qualitative Disclosures About Market Risk This section addresses the company's exposure to market risk, primarily interest rate sensitivity on its cash and cash equivalents - The company's primary market risk exposure is interest rate sensitivity, affecting its cash, cash equivalents, and restricted cash[216] - As of March 31, 2025, cash, cash equivalents, and restricted cash totaled $182.1 million, held in non-interest and interest-bearing money market accounts[216] - An immediate 100 basis point change in interest rates is not expected to materially affect the fair market value of these short-term, low-risk investments[216] Item 4. Controls and Procedures This section reports on the effectiveness of the company's disclosure controls and procedures, concluding they were effective as of March 31, 2025 - Management concluded that disclosure controls and procedures were effective at the reasonable assurance level as of March 31, 2025[217] - Internal controls over financial reporting have inherent limitations, providing only reasonable assurance, and are subject to risks like cost limitations, human error, and fraud[218] - No material changes in internal control over financial reporting occurred during the three months ended March 31, 2025[219] PART II. OTHER INFORMATION This part contains other information, including legal proceedings, risk factors, equity sales, defaults, mine safety, and exhibits Item 1. Legal Proceedings This section states that there are no legal proceedings to report - No legal proceedings are reported[221] Item 1A. Risk Factors This section refers to previously disclosed risk factors, confirming no material changes since the last annual report - No material changes to risk factors have occurred since those disclosed in the Annual Report on Form 10-K for the year ended December 31, 2024[222] Item 2. Unregistered Sales of Equity Securities and Use of Proceeds This section indicates that there are no unregistered sales of equity securities or use of proceeds to report - Not applicable for this reporting period[223] Item 3. Defaults Upon Senior Securities This section states that there are no defaults upon senior securities to report - No defaults upon senior securities are reported[224] Item 4. Mine Safety Disclosures This section indicates that there are no mine safety disclosures to report - No mine safety disclosures are reported[225] Item 5. Other Information This section reports no adoption or termination of Rule 10b5-1 trading arrangements by directors or officers during the quarter - No director or officer adopted or terminated any Rule 10b5-1 trading arrangements or non-Rule 10b5-1 trading arrangements during the quarter ended March 31, 2025[226] Item 6. Exhibits This section provides a comprehensive list of exhibits filed with the Quarterly Report, including corporate documents, agreements, and certifications - The exhibit index includes the Agreement and Plan of Merger, various corporate governance documents (Restated Certificate of Incorporation, Certificates of Amendment, By-laws, Certificates of Designation for Preferred Stock)[229] - Recent filings include the Third Amendment to Lease Agreement (March 12, 2025) and a Separation Agreement and Release (April 29, 2025)[229] - Certifications from the Principal Executive Officer and Principal Financial Officer (pursuant to Rule 13a-14(a) and 18 U.S.C. Section 1350) are included[229] - Inline XBRL documents (Instance, Schema, Calculation, Definition, Label, Presentation Linkbase Documents) are also part of the exhibits[229][230] Signatures Signatures Section This section contains the signatures of the company's President and Chief Executive Officer and Chief Financial Officer, certifying the report on May 8, 2025 - The report is signed by Carsten Brunn, Ph.D., President and Chief Executive Officer (Principal Executive Officer), and Blaine Davis, Chief Financial Officer (Principal Financial Officer), on May 8, 2025[236]