Entrada Therapeutics(TRDA) - 2025 Q2 - Quarterly Report

PART I — FINANCIAL INFORMATION This section provides the unaudited condensed consolidated financial statements, management's discussion and analysis, market risk disclosures, and controls and procedures for the reported period Item 1. Financial Statements (Unaudited) This section presents the unaudited condensed consolidated financial statements, including the balance sheets, statements of operations and comprehensive income (loss), statements of stockholders' equity, and statements of cash flows, along with their accompanying notes. Key financial figures show a shift from net income to net loss year-over-year, primarily due to decreased collaboration revenue, while R&D and G&A expenses increased Condensed Consolidated Balance Sheets The balance sheet reflects a decrease in cash, marketable securities, and total assets, alongside a reduction in total liabilities and stockholders' equity | Metric | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | Change (in thousands) | | :--------------------------------- | :----------------------------- | :------------------------------- | :-------------------- | | Cash and cash equivalents | $72,482 | $101,211 | $(28,729) | | Marketable securities | $281,530 | $318,787 | $(37,257) | | Total current assets | $369,146 | $440,039 | $(70,893) | | Total assets | $448,783 | $526,321 | $(77,538) | | Total current liabilities | $19,803 | $39,482 | $(19,679) | | Total liabilities | $69,278 | $97,643 | $(28,365) | | Total stockholders' equity | $379,505 | $428,678 | $(49,173) | | Accumulated deficit | $(189,796) | $(129,344) | $(60,452) | Condensed Consolidated Statements of Operations and Comprehensive Income (Loss) This statement shows a significant shift from net income to net loss year-over-year, driven by decreased collaboration revenue and increased operating expenses | Metric (in thousands) | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Change (YoY) | | :-------------------- | :------------------------------- | :------------------------------- | :----------- | | Collaboration revenue | $1,950 | $94,694 | $(92,744) | | Research and development | $37,877 | $32,035 | $5,842 | | General and administrative | $10,922 | $9,236 | $1,686 | | Net (loss) income | $(43,103) | $55,031 | $(98,134) | | Metric (in thousands) | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Change (YoY) | | :-------------------- | :----------------------------- | :----------------------------- | :----------- | | Collaboration revenue | $22,508 | $153,814 | $(131,306) | | Research and development | $69,951 | $60,643 | $9,308 | | General and administrative | $21,196 | $18,635 | $2,561 | | Net (loss) income | $(60,452) | $78,527 | $(138,979) | Condensed Consolidated Statements of Stockholders' Equity This statement details changes in stockholders' equity, primarily reflecting the impact of net loss, stock-based compensation, and other comprehensive income - Total stockholders' equity decreased from $428.678 million as of December 31, 2024, to $379.505 million as of June 30, 2025, primarily due to a net loss of $60.452 million for the six months ended June 30, 2025, partially offset by stock-based compensation and other comprehensive income[29] | Metric (in thousands) | December 31, 2024 | June 30, 2025 | | :-------------------- | :---------------- | :------------ | | Additional Paid-in Capital | $558,061 | $568,897 | | Accumulated Other Comprehensive Income (Loss) | $(43) | $400 | | Accumulated Deficit | $(129,344) | $(189,796) | | Total Stockholders' Equity | $428,678 | $379,505 | Condensed Consolidated Statements of Cash Flows This statement highlights a shift from positive to negative operating cash flow, influenced by net loss and changes in working capital | Cash Flow Activity (in thousands) | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :-------------------------------- | :----------------------------- | :----------------------------- | | Net cash (used in) provided by operating activities | $(67,996) | $14,314 | | Net cash provided by investing activities | $38,580 | $1,764 | | Net cash provided by financing activities | $687 | $101,573 | | Net (decrease) increase in cash, cash equivalents, and restricted cash | $(28,729) | $117,651 | - The company experienced a significant shift from net cash provided by operating activities in H1 2024 to net cash used in operating activities in H1 2025, primarily due to a net loss of $60.5 million in 2025 compared to net income of $78.5 million in 2024, and changes in operating assets and liabilities[32][144][145] Notes to Condensed Consolidated Financial Statements These notes provide essential details and context for the unaudited condensed consolidated financial statements 1. Nature of the Business This note describes the company's biopharmaceutical focus, accumulated deficit, and liquidity outlook for future operations - Entrada Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing Endosomal Escape Vehicle (EEV™)-therapeutics to engage intracellular targets. The company has incurred significant net losses since inception, with an accumulated deficit of $189.8 million as of June 30, 2025[33][35] - The company expects its cash, cash equivalents and marketable securities of $354.0 million as of June 30, 2025, to fund operations and capital expenditure requirements for at least the next twelve months, but will need additional financing to support its continuing operations and pursue its business strategy[36] 2. Summary of Significant Accounting Policies This note outlines the accounting principles used in preparing the financial statements and discusses the impact of new accounting pronouncements - The condensed consolidated financial statements are unaudited and prepared in conformity with GAAP, consistent with the Annual Report on Form 10-K for FY2024, with certain information condensed or omitted as permitted[39] - New accounting pronouncements, ASU 2023-09 (Income Taxes) and ASU 2024-03 (Expense Disaggregation Disclosures), are expected to impact future disclosures and presentation of financial statements upon adoption in 2025 and 2026/2027, respectively[42][43] 3. Cash, Cash Equivalents and Restricted Cash This note details the composition of cash and cash equivalents, including highly liquid investments and restricted cash balances - Cash and cash equivalents include highly liquid investments with original maturities of 90 days or less, primarily U.S. government-backed securities and treasuries[44] | Metric (in thousands) | June 30, 2025 | December 31, 2024 | | :-------------------- | :------------ | :---------------- | | Cash and cash equivalents | $72,482 | $101,211 | | Restricted cash | $3,950 | $3,950 | | Total cash, cash equivalents and restricted cash | $76,432 | $105,161 | 4. Marketable Securities This note provides a breakdown of marketable securities, their fair values, and the company's assessment of unrealized losses | Security Type (in thousands) | Amortized Cost (June 30, 2025) | Fair Value (June 30, 2025) | Amortized Cost (Dec 31, 2024) | Fair Value (Dec 31, 2024) | | :--------------------------- | :----------------------------- | :------------------------- | :---------------------------- | :------------------------ | | U.S. government agency securities and treasuries | $247,504 | $247,848 | $253,818 | $253,628 | | Corporate debt securities | $33,626 | $33,682 | $65,011 | $65,159 | | Total available-for-sale securities | $281,130 | $281,530 | $318,829 | $318,787 | - As of June 30, 2025, the Company had $82.0 million in marketable securities with unrealized losses, which are considered temporary, and no allowance for credit losses was recognized[48][49] 5. Fair Value Measurements This note categorizes financial assets measured at fair value using a three-level hierarchy based on observable inputs | Asset Type (in thousands) | Level 1 (June 30, 2025) | Level 2 (June 30, 2025) | Total (June 30, 2025) | | :------------------------ | :---------------------- | :---------------------- | :-------------------- | | Money market funds | $71,982 | — | $71,982 | | U.S. government agency securities and treasuries | — | $247,848 | $247,848 | | Corporate bonds | — | $33,682 | $33,682 | | Total | $71,982 | $281,530 | $353,512 | - Money market funds are classified as Level 1, while debt securities (U.S. government agency securities, treasuries, and corporate bonds) are classified as Level 2, reflecting valuation using observable market data[52] 6. Property and Equipment, Net This note presents the breakdown of property and equipment, net of accumulated depreciation, and related depreciation expenses | Category (in thousands) | June 30, 2025 | December 31, 2024 | | :---------------------- | :------------ | :---------------- | | Laboratory equipment | $16,366 | $15,686 | | Furniture and fixtures | $2,303 | $2,303 | | Computer equipment | $431 | $431 | | Leasehold improvements | $2,046 | $1,932 | | Total property and equipment | $21,146 | $20,352 | | Less: accumulated depreciation | $(11,339) | $(9,424) | | Property and equipment, net | $9,807 | $10,928 | - Depreciation expense for the three months ended June 30, 2025, increased to $1.1 million from $0.9 million in the prior year, and for the six months, it increased to $2.0 million from $1.8 million[54] 7. Accrued Expenses and Other Current Liabilities This note details the components of accrued expenses and other current liabilities, highlighting changes in employee compensation and R&D expenses | Category (in thousands) | June 30, 2025 | December 31, 2024 | | :---------------------- | :------------ | :---------------- | | Employee compensation and benefits | $4,220 | $7,391 | | External research and development expenses | $6,180 | $4,265 | | General and administrative professional service expenses | $922 | $648 | | Other | $473 | $1,039 | | Total accrued expenses and other current liabilities | $11,795 | $13,343 | - Total accrued expenses and other current liabilities decreased from $13.343 million at December 31, 2024, to $11.795 million at June 30, 2025, primarily due to a decrease in employee compensation and benefits[55] 8. Common Stock and Preferred Stock This note outlines the authorized and outstanding common stock, recent equity offerings, and shares reserved for future issuance - As of June 30, 2025, the company had 38,033,970 shares of common stock issued and outstanding, with 150,000,000 shares authorized[24][56] - In June 2024, the company completed a registered direct offering, raising approximately $99.6 million net proceeds from the sale of 3,367,003 shares of common stock and pre-funded warrants[37][58][142] | Shares Reserved for Future Issuance | June 30, 2025 | December 31, 2024 | | :---------------------------------- | :------------ | :---------------- | | Exercise of outstanding stock options | 6,008,644 | 5,336,407 | | Vesting of outstanding restricted stock units | 2,484,038 | 1,968,374 | | Future awards under the 2021 Stock Option and Incentive Plan | 1,195,876 | 1,264,247 | | Total shares reserved for future issuance | 11,826,677 | 10,135,623 | 9. Stock-Based Compensation This note details stock-based compensation expenses, unrecognized costs for options, RSUs, and PSUs, and their vesting periods | Expense Category (in thousands) | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :------------------------------ | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Research and development expenses | $2,141 | $2,014 | $4,507 | $3,627 | | General and administrative expenses | $2,907 | $2,608 | $5,628 | $4,728 | | Total | $5,048 | $4,622 | $10,135 | $8,355 | - Total stock-based compensation expense increased by $0.426 million (9.2%) for the three months ended June 30, 2025, and by $1.78 million (21.3%) for the six months ended June 30, 2025, compared to the prior year periods[67] - As of June 30, 2025, there was $14.8 million of unrecognized compensation cost related to unvested stock options, expected to be recognized over a weighted-average period of 2.4 years[71] - Unrecognized stock-based compensation expense for restricted stock units (RSUs) was $28.4 million as of June 30, 2025, with a weighted-average remaining vesting period of 2.9 years[73] - No stock-based compensation expense was recognized for Performance Stock Units (PSUs) as of June 30, 2025, as performance conditions were not deemed probable, with $6.5 million unrecognized[74] 10. Income Taxes This note explains the income tax expense, the impact of a prior-year milestone, and the company's valuation allowance against deferred tax assets - The company recorded income tax expense of $0.2 million for both the three and six months ended June 30, 2025, a significant decrease from $2.8 million and $4.6 million for the corresponding periods in 2024[76] - The higher income tax provision in 2024 was primarily driven by a $75 million clinical advancement milestone achieved in Q1 2024. The company maintains a valuation allowance against all remaining deferred tax assets due to expected future operating losses[76][77] 11. Commitments and Contingencies This note describes significant commitments, including a workforce reduction plan and associated severance charges - In April 2025, the company approved a strategic plan including a workforce reduction of approximately 20%, resulting in a total charge of $1.9 million for severance and termination benefits during Q2 2025[80][81] - Of the $1.9 million charge, $1.7 million was recorded as R&D expenses and $0.2 million as G&A expenses. As of June 30, 2025, $1.6 million of these costs were paid[81] 12. Collaboration and License Agreements This note details the Vertex Agreement, collaboration revenue recognition, and the impact of research activity completion - The Vertex Agreement, amended in October 2023, grants Vertex an exclusive worldwide license for VX-670 and other EEV-based therapeutic candidates for DM1, with Entrada performing preclinical development and research activities[82][83] | Metric (in thousands) | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :-------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Collaboration revenue | $2,000 | $94,700 | $22,500 | $152,800 | | Cost reimbursements | $1,200 | $7,500 | $2,800 | $9,600 | | Deferred revenue recognized | $800 | $87,200 | $19,700 | $125,800 | - Collaboration revenue significantly decreased in 2025 compared to 2024, primarily because research plan activities for VX-670 were substantially completed in Q1 2025. A cumulative catch-up adjustment of $12.3 million was recorded in H1 2025 due to revised forecasted costs[85] 13. Net (Loss) Income per Share This note presents the calculation of basic and diluted net (loss) income per share, including the treatment of anti-dilutive securities | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :---------------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Net (loss) income | $(43,103) | $55,031 | $(60,452) | $78,527 | | Weighted-average common shares outstanding, basic | 41,338,752 | 34,180,549 | 41,206,974 | 33,838,811 | | Net (loss) income per share, basic | $(1.04) | $1.61 | $(1.47) | $2.32 | | Net (loss) income per share, diluted | $(1.04) | $1.55 | $(1.47) | $2.23 | - Potential common shares from unvested restricted stock units, performance stock units, and stock options were excluded from diluted EPS calculation for periods with net loss (H1 2025) as their inclusion would be anti-dilutive[90] 14. Segment Information This note clarifies that the company operates as a single segment, with the CEO as the primary decision-maker for performance assessment - The company manages its operations as a single segment, with the CEO acting as the Chief Operating Decision Maker (CODM) for performance assessment and operating decisions[91] - The segment measure of profit or loss is consolidated net (loss) income, and all tangible assets are held in the United States[91] 15. Subsequent Events This note describes significant events occurring after the balance sheet date, including new legislation and its potential financial impact - On July 4, 2025, President Trump signed the One Big Beautiful Bill Act of 2025 (OBBBA) into law, making permanent 100% bonus depreciation and domestic research cost expensing, and eliminating restrictions on orphan drug exemptions from Medicare price negotiation[92][322] - The company is currently assessing the financial impact of the OBBBA, which will be reflected in the financial statements for the three months ended September 30, 2025[93] 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 of operations, highlighting the shift to a net loss due to decreased collaboration revenue and increased R&D expenses. It details pipeline progress, liquidity, and future funding requirements, emphasizing the need for additional capital to support ongoing clinical development and commercialization efforts Overview This overview introduces Entrada Therapeutics as a clinical-stage biopharmaceutical company, its lead programs, and current financial position - Entrada Therapeutics is a clinical-stage biopharmaceutical company focused on EEV™-therapeutics for intracellular targets, with lead oligonucleotide programs for Duchenne muscular dystrophy (DMD) amenable to exon 44, 45, 50, and 51 skipping[95] - The company has ENTR-601-44 and ENTR-601-45 in clinical development, with ENTR-601-50 expected to file for regulatory clearance in Q4 2025. A partnered program, VX-670, is in development with Vertex for myotonic dystrophy type 1[95][96][97][98] - As of June 30, 2025, the company had $354.0 million in cash, cash equivalents and marketable securities, projected to fund operations into Q2 2027[95] Components of Our Results of Operations This section describes the primary drivers of the company's revenue and expenses, including collaboration agreements and operational costs - Revenue is primarily from the Vertex Agreement, with no product sales expected until regulatory approval. Future revenue may come from product sales or collaboration/license arrangements[99] - Research and development expenses include personnel, third-party contractor costs (CROs, CMOs), manufacturing process development, and costs under the Vertex Agreement. These costs are expensed as incurred[101][102] - General and administrative expenses cover personnel, legal, accounting, consulting, and facility costs. These are expected to increase with headcount and facility expansion[109][110] Results of Operations This section analyzes the company's financial performance, highlighting significant changes in collaboration revenue, R&D, G&A expenses, and net income/loss | Metric (in thousands) | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Change (YoY) | | :-------------------- | :------------------------------- | :------------------------------- | :----------- | | Collaboration revenue | $1,950 | $94,694 | $(92,744) | | Research and development | $37,877 | $32,035 | $5,842 | | General and administrative | $10,922 | $9,236 | $1,686 | | Net (loss) income | $(43,103) | $55,031 | $(98,134) | - Collaboration revenue decreased by $92.7 million for Q2 2025 due to substantial completion of VX-670 research plan activities. R&D expenses increased by $5.9 million, driven by higher personnel costs (including $1.7 million from workforce reduction) and increased direct R&D for Duchenne programs[117][118][119] | Metric (in thousands) | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Change (YoY) | | :-------------------- | :----------------------------- | :----------------------------- | :----------- | | Collaboration revenue | $22,508 | $153,814 | $(131,306) | | Research and development | $69,951 | $60,643 | $9,308 | | General and administrative | $21,196 | $18,635 | $2,561 | | Net (loss) income | $(60,452) | $78,527 | $(138,979) | - For H1 2025, collaboration revenue decreased by $131.3 million due to VX-670 research completion. R&D expenses increased by $9.3 million, primarily from a $6.2 million rise in personnel costs (including $1.7 million from workforce reduction) and a $2.7 million increase in direct R&D for Duchenne programs[124][125][126] Liquidity and Capital Resources This section assesses the company's ability to meet its financial obligations, detailing its cash position, funding requirements, and historical capital raises - The company has an accumulated deficit of $189.8 million as of June 30, 2025, and expects continued operating losses and negative cash flows[134] - Cash, cash equivalents, and marketable securities totaled $354.0 million as of June 30, 2025, estimated to fund operations into Q2 2027. Additional financing will be required to support ongoing operations and growth strategy[138][139][150] - Since inception, the company has raised over $850.0 million gross proceeds from equity sales and the Vertex Agreement. A $150.0 million 'at the market offering' program was established in September 2023, but no shares have been sold under it as of June 30, 2025[139][140][141] | Cash Flow Activity (in thousands) | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :-------------------------------- | :----------------------------- | :----------------------------- | | Net cash (used in) provided by operating activities | $(67,996) | $14,314 | | Net cash provided by investing activities | $38,580 | $1,764 | | Net cash provided by financing activities | $687 | $101,573 | Contractual Obligations and Commitments This section outlines the company's contractual obligations and commitments, including any material changes during the reporting period - No material changes to lease commitments or license agreements were reported during the six months ended June 30, 2025[154][156] - The workforce reduction in April 2025 resulted in a $1.9 million charge for severance and benefits, with $1.6 million paid by June 30, 2025[157][158] Emerging Growth Company Status This section explains the company's status as an 'emerging growth company' and the associated reduced reporting requirements under the JOBS Act - The company is an 'emerging growth company' (EGC) under the JOBS Act, allowing it to take advantage of reduced reporting requirements, such as providing only two years of audited financial statements and delaying adoption of new accounting standards[159][160][473][474] - EGC status allows exemption from auditor attestation on internal controls over financial reporting and reduced executive compensation disclosures[166][479] Recently Issued Accounting Pronouncements This section directs readers to relevant notes for details on recently issued accounting pronouncements - Refer to Note 2, Summary of Significant Accounting Policies, for details on recently issued accounting pronouncements[161] Item 3. Quantitative and Qualitative Disclosures about Market Risk This section discusses the company's exposure to market risks, including interest rate risk, inflation, and foreign currency exchange risk. It notes that changes in these factors have not had a material impact on historical financial position but acknowledges potential future effects - The company is exposed to interest rate market risk through its cash, cash equivalents, and marketable securities, which are primarily U.S. government securities, investment-grade corporate bonds, and money market funds[162] - An immediate 5% change in interest rates is not expected to have a material effect on the fair market value of the investment portfolio due to its short-term duration and low-risk profile[163] - Inflation generally affects labor and contract costs but has not had a material effect on results of operations. Foreign currency exchange rate fluctuations from international vendor contracts have also not had a material effect[164][165] Item 4. Controls and Procedures Management, including the CEO and CFO, evaluated the effectiveness of disclosure controls and procedures as of June 30, 2025, concluding they were effective at a reasonable assurance level. No material changes in internal control over financial reporting occurred during the quarter - As of June 30, 2025, the company's disclosure controls and procedures were evaluated and deemed effective at the reasonable assurance level by management, including the CEO and CFO[167][168] - There were no changes in internal control over financial reporting during the quarter ended June 30, 2025, that have materially affected, or are reasonably likely to materially affect, internal control over financial reporting[169] PART II — OTHER INFORMATION This section covers legal proceedings, risk factors, equity sales, defaults, mine safety, other information, and exhibits Item 1. Legal Proceedings The company is involved in ordinary course legal claims and a specific lawsuit filed by Ohio State Innovation Foundation (OSIF) in February 2025, alleging breach of contract related to sublicensing fees. Management believes the resolution of these matters will not materially impact the company's financial position or results of operations - OSIF filed a complaint against the company on February 7, 2025, alleging breach of contract regarding sublicensing fees from a December 2018 license agreement[173] - The complaint seeks compensatory damages, pre-judgment and post-judgment interest, and attorneys' fees and costs, as well as an order for specific performance. The company disputes the claims and intends to vigorously defend against them[173] Item 1A. Risk Factors This section details numerous risks associated with the company's business, including its limited operating history, significant losses, dependence on additional financing, and the inherent uncertainties of developing novel EEV-based therapeutic candidates. It also covers risks related to regulatory approvals, reliance on third parties, market acceptance, competition, business operations, intellectual property, and common stock ownership Risks Related to Our Limited Operating History, Financial Position and Capital Requirements This section outlines risks stemming from the company's short operational history, substantial financial losses, and ongoing need for external capital - The company has a limited operating history since its inception in 2016, has incurred significant operating losses ($189.8 million accumulated deficit as of June 30, 2025), and expects to continue incurring losses for the foreseeable future[175][176] - Additional financing will be required to achieve goals, and failure to obtain necessary capital could force delays or termination of development programs and commercialization efforts[181] - Operating results may fluctuate significantly due to factors like collaboration agreements, milestone payments, stock-based compensation, and the timing/cost of R&D activities, making future predictions difficult[185][187][188] Risks Related to the Discovery, Development and Regulatory Approval of Our Therapeutic Candidates This section details the substantial risks involved in the discovery, development, and regulatory approval processes for the company's novel therapeutic candidates - The company is in early development stages, with lead candidates ENTR-601-44, ENTR-601-45 in early clinical stage, and ENTR-601-50, ENTR-601-51 in preclinical/discovery. Commercialization is years away, if ever, and depends heavily on successful development and regulatory approval[189][191] - Development of EEV therapeutic candidates is based on a novel, unproven approach, making it difficult to predict development time, cost, and regulatory approval outcomes. Preclinical results are not necessarily predictive of clinical trial outcomes[197][201] - Substantial delays in clinical trials or failure to demonstrate safety and efficacy could prevent commercialization. Side effects or adverse events could delay or preclude approval, or limit commercial profile[204][218] - The company may pursue accelerated approval pathways (Fast Track, Breakthrough Therapy, Orphan Drug designation) but cannot guarantee obtaining or maintaining benefits, or that such pathways will lead to faster approval[236][240][241][243][244] Risks Related to Our Reliance on Third Parties This section highlights the risks associated with the company's heavy dependence on third-party contractors for manufacturing, research, and clinical testing activities - The company relies heavily on third parties (CMOs, CROs) for manufacturing, research, and preclinical/clinical testing, which reduces direct control and poses risks if these parties do not perform satisfactorily or terminate engagements[262][263][264] - Manufacturing EEV-based therapeutic candidates is complex and novel, potentially leading to difficulties in scaling, product release, shelf life, and supply chain management, which could delay clinical trials or product supply[272][274][275][276] - Reliance on single- or limited-source suppliers for raw materials and components creates risks of supply disruptions, price increases, or delays, potentially impacting clinical and commercial supply[283][284] - Collaborations with third parties, such as the Vertex Agreement, involve risks including limited control over partner resources, potential for non-performance, delays, or termination, which could impact funding and development[285][286][287][288] Risks Related to Commercialization of Our Therapeutic Candidates This section addresses the challenges and uncertainties involved in achieving market acceptance and commercial success for the company's therapeutic candidates - Commercial success depends on market acceptance by physicians, patients, and healthcare payors, which is influenced by efficacy, safety, pricing, cost-effectiveness, and reimbursement policies[297][298][299][300] - The company faces significant competition from larger, better-funded pharmaceutical and biotechnology companies developing treatments for DMD and DM1, including existing therapies and products in development[306][307][309][310] - Unfavorable pricing regulations or third-party coverage and reimbursement policies, both domestically and internationally, could hinder the ability to generate sufficient revenue and recoup investment[299][301][305] Risks Related to Our Business Operations and Industry This section covers various operational and industry-specific risks, including human capital management, regulatory compliance, and the impact of new technologies - Future success depends on retaining key employees and attracting qualified personnel in a highly competitive industry, particularly in the Boston area[311][312] - The strategic plan and 20% workforce reduction in April 2025 may not yield anticipated cost savings, could incur greater-than-expected costs, and may disrupt business operations or affect employee morale and retention[316] - Compliance with environmental, health, and safety laws is crucial; failure could lead to fines, penalties, and significant costs. The company does not maintain insurance for environmental liability or toxic tort claims[333][335][336] - Relationships with healthcare providers and payors are subject to anti-kickback, fraud and abuse, and other healthcare laws, which could expose the company to criminal sanctions, civil penalties, and reputational harm[339][342] - The use of new and evolving technologies, such as artificial intelligence, presents risks including security vulnerabilities, ethical concerns, and increased compliance costs due to emerging regulations like the EU's AI Act and state-level laws[440][441][442] Risks Related to Our Intellectual Property This section details the risks associated with obtaining, maintaining, and enforcing intellectual property rights crucial for the company's therapeutic programs and technologies - The company's success relies on obtaining and maintaining patent protection for its therapeutic programs and technologies. Failure to do so, or if patent scope is insufficient, could allow competitors to commercialize similar products[362][365] - Patent prosecution is expensive and complex, with no guarantee that applications will issue as patents or provide meaningful protection. Issued patents can be challenged, invalidated, or circumvented[367][368][369][370] - Reliance on third-party licenses (e.g., OSIF) means compliance with obligations is critical; failure could lead to loss of intellectual property rights, liability, or inability to develop/commercialize therapeutic candidates[372][373][375] - Protecting intellectual property globally is expensive and challenging, as foreign laws may not offer the same level of protection as the U.S., potentially allowing competitors to use technologies in other jurisdictions[382][383] - The company relies on trade secrets and confidentiality agreements for unpatented know-how. Breaches or independent development by competitors could harm its competitive position[394][395] Risks Related to Ownership of Our Common Stock This section addresses risks pertinent to the company's common stock, including market volatility, potential dilution, and anti-takeover provisions - An active, liquid, and orderly trading market for common stock may not be sustained, making it difficult for stockholders to sell shares and potentially reducing fair market value[451] - The market price of common stock is highly volatile, influenced by factors such as clinical trial results, regulatory actions, competition, and macroeconomic conditions, leading to potential loss of investment[453][455] - Future sales and issuances of common stock or rights to purchase common stock (e.g., under the 2021 Plan and 2025 Inducement Equity Plan) could result in additional dilution for existing stockholders and cause the stock price to fall[468][469] - Anti-takeover provisions in the company's charter, bylaws, and Delaware law could discourage, delay, or prevent a change in control or management, potentially depressing the stock price[478][481][482] General Risk Factors This section covers broad risks applicable to the company's operations as a public entity, including compliance costs, internal controls, and government agency disruptions - Operating as a public company incurs significant legal, accounting, and compliance costs, requiring substantial management time and potentially increasing director and officer liability insurance expenses[485] - The company's internal control over financial reporting may not prevent or detect all errors or fraud, and failure to maintain effective controls could adversely affect investor confidence and stock value[488][490][491] - Disruptions at the FDA and other government agencies (e.g., staffing reductions, funding shortages, global health concerns) could delay product development, approval, or commercialization[494][495][496] Item 2. Unregistered Sales of Equity Securities and Use of Proceeds This section confirms that there were no unregistered sales of equity securities or purchases of equity securities by the company during the three months ended June 30, 2025 - No unregistered equity securities were sold during the three months ended June 30, 2025[498] - No equity securities were purchased by the company during the three months ended June 30, 2025[499] Item 3. Defaults Upon Senior Securities This section states that there were no defaults upon senior securities during the reported period - There were no defaults upon senior securities during the period[500] Item 4. Mine Safety Disclosures This item is not applicable to the company - This item is not applicable to the company[501] Item 5. Other Information This section reports on Rule 10b5-1 trading arrangements, stating that no officers or directors adopted or terminated such plans for the purchase or sale of company securities during the three months ended June 30, 2025 - No officers or directors adopted or terminated Rule 10b5-1 trading arrangements for company securities during the three months ended June 30, 2025[502][503] Item 6. Exhibits This section lists all exhibits filed or furnished as part of this Quarterly Report on Form 10-Q, including organizational documents, specimen stock certificates, investor rights agreements, pre-funded warrants, compensation policies, and certifications - Exhibits include the Fourth Amended and Restated Certificate of Incorporation, Amended and Restated Bylaws, Specimen Common Stock Certificate, Amended and Restated Investors' Rights Agreement, Form of Pre-Funded Warrant, Third Amended and Restated Non-Employee Director Compensation Policy, and various certifications[505] SIGNATURES This section confirms the official signing of the report by the Chief Executive Officer and Chief Financial Officer - The report was signed on August 6, 2025, by Dipal Doshi, Chief Executive Officer and Director, and Kory Wentworth, Chief Financial Officer[510]