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Core Insights - Edgewise Therapeutics, Inc. is participating in the 30th International Annual Congress of the World Muscle Society (WMS) to present long-term data on sevasemten, demonstrating sustained disease stabilization in Becker muscular dystrophy compared to natural history [1][2]. Company Overview - Edgewise Therapeutics is a biopharmaceutical company focused on developing novel therapeutics for muscular dystrophies and serious cardiac conditions, with a strong emphasis on muscle physiology [7]. - The company is advancing sevasemten, a first-in-class fast skeletal myosin inhibitor, currently in late-stage clinical trials for Becker and Duchenne muscular dystrophies [7]. Product Information - Sevasemten is designed to prevent contraction-induced muscle damage in muscular dystrophies, with a unique mechanism of action that limits muscle damage due to the absence of functional dystrophin [5]. - The product has received several regulatory designations, including FDA Orphan Drug Designation and Fast Track designations for Becker and Duchenne muscular dystrophies, as well as EMA Orphan Drug Designations [5]. Upcoming Events - The Edgewise symposium at WMS will take place on October 8, 2025, featuring key opinion leaders discussing the clinical progress of sevasemten and results from the CANYON and MESA trials [2]. - Scientific posters detailing trial results will be presented on October 10, 2025, highlighting the efficacy of sevasemten in reducing muscle damage biomarkers and stabilizing function in Becker muscular dystrophy [3].

Core Viewpoint - Edgewise Therapeutics, Inc. (EWTX) shows significant upside potential with a mean price target of $39.17, indicating an 182% increase from the current price of $13.89 [1][11]. Price Targets and Estimates - The mean estimate consists of 12 short-term price targets with a standard deviation of $12.96, suggesting variability among analysts [2]. - The lowest estimate is $14.00, indicating a modest 0.8% increase, while the highest estimate is $51.00, reflecting a potential surge of 267.2% [2]. - A low standard deviation indicates a high degree of agreement among analysts regarding the stock's price movement [9]. Earnings Estimates - Analysts have shown increasing optimism about EWTX's earnings prospects, with a strong consensus on higher EPS estimates [11]. - The Zacks Consensus Estimate for the current year has increased by 1% over the past month, with one estimate rising and no negative revisions [12]. - EWTX holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimates [13]. Analyst Behavior and Price Target Reliability - Analysts often set overly optimistic price targets due to business incentives, which can lead to inflated estimates [8]. - While price targets can provide insights, they should be approached with skepticism as they may not accurately predict stock price movements [10].

Core Viewpoint - Edgewise Therapeutics, Inc. (EWTX) shows significant upside potential with a mean price target of $39.17, indicating a 173% increase from its last trading price of $14.35 [1][11]. Price Targets and Analyst Consensus - The average price target for EWTX is based on 12 short-term estimates, ranging from a low of $14.00 to a high of $51.00, with a standard deviation of $12.96, suggesting variability in analyst predictions [2][9]. - The lowest estimate indicates a potential decline of 2.4%, while the highest suggests a 255.4% upside [2]. - A low standard deviation among price targets indicates strong agreement among analysts regarding the stock's price direction [9]. Earnings Estimates and Analyst Optimism - Analysts have shown increasing optimism about EWTX's earnings prospects, as evidenced by a positive trend in earnings estimate revisions [11]. - The Zacks Consensus Estimate for the current year has risen by 9.1% over the past month, with eight estimates increasing and no negative revisions [12]. - EWTX holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimate factors [13]. Caution on Price Targets - While price targets are a common tool for investors, they can often mislead, as empirical research indicates that they rarely predict actual stock price movements accurately [7][10]. - Analysts may set overly optimistic price targets due to business incentives, which can inflate expectations [8].

Core Viewpoint - Edgewise Therapeutics, Inc. is under investigation for possible securities fraud following a decline in stock price after the announcement of clinical trial results for its lead asset, sevasemten [3][4]. Group 1: Company Overview - Edgewise Therapeutics, Inc. is focused on developing treatments for Becker and Duchenne muscular dystrophies [3]. - The company's lead asset is sevasemten, which recently reported clinical trial data [3]. Group 2: Recent Developments - On June 26, 2025, Edgewise Therapeutics announced clinical trial results that it characterized as "positive," but the FDA deemed the data insufficient for accelerated approval [3]. - Following this announcement, Edgewise's stock price fell by $1.27 per share, approximately 8.86%, closing at $14.33 per share [4]. Group 3: Legal Actions - The Portnoy Law Firm has initiated an investigation into Edgewise Therapeutics and may file a class action on behalf of investors who lost money [1]. - Investors are encouraged to contact the law firm for a complimentary case evaluation regarding their legal rights and options for recovering losses [2].

[Special Note Regarding Forward-Looking Statements](index=3&type=section&id=Special%20Note%20Regarding%20Forward-Looking%20Statements) This section highlights that the Quarterly Report contains forward-looking statements about the company's future operations, financial position, business strategy, and product development. It cautions readers that these statements involve risks and uncertainties, are not guarantees of future performance, and actual results could differ materially from projections - The report contains forward-looking statements regarding future results of operations, financial position, business strategy, development plans, preclinical studies, clinical trials, research and development costs, regulatory strategy, and management objectives[8](index=8&type=chunk) - Readers are cautioned not to rely on these statements as predictions of future events, as they are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified[11](index=11&type=chunk) - Key areas covered by forward-looking statements include the safety and efficacy of product candidates (sevasemten, EDG-7500, EDG-003 program), timing of clinical trials and regulatory approvals, market opportunity, intellectual property, and financial performance[8](index=8&type=chunk)[9](index=9&type=chunk)[10](index=10&type=chunk) [Part I. Financial Information](index=8&type=section&id=Part%20I%20Financial%20Information) [Item 1. Financial Statements (Unaudited)](index=8&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) This section presents the unaudited condensed financial statements, including the balance sheets, statements of operations and comprehensive loss, statements of stockholders' equity, and statements of cash flows, along with their accompanying notes, providing a snapshot of the company's financial position and performance for the reported interim periods [Condensed Balance Sheets](index=8&type=section&id=Condensed%20Balance%20Sheets) The Condensed Balance Sheets provide a summary of the company's assets, liabilities, and stockholders' equity as of June 30, 2025, and December 31, 2024, showing an increase in total assets and stockholders' equity, primarily driven by an increase in marketable securities and additional paid-in capital | Metric | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | Change (in thousands) | | :-------------------------------- | :----------------------------- | :------------------------------- | :-------------------- | | Total assets | $613,334 | $486,817 | $126,517 | | Total liabilities | $24,263 | $27,601 | $(3,338) | | Total stockholders' equity | $589,071 | $459,216 | $129,855 | - Marketable securities, available for sale, significantly increased from **$428,504 thousand** as of December 31, 2024, to **$550,588 thousand** as of June 30, 2025[16](index=16&type=chunk) - Additional paid-in capital rose from **$837,363 thousand** to **$1,044,346 thousand**, contributing to the increase in stockholders' equity[16](index=16&type=chunk) [Condensed Statements of Operations and Comprehensive Loss](index=10&type=section&id=Condensed%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) The Condensed Statements of Operations and Comprehensive Loss show an increase in net loss for both the three and six months ended June 30, 2025, compared to the same periods in 2024, primarily due to higher operating expenses, particularly in research and development | Metric | Three months ended June 30, 2025 (in thousands) | Three months ended June 30, 2024 (in thousands) | Six months ended June 30, 2025 (in thousands) | Six months ended June 30, 2024 (in thousands) | | :------------------------------------ | :------------------------------------------ | :------------------------------------------ | :---------------------------------------- | :---------------------------------------- | | Research and development expenses | $33,558 | $30,680 | $70,315 | $58,374 | | General and administrative expenses | $9,052 | $7,427 | $18,254 | $14,486 | | Total operating expenses | $42,610 | $38,107 | $88,569 | $72,860 | | Interest income | $6,495 | $6,610 | $11,656 | $12,838 | | Net loss | $(36,115) | $(31,497) | $(76,913) | $(60,022) | | Net loss per share, basic and diluted | $(0.34) | $(0.34) | $(0.77) | $(0.66) | - Research and development expenses increased by **$2,878 thousand** for the three months ended June 30, 2025, and by **$11,941 thousand** for the six months ended June 30, 2025, compared to the respective prior periods[17](index=17&type=chunk) - General and administrative expenses increased by **$1,625 thousand** for the three months and **$3,768 thousand** for the six months ended June 30, 2025, compared to the respective prior periods[17](index=17&type=chunk) [Condensed Statements of Stockholders' Equity](index=11&type=section&id=Condensed%20Statements%20of%20Stockholders'%20Equity) The Condensed Statements of Stockholders' Equity detail changes in common stock, additional paid-in capital, accumulated other comprehensive income, and accumulated deficit, reflecting significant capital raises and ongoing net losses, leading to an overall increase in total stockholders' equity | Metric | December 31, 2024 (in thousands) | June 30, 2025 (in thousands) | | :-------------------------- | :------------------------------- | :--------------------------- | | Common Stock (shares) | 94,838,466 | 105,341,296 | | Common Stock (amount) | $9 | $10 | | Additional Paid-In Capital | $837,363 | $1,044,346 | | Accumulated Deficit | $(378,576) | $(455,489) | | Total Stockholders' Equity | $459,216 | $589,071 | - Issuance of common stock, net of offering costs, contributed **$187,101 thousand** to additional paid-in capital during the six months ended June 30, 2025[20](index=20&type=chunk) - Stock-based compensation expense added **$16,792 thousand** to additional paid-in capital for the six months ended June 30, 2025[20](index=20&type=chunk)[56](index=56&type=chunk) [Condensed Statements of Cash Flows](index=12&type=section&id=Condensed%20Statements%20of%20Cash%20Flows) The Condensed Statements of Cash Flows indicate that the company continued to use cash in operating and investing activities, but this was more than offset by significant cash provided by financing activities, primarily from common stock offerings, resulting in a net increase in cash and cash equivalents for the six months ended June 30, 2025 | Cash Flow Activity | Six months ended June 30, 2025 (in thousands) | Six months ended June 30, 2024 (in thousands) | | :---------------------------------- | :------------------------------------------ | :------------------------------------------ | | Net cash used in operating activities | $(70,448) | $(54,697) | | Net cash used in investing activities | $(118,268) | $(230,940) | | Net cash provided by financing activities | $190,454 | $243,025 | | Net change in cash and cash equivalents | $1,738 | $(42,612) | - Cash used in operating activities increased to **$70.4 million** in 2025 from **$54.7 million** in 2024, primarily due to a higher net loss[23](index=23&type=chunk)[98](index=98&type=chunk)[99](index=99&type=chunk) - Cash provided by financing activities was **$190.5 million** in 2025, mainly from the April 2025 offering (**$187.1 million** net proceeds), compared to **$243.0 million** in 2024[23](index=23&type=chunk)[102](index=102&type=chunk)[103](index=103&type=chunk) [Notes to Condensed Financial Statements](index=13&type=section&id=Notes%20to%20Condensed%20Financial%20Statements) These notes provide detailed disclosures and explanations for the condensed financial statements, covering the company's business, significant accounting policies, equity structure, stock-based compensation, commitments, contingencies, net loss per share, property and equipment, accrued expenses, and segment reporting [NOTE 1 DESCRIPTION OF BUSINESS](index=13&type=section&id=NOTE%201%20DESCRIPTION%20OF%20BUSINESS) Edgewise Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on severe muscle diseases, with lead product candidates sevasemten (for dystrophinopathies) and EDG-7500 (for hypertrophic cardiomyopathy). The company has an accumulated deficit of $455.5 million but believes its $594.0 million in cash, cash equivalents, and marketable securities as of June 30, 2025, will fund operations for at least the next 12 months, supported by recent equity offerings - Edgewise Therapeutics is a clinical-stage biopharmaceutical company developing treatments for severe muscle diseases, with lead candidates sevasemten (Phase 2 for dystrophinopathies) and EDG-7500 (Phase 2 for hypertrophic cardiomyopathy)[25](index=25&type=chunk) | Financial Metric | As of June 30, 2025 (in millions) | | :------------------------------------ | :-------------------------------- | | Accumulated Deficit | $(455.5) | | Cash, Cash Equivalents, and Marketable Securities | $594.0 | - The company raised **$187.1 million** net proceeds from an underwritten registered direct offering of 9,935,419 shares of common stock at **$20.13 per share** in April 2025[33](index=33&type=chunk) [NOTE 2 SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES](index=15&type=section&id=NOTE%202%20SUMMARY%20OF%20SIGNIFICANT%20ACCOUNTING%20POLICIES) This note outlines the basis of presentation for the interim financial statements, confirming adherence to U.S. GAAP and no material changes to significant accounting policies since the last annual report. It details the classification and valuation of marketable securities as available-for-sale and discusses recently issued accounting pronouncements, ASU 2023-09 and ASU 2024-03, which are being evaluated for impact - Interim financial statements are prepared in accordance with U.S. GAAP, with no material changes to significant accounting policies since the December 31, 2024 Annual Report on Form 10-K[36](index=36&type=chunk)[37](index=37&type=chunk) - All marketable securities are classified as 'available-for-sale' and carried at fair value, with unrealized gains and losses excluded from earnings and included in other comprehensive income (loss)[38](index=38&type=chunk) | Fair Value Hierarchy | As of June 30, 2025 (in thousands) | As of December 31, 2024 (in thousands) | | :------------------- | :--------------------------------- | :----------------------------------- | | Level 1 (Money market funds) | $43,218 | $41,474 | | Level 2 (Marketable securities) | $550,588 | $428,504 | | Total Fair Market Value | $593,806 | $469,978 | [NOTE 3 PREFERRED STOCK AND COMMON STOCK](index=19&type=section&id=NOTE%203%20PREFERRED%20STOCK%20AND%20COMMON%20STOCK) The company is authorized to issue common stock and preferred stock, with 1,000,000,000 shares of common stock and 200,000,000 shares of preferred stock authorized, each with a par value of $0.0001 per share. As of June 30, 2025, 105,341,296 shares of common stock were issued and outstanding, with no preferred stock issued - Total authorized shares are **1,200,000,000**, comprising **1,000,000,000 common stock shares** and **200,000,000 preferred stock shares**, each with a **$0.0001 par value**[45](index=45&type=chunk) - As of June 30, 2025, **105,341,296 shares of common stock** were issued and outstanding, while no preferred stock shares were issued or outstanding[16](index=16&type=chunk)[45](index=45&type=chunk) - Common stockholders are entitled to dividends when declared by the board and after any convertible preferred share dividends are paid, with each share carrying one vote[46](index=46&type=chunk) [NOTE 4 STOCK-BASED COMPENSATION AWARDS](index=19&type=section&id=NOTE%204%20STOCK-BASED%20COMPENSATION%20AWARDS) This note details the company's equity incentive plans, including the 2021 Equity Incentive Plan, the 2024 Inducement Equity Incentive Plan, Founder Stock Options, and the 2021 Employee Stock Purchase Plan (ESPP). It also provides a breakdown of stock-based compensation expense allocated to research and development and general and administrative functions - The 2021 Equity Incentive Plan, effective March 2021, reserves shares for incentive and nonstatutory stock options, restricted stock, RSUs, and other awards, with **5,519,964 shares** available for future issuance as of June 30, 2025[47](index=47&type=chunk)[48](index=48&type=chunk)[49](index=49&type=chunk) - The 2024 Inducement Equity Incentive Plan, adopted August 2024, reserved **2,000,000 shares** for issuance to new employees as an inducement, with all shares remaining available as of June 30, 2025[50](index=50&type=chunk)[51](index=51&type=chunk)[52](index=52&type=chunk) | Expense Category | Three months ended June 30, 2025 (in thousands) | Three months ended June 30, 2024 (in thousands) | Six months ended June 30, 2025 (in thousands) | Six months ended June 30, 2024 (in thousands) | | :------------------------------- | :------------------------------------------ | :------------------------------------------ | :---------------------------------------- | :---------------------------------------- | | Research and development | $4,489 | $2,924 | $10,356 | $5,624 | | General and administrative | $3,244 | $2,300 | $6,436 | $4,471 | | Total stock-based compensation expense | $7,733 | $5,224 | $16,792 | $10,095 | [NOTE 5 COMMITMENTS AND CONTINGENCIES](index=23&type=section&id=NOTE%205%20COMMITMENTS%20AND%20CONTINGENCIES) The company records liabilities for loss contingencies when probable and estimable, but was not subject to any material legal proceedings during the three and six months ended June 30, 2025. It also provides indemnification to various parties in the ordinary course of business, with no material costs incurred to date - The company was not subject to any material legal proceedings during the three and six months ended June 30, 2025, and no material legal proceedings are currently pending or threatened[58](index=58&type=chunk) - The company provides indemnification to vendors, lessors, business partners, and directors, with maximum potential future payments often unlimited, but no material costs have been incurred to date[59](index=59&type=chunk) [NOTE 6 NET LOSS PER SHARE](index=23&type=section&id=NOTE%206%20NET%20LOSS%20PER%20SHARE) This note details the computation of basic and diluted net loss per share. Due to net losses in all periods presented, diluted net loss per share is the same as basic net loss per share, as potentially dilutive securities would be anti-dilutive | 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 | $(36,115) | $(31,497) | $(76,913) | $(60,022) | | Weighted-average shares outstanding, basic and diluted | 104,940,493 | 93,515,356 | 100,062,373 | 90,541,332 | | Net loss per share, basic and diluted | $(0.34) | $(0.34) | $(0.77) | $(0.66) | | Potentially Dilutive Securities | Three months ended June 30, 2025 | Three months ended June 30, 2024 | Six months ended June 30, 2025 | Six months ended June 30, 2024 | | :------------------------------ | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Options to purchase common stock | 16,825,819 | 15,656,198 | 16,809,703 | 15,735,754 | | Unvested restricted stock units | 633,509 | 113,796 | 651,722 | 142,800 | | Total | 17,459,328 | 15,769,994 | 17,461,425 | 15,878,554 | - For all periods presented, diluted net loss per common share is the same as basic net loss per common share because the company reported a net loss, making common stock options and unvested restricted stock units anti-dilutive[60](index=60&type=chunk) [NOTE 7 PROPERTY AND EQUIPMENT](index=25&type=section&id=NOTE%207%20PROPERTY%20AND%20EQUIPMENT) This note provides a breakdown of property and equipment, net of accumulated depreciation, showing a decrease in net value from December 31, 2024, to June 30, 2025, primarily due to depreciation expense | Property and Equipment (in thousands) | As of June 30, 2025 | As of December 31, 2024 | | :------------------------------------ | :------------------ | :---------------------- | | Leasehold improvements | $9,646 | $9,646 | | Laboratory equipment | $3,863 | $3,735 | | Computers and software | $296 | $296 | | Furniture and fixtures | $511 | $511 | | Property and equipment, at cost | $14,316 | $14,188 | | Less: accumulated depreciation | $(5,721) | $(4,685) | | Property and equipment, net | $8,595 | $9,503 | - Depreciation expense was **$0.5 million** for the three months ended June 30, 2025, and **$1.0 million** for the six months ended June 30, 2025[62](index=62&type=chunk) [NOTE 8 ACCRUED OTHER EXPENSES](index=25&type=section&id=NOTE%208%20ACCRUED%20OTHER%20EXPENSES) This note details the components of accrued other expenses, which primarily consist of accrued research and development costs and other accrued expenses, showing a decrease from December 31, 2024, to June 30, 2025 | Accrued Other Expenses (in thousands) | As of June 30, 2025 | As of December 31, 2024 | | :------------------------------------ | :------------------ | :---------------------- | | Accrued research and development costs | $5,254 | $6,488 | | Accrued other | $682 | $741 | | Total accrued other expenses | $5,936 | $7,229 | - Accrued research and development costs decreased by **$1,234 thousand** from December 31, 2024, to June 30, 2025[63](index=63&type=chunk) [NOTE 9 SEGMENT REPORTING](index=25&type=section&id=NOTE%209%20SEGMENT%20REPORTING) This note confirms no changes in the company's basis of segmentation or measurement of segment profit or loss since the last annual report. It provides a breakdown of operating expenses and reconciliation to net loss, highlighting increases in personnel and stock-based compensation expenses - No changes in the company's assessment of segmentation or measurement of segment profit or loss since the Annual Report on Form 10-K filed March 3, 2025[64](index=64&type=chunk) | Operating Expenses (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 | | :-------------------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Contracted research expense | $19,017 | $20,313 | $40,316 | $38,118 | | Personnel expense | $10,894 | $8,002 | $21,518 | $15,735 | | Stock-based compensation expense | $7,733 | $5,224 | $16,792 | $10,095 | | Other segment expense | $4,443 | $4,088 | $8,907 | $7,959 | | Depreciation | $523 | $480 | $1,036 | $953 | | Segment net loss | $(42,610) | $(38,107) | $(88,569) | $(72,860) | | Interest income | $6,495 | $6,610 | $11,656 | $12,838 | | Net Loss | $(36,115) | $(31,497) | $(76,913) | $(60,022) | - Personnel expense increased by **$2,892 thousand** for the three months and **$5,783 thousand** for the six months ended June 30, 2025, compared to the respective prior periods[65](index=65&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=27&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) This section provides management's perspective on the company's financial condition and results of operations, discussing key financial metrics, operational highlights, macroeconomic factors, and future outlook. It details the drivers behind changes in operating expenses, net loss, and liquidity, emphasizing the company's clinical-stage focus and ongoing capital requirements [Overview](index=27&type=section&id=Overview) Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on severe muscle diseases, advancing two clinical-stage programs: sevasemten for muscular dystrophies and EDG-7500 for hypertrophic cardiomyopathy. The company has incurred significant net losses ($36.1M for Q2 2025, $76.9M for H1 2025) and expects continued losses, but believes its $594.0 million in cash, cash equivalents, and marketable securities as of June 30, 2025, will fund operations for at least the next 12 months - Edgewise Therapeutics is a clinical-stage biopharmaceutical company with a precision medicine muscle platform, advancing sevasemten (Phase 2 for muscular dystrophies) and EDG-7500 (Phase 2 for hypertrophic cardiomyopathy)[67](index=67&type=chunk) | Metric | Three months ended June 30, 2025 (in millions) | Six months ended June 30, 2025 (in millions) | | :------- | :----------------------------------------- | :---------------------------------------- | | Net Loss | $(36.1) | $(76.9) | | Accumulated Deficit (as of June 30, 2025) | $(455.5) | $(455.5) | | Cash, Cash Equivalents and Marketable Securities (as of June 30, 2025) | $594.0 | $594.0 | - The company expects to continue incurring significant losses as it advances product candidates through preclinical development and clinical trials, and seeks regulatory approval[69](index=69&type=chunk) [Macroeconomic and Geopolitical Developments](index=29&type=section&id=Macroeconomic%20and%20Geopolitical%20Developments) The company is actively monitoring macroeconomic and geopolitical developments, including inflation, banking sector instability, international conflicts, and public health pandemics, to assess their potential impact on its business, operations, and research and development timelines - The company monitors macroeconomic and geopolitical developments such as inflation, banking sector instability, tightening credit markets, changes in U.S. government policy, international conflicts, and public health pandemics[71](index=71&type=chunk) - The extent, severity, and duration of these impacts on business, operations, and R&D timelines are unpredictable[71](index=71&type=chunk) [Components of Our Results of Operations](index=29&type=section&id=Components%20of%20Our%20Results%20of%20Operations) This section defines the primary components of the company's results of operations: research and development expenses, general and administrative expenses, and interest income. It explains what each category includes and how they are expected to trend in the future [Research and development expenses](index=29&type=section&id=Research%20and%20development%20expenses) Research and development expenses primarily consist of costs for discovering and developing product candidates, including employee-related expenses, external consultant fees, manufacturing costs for clinical supply, preclinical development services, and laboratory supplies. These expenses are expected to increase substantially as lead candidates (sevasemten, EDG-7500, EDG-003 program) advance through clinical trials - R&D expenses include employee costs (salaries, bonuses, stock-based compensation), external consultant/CRO fees, manufacturing costs for clinical trials, preclinical development services, laboratory supplies, and regulatory compliance[72](index=72&type=chunk)[74](index=74&type=chunk) - The majority of R&D expenses are incurred to advance sevasemten (Phase 2 trials: CANYON, LYNX, FOX, DUNE, GRAND CANYON pivotal cohort) and EDG-7500 (multipart Phase 2 CIRRUS-HCM trial)[72](index=72&type=chunk)[77](index=77&type=chunk) - R&D expenses are expected to increase substantially over the next several years due to advancing clinical trials, developing the drug discovery platform, discovering new candidates, and hiring additional personnel[77](index=77&type=chunk) [General and administrative expenses](index=33&type=section&id=General%20and%20administrative%20expenses) General and administrative expenses primarily cover salaries, benefits, and stock-based compensation for executive, finance, accounting, legal, and administrative personnel, along with facilities and professional fees. These expenses are anticipated to increase with future headcount growth to support program development - G&A expenses consist mainly of personnel-related costs (salaries, benefits, stock-based compensation) for executive, finance, accounting, legal, and administrative functions[80](index=80&type=chunk) - Other G&A costs include facilities, insurance, and professional fees for legal, patent, consulting, investor relations, accounting, and audit services[80](index=80&type=chunk) - G&A expenses are expected to increase in the future as the company increases headcount to support the continued research and development of its programs[81](index=81&type=chunk) [Interest income](index=33&type=section&id=Interest%20income) Interest income is generated primarily from the company's cash, cash equivalents, and marketable securities - Interest income is primarily derived from investments in cash, cash equivalents, and marketable securities[82](index=82&type=chunk) [Results of Operations](index=33&type=section&id=Results%20of%20Operations) This section provides a detailed comparison of the company's financial results for the three and six months ended June 30, 2025, versus the same periods in 2024, highlighting the changes in operating expenses, net loss, and interest income [Comparison of the three months ended June 30, 2025 and 2024](index=33&type=section&id=Comparison%20of%20the%20three%20months%20ended%20June%2030,%202025%20and%202024) For the three months ended June 30, 2025, the company experienced an increased net loss of $4.6 million compared to the prior year, driven by higher research and development expenses (up $2.9 million) and general and administrative expenses (up $1.6 million), partially offset by a slight decrease in interest income | Metric | 2025 (in thousands) | 2024 (in thousands) | Change (in thousands) | | :-------------------------- | :------------------ | :------------------ | :-------------------- | | Research and development | $33,558 | $30,680 | $2,878 | | General and administrative | $9,052 | $7,427 | $1,625 | | Total operating expenses | $42,610 | $38,107 | $4,503 | | Interest income | $6,495 | $6,610 | $(115) | | Net loss | $36,115 | $31,497 | $4,618 | - The increase in R&D expenses was primarily due to a **$4.4 million** increase in internal costs (personnel-related, including stock-based compensation) and a **$0.4 million** increase in sevasemten clinical program expenses, partially offset by a **$2.0 million** decrease in EDG-7500 clinical program expenses[84](index=84&type=chunk)[89](index=89&type=chunk) - General and administrative expenses increased mainly due to **$1.4 million** in higher personnel-related costs and **$0.3 million** in increased professional and consulting costs[86](index=86&type=chunk) [Comparison of the six months ended June 30, 2025 and 2024](index=35&type=section&id=Comparison%20of%20the%20six%20months%20ended%20June%2030,%202025%20and%202024) For the six months ended June 30, 2025, the net loss increased by $16.9 million compared to the prior year, driven by a $11.9 million rise in research and development expenses and a $3.8 million increase in general and administrative expenses, while interest income saw a slight decrease | Metric | 2025 (in thousands) | 2024 (in thousands) | Change (in thousands) | | :-------------------------- | :------------------ | :------------------ | :-------------------- | | Research and development | $70,315 | $58,374 | $11,941 | | General and administrative | $18,254 | $14,486 | $3,768 | | Total operating expenses | $88,569 | $72,860 | $15,709 | | Interest income | $11,656 | $12,838 | $(1,182) | | Net loss | $76,913 | $60,022 | $16,891 | - The increase in R&D expenses was primarily due to a **$10.1 million** increase in internal costs (personnel-related) and a **$2.3 million** increase in sevasemten clinical program expenses, partially offset by a **$1.8 million** decrease in EDG-7500 clinical program expenses[91](index=91&type=chunk)[94](index=94&type=chunk) - General and administrative expenses increased mainly due to **$3.0 million** in higher personnel-related costs and **$0.8 million** in increased professional and consulting costs[92](index=92&type=chunk) [Liquidity and Capital Resources](index=37&type=section&id=Liquidity%20and%20Capital%20Resources) This section discusses the company's liquidity, capital-raising efforts, cash flow activities, and future funding requirements. It highlights the reliance on equity offerings to finance operations and the expectation of needing substantial additional capital to advance product candidates and commercialization [Sources of liquidity](index=37&type=section&id=Sources%20of%20liquidity) Since inception, the company has primarily financed operations through private placements and public offerings of common stock, generating $793.7 million in net proceeds from various offerings as of June 30, 2025. The company held $594.0 million in cash, cash equivalents, and marketable securities as of June 30, 2025, which is expected to fund operations for at least the next 12 months - From inception to June 30, 2025, the company generated **$793.7 million** in net proceeds from its IPO, follow-on public offering, ATM Program, and January 2024 and April 2025 underwritten registered direct offerings[95](index=95&type=chunk)[96](index=96&type=chunk) - As of June 30, 2025, the company had **$594.0 million** in cash, cash equivalents, and marketable securities[96](index=96&type=chunk) - The company believes its existing cash, cash equivalents, and marketable securities will be sufficient to fund planned operating expenses and capital expenditure requirements through at least the next 12 months[70](index=70&type=chunk) [Cash flows](index=39&type=section&id=Cash%20flows) For the six months ended June 30, 2025, the company used $70.4 million in operating activities and $118.3 million in investing activities, but these outflows were offset by $190.5 million provided by financing activities, primarily from equity offerings, resulting in a net increase of $1.7 million in cash and cash equivalents | Cash Flow Activity | Six months ended June 30, 2025 (in thousands) | Six months ended June 30, 2024 (in thousands) | | :---------------------------------- | :------------------------------------------ | :------------------------------------------ | | Net cash used in operating activities | $(70,448) | $(54,697) | | Net cash used in investing activities | $(118,268) | $(230,940) | | Net cash provided by financing activities | $190,454 | $243,025 | | Net change in cash and cash equivalents | $1,738 | $(42,612) | - Cash used in operating activities for the six months ended June 30, 2025, was **$70.4 million**, primarily due to a net loss of **$76.9 million**, partially offset by non-cash charges[98](index=98&type=chunk) - Cash provided by financing activities for the six months ended June 30, 2025, was **$190.5 million**, mainly from **$200.0 million** in gross proceeds from the April 2025 offering[102](index=102&type=chunk) [Funding requirements](index=41&type=section&id=Funding%20requirements) The company will require substantial additional capital to develop product candidates and fund operations, with expenses expected to increase as clinical development progresses and regulatory approvals are sought. Future funding needs are highly uncertain and depend on factors such as clinical trial scope, regulatory outcomes, commercialization efforts, and intellectual property protection - The company will continue to require substantial additional capital to develop product candidates and fund operations for the foreseeable future[105](index=105&type=chunk) - Expenses are expected to increase with ongoing activities, particularly as the company continues development and seeks regulatory approvals for its product candidates and begins commercialization[105](index=105&type=chunk) - Future funding requirements depend on numerous factors, including the scope and costs of preclinical and clinical development, regulatory review outcomes, commercialization infrastructure, intellectual property costs, and potential acquisitions[107](index=107&type=chunk)[108](index=108&type=chunk)[109](index=109&type=chunk)[114](index=114&type=chunk) [Operating and Capital Expenditure Requirements and Contractual Obligations](index=45&type=section&id=Operating%20and%20Capital%20Expenditure%20Requirements%20and%20Contractual%20Obligations) The company anticipates its existing cash, cash equivalents, and marketable securities will cover operating and capital expenditure requirements for at least the next 12 months. Short-term cash needs are primarily for R&D, while long-term obligations include lease liabilities totaling $4.4 million as of June 30, 2025 - Existing cash, cash equivalents, and marketable securities are expected to be sufficient to fund planned operating expenses and capital expenditure requirements through at least the next 12 months[116](index=116&type=chunk) - Short-term material cash requirements are primarily for research and development expenses[117](index=117&type=chunk) - Total operating lease liability balance is **$4.4 million** as of June 30, 2025, with **$1.0 million** classified as a current liability[118](index=118&type=chunk) [Critical Accounting Estimates](index=45&type=section&id=Critical%20Accounting%20Estimates) This section states that the company's financial statements are prepared using U.S. GAAP, requiring estimates and judgments. It confirms that there have been no material changes to its critical accounting estimates from those discussed in the Annual Report on Form 10-K filed on March 3, 2025 - The preparation of financial statements requires estimates and judgments affecting reported amounts of assets, liabilities, costs, and expenses[119](index=119&type=chunk) - There were no material changes to critical accounting estimates during the three and six months ended June 30, 2025, from those discussed in the Annual Report on Form 10-K filed March 3, 2025[120](index=120&type=chunk) [Recently Issued Accounting Pronouncements](index=45&type=section&id=Recently%20Issued%20Accounting%20Pronouncements) The company is evaluating the impact of recently issued accounting pronouncements, ASU 2023-09 (Income Taxes) and ASU 2024-03 (Expense Disaggregation Disclosures), which are effective for fiscal years 2025 and 2027, respectively - ASU 2023-09, 'Income Taxes (Topic 740): Improvements to Income Tax Disclosures,' is effective for the company's **2025 fiscal year** annual reporting period[41](index=41&type=chunk)[43](index=43&type=chunk) - ASU 2024-03, 'Income Statement–Reporting Comprehensive Income–Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses,' is effective for the company's **2027 fiscal year** annual reporting period[44](index=44&type=chunk) - The company is currently evaluating the impact that the adoption of these standards will have on its financial statements[43](index=43&type=chunk)[44](index=44&type=chunk) [Transition from Emerging Growth Company and Smaller Reporting Company Status](index=45&type=section&id=Transition%20from%20Emerging%20Growth%20Company%20and%20Smaller%20Reporting%20Company%20Status) As of December 31, 2024, the company ceased to be an 'emerging growth company' and a 'smaller reporting company' due to its large accelerated filer status and market value, respectively. This transition means it can no longer take advantage of reduced reporting requirements, such as the extended transition period for new accounting standards or exemption from auditor attestation of internal controls - The company ceased to be an 'emerging growth company' as of **December 31, 2024**, due to its large accelerated filer status[122](index=122&type=chunk) - The company also ceased to be a 'smaller reporting company' as of **December 31, 2024**, because the market value of its common stock held by non-affiliates exceeded **$700 million** as of **June 30, 2024**[123](index=123&type=chunk)[124](index=124&type=chunk) - This transition means the company can no longer use the extended transition period for new accounting standards or be exempt from auditor attestation of internal control over financial reporting under Sarbanes-Oxley Act Section 404(b)[122](index=122&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=47&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company is exposed to market risk primarily related to changes in interest rates, affecting its portfolio of cash, cash equivalents, and marketable securities. Due to the short-term duration and low-risk profile of its investments, an immediate 10% change in interest rates is not expected to have a material effect on the fair market value of its investment portfolio - The company's primary exposure to market risk is interest rate sensitivity, affecting its investments in short-term available-for-sale marketable securities[125](index=125&type=chunk) | Asset Category | As of June 30, 2025 (in millions) | As of December 31, 2024 (in millions) | | :--------------- | :-------------------------------- | :------------------------------------ | | Cash, cash equivalents and marketable securities | $594.0 | $470.2 | - An immediate **10% change** in interest rates would not have a material effect on the fair market value of the investment portfolio due to its short-term duration and low-risk profile[125](index=125&type=chunk) [Item 4. Controls and Procedures](index=47&type=section&id=Item%204.%20Controls%20and%20Procedures) Management, including the CEO and CFO, evaluated the effectiveness of the company's disclosure controls and procedures as of June 30, 2025, concluding they were effective at a reasonable assurance level. There were no material changes in internal control over financial reporting during the three and six months ended June 30, 2025 - The CEO and CFO concluded that disclosure controls and procedures were effective at a reasonable assurance level as of **June 30, 2025**[126](index=126&type=chunk) - No change in internal control over financial reporting occurred during the three and six months ended June 30, 2025, that materially affected, or is reasonably likely to materially affect, internal control over financial reporting[127](index=127&type=chunk) [Part II. Other Information](index=48&type=section&id=Part%20II%20Other%20Information) [Item 1. Legal Proceedings](index=48&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently a party to any material legal proceedings, though it may be involved in various claims in the ordinary course of business. The outcome of litigation is uncertain and could adversely affect financial condition - The company is not currently a party to any material legal proceedings[130](index=130&type=chunk) - The outcome of litigation cannot be predicted with certainty, and unfavorable outcomes could materially affect financial condition or results of operations[130](index=130&type=chunk) [Item 1A. Risk Factors](index=48&type=section&id=Item%201A.%20Risk%20Factors) This section details the significant risks associated with investing in the company's common stock, encompassing financial position, capital needs, operating history, product development and commercialization, regulatory approvals, intellectual property, and dependence on third parties. These risks could materially harm the business, financial condition, results of operations, and growth prospects [Risk Factors Summary](index=48&type=section&id=Risk%20Factors%20Summary) Investing in the company's common stock involves a high degree of risk due to its limited operating history, significant net losses, substantial capital requirements, and dependence on the success of its lead product candidates, sevasemten and EDG-7500. Other key risks include the lengthy and uncertain clinical development process, intense competition, and the need to protect intellectual property - The company has a limited operating history, no products approved for commercial sale, and has incurred significant net losses since inception, expecting to continue to do so[132](index=132&type=chunk) - Future success is substantially dependent on the success of lead product candidates, sevasemten and EDG-7500, which are in early development stages[132](index=132&type=chunk) - Clinical drug development is lengthy, expensive, and uncertain, with no guarantee of demonstrating safety and efficacy or producing positive results[132](index=132&type=chunk) [Risks Related to Our Financial Position, Need for Additional Capital and Limited Operating History](index=52&type=section&id=Risks%20Related%20to%20Our%20Financial%20Position,%20Need%20for%20Additional%20Capital%20and%20Limited%20Operating%20History) This section outlines risks stemming from the company's early operational stage, including a history of significant net losses and the expectation of continued losses. It emphasizes the substantial need for additional capital to fund operations and product development, with potential consequences such as dilution for stockholders or delays in programs if funding is not secured on acceptable terms. Macroeconomic factors and public health pandemics are also noted as potential adverse impacts [Limited Operating History and No Approved Products](index=52&type=section&id=Limited%20Operating%20History%20and%20No%20Approved%20Products) The company has a limited operating history since its inception in 2017 and no products approved for commercial sale, making it difficult for investors to evaluate its future viability. It is transitioning from an R&D focus to commercial activities, a process fraught with unforeseen expenses, difficulties, and delays - The company is a clinical-stage biopharmaceutical company with a limited operating history since **2017** and no products approved for commercial sale[135](index=135&type=chunk) - It has not yet demonstrated the ability to obtain marketing approvals, manufacture commercial-scale products, or conduct sales and marketing activities[136](index=136&type=chunk) - The transition from an R&D focus to commercial activities involves inherent risks, including unforeseen expenses, difficulties, complications, and delays[137](index=137&type=chunk) [Significant Net Losses and Future Profitability Uncertainty](index=52&type=section&id=Significant%20Net%20Losses%20and%20Future%20Profitability%20Uncertainty) The company has incurred significant net losses since inception, with $36.1 million for the three months and $76.9 million for the six months ended June 30, 2025, and an accumulated deficit of $455.5 million. It expects to continue incurring substantial losses for the foreseeable future, and profitability is uncertain, depending on successful product development and commercialization | Metric | Three months ended June 30, 2025 (in millions) | Six months ended June 30, 2025 (in millions) | | :------- | :----------------------------------------- | :---------------------------------------- | | Net Loss | $(36.1) | $(76.9) | | Accumulated Deficit (as of June 30, 2025) | $(455.5) | $(455.5) | - The company has not generated any revenue to date and expects to continue incurring significant expenses and increasing operating losses for the foreseeable future[138](index=138&type=chunk)[139](index=139&type=chunk) - Achieving profitability depends on successful discovery, development, regulatory approval, and commercialization of product candidates, which is highly uncertain[142](index=142&type=chunk)[143](index=143&type=chunk) [Need for Substantial Additional Capital](index=56&type=section&id=Need%20for%20Substantial%20Additional%20Capital) The company will require substantial additional capital beyond its current $594.0 million in cash, cash equivalents, and marketable securities to fund operations and advance product candidates through regulatory approval and commercialization. Future capital requirements are highly uncertain and depend on various factors, including the scope and costs of clinical trials, regulatory review, and market conditions, with potential delays or program reductions if funding is not secured - As of **June 30, 2025**, the company had **$594.0 million** in cash, cash equivalents, and marketable securities, expected to fund operations for at least the next **12 months**[145](index=145&type=chunk) - Substantial additional funding will be needed to complete the development and commercialization of product candidates, as current capital is insufficient to fund through regulatory approval[149](index=149&type=chunk) - Future capital requirements depend on factors such as the scope and costs of preclinical and clinical development, regulatory review, commercialization activities, and intellectual property protection[150](index=150&type=chunk)[151](index=151&type=chunk) [Dilution and Restrictions from Capital Raising](index=60&type=section&id=Dilution%20and%20Restrictions%20from%20Capital%20Raising) Future capital raising efforts, through equity or convertible debt securities, may dilute existing stockholders' ownership and could involve agreements with covenants that restrict the company's operations. The company recently closed an underwritten registered direct offering in April 2025, raising $187.1 million in net proceeds - Raising additional capital through equity or convertible debt securities will likely dilute stockholders' ownership interest[157](index=157&type=chunk) - Debt financing and preferred equity financing may include covenants limiting the company's ability to incur additional debt, make acquisitions, or engage in certain transactions[157](index=157&type=chunk)[159](index=159&type=chunk) - On **April 3, 2025**, the company closed an underwritten registered direct offering of **9,935,419 shares** of common stock for net proceeds of **$187.1 million**[156](index=156&type=chunk) [Limitations on Net Operating Loss Carryforwards](index=62&type=section&id=Limitations%20on%20Net%20Operating%20Loss%20Carryforwards) The company's ability to use its net operating loss (NOL) carryforwards to offset future taxable income may be limited by U.S. tax law, particularly due to potential 'ownership changes' under Sections 382 and 383 of the Internal Revenue Code. As of December 31, 2024, the company had $146.3 million in federal NOL carryforwards and $155.6 million in state NOL carryforwards | NOL Type | Amount as of December 31, 2024 (in millions) | | :--------- | :--------------------------------------- | | Federal NOL carryforwards | $146.3 | | State NOL carryforwards | $155.6 | - Federal NOLs generated after **December 31, 2017**, can be carried forward indefinitely but are limited to **80%** of current year taxable income for tax years beginning after **December 31, 2020**[161](index=161&type=chunk) - An 'ownership change' under Sections 382 and 383 could limit the company's ability to use pre-change NOLs and other tax attributes, potentially having a material adverse effect on cash flows and results of operations[162](index=162&type=chunk) [Impact of Changes in Tax Laws](index=62&type=section&id=Impact%20of%20Changes%20in%20Tax%20Laws) Changes in tax laws, regulations, and policies, such as the requirement to capitalize and amortize R&D expenditures (though temporarily suspended by the OBBB Act) and proposed global minimum taxes, could adversely affect the company's tax obligations, effective tax rates, cash flow, and financial condition - Changes in tax laws, including the requirement to capitalize and amortize R&D expenditures (effective **2022**), could impact the effective tax rate and cash tax liability[163](index=163&type=chunk) - The OBBB Act, enacted **July 4, 2025**, permanently suspends the R&D capitalization requirement, permitting current deductions, but its full impact is still being evaluated[163](index=163&type=chunk) - Proposed global minimum tax (Pillar Two) and other international tax changes could adversely affect the company's effective tax rate and operating results[163](index=163&type=chunk)[165](index=165&type=chunk) [Market Conditions and Access to Capital](index=64&type=section&id=Market%20Conditions%20and%20Access%20to%20Capital) Market conditions and changing circumstances, such as instability in the banking and financial services sector (e.g., Silicon Valley Bank failure), could impair the company's ability to access its existing cash, cash equivalents, and investments, or timely pay vendors. This could lead to operational disruptions and a need for additional capital sooner than anticipated - Market conditions and banking sector instability (e.g., Silicon Valley Bank failure in **March 2023**) could impair the company's ability to access cash, cash equivalents, and investments[166](index=166&type=chunk) - The company regularly maintains cash balances that are not insured or exceed the FDIC's insurance limit, posing a risk of loss if a banking institution becomes insolvent[166](index=166&type=chunk) - Any delay in accessing funds or paying vendors could have a material adverse effect on operations and necessitate seeking additional capital sooner than planned[166](index=166&type=chunk) [Impact of Public Health Pandemics](index=64&type=section&id=Impact%20of%20Public%20Health%20Pandemics) Public health pandemics, such as COVID-19, can adversely impact the company's operations and financial results by causing delays or difficulties in clinical trial enrollment, increasing operational expenses, and disrupting supply chains. These disruptions can affect preclinical studies, clinical trial sites, and interactions with regulators, potentially leading to increased costs and delays in product development - Public health pandemics can cause delays or difficulties in enrolling and retaining patients in clinical trials, and increase operational expenses[167](index=167&type=chunk)[168](index=168&type=chunk) - Disruptions can affect third-party contractors, manufacturers, suppliers, and clinical trial sites, potentially leading to supply chain issues and delays in preclinical studies[170](index=170&type=chunk) - Any prolonged public health crisis could have a material negative impact on the company's business, financial condition, and operating results, exacerbating other risks[172](index=172&type=chunk) [Risks Related to the Discovery, Development and Commercialization of Our Product Candidates](index=68&type=section&id=Risks%20Related%20to%20the%20Discovery,%20Development%20and%20Commercialization%20of%20Our%20Product%20Candidates) This section details the extensive and uncertain risks involved in the discovery, development, and commercialization of the company's product candidates. It highlights the substantial dependence on lead candidates, the high failure rate in drug development, the lengthy and expensive clinical trial process, potential adverse events, and intense competition. Challenges in market acceptance, small patient populations for rare diseases, and securing favorable reimbursement are also significant concerns [Dependence on Lead Product Candidates (Sevasemten and EDG-7500)](index=68&type=section&id=Dependence%20on%20Lead%20Product%20Candidates%20(Sevasemten%20and%20EDG-7500)) The company's future success is highly dependent on the timely and successful completion of clinical trials, regulatory approval, and commercialization of its lead product candidates, sevasemten (for muscular dystrophies) and EDG-7500 (for hypertrophic cardiomyopathy). Failure to achieve these objectives for either candidate would materially harm the business - The company's future success is dependent on the timely and successful completion of clinical trials, marketing approval, and commercialization of sevasemten and EDG-7500[175](index=175&type=chunk) - Sevasemten is in advanced Phase 2 clinical trials for Becker, Duchenne, Limb-Girdle muscular dystrophies, and McArdle Disease, including a pivotal cohort (GRAND CANYON)[175](index=175&type=chunk)[177](index=177&type=chunk) - EDG-7500 is in advanced Phase 2 trials for hypertrophic cardiomyopathy (HCM)[175](index=175&type=chunk)[177](index=177&type=chunk) [Uncertainty of Other Product Candidates and Programs](index=70&type=section&id=Uncertainty%20of%20Other%20Product%20Candidates%20and%20Programs) Beyond sevasemten and EDG-7500, the company's prospects rely on developing and commercializing product candidates from its EDG-003 cardiometabolic discovery program and other future programs. Research and development for novel therapeutics is inherently risky, with a high failure rate due to safety, efficacy, and clinical execution challenges, and early-stage results may not predict later success - Future operating results depend on successfully developing, obtaining regulatory approval for, and commercializing product candidates from the EDG-003 cardiometabolic discovery program[179](index=179&type=chunk) - Research and development related to novel therapeutics is inherently risky, with a high historical failure rate due to safety, efficacy, clinical execution, and other unpredictable variables[179](index=179&type=chunk) - The success of other product candidates depends on factors such as patent protection, market acceptance, patient identification, and the ability to compete with other therapies[180](index=180&type=chunk) [Lengthy and Expensive Clinical Drug Development Process](index=72&type=section&id=Lengthy%20and%20Expensive%20Clinical%20Drug%20Development%20Process) Clinical drug development is a lengthy, expensive, and uncertain process with a high risk of failure at any stage. The outcome of preclinical studies and early clinical trials may not predict success in later stages, and the company may incur additional costs or delays due to regulatory feedback, patient enrollment issues, manufacturing problems, or unforeseen events during trials - Clinical testing is expensive, difficult to design and implement, can take many years, and its ultimate outcome is uncertain, with a high risk of failure at any stage[185](index=185&type=chunk) - The outcome of preclinical studies and early-stage clinical trials may not be predictive of the success of later clinical trials; for example, the GRAND CANYON cohort's primary endpoint may not be met despite earlier positive trends[185](index=185&type=chunk) - Numerous unforeseen events during clinical trials, such as regulatory feedback, patient enrollment issues, manufacturing problems, or geopolitical conflicts, could delay or prevent marketing approval[189](index=189&type=chunk) [Adverse Events and Side Effects of Product Candidates](index=76&type=section&id=Adverse%20Events%20and%20Side%20Effects%20of%20Product%20Candidates) The company's novel small molecules for muscle diseases carry uncertainty regarding their safety profile. Product candidates may cause serious adverse events, toxicities, or undesirable side effects, alone or in combination with other drugs, which could prevent regulatory approval, limit market acceptance, or lead to significant negative consequences. Past observations include somnolence, dizziness, fall, arthralgia, upper respiratory tract infection, and atrial fibrillation - Product candidates may cause serious adverse events, toxicities, or undesirable side effects, alone or in combination with other drugs, which could prevent regulatory approval or market acceptance[195](index=195&type=chunk)[198](index=198&type=chunk) - Observed adverse events for sevasemten include dose-limiting somnolence (SAD trial), dizziness and somnolence (MAD trial), and dizziness, fall, and arthralgia (ARCH trial)[199](index=199&type=chunk) - For EDG-7500, frequently reported adverse events in the CIRRUS-HCM trial were dizziness, upper respiratory tract infection, and atrial fibrillation, mostly mild to moderate[199](index=199&type=chunk) [Predictive Value of Preclinical and Early Clinical Trial Results](index=78&type=section&id=Predictive%20Value%20of%20Preclinical%20and%20Early%20Clinical%20Trial%20Results) Success in preclinical studies and early-stage clinical trials does not guarantee success in later clinical trials, as product candidates may fail to demonstrate sufficient safety and efficacy. There can be significant variability in results between trials due to factors like study design, patient populations, and adherence to protocols, increasing the uncertainty of clinical trial outcomes and potentially delaying or preventing marketing approval - Success in preclinical studies and early-stage clinical trials does not mean that future clinical trials will be successful, and product candidates may fail to show desired safety and efficacy in later stages[201](index=201&type=chunk) - There can be significant variability in safety and efficacy results between different clinical trials of the same product candidate due to factors such as changes in trial protocols, patient populations, and dosing regimens[204](index=204&type=chunk) - Regulatory authorities may limit the scope of later-stage trials until satisfactory safety is demonstrated, which could delay approval or restrict the target patient population[203](index=203&type=chunk) [Delays in Patient Enrollment and Maintenance](index=80&type=section&id=Delays%20in%20Patient%20Enrollment%20and%20Maintenance) The company may face significant delays or be unable to initiate or continue clinical trials if it cannot enroll and maintain a sufficient number of eligible patients, particularly for rare diseases like Duchenne and Becker. Patient enrollment is affected by factors such as the size of the patient population, perceived risks and benefits of novel therapies, and competition from other clinical trials, which could increase development costs and jeopardize marketing approval - The company may not be able to initiate or continue clinical trials if it cannot locate and enroll a sufficient number of eligible patients, especially for severe muscle diseases with limited patient pools[206](index=206&type=chunk)[208](index=208&type=chunk) - Patient enrollment is affected by factors including the size and nature of the patient population, perceived risks and benefits of novel approaches, availability of approved drugs, and competition from other clinical trials[210](index=210&type=chunk) - Inability to enroll sufficient patients would result in significant delays, increased development costs, and could jeopardize marketing approval for product candidates[210](index=210&type=chunk) [Limited Resources and Focus on Specific Indications](index=82&type=section&id=Limited%20Resources%20and%20Focus%20on%20Specific%20Indications) With limited resources, the company is primarily focusing its efforts on developing sevasemten and EDG-7500 for specific indications, which may lead to foregoing or delaying opportunities for other indications or product candidates with potentially greater commercial potential, including those from the EDG-003 cardiometabolic discovery program - The company is focusing the majority of its resources on developing sevasemten and EDG-7500, potentially foregoing or delaying other opportunities[211](index=211&type=chunk) - Resource allocation decisions may cause the company to fail to capitalize on viable commercial drugs or profitable market opportunities, including product candidates from the EDG-003 cardiometabolic discovery program[211](index=211&type=chunk)[214](index=214&type=chunk) [Significant Competition](index=84&type=section&id=Significant%20Competition) The company faces intense competition from major pharmaceutical and biotechnology companies, as well as emerging firms, in developing treatments for muscle diseases. Competitors have existing products and numerous candidates in development for Duchenne and HCM, potentially offering more effective, safer, or less expensive alternatives, which could negatively impact the company's commercial opportunities and market penetration - The biotechnology and biopharmaceutical industries are characterized by rapidly advancing technologies, intense competition, and a strong emphasis on proprietary and novel products[215](index=215&type=chunk) - For Duchenne, competitors include companies marketing corticosteroids (Deflazacort, Prednisone, Vamorolone), exon skipping drugs (EXONDYS 51, AMONDYS 45, VYONDYS 53, VILTEPSO), and gene therapies (Elevidys)[217](index=217&type=chunk)[218](index=218&type=chunk)[219](index=219&type=chunk)[220](index=220&type=chunk)[221](index=221&type=chunk) - For HCM, competitors include companies developing cardiac myosin inhibitors (Camzyos, aficamten) and other non-myosin targeting drugs (ninerafaxstat, CT-G20, sotagliflozin, trientine dihydrochloride, gene therapies)[223](index=223&type=chunk)[224](index=224&type=chunk)[225](index=225&type=chunk) [Changes in Interim, Topline, and Preliminary Data](index=88&type=section&id=Changes%20in%20Interim,%20Topline,%20and%20Preliminary%20Data) Publicly disclosed preliminary, interim, or topline data from clinical trials are subject to change upon more comprehensive review and audit. Such data may differ from final results, or regulatory agencies may interpret data differently, potentially impacting program value, approvability, and the company's financial condition - Preliminary, interim, or topline data from clinical trials (e.g., sevasemten's ARCH, CANYON, LYNX, FOX, MESA; EDG-7500's Phase 1, CIRRUS-HCM) are subject to change following comprehensive review and audit[229](index=229&type=chunk) - Topline data should be viewed with caution until final data are available, as material differences could arise[229](index=229&type=chunk) - Regulatory agencies or others may disagree with the company's interpretations of data, potentially harming the approvability or commercialization of product candidates[232](index=232&type=chunk) [Uncertainty in Developing Proprietary Drug Discovery Platform](index=90&type=section&id=Uncertainty%20in%20Developing%20Proprietary%20Drug%20Discovery%20Platform) The company's strategy relies on leveraging its proprietary drug discovery platform to build a pipeline of precision medicine product candidates. However, there is no guarantee that these candidates will be safe and effective, or that the platform will successfully yield a diverse pipeline. The rapidly advancing biotechnology industry also poses a risk of technological obsolescence - A key strategy is to leverage the proprietary drug discovery platform to develop a pipeline of precision medicine product candidates for muscle diseases[233](index=233&type=chunk) - There is no assurance that product candidates identified through the platform will be safe and effective or suitable for clinical development[233](index=233&type=chunk) - The rapidly advancing biotechnology industry means competitors could render the company's approach obsolete, limiting the commercial value of its product candidates[234](index=234&type=chunk) [Risks of Combination Therapies](index=90&type=section&id=Risks%20of%20Combination%20Therapies) Developing product candidates, such as sevasemten, in combination with other therapies exposes the company to additional risks, including patient tolerability issues, unexpected consequences from dosing, and the potential for regulatory authorities to revoke approval or for supply issues with the co-therapies. The success of such combination therapies depends on the approval and continued viability of all components - Developing product candidates in combination with other therapies (approved or investigational) exposes the company to risks such as patient tolerability issues and unexpected consequences from dosing[235](index=235&type=chunk)[236](index=236&type=chunk) - Regulatory approval of combination therapies could be revoked, or safety, efficacy, manufacturing, or supply issues could arise with the existing therapies[237](index=237&type=chunk) - If third-party providers of co-therapies cannot produce sufficient quantities or if costs are prohibitive, development and commercialization efforts would be impaired[240](index=240&type=chunk) [Manufacturing Difficulties by Third-Party Manufacturers](index=92&type=section&id=Manufacturing%20Difficulties%20by%20Third-Party%20Manufacturers) The company relies on third-party manufacturers for the production of its product candidates for clinical trials and future commercialization, increasing the risk of insufficient quality or quantity, or unacceptable costs. Manufacturing drugs is complex, requiring strict compliance with cGMP regulations, and any difficulties or non-compliance could delay or prevent development and commercialization efforts - The company relies on third-party manufacture

[Executive Summary & Q2 2025 Highlights](index=1&type=section&id=News%20Release) [Q2 2025 Highlights and CEO Commentary](index=1&type=section&id=Q2%202025%20Highlights%20and%20CEO%20Commentary) Edgewise Therapeutics reported Q2 2025 results, highlighting positive sevasemten data and EDG-7500 progress - Announced positive top-line data from the open-label extension MESA trial of sevasemten in participants with Becker muscular dystrophy[1](index=1&type=chunk) - Announced encouraging top-line data from the Phase 2 LYNX and FOX trials of sevasemten in Duchenne muscular dystrophy[1](index=1&type=chunk) - Advanced the Phase 2 CIRRUS-HCM trial of EDG-7500 in Hypertrophic Cardiomyopathy (HCM)[1](index=1&type=chunk) - Company is advancing skeletal and cardiac muscle programs and building commercial infrastructure for a potential U.S. launch of sevasemten in Becker, while exploring Phase 3 trial designs in HCM and Duchenne[2](index=2&type=chunk) [Recent Business Highlights](index=1&type=section&id=Recent%20Highlights) [Muscular Dystrophy Program](index=1&type=section&id=Muscular%20Dystrophy%20Program) Edgewise advanced its muscular dystrophy program with positive sevasemten trial results for Becker and Duchenne [Sevasemten and Becker Muscular Dystrophy](index=1&type=section&id=Sevasemten%20and%20Becker) Sevasemten showed sustained disease stabilization in Becker muscular dystrophy, with GRAND CANYON data expected Q4 2026 - Sevasemten is an orally administered first-in-class fast skeletal myosin inhibitor designed to protect against contraction-induced muscle damage in muscular dystrophies[3](index=3&type=chunk) - Becker muscular dystrophy is a rare, genetic, life-shortening, debilitating, and degenerative neuromuscular disorder with no currently approved therapies[4](index=4&type=chunk) - Positive data from the MESA open-label extension trial showed sustained disease stabilization in NSAA scores for Becker participants, with a favorable safety profile after up to **three years of treatment**[5](index=5&type=chunk)[6](index=6&type=chunk) - The global pivotal placebo-controlled GRAND CANYON trial completed enrollment of **175 adults** in February 2025 and is on track for top-line data in **Q4 2026**[7](index=7&type=chunk)[8](index=8&type=chunk) - The Company completed a successful Type C meeting with the FDA, providing a clear path to registration of sevasemten as the first therapy for Becker[8](index=8&type=chunk) [Sevasemten and Duchenne Muscular Dystrophy](index=2&type=section&id=Duchenne) Sevasemten Phase 2 trials in Duchenne showed encouraging results, identifying a **10 mg** dose for Phase 3 - Duchenne is the most common type of muscular dystrophy, a severe degenerative muscle disorder with high unmet medical need despite existing therapies[9](index=9&type=chunk) - LYNX Phase 2 trial showed encouraging observations across functional measures (SV95C, NSAA, 4 stair-climb) and identified a **10 mg dose** for Phase 3[10](index=10&type=chunk) - FOX Phase 2 trial in Duchenne boys previously treated with gene therapy supports the potential of sevasemten **10 mg** to reduce the rate of functional decline[11](index=11&type=chunk) - The Company plans to meet with the FDA in **Q4 2025** to discuss Phase 3 design and initiate a pivotal study in **2026**[12](index=12&type=chunk) [Cardiovascular and Cardiometabolic Programs](index=3&type=section&id=Cardiovascular%20and%20Cardiometabolic%20Programs) Edgewise advances cardiovascular programs with EDG-7500 in Phase 2 for HCM and EDG-15400 entering Phase 1 [EDG-7500 and Hypertrophic Cardiomyopathy (HCM)](index=3&type=section&id=EDG-7500%20and%20HCM) The CIRRUS-HCM Phase 2 trial for EDG-7500 is recruiting for Part D, with an update expected in Q4 2025 - EDG-7500 is a novel oral, selective, cardiac sarcomere modulator designed to slow early contraction velocity and address impaired cardiac relaxation associated with HCM[14](index=14&type=chunk) - HCM is the most common form of genetic heart disease, affecting approximately **one in 500 people**, with significant unmet needs for additional therapeutic approaches, particularly for nonobstructive HCM (nHCM)[14](index=14&type=chunk) - The multi-part, open-label CIRRUS-HCM Phase 2 trial is actively recruiting participants for Part D (**12-week study** in oHCM and nHCM), with an update expected in **Q4 2025**[15](index=15&type=chunk) [Heart Failure and Preclinical Programs](index=3&type=section&id=Heart%20failure%20and%20preclinical%20programs) Edgewise plans to initiate a Phase 1 trial for EDG-15400 in heart failure in Q3 2025 - The Company expects to begin dosing in a first-in-human Phase 1 trial of EDG-15400 for heart failure during **Q3 2025**[16](index=16&type=chunk) - Edgewise continues to advance its preclinical cardiometabolic programs[16](index=16&type=chunk) [Strengthened Engagement with the Scientific and Patient Communities](index=3&type=section&id=Strengthened%20Engagement%20with%20the%20Scientific%20and%20Patient%20Communities) Edgewise continued engagement with muscular dystrophy and HCM communities through educational outreach and conferences - The Company participated in the Parent Project Muscular Dystrophy annual conference, CureDuchenne Futures conference, and the Annual Congress of the Heart Failure Association of the ESC[17](index=17&type=chunk) - Edgewise continues to sponsor and participate in numerous other clinician and patient-focused events[17](index=17&type=chunk) [Second Quarter Financial Results](index=3&type=section&id=Second%20Quarter%20Financial%20Results) [Financial Position (Cash, Cash Equivalents, Marketable Securities)](index=3&type=section&id=Financial%20Position%20(Cash%2C%20Cash%20Equivalents%2C%20Marketable%20Securities)) Edgewise Therapeutics maintained a strong liquidity position with **$594.0 million** in cash, cash equivalents, and marketable securities Cash, Cash Equivalents and Marketable Securities | Metric | June 30, 2025 | | :----- | :------------ | | Cash, cash equivalents and marketable securities | **$594.0 million** | [Operating Expenses](index=3&type=section&id=Operating%20expenses) Total operating expenses for Q2 2025 decreased to **$42.6 million**, primarily due to reduced R&D activities Total Operating Expenses (QoQ) | Metric | Q2 2025 | Q1 2025 | Change | | :----- | :------ | :------ | :----- | | Total operating expenses | **$42.610 million** | **$45.959 million** | **-$3.349 million** | [Research and Development (R&D) Expenses](index=3&type=section&id=Research%20and%20development) R&D expenses decreased by **$3.2 million** to **$33.6 million** in Q2 2025, driven by reduced clinical development and personnel costs Research and Development Expenses (QoQ) | Metric | Q2 2025 | Q1 2025 | Change | | :----- | :------ | :------ | :----- | | R&D expenses | **$33.6 million** | **$36.8 million** | **-$3.2 million** | - Decrease primarily driven by a **$2.6 million** decrease in sevasemten clinical development, a **$0.9 million** decrease in EDG-7500 clinical development, and a **$1.1 million** decrease in personnel-related costs, offset by a **$1.7 million** increase in manufacturing expenses[19](index=19&type=chunk) [General and Administrative (G&A) Expenses](index=4&type=section&id=General%20and%20administrative) G&A expenses remained stable at **$9.1 million** for Q2 2025 General and Administrative Expenses (QoQ) | Metric | Q2 2025 | Q1 2025 | Change | | :----- | :------ | :------ | :----- | | G&A expenses | **$9.1 million** | **$9.2 million** | **-$0.1 million** | [Net Loss and Net Loss Per Share](index=4&type=section&id=Net%20loss) Edgewise reported a net loss of **$36.1 million** (or **$0.34 per share**) for Q2 2025, an improvement Net Loss and Net Loss Per Share (QoQ) | Metric | Q2 2025 | Q1 2025 | Change | | :----- | :------ | :------ | :----- | | Net loss | **$(36.1) million** | **$(40.8) million** | **+$4.7 million** | | Net loss per share | **$(0.34)** | **$(0.43)** | **+$0.09** | [Company Overview](index=4&type=section&id=About%20Edgewise%20Therapeutics) [Company Mission and Product Pipeline](index=4&type=section&id=Company%20Mission%20and%20Product%20Pipeline) Edgewise Therapeutics develops novel therapeutics for muscular dystrophies and cardiac conditions, with sevasemten and EDG-7500 as key candidates - Edgewise Therapeutics is a leading muscle disease biopharmaceutical company developing novel therapeutics for muscular dystrophies and serious cardiac conditions[21](index=21&type=chunk) - Sevasemten is an orally administered first-in-class fast skeletal myosin inhibitor in late-stage clinical trials for Becker and Duchenne muscular dystrophies[21](index=21&type=chunk) - EDG-7500 is a novel cardiac sarcomere modulator for hypertrophic cardiomyopathy and other diseases of diastolic dysfunction, currently in Phase 2 clinical development[21](index=21&type=chunk) [Financial Statements](index=6&type=section&id=Financial%20Statements) [Condensed Statement of Operations](index=6&type=section&id=Condensed%20Statement%20of%20Operations) The condensed statement of operations shows a net loss of **$36.1 million** for Q2 2025, an improvement driven by reduced R&D expenses Condensed Statement of Operations (Three months ended, in thousands) | Operating expenses: | June 30, 2025 | March 31, 2025 | | :------------------ | :------------ | :------------- | | Research and development | **$33,558** | **$36,757** | | General and administrative | **$9,052** | **$9,202** | | Total operating expenses | **$42,610** | **$45,959** | | Loss from operations | **$(42,610)** | **$(45,959)** | | Interest income | **$6,495** | **$5,161** | | Net loss | **$(36,115)** | **$(40,798)** | | Net loss per share - basic and diluted | **$(0.34)** | **$(0.43)** | | Weighted-average shares outstanding, basic and diluted | **104,940,493** | **95,130,053** | [Condensed Balance Sheet Data](index=6&type=section&id=Condensed%20Balance%20Sheet%20Data) Total assets increased to **$613.3 million** as of June 30, 2025, driven by increased cash and marketable securities Condensed Balance Sheet Data (in thousands) | Assets | June 30, 2025 | December 31, 2024 | | :----- | :------------ | :---------------- | | Cash, cash equivalents and marketable securities | **$593,992** | **$470,170** | | Other assets | **$19,342** | **$16,647** | | Total assets | **$613,334** | **$486,817** | | Liabilities | **$24,263** | **$27,601** | | Stockholders' equity | **$589,071** | **$459,216** | | Total liabilities and stockholders' equity | **$613,334** | **$486,817** | [Additional Information](index=4&type=section&id=Additional%20Information) [Cautionary Note Regarding Forward-Looking Statements](index=4&type=section&id=Cautionary%20Note%20Regarding%20Forward-Looking%20Statements) This section outlines forward-looking statements, emphasizing inherent risks and uncertainties that could cause actual results to differ - The press release contains forward-looking statements regarding product candidates, clinical trials, potential launches, market opportunities, and financial expectations[22](index=22&type=chunk) - Actual results could differ materially due to numerous risks and uncertainties, including those related to early development, lack of approved products, need for capital, dependence on product candidates, clinical trial outcomes, regulatory approvals, and intellectual property[22](index=22&type=chunk) [Company Contacts](index=6&type=section&id=Edgewise%20Contacts) Contact information is provided for investor relations and media inquiries - Investors can contact Michael Carruthers, Chief Financial Officer, at ir@edgewisetx.com[28](index=28&type=chunk) - Media inquiries can be directed to Maureen Franco, VP Corporate Communications, at media@edgewisetx.com[28](index=28&type=chunk)

Core Viewpoint - Pomerantz LLP is investigating potential securities fraud or unlawful business practices involving Edgewise Therapeutics and its officers or directors [1] Group 1: Company Performance - On June 26, 2025, Edgewise reported clinical trial data for its lead asset, sevasemten, targeting Becker and Duchenne muscular dystrophies, which it described as "positive" [3] - Despite the positive characterization, the U.S. FDA indicated that the CANYON data was insufficient for accelerated approval during a Type C meeting [3] - Following this announcement, Edgewise's stock price decreased by $1.27 per share, or 8.86%, closing at $14.33 per share on the same day [3] Group 2: Legal Investigation - Pomerantz LLP is reaching out to investors of Edgewise regarding claims of potential securities fraud [1] - The firm is recognized for its expertise in corporate, securities, and antitrust class litigation, having a history of recovering multimillion-dollar damages for victims of securities fraud [4]

Core Viewpoint - Pomerantz LLP is investigating potential securities fraud or unlawful business practices involving Edgewise Therapeutics and its officers or directors, following a significant stock price drop after clinical trial data was deemed insufficient for accelerated FDA approval [1][3]. Group 1: Company Overview - Edgewise Therapeutics, Inc. is publicly traded on NASDAQ under the ticker EWTX [1]. - The company is focused on developing treatments for Becker and Duchenne muscular dystrophies, with its lead asset being sevasemten [3]. Group 2: Recent Developments - On June 26, 2025, Edgewise reported clinical trial data for sevasemten, which it described as "positive," but the FDA indicated that the data was insufficient for accelerated approval [3]. - Following the announcement, Edgewise's stock price decreased by $1.27, or 8.86%, closing at $14.33 per share [3]. Group 3: Legal Context - Pomerantz LLP is recognized for its expertise in corporate, securities, and antitrust class litigation, having a long history of advocating for victims of securities fraud and corporate misconduct [4].

Group 1 - Edgewise Therapeutics is under investigation for potential securities fraud or unlawful business practices involving its officers and/or directors [1] - The company reported clinical trial data for its lead asset, sevasemten, indicating "positive" results for Becker and Duchenne muscular dystrophies, but the FDA deemed the data insufficient for accelerated approval [2] - Following the announcement, Edgewise's stock price decreased by $1.27 per share, or 8.86%, closing at $14.33 per share [3] Group 2 - Pomerantz LLP is a prominent firm specializing in corporate, securities, and antitrust class litigation, with a history of recovering multimillion-dollar damages for victims of securities fraud and corporate misconduct [4]