Financial Data and Key Metrics Changes - The company is in Phase II and Phase III studies for Becker muscular dystrophy and Duchenne muscular dystrophy, with a strong safety profile and significant biomarker changes reported [4][5] - Enrollment for the Phase III confirmatory study in Becker muscular dystrophy was completed with 175 patients, achieving over 96% power to hit a 1.6 change in North Star [4] Business Line Data and Key Metrics Changes - The company reported positive data in hypertrophic cardiomyopathy (HCM) with a novel drug that shows no concentration relationship with ejection fraction changes, differentiating it from competitor drugs [7][11] - The drug demonstrated significant improvements in KCCQ scores and New York Heart Association classifications, indicating a strong functional benefit [11][18] Market Data and Key Metrics Changes - The non-obstructive HCM patient population is growing, constituting a larger proportion of diagnosed HCM patients, which presents a market opportunity for the company's drug [29] - The company anticipates launching its drug for Becker muscular dystrophy in Q1 2028, with a focus on a small patient population, allowing for flexible pricing strategies [49] Company Strategy and Development Direction - The company aims to differentiate its drug by eliminating the need for extensive echo monitoring, which is a requirement for current competitor drugs [22][23] - The strategy includes targeting community practices for commercialization, as these practices face challenges in managing echo monitoring for patients [26][28] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the potential for accelerated approval pathways with the FDA based on the data from the Grand Canyon study [54] - The company is exploring the possibility of a rolling submission strategy to present additional supportive data to the FDA [54][55] Other Important Information - The company is preparing to report data from its Phase II study in Duchenne muscular dystrophy, focusing on identifying appropriate dosing and patient populations for future studies [6][44] - There is a significant unmet need in the Duchenne muscular dystrophy space, with management noting that patients may still require treatment even after gene therapy [46][48] Q&A Session Summary Question: Can you walk us through the mechanism of action for the HCM program? - The drug was designed to decouple the relationship between drug concentration and ejection fraction change, focusing on diastolic activity and partial inhibition [10][11] Question: How do you view the competitive landscape for HCM treatments? - The company sees an opportunity in the non-obstructive HCM space, especially as current treatments have not effectively addressed this population [20][21] Question: What impact has the recent patient death in the DMD space had on your programs? - Management noted that there hasn't been a significant change in patient interest or clinician engagement in their DMD program despite the incident [37][39] Question: What are the primary endpoints you are considering for your Phase III study in Duchenne muscular dystrophy? - The company is inclined towards using SV95 as a primary endpoint, alongside traditional measures like North Star and Time to Rise [43][44] Question: How does the company plan to approach pricing for its Becker muscular dystrophy treatment? - The company plans to set a price based on the value provided, considering the small patient population and the lack of existing treatments [49][50]

Summary of Edgewise Therapeutics (EWTX) Conference Call Company Overview - **Company**: Edgewise Therapeutics (EWTX) - **Event**: 2025 Conference on May 20, 2025 Key Points on EDG-7500 in Hypertrophic Cardiomyopathy (HCM) 1. **Efficacy Profile**: EDG-7500 shows a unique efficacy profile with no changes in ejection fraction despite increased drug concentration, indicating a differentiated mechanism of action [3][6][10] 2. **Symptomatic Benefits**: 70% of patients became asymptomatic according to New York Heart Association (NYHA) classifications, with KCCQ scores improving by an average of over 20 points [6][9] 3. **Mechanism of Action**: The drug directly addresses hypertrophy without lowering ejection fraction, improving diastolic function and patient symptoms [5][6] 4. **Clinical Observations**: The absence of a placebo arm raised questions, but the deepening response at higher doses supports the drug's efficacy [7][8] 5. **Ejection Fraction Stability**: No relationship was found between drug concentration and changes in ejection fraction, reinforcing the drug's safety profile [10][12][15] 6. **Atrial Fibrillation Cases**: Four cases of atrial fibrillation were noted, but extensive preclinical studies showed no correlation with the drug's mechanism [16][19] 7. **Patient Enrollment Strategy**: Future trials will involve stricter patient selection criteria to minimize risks associated with atrial fibrillation [20][21][23] 8. **Titration Scheme**: The company is exploring a titration scheme that allows for individualized dosing based on patient response rather than relying solely on ejection fraction measurements [28][30][33] Upcoming Data Expectations 1. **Twelve-Week Data**: Anticipated trends in NT proBNP levels and potential deepening of efficacy in non-obstructive patients [25][27] 2. **Individualized Treatment Goals**: The aim is to achieve optimal dosing for each patient based on symptom relief and biomarker normalization [29][33] Sevasemten and DMD Data 1. **Patient Breakdown**: Upcoming data will include approximately 60-70 patients in the LINX group and 20-30 in the FOX group, focusing on biomarker and functional outcomes [38][39] 2. **Long-Term Efficacy**: Observations from previous studies indicate that benefits from gene therapy may diminish after two years, highlighting the need for ongoing assessment [40][41][42] 3. **FDA Discussions**: The company is on track for discussions regarding accelerated approval for Becker by the end of the quarter [44] Conclusion - Edgewise Therapeutics is advancing its clinical programs with a focus on individualized treatment strategies and robust data collection to support the efficacy and safety of its therapies in HCM and DMD. The upcoming data releases and regulatory discussions are critical for the company's future trajectory.

Core Viewpoint - Edgewise Therapeutics, Inc. (EWTX) has received a Zacks Rank 2 (Buy) upgrade due to an upward trend in earnings estimates, which is a significant factor influencing stock prices [1][4]. Earnings Estimates and Ratings - The Zacks rating system is primarily based on a company's changing earnings picture, specifically tracking the Zacks Consensus Estimate for EPS from sell-side analysts [2]. - The Zacks rating upgrade for Edgewise Therapeutics reflects a positive outlook on its earnings, which could positively affect its stock price [4][6]. Impact of Earnings Estimates on Stock Prices - Changes in a company's future earnings potential, as indicated by earnings estimate revisions, are strongly correlated with near-term stock price movements [5]. - Institutional investors utilize earnings estimates to determine the fair value of a company's shares, influencing their buying or selling actions, which subsequently affects stock prices [5]. Recent Performance of Edgewise Therapeutics - Edgewise Therapeutics is projected to earn -$1.82 per share for the fiscal year ending December 2025, indicating a year-over-year decline of 25.5% [9]. - Over the past three months, the Zacks Consensus Estimate for Edgewise Therapeutics has increased by 2.1%, suggesting a positive trend in earnings estimates [9]. Zacks Rank System Overview - The Zacks Rank system classifies stocks into five groups based on earnings estimates, with Zacks Rank 1 (Strong Buy) stocks historically generating an average annual return of +25% since 1988 [8]. - The upgrade of Edgewise Therapeutics to a Zacks Rank 2 places it in the top 20% of Zacks-covered stocks, indicating a strong potential for market-beating returns in the near term [11].

Group 1 - Edgewise Therapeutics, Inc. (EWTX) shares have increased by 14.4% over the past four weeks, closing at $14.89, with a mean price target of $40.90 indicating a potential upside of 174.7% [1] - The average of 10 short-term price targets ranges from a low of $14 to a high of $52, with a standard deviation of $14.28, suggesting variability in analyst estimates [2] - Analysts show strong agreement on EWTX's ability to report better earnings than previously predicted, which supports the potential for stock upside [4][11] Group 2 - The Zacks Consensus Estimate for EWTX's current year earnings has increased by 6.8% over the past month, with four estimates rising and one falling [12] - EWTX holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimates, indicating strong potential for near-term upside [13] - While consensus price targets may not be reliable for predicting exact stock gains, they can provide a directional guide for price movement [13]

Part I [Item 1. Financial Statements (Unaudited)](index=8&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) The company reported a net loss of $40.8 million for Q1 2025, an increase from $28.5 million in Q1 2024, driven by higher R&D expenses, and saw total assets decrease to $454.4 million [Condensed Balance Sheets](index=8&type=section&id=Condensed%20Balance%20Sheets) As of March 31, 2025, total assets decreased to $454.4 million from $486.8 million, primarily due to reduced marketable securities, while stockholders' equity fell to $429.7 million Condensed Balance Sheet Data (in thousands) | Account | March 31, 2025 (unaudited) | December 31, 2024 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $49,921 | $41,666 | | Marketable securities, available for sale | $386,466 | $428,504 | | Total current assets | $443,178 | $475,483 | | Total assets | $454,391 | $486,817 | | **Liabilities and Stockholders' Equity** | | | | Total current liabilities | $21,115 | $23,860 | | Total liabilities | $24,672 | $27,601 | | Total stockholders' equity | $429,719 | $459,216 | | Total liabilities and stockholders' equity | $454,391 | $486,817 | [Condensed Statements of Operations and Comprehensive Loss](index=10&type=section&id=Condensed%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Net loss for Q1 2025 increased to $40.8 million ($0.43 per share) from $28.5 million ($0.33 per share) in Q1 2024, driven by higher operating expenses, particularly R&D Statement of Operations Summary (in thousands, except per share data) | Metric | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | Research and development | $36,757 | $27,694 | | General and administrative | $9,202 | $7,059 | | Total operating expenses | $45,959 | $34,753 | | Loss from operations | ($45,959) | ($34,753) | | Interest income | $5,161 | $6,228 | | Net loss | ($40,798) | ($28,525) | | Net loss per share, basic and diluted | ($0.43) | ($0.33) | [Condensed Statements of Stockholders' Equity](index=11&type=section&id=Condensed%20Statements%20of%20Stockholders'%20Equity) Total stockholders' equity decreased to $429.7 million from $459.2 million, primarily due to a $40.8 million net loss, partially offset by stock-based compensation and option exercises - Key changes in stockholders' equity for Q1 2025 include a **$40.8 million net loss**, a **$9.1 million increase** from stock-based compensation, and a **$2.3 million increase** from the exercise of stock options[20](index=20&type=chunk) [Condensed Statements of Cash Flows](index=12&type=section&id=Condensed%20Statements%20of%20Cash%20Flows) Net cash used in operating activities was $37.9 million, while investing activities provided $43.9 million and financing activities provided $2.2 million, resulting in a net $8.3 million increase in cash Cash Flow Summary (in thousands) | Activity | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | ($37,866) | ($28,598) | | Net cash provided by (used in) investing activities | $43,898 | ($268,308) | | Net cash provided by financing activities | $2,223 | $241,073 | | Net change in cash and cash equivalents | $8,255 | ($55,833) | | Cash and cash equivalents at end of period | $49,921 | $30,264 | [Notes to Condensed Financial Statements](index=13&type=section&id=Notes%20to%20Condensed%20Financial%20Statements) Notes detail the company's clinical-stage biopharmaceutical focus, significant accumulated deficit, reliance on equity financing, and sufficient cash reserves of $436.4 million to fund operations for at least 12 months - The company is a clinical-stage biopharmaceutical company focused on treatments for severe muscle diseases, with lead candidates **sevasemten** (for Duchenne and Becker muscular dystrophy) and **EDG-7500** (for hypertrophic cardiomyopathy)[24](index=24&type=chunk) - As of March 31, 2025, the company had an accumulated deficit of **$419.4 million** and cash, cash equivalents, and marketable securities of **$436.4 million**, which management believes is sufficient to fund operations for at least the next 12 months[26](index=26&type=chunk)[33](index=33&type=chunk) - The company raised net proceeds of **$231.9 million** from a direct offering in January 2024 and an additional **$188.0 million** (before expenses) from a direct offering in April 2025[28](index=28&type=chunk)[31](index=31&type=chunk) Stock-Based Compensation Expense (in thousands) | Category | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | Research and development | $5,867 | $2,700 | | General and administrative | $3,192 | $2,171 | | **Total** | **$9,059** | **$4,871** | [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=26&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's clinical program advancements, increased net loss to $40.8 million in Q1 2025 due to higher R&D expenses, and its belief that current cash, supplemented by a recent offering, will fund operations for at least 12 months [Overview](index=26&type=section&id=Overview) Edgewise is a clinical-stage biopharmaceutical company advancing sevasemten and EDG-7500, incurring a $40.8 million net loss in Q1 2025, but expects its $436.4 million cash reserves to fund operations for at least 12 months - The company is advancing two clinical-stage programs: **sevasemten** for muscular dystrophies and **EDG-7500** for hypertrophic cardiomyopathy (HCM)[64](index=64&type=chunk) - Net loss for Q1 2025 was **$40.8 million**, and the company expects to continue incurring significant losses as it advances its product candidates[66](index=66&type=chunk) - The company believes its cash, cash equivalents, and marketable securities of **$436.4 million** as of March 31, 2025, will fund operations for at least the next 12 months[67](index=67&type=chunk) [Results of Operations](index=32&type=section&id=Results%20of%20Operations) Operating expenses increased by $11.2 million to $46.0 million in Q1 2025, primarily due to a $9.1 million rise in R&D expenses driven by clinical trial advancements and personnel costs Comparison of Results of Operations (in thousands) | Account | Q1 2025 | Q1 2024 | Change | | :--- | :--- | :--- | :--- | | Research and development | $36,757 | $27,694 | $9,063 | | General and administrative | $9,202 | $7,059 | $2,143 | | **Total operating expenses** | **$45,959** | **$34,753** | **$11,206** | | Net loss | ($40,798) | ($28,525) | ($12,273) | - The **$9.1 million increase** in R&D expenses was due to: **$1.9 million** for the sevasemten program due to increased trial activity, **$0.2 million** for the EDG-7500 program related to the Phase 2 CIRRUS-HCM trial, **$1.3 million** for discovery and preclinical expenses for the EDG-003 program, and **$5.7 million** in internal costs, mainly from increased employee headcount and stock-based compensation[83](index=83&type=chunk)[86](index=86&type=chunk) [Liquidity and Capital Resources](index=34&type=section&id=Liquidity%20and%20Capital%20Resources) The company funds operations through equity offerings, holding $436.4 million in cash and marketable securities as of March 31, 2025, and expects these funds to cover operating expenses for at least the next 12 months - As of March 31, 2025, the company had **$436.4 million** in cash, cash equivalents, and marketable securities, with an additional **$188.0 million** raised in April 2025[85](index=85&type=chunk) Cash Flow Summary (in thousands) | Activity | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | ($37,866) | ($28,598) | | Net cash provided by (used in) investing activities | $43,898 | ($268,308) | | Net cash provided by financing activities | $2,223 | $241,073 | - The company expects its existing cash and marketable securities to be sufficient to fund planned operating expenses and capital expenditure requirements for at least the next 12 months[105](index=105&type=chunk) - Long-term cash requirements include total operating lease liabilities of **$4.6 million** as of March 31, 2025[110](index=110&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=43&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company faces interest rate risk on its $436.4 million portfolio of short-term, low-risk investments, but a 10% interest rate change is not expected to materially affect its fair value - The company's primary market risk is interest rate sensitivity on its **$436.4 million** portfolio of cash and marketable securities[116](index=116&type=chunk) - Due to the short-term duration and low-risk profile of investments, a **10% change** in interest rates is not expected to have a material effect on the portfolio's fair market value[116](index=116&type=chunk) [Item 4. Controls and Procedures](index=43&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of March 31, 2025, with no material changes to internal control over financial reporting during the quarter - The CEO and CFO concluded that as of March 31, 2025, the company's disclosure controls and procedures were effective[117](index=117&type=chunk) - No material changes occurred in the company's internal control over financial reporting during the first quarter of 2025[118](index=118&type=chunk) Part II [Item 1. Legal Proceedings](index=44&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 ordinary course claims not deemed material - The company is not currently a party to any material legal proceedings[121](index=121&type=chunk) [Item 1A. Risk Factors](index=44&type=section&id=Item%201A.%20Risk%20Factors) This section outlines numerous risks, including the company's limited operating history, net losses, dependence on lead product candidates, clinical development uncertainties, regulatory hurdles, competition, reliance on third parties, intellectual property protection, and stock market volatility - The company has a limited operating history, has incurred significant net losses since inception (**$419.4 million** accumulated deficit as of March 31, 2025), and expects to continue incurring losses[127](index=127&type=chunk)[130](index=130&type=chunk) - The business is substantially dependent on the success of its two lead product candidates, **sevasemten** and **EDG-7500**[123](index=123&type=chunk)[166](index=166&type=chunk) - Clinical drug development is a lengthy, expensive, and uncertain process; positive results from early trials may not be predictive of future results, and product candidates may cause serious adverse events[123](index=123&type=chunk)[175](index=175&type=chunk)[186](index=186&type=chunk) - The company relies on third parties to conduct clinical trials and manufacture its product candidates, which increases risks related to quality, quantity, and cost[125](index=125&type=chunk)[428](index=428&type=chunk)[433](index=433&type=chunk) - The company faces significant competition from major pharmaceutical and biotechnology companies with greater resources, particularly in the development of treatments for Duchenne and HCM[203](index=203&type=chunk)[205](index=205&type=chunk)[210](index=210&type=chunk) - The company's success depends on its ability to obtain and maintain patent protection and trade secret protection for its product candidates and technologies[125](index=125&type=chunk)[351](index=351&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=183&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) This section details net proceeds from multiple public offerings, including a March 2021 IPO ($186.1 million) and an April 2025 direct offering ($188.0 million), with no material change in the planned use of proceeds - There were no unregistered sales of equity securities during the period[488](index=488&type=chunk) Summary of Net Proceeds from Public Offerings | Offering | Date | Net Proceeds (approx.) | | :--- | :--- | :--- | | Initial Public Offering (IPO) | Mar 2021 | $186.1 million | | Follow-On Offering | Sep 2022 | $129.2 million | | ATM Program | Jun 2023 - Jan 2024 | $59.4 million | | Underwritten Direct Offering | Jan 2024 | $231.9 million | | Underwritten Direct Offering | Apr 2025 | $188.0 million | - There has been no material change in the use of proceeds from these offerings; funds were invested in interest-bearing, investment-grade securities[493](index=493&type=chunk)[495](index=495&type=chunk)[497](index=497&type=chunk)[499](index=499&type=chunk)[501](index=501&type=chunk) [Item 3. Defaults Upon Senior Securities](index=187&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) The company reports no defaults upon senior securities - None[501](index=501&type=chunk) [Item 4. Mine Safety Disclosures](index=187&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to the company - Not applicable[502](index=502&type=chunk) [Item 5. Other Information](index=187&type=section&id=Item%205.%20Other%20Information) No directors or officers adopted or terminated a Rule 10b5-1 or non-Rule 10b5-1 trading arrangement during the last fiscal quarter - No directors or officers adopted or terminated a Rule 10b5-1 or non-Rule 10b5-1 trading arrangement during the last fiscal quarter[503](index=503&type=chunk) [Item 6. Exhibits](index=187&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the quarterly report, including director compensation policy, officer certifications, and XBRL data files - The report includes several exhibits, such as officer certifications (31.1, 31.2, 32.1, 32.2) and Inline XBRL documents[507](index=507&type=chunk)

News Release Edgewise Therapeutics Reports First Quarter 2025 Financial Results and Recent Business Highlights – Announced positive top-line results from Phase 2 CIRRUS-HCM trial of EDG-7500 in Hypertrophic Cardiomyopathy (HCM) – – Strengthened balance sheet with net proceeds of approximately $188 million from the April 2025 public of ering; pro-forma cash balance exceeds $624 million – Exhibit 99.1 – On track to report data from the Phase 2 LYNX and FOX trials of sevasemten in Duchenne muscular dystrophy ( ...

Shares of Edgewise Therapeutics (EWTX) plunged 47.7% in a week, likely due to safety concerns for its investigational candidate, EDG-7500, in patients with obstructive or nonobstructive hypertrophic cardiomyopathy (HCM), despite positive mid-stage study results.EWTX is evaluating EDG-7500, a novel oral, selective, cardiac sarcomere modulator with a unique mechanism of action, in the ongoing phase II CIRRUS-HCM study. The latest data readout is from Part B and Part C of the CIRRUS-HCM study, which included 1 ...

This article is published by Terry Chrisomalis, who runs the Biotech Analysis Central pharmaceutical service on Seeking Alpha Marketplace. If you like what you read here and would like to subscribe to, I'm currently offering a two-week free trial period for subscribers to take advantage of. My service offers a deep-dive analysis of many pharmaceutical companies. The Biotech Analysis Central SA marketplace is $49 per month, but for those who sign up for the yearly plan will be able to take advantage of a 33. ...

BOULDER, Colo., April 2, 2025 /PRNewswire/ -- Edgewise Therapeutics, Inc. (NASDAQ: EWTX), a leading muscle disease biopharmaceutical company, today announced the pricing of an underwritten offering of 9,935,419 shares of its common stock at an offering price of $20.13 per share. Edgewise anticipates gross proceeds from the offering to be approximately $200 million, before deducting underwriting discounts and commissions and offering expenses. The closing of the offering is expected to occur on April 3, 2025 ...

Core Insights - Edgewise Therapeutics announced positive top-line data from the Phase 2 CIRRUS-HCM trial for EDG-7500, showing significant reductions in left ventricular outflow tract gradients in patients with obstructive hypertrophic cardiomyopathy (HCM) [1][2] - The treatment demonstrated improvements in cardiac function and biomarkers without adversely affecting left ventricular ejection fraction (LVEF) [1][4] Group 1: Trial Overview - CIRRUS-HCM is a multi-part, open-label trial assessing EDG-7500 in individuals with both obstructive and nonobstructive HCM [2][10] - Part B included 17 participants with obstructive HCM, while Part C included 12 participants with nonobstructive HCM, evaluating the safety and efficacy of daily doses of 50 or 100 mg over four weeks [3][10] Group 2: Efficacy Results - In obstructive HCM participants, EDG-7500 led to a 71% reduction in resting LVOT gradient and a 58% reduction in provokable gradient at the 100 mg dose, alongside a 62% mean reduction in NT-proBNP, a heart failure biomarker [4][6] - Participants also showed a substantial mean increase of 23 points in the Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ-OSS) at the 100 mg dose [4][5] Group 3: Safety Profile - EDG-7500 was generally well-tolerated, with no participants experiencing LVEF values below 50% [1][6] - Common adverse events included dizziness and upper respiratory infections, with most being mild to moderate in severity [6] Group 4: Future Directions - The company plans to optimize dosing strategies in Part D of the trial, with initial data expected in the second half of 2025 and Phase 3 initiation planned for the first half of 2026 [8][10]