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Core Insights - Edgewise Therapeutics, Inc. reported topline data from the Phase 2 CIRRUS-HCM trial for EDG-7500, targeting obstructive and nonobstructive Hypertrophic Cardiomyopathy (HCM) [1][2] Group 1: Trial Results - In Part A of the trial, a single oral dose of EDG-7500 showed significant reductions in left ventricular outflow tract gradient (LVOT-G) without affecting left ventricular ejection fraction (LVEF) [2] - Part B included 17 participants with obstructive HCM, while Part C included 12 with nonobstructive HCM, both evaluating the safety and efficacy of once-daily doses of 50 or 100 mg of EDG-7500 for four weeks [3] - Participants receiving 100 mg experienced mean reductions of 71% in resting LVOT-G and 58% in provokable (Valsalva) gradients, achieved without meaningful changes in LVEF [6] Group 2: Biomarker and Clinical Improvements - In nonobstructive HCM participants, a 42% mean decrease in NT-proBNP was observed at the 100 mg dose [4] - Significant improvements in Kansas City Cardiomyopathy Questionnaire Overall Summary Score (KCCQ-OSS) and Clinical Summary Scores were noted, with mean increases of 17 points and 22 points, respectively, at the 100 mg dose after four weeks [4] - Treatment with 100 mg of EDG-7500 resulted in a 62% mean reduction in NT-proBNP, a key heart failure biomarker, and 78% of participants improved by at least one NYHA Class, with 67% improving to NYHA Class I [6] Group 3: Future Outlook - Initial data from Part D of the trial is expected in the second half of 2025, with Phase 3 initiation planned for the first half of 2026 [4]

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]

Group 1 - Edgewise Therapeutics will hold a live webcast on April 2, 2025, to discuss top-line data of EDG-7500 from the Phase 2 CIRRUS-HCM trial [1] - The webcast will feature two leading investigators in the field of hypertrophic cardiomyopathy (HCM) [1] - An accompanying slide presentation will be available for participants [1] Group 2 - EDG-7500 is a novel oral, selective cardiac sarcomere modulator aimed at addressing impaired cardiac relaxation in HCM and other diastolic dysfunction diseases [2] - The drug is currently in Phase 2 clinical development [2][3] - Edgewise Therapeutics is focused on developing therapeutics for muscular dystrophies and serious cardiac conditions, with a commitment to improving patient lives [3]

Group 1 - The stock of Edgewise Therapeutics (NASDAQ: EWTX) nearly doubled after strong midstage data was released from an ongoing late-stage study targeting Becker's muscular dystrophy [1] - The company has a program that is currently in late-stage study, indicating potential for further growth and investment interest [1] Group 2 - The Total Pharma Tracker offers tools for DIY investors, including a software that provides extensive curated research material for any ticker [2] - In-house experts are available to assist investors by identifying the best investable stocks along with buy/sell strategies and alerts [2]

Financial Performance - Net losses for Edgewise Therapeutics were $133.8 million in 2024, up from $100.2 million in 2023, reflecting an increase of $33.6 million [655]. - The net loss for 2024 was $133.8 million, compared to a net loss of $100.2 million in 2023, indicating a 33% increase in losses [734]. - The total comprehensive loss for 2024 was $133.5 million, compared to $98.7 million in 2023, representing a 35% increase [734]. - The Company reported a net loss of $133.8 million for the year ended December 31, 2024, compared to a net loss of $100.2 million for 2023, representing a 33.5% increase in net loss year-over-year [816]. Research and Development Expenses - Research and development expenses rose to $127.0 million in 2024 from $90.9 million in 2023, an increase of $36.1 million [672]. - Research and development expenses for the year ended December 31, 2024, were $127.0 million, up 40% from $90.9 million in 2023 [734]. - The sevasemten clinical program incurred $51.7 million in expenses in 2024, up from $34.8 million in 2023, a rise of $16.8 million [673]. - EDG-7500 clinical program expenses increased by $8.9 million to $17.9 million in 2024, primarily due to the activation of the Phase 2 CIRRUS-HCM trial [673]. - Research and development expenses increased to $80.2 million in 2024 from $57.5 million in 2023, reflecting a 39.4% increase [824]. - Accrued research and development costs were $6.5 million as of December 31, 2024, compared to $5.7 million in 2023, indicating an increase of 14.4% [820]. Cash Flow and Liquidity - As of December 31, 2024, Edgewise Therapeutics had cash, cash equivalents, and marketable securities totaling $470.2 million [679]. - Cash used in investing activities in 2024 amounted to $184.7 million, primarily due to $477.1 million in purchases of marketable securities, offset by $277.9 million in maturities of marketable securities [685]. - Cash provided by financing activities in 2024 was $249.3 million, significantly higher than $53.2 million in 2023, driven by $239.1 million in net proceeds from the issuance of common stock [687][688]. - Cash flows from operating activities resulted in a net cash used of $109.0 million in 2024, up from $91.9 million in 2023, indicating an 18.5% increase in cash outflow [739]. - Cash and cash equivalents decreased to $41.7 million in 2024 from $86.1 million in 2023, a decline of 52% [733]. - The company believes its cash and marketable securities will be sufficient to fund operations for at least the next 12 months [749]. Capital and Financing - The company expects significant additional spending to progress its product candidates through clinical trials and regulatory approval [660]. - The company expects to require substantial additional capital to develop product candidates and fund operations, with a shelf registration statement filed for equity and debt offerings of up to $175 million [689]. - The company raised $240.0 million in gross proceeds from an underwritten registered direct offering of 21,818,182 shares at $11.00 per share in January 2024 [745]. - The company has entered into a sales agreement allowing for the potential sale of up to $175.0 million in common stock through an at-the-market offering program [746]. Operational Expenses - General and administrative expenses increased to $31.9 million in 2024 from $23.5 million in 2023, an increase of $8.4 million [677]. - The company reported total operating expenses of $158.8 million for 2024, a 39% increase from $114.4 million in 2023 [734]. - Non-cash charges in operating activities for 2024 included stock-based compensation expense of $24.7 million and depreciation of $2.1 million [683]. Shareholder Equity and Stock Options - The weighted-average shares outstanding increased to 92.4 million in 2024 from 63.7 million in 2023, reflecting a 45% increase [734]. - Additional paid-in capital rose to $837.4 million in 2024 from $563.5 million in 2023, marking a 48% increase [733]. - The company has 15,416,761 stock options outstanding with a weighted average exercise price of $9.40 and total intrinsic value of $267.4 million [787]. - The total stock-based compensation expense for the year ended December 31, 2024 was $24.7 million, an increase from $17.6 million in 2023 [800]. - The company granted 2,817,497 stock options in 2024 at a weighted average exercise price of $17.67 [787]. Compliance and Regulatory - The company ceased to be an "emerging growth company" and "smaller reporting company" as of December 31, 2024, which is expected to increase public company compliance costs [711][712]. - The company maintained effective internal control over financial reporting as of December 31, 2024, according to the auditor's opinion [719]. Marketable Securities - As of December 31, 2024, the total fair value of marketable securities was $469,978,000, with unrealized gains of $530,000 and unrealized losses of $110,000 [768]. - The remaining contractual maturities of $394.4 million of marketable securities were less than one year, and $33.7 million were between 1 and 2 years as of December 31, 2024 [769]. - The company has not recognized any impairment charges for long-lived assets during the years ended December 31, 2024, and 2023 [762].

Financial Performance - Edgewise Therapeutics reported a net loss of $39.7 million or $0.42 per share for Q4 2024, compared to a net loss of $34.1 million or $0.36 per share in the previous quarter[18]. - Total operating expenses for Q4 2024 were $45.5 million, compared to $40.4 million in the previous quarter[23]. - Cash, cash equivalents, and marketable securities increased to approximately $470.2 million as of December 31, 2024, up from $318.4 million in the previous year[16][25]. Research and Development - Research and development expenses for Q4 2024 were $36.4 million, an increase from $32.2 million in Q3 2024, driven by higher personnel costs and clinical development activities[17]. - The GRAND CANYON study completed enrollment with 175 adults across 51 sites in 12 countries, with data expected in Q4 2026[6]. - Positive top-line results were announced from the Phase 2 CANYON trial, which evaluated 40 adults and 29 adolescents over a 12-month treatment period[5]. - The CIRRUS-HCM trial is on track to report data in Q1 2025, following positive topline results from Part A of the trial[12][13]. - The LYNX and FOX trials for Duchenne muscular dystrophy are ongoing, with data expected in the first half of 2025 to inform Phase 3 strategy[10]. - Edgewise Therapeutics aims to file an investigational new drug application for a novel heart failure candidate in 2025[14]. Regulatory Engagement - The company plans to engage the U.S. FDA in the first half of 2025 to discuss marketing authorization strategies for sevasemten in Becker muscular dystrophy[5].

Core Viewpoint - Edgewise Therapeutics (EWTX) shares surged over 18% following positive mid-stage results from the CANYON study for its drug sevasemten, aimed at treating Becker Muscular Dystrophy (BMD) [1][5]. Group 1: Study Results - The CANYON study achieved its primary endpoint, showing a significant 28% reduction in creatine kinase (CK) levels in the sevasemten-treated group compared to placebo over six to twelve months [2]. - Sevasemten treatment indicated a trend towards improvement in the North Star Ambulatory Assessment, a key secondary endpoint, and showed a significant reduction in plasma levels of fast skeletal muscle troponin I (TNNI2) [3]. - The sevasemten-treated group had patients with more advanced disease compared to the placebo group, suggesting the drug's efficacy in a challenging patient population [3]. Group 2: Future Plans and Pipeline - Edgewise is on track to complete recruitment for the pivotal GRAND CANYON cohort by the first quarter of 2025, with plans to seek FDA and EMA approval for sevasemten if results are positive [4]. - Besides BMD, sevasemten is being evaluated for other muscular disorders, including Duchenne muscular dystrophy (DMD), Limb-Girdle muscular dystrophy (LGMD), and McArdle Disease [8]. - Edgewise is also developing another candidate, EDG-7500, for obstructive hypertrophic cardiomyopathy (HCM), with initial data expected in the first quarter of 2025 [9][11]. Group 3: Stock Performance - EWTX shares have increased nearly 200% year to date, contrasting with an 11.4% decline in the industry, reflecting investor confidence in the CANYON study results and the potential of sevasemten [6].

Core Insights - Edgewise Therapeutics, Inc. announced positive topline results from the Phase 2 CANYON trial of sevasemten for Becker muscular dystrophy (BMD), marking it as the largest interventional trial in this area and the first to meet its primary endpoint [1][2] Group 1: Trial Results - The trial met its primary endpoint with a significant 28% average decrease in creatine kinase (CK) levels in the sevasemten-treated group compared to placebo over months 6 through 12 (p=0.02) [2] - Secondary endpoint, North Star Ambulatory Assessment (NSAA), indicated a trend towards improvement in motor function in the sevasemten-treated group [2] - Plasma fast skeletal muscle troponin I (TNNI2) showed a significant reduction in the treatment group compared to placebo, indicating muscle damage reduction [3] Group 2: Functional Measures - Additional functional measures, including the 10-meter walk/run, 4-stair climb, and 100-meter timed test, also showed trends towards improvement in the sevasemten group compared to placebo [3] Group 3: Patient Population and Safety - The treatment population had more advanced disease compared to the placebo group, suggesting a challenging patient demographic [4] - Sevasemten was well-tolerated with no new safety concerns reported during the trial [5] Group 4: Future Plans and Market Reaction - The company is on track to complete recruitment for the GRAND CANYON cohort by the first quarter of 2025 [6] - Following the announcement, EWTX stock rose by 8.62% to $30 [7]