EDG-7500

– 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 offering; pro-forma cash balance exceeds $624 million – – On track to report data from the Phase 2 LYNX and FOX trials of sevasemten in Duchenne muscular dystrophy (Duchenne) in the second quarter of 2025 – – Expect to report data from Part D of the Phase 2 CIRRUS-HCM trial in the second half of 2025 – BOULDER, Colo., May 8, 2025 /PRNewswire/ -- Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the first quarter of 2025 and recent business highlights. "We're seeing strong progress across our skeletal and cardiac muscle programs," said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise. "Most recently, we announced positive top-line results from CIRRUS-HCM and completed a $200 million offering enabling the execution of our near and long-term goals. Our goals include commercial readiness for a potential U.S. approval of sevasemten in Becker, advancement of a Phase 3 trial in Duchenne, a Phase 3 program in HCM and our ongoing research and development activities. We anticipate several key milestones this year in both our sevasemten and EDG-7500 programs." Recent Highlights Strengthened Financial Position On April 3, 2025, the Company closed an underwritten registered direct offering of 9,935,419 shares of common stock at an offering price of $20.13 per share. The aggregate gross proceeds from the offering were $200 million. The net proceeds after deducting underwriting discounts and commissions but before offering expenses were $188 million. Muscular Dystrophy Program / Sevasemten Sevasemten and Becker Sevasemten is an orally administered first-in-class fast skeletal myosin inhibitor designed to protect against contraction-induced muscle damage in muscular dystrophies including Becker and Duchenne. Becker is a rare, genetic, life-shortening, debilitating and degenerative neuromuscular disorder. Genetic mutations in the dystrophin gene result in contraction-induced muscle damage, which is the primary driver of irreversible muscle loss and impaired motor function. The disease predominantly affects males, with functional decline beginning at any age. Once that muscle loss occurs, the decline in function is irreversible and continues throughout the individual's life. Currently, there are no approved therapies on the market to treat Becker. CANYON Phase 2 placebo-controlled trial in adults with Becker: In December 2024, the Company announced positive topline results from CANYON, the largest interventional Becker trial to date, which evaluated 40 adults and 29 adolescents with a sevasemten treatment period of 12 months. Data from CANYON was presented at the 2025 MDA Clinical and Scientific Conference. The Company is engaging with the U.S. FDA to discuss potential marketing authorization filing strategies for sevasemten in Becker. GRAND CANYON, a global pivotal placebo-controlled cohort in Becker: In February 2025, the Company completed enrollment in GRAND CANYON, an expansion of the CANYON placebo-controlled trial. The 18-month GRAND CANYON study is active in 12 countries, including the United States, United Kingdom, Netherlands, Denmark, Belgium, France, Spain, Germany, Italy, Israel, New Zealand and Australia. GRAND CANYON enrolled 175 adults, reflective of the Becker community's enthusiasm to have access to a therapy with the potential to stabilize their debilitating decline in function and ability to perform everyday activities. Data from the study are expected in Q4 2026. To learn more, go to clinicaltrials.gov (NCT05291091). MESA Phase 2 open label extension trial in adults with Becker: The Company is advancing MESA, an open-label extension trial to assess the long-term effect of sevasemten in individuals with Becker. MESA provides continued access to sevasemten to participants who were previously enrolled in ARCH, or completed CANYON, GRAND CANYON, or DUNE. MESA has continuing enrollment of 99% of eligible participants completing these prior trials. Duchenne Duchenne, a severe degenerative muscle disorder, is the most common type of muscular dystrophy with a median life expectancy of around 30 years. Genetic mutations in the dystrophin gene result in contraction-induced muscle damage, which is the primary driver of irreversible muscle loss and impaired motor function. While there are approved therapies on the market aimed to treat the disease, there remains a high unmet need for additional therapies. LYNX and FOX Phase 2 trials in boys with Duchenne: LYNX is an ongoing multi-center, dose-finding Phase 2 trial to evaluate the effect of sevasemten on safety, biomarkers of muscle damage and function in children with Duchenne treated with oral, once-daily sevasemten. FOX is a Phase 2 ongoing placebo-controlled trial to assess the effect of sevasemten on safety, biomarkers of muscle damage and function in children and adolescents with Duchenne who have been previously treated with gene therapy. Based on collective dose finding observations from both LYNX and FOX, the Company will form its Phase 3 strategy for sevasemten in Duchenne including patient and dose selection. The Company expects to report data from LYNX and FOX as well as its future clinical trial plans in the second quarter of 2025. For more information, go to clinicaltrials.gov to learn more about LYNX (NCT05540860) and FOX (NCT06100887). Cardiovascular and Cardiometabolic Programs EDG-7500 and HCM EDG-7500 is a novel oral, selective, cardiac sarcomere modulator, specifically designed to slow early contraction velocity and address impaired cardiac relaxation associated with HCM and other diseases of diastolic dysfunction. HCM is the most common form of genetic heart disease, affecting approximately one in 500 people, and is associated with reduced quality of life and an elevated risk of heart failure, abnormal heart rhythms, and sudden cardiac death. There are two major forms of HCM: obstructive and nonobstructive. Despite advancements in treatment options for some HCM patients, there remains a significant unmet need for additional therapeutic approaches for patients, including nonobstructive HCM patients, for which there are no approved treatment options. CIRRUS-HCM Phase 2 trial in adults with HCM: The Company is advancing CIRRUS-HCM, a multi-part, open-label trial, in participants with HCM at up to 20 clinical sites in the U.S. Part A of the trial was designed to evaluate the safety and tolerability of a single oral dose of EDG-7500 in obstructive HCM. In September 2024, the Company announced positive top-line data from Part A of the trial showing that a single oral dose of EDG-7500 in participants with obstructive HCM demonstrated robust reductions in left ventricular outflow tract gradient (LVOT-G) without meaningful changes in left ventricular ejection fraction (LVEF). Part B and Part C of the trial were designed to evaluate the safety and efficacy of once-daily doses of EDG-7500 for four weeks in participants with obstructive HCM (Part B) and in participants with nonobstructive HCM (Part C). In April 2025, the Company announced positive top-line data from Parts B and C. EDG-7500 over four weeks demonstrated rapid and sustained clinically meaningful reductions in LVOT-G. Further, four-week treatment with EDG-7500 demonstrated substantial improvements in measures of feel and function (Kansas City Cardiomyopathy Questionnaire and/or New York Heart Association functional class scores), reductions in NT-proBNP, a key biomarker of heart failure, and positive trends in measures of diastolic function. EDG-7500 was generally well-tolerated; clinical activity was observed without meaningful changes in LVEF, including no participant with a value below 50% at any measurement. The most frequently reported adverse events were dizziness, upper respiratory tract infection and atrial fibrillation (AF); nearly all were considered mild to moderate in severity. The Company expects to report data from the 12-week (Part D) CIRRUS-HCM trial in participants with obstructive HCM and nonobstructive HCM in the second half of 2025. To learn more about CIRRUS-HCM, visit clinicaltrials.gov, NCT06347159 (Phase 2). Preclinical programs: During 2025, the Company expects to file an investigational new drug application for a novel candidate for the treatment of heart failure and continue to advance a novel cardiometabolic program. Strengthened Engagement with the Scientific and Patient Communities The Company continued its education and outreach with the muscular dystrophy and HCM medical and patient communities. The team participated in the Muscular Dystrophy Association annual conference and the American College of Cardiology Scientific Sessions. The Company continues to sponsor and participate in numerous other clinician and patient-focused events. First Quarter Financial Results Cash, cash equivalents and marketable securities were approximately $436.4 million as of March 31, 2025. Our cash and cash equivalents at March 31, 2025 do not include the $188 million in net proceeds after deducting underwriting discounts and commissions but before offering expenses from our underwritten registered direct offering that closed on April 3, 2025. Combining the actual balance at March 31, 2025 with the net proceeds of the offering provides for a proforma balance of $624.4 million. Research and development (R&D) expenses were $36.8 million for the first quarter of 2025, compared to $36.4 million for the preceding quarter. The increase of $0.4 million was primarily driven by an additional $1.0 million higher personnel related costs, $0.9 million increase in clinical development activities, offset by a $1.2 million decrease in manufacturing expenses and a $0.4 million decrease in professional fees and other research costs. General and Administrative (G&A) expenses were $9.2 million for the first quarter of 2025, compared to $9.2 million for the preceding quarter. There were no significant changes between the first quarter of 2025 and the immediately preceding quarter. Net loss and net loss per share for the first quarter of 2025 was $40.8 million or $0.43 per share, compared to $39.7 million or $0.42 per share for the preceding quarter. About Edgewise Therapeutics Edgewise Therapeutics is a leading muscle disease biopharmaceutical company developing novel therapeutics for muscular dystrophies and serious cardiac conditions. The Company's deep expertise in muscle physiology is driving a new generation of novel therapeutics. Sevasemten is an orally administered first-in-class fast skeletal myosin inhibitor in late-stage clinical trials in Becker and Duchenne muscular dystrophies. EDG-7500 is a novel cardiac sarcomere modulator for the treatment of hypertrophic cardiomyopathy and other diseases of diastolic dysfunction, currently in Phase 2 clinical development. The entire team at Edgewise is dedicated to our mission: changing the lives of patients and families affected by serious muscle diseases. To learn more, go to: www.edgewisetx.com or follow us on LinkedIn, X, Facebook and Instagram. Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements as that term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Statements in this press release that are not purely historical are forward-looking statements. Such forward-looking statements include, among other things, statements regarding the potential of, and expectations regarding, Edgewise's product candidates and programs, including sevasemten, EDG-7500 and its novel cardiometabolic program; statements regarding Edgewise's expectations relating to its clinical trials, including timing of reporting data (including the presentation of data from the GRAND CANYON trial, the LYNX trial, the FOX trial, and the CIRRUS-HCM trial); timing of reporting Edgewise's future clinical trial plans; statements regarding the advancement of Edgewise's research and development programs; statements regarding Edgewise's ability to execute its near and long-term goals; statements regarding Edgewise's commercial readiness; statements regarding the outcome of Edgewise's discussions with the U.S. FDA on marketing authorization filing strategies for sevasemten in Becker; the timing of filing an investigational new drug application for a novel candidate; statements regarding Edgewise's anticipated milestones; and statements by Edgewise's President and Chief Executive Officer. Words such as "believes," "anticipates," "plans," "expects," "intends," "will," "goal," "potential" and similar expressions are intended to identify forward-looking statements. The forward-looking statements contained herein are based upon Edgewise's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those projected in any forward-looking statements due to numerous risks and uncertainties, including but not limited to: risks associated with Edgewise's limited operating history, its products being early in development and not having products approved for commercial sale; risks associated with Edgewise not having generated any revenue to date; Edgewise's ability to achieve objectives relating to the discovery, development and commercialization of its product candidates, if approved; Edgewise's need for substantial additional capital to finance its operations; Edgewise's substantial dependence on the success of sevasemten and EDG-7500; Edgewise's ability to develop and commercialize sevasemten and EDG-7500 and discover, develop and commercialize product candidates in future programs; risks related to Edgewise's clinical trials of its product candidates not demonstrating safety and efficacy; risks related to Edgewise's product candidates causing serious adverse events, toxicities or other undesirable side effects; the outcome of preclinical testing and early clinical trials not being predictive of the success of later clinical trials and the risks related to the results of Edgewise's clinical trials not satisfying the requirements of regulatory authorities; delays or difficulties in the enrollment and/or maintenance of patients in clinical trials; risks related to failure to capitalize on other indications or product candidates; risks related to competition; risks relating to interim, topline and preliminary data from Edgewise's clinical trials changing as more patient data becomes available; risks related to the regulatory approval processes of domestic and foreign authorities being lengthy, time consuming and inherently unpredictable; risks related to production of drugs by Edgewise's third-party manufacturers; risks related to changes in methods of product candidate manufacturing or formulation; risks related to not achieving adequate market acceptance; risks related to the patient population for our product candidates having a small patient population; risks related to regulatory authorities not accepting data from trials conducted in locations outside of their jurisdiction; risks relating to Edgewise's ability to attract and retain highly skilled executive officers and employees; Edgewise's ability to obtain and maintain intellectual property protection for its product candidates; Edgewise's reliance on third parties; general economic and market conditions; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled "Risk Factors" in documents that Edgewise files from time to time with the U.S. Securities and Exchange Commission. These forward-looking statements are made as of the date of this press release, and Edgewise assumes no obligation to update the forward-looking statements, or to update the reasons why actual results could differ from those projected in the forward-looking statements, except as required by law. This press release contains hyperlinks to information that is not deemed to be incorporated by reference into this press release. Edgewise Therapeutics, Inc. Condensed Statement of Operations (in thousands except share and per share amounts, unaudited) Three months ended March 31, 2025 December 31, 2024 Operating expenses: Research and development $ 36,757 $ 36,370 General and administrative 9,202 9,170 Total operating expenses 45,959 45,540 Loss from operations (45,959) (45,540) Interest income 5,161 5,878 Net loss $ (40,798) $ (39,662) Net loss per share - basic and diluted $ (0.43) $ (0.42) Weighted-average shares outstanding, basic and diluted 95,130,053 94,721,769 Edgewise Therapeutics, Inc. Condensed Balance Sheet Data (in thousands, unaudited) March 31 December 31, 2025 2024 Assets Cash, cash equivalents and marketable securities $ 436,387 $ 470,170 Other assets 18,004 16,647 Total assets $ 454,391 $ 486,817 Liabilities and stockholders' equity Liabilities 24,672 27,601 Stockholders' equity 429,719 459,216 Total liabilities and stockholders' equity $ 454,391 $ 486,817 View original content to download multimedia:https://www.prnewswire.com/news-releases/edgewise-therapeutics-reports-first-quarter-2025-financial-results-and-recent-business-highlights-302449035.html SOURCE Edgewise Therapeutics