EWTX

– Announced positive top-line data from 12-week Phase 2 CIRRUS-HCM trial with EDG-7500 in obstructive and nonobstructive hypertrophic cardiomyopathy (HCM) – – Completed sale of sevasemten and muscular dystrophy business to Servier for up to $2.65 billion in July 2026, strengthening the balance sheet, sharpening focus on the cardiovascular pipeline – BOULDER, Colo., Aug. 6, 2026 /PRNewswire/ -- Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading clinical-stage biopharmaceutical company focused on cardiovascular diseases, today reported financial results for the second quarter of 2026 and recent business highlights. "In the second quarter, Edgewise entered into a deal to sell sevasemten and our muscular dystrophy business to Servier. Our post-deal close financial position enhances our ability to advance EDG-7500 and our cardiovascular-focused pipeline toward important clinical and regulatory milestones as an independent company," said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise Therapeutics. "With positive Phase 2 CIRRUS-HCM results for EDG-7500 and continued progress with EDG-15400, we are well positioned to advance a differentiated pipeline for patients with serious cardiovascular disease." Recent Highlights Sevasemten and Muscular Dystrophy Program SaleIn July 2026, Edgewise completed the previously announced transaction under which Servier, an independent international pharmaceutical group governed by a foundation, acquired sevasemten and Edgewise's muscular dystrophy business for $1.55 billion in upfront cash consideration and up to $1.1 billion in additional milestone payments, for aggregate potential consideration of up to $2.65 billion. Servier brings the global development, regulatory, and commercial capabilities required to fully realize sevasemten's potential for patients. The transaction strengthens Edgewise's balance sheet, enhances financial flexibility and sharpens the company's strategic focus on advancing its cardiovascular pipeline. This transaction marks Edgewise's transition to a cardiovascular-focused company, with a pipeline comprising EDG-7500 for hypertrophic cardiomyopathy, EDG-15400 for heart failure with preserved ejection fraction (HFpEF) and EDG-003 for an undisclosed target. Cardiovascular Programs CIRRUS-HCM Phase 2 trial in adults with symptomatic HCM: The company announced positive top-line results from the 12-week Phase 2 Part D CIRRUS-HCM open label trial of EDG-7500 in obstructive (oHCM) and nonobstructive (nHCM) HCM, which was designed to inform a Phase 3 trial. Results from the study are available in the company's June 2026 press release, which can be found here. The company expects to initiate a Phase 3 trial in the fourth quarter of 2026. To learn more about CIRRUS-HCM, visit ClinicalTrials.gov, NCT06347159. EDG-15400 and heart failure: EDG-15400 is a novel, oral, selective cardiac sarcomere modulator being developed for the treatment of heart failure. EDG-15400 has completed dosing in a Phase 1 randomized, double-blind, placebo-controlled, single and multiple ascending dose study in healthy adults evaluating safety, tolerability, pharmacokinetics and pharmacodynamics. The company expects to initiate a Phase 2 trial in participants with heart failure with preserved ejection fraction (HFpEF) in the second half of 2026. To learn more about this study, go to ClinicalTrials.gov (NCT07177066). Second Quarter 2026 Financial ResultsCash, cash equivalents and marketable securities were approximately $460.7 million as of June 30, 2026. Our cash and cash equivalents at June 30, 2026 do not include the $1,550.0 million in upfront cash proceeds for the sale of sevasemten on July 10, 2026. Combining the actual balance at June 30, 2026 with the upfront cash proceeds of the sale provides for a pro forma balance of $2,010.7 million before taxes and transaction related costs. Research and development (R&D) expenses were $47.5 million for the second quarter of 2026, compared to $33.6 million for the same period in 2025. The increase was primarily driven by increased clinical development activity related to EDG‑7500, EDG‑15400 and patient rollover activity in the MESA open-label extension study, as well as higher personnel‑related costs to support the advancement of the Company's clinical‑stage programs. General and Administrative (G&A) expenses were $14.4 million for the second quarter of 2026, compared to $9.1 million for the same period in 2025. The increase was primarily due to higher personnel‑related costs, including stock‑based compensation, as the Company expanded its organizational capabilities to support clinical development, as well as increased professional fees and other administrative costs. Net loss and net loss per share were $57.3 million or $0.53 per share for the second quarter of 2026, compared to $36.1 million or $0.34 per share for the same period in 2025. About Edgewise Therapeutics Edgewise Therapeutics is a leading biopharmaceutical company focused on novel, muscle-targeted therapeutics. Leveraging its expertise in muscle biology and small molecule drug development, Edgewise is advancing a cardiovascular pipeline targeting significant unmet needs, including hypertrophic cardiomyopathy, heart failure and other serious cardiovascular and cardiometabolic conditions. To learn more, go to edgewisetx.com or follow Edgewise 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 EDG-7500, EDG-15400 and its cardiovascular programs; statements regarding Edgewise's expectations relating to its clinical trials; statements regarding timing of Edgewise's initiation of a Phase 3 trial of EDG-7500 in HCM and a Phase 2 trial of EDG-15400 in participants with HFpEF; statements regarding Edgewise's ability to advance a differentiated pipeline for patients with serious cardiovascular disease; statements regarding the sale of sevasemten and muscular dystrophy business to Servier (Transaction), including potential payments which may become owing to Edgewise; 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 potential need for substantial additional capital to finance its operations; Edgewise's substantial dependence on the success of EDG-7500; Edgewise's ability to develop and commercialize EDG-7500 and EDG-15400, and discover, develop and commercialize product candidates in its cardiovascular, cardiometabolic and 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, top-line and preliminary data from Edgewise's clinical trials changing as more patient data becomes available; risks related to failure to develop a proprietary drug discovery platform; 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 regulatory approval processes of domestic and foreign authorities being lengthy, time consuming and inherently unpredictable; risks relating to disruptions at the FDA, the SEC and other government agencies; 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; risks related to future acquisitions or strategic partnerships; risks related to Edgewise's future operations following the sale of sevasemten; risks relating to Edgewise not receiving milestone payments related to the Transaction; risks related to 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. View original content to download multimedia:https://www.prnewswire.com/news-releases/edgewise-therapeutics-reports-second-quarter-2026-financial-results-302844178.html

We expect investors to focus on updates related to Viking Therapeutics’ VKTX pipeline when it reports second-quarter 2026 earnings. In the last reported quarter, the company’s earnings missed expectations by more than 44%. Since the company lacks a marketed drug in its portfolio, no revenues are expected to have been recorded. The Zacks Consensus Estimate for earnings is pegged at a loss of $1.21 per share. Investor focus will likely be on pipeline updates. Viking Therapeutics’ lead candidate is VK2735, which is being developed to treat obesity. The company is conducting two late-stage studies (VANQUISH-1 and VANQUISH-2) on the subcutaneous (SC) formulation of VK2735. While VANQUISH-1 is evaluating the drug in obese adults with at least one weight-related co-morbid condition and without type II diabetes (T2D), VANQUISH-2 is assessing its efficacy in obese or overweight adults with T2D. Data from these studies is not expected before next year. Investors will likely be seeking updates on the ongoing maintenance dosing study on VK2735, which was initiated last year. This study is evaluating multiple regimens — monthly SC, weekly oral and daily oral dosing — to determine whether the initial weight loss achieved with weekly SC dosing can be sustained. The company had previously announced that it would report SC maintenance results in the third quarter of 2026, followed by oral maintenance results in the first half of 2027. Investors will also likely be interested in seeking updates from VKTX on the study design for the late-stage program on the oral version of VK2735. Viking Therapeutics had previously announced plans to start the program in the fourth quarter of 2026. The biotech firm’s performance has been dismal over the past four quarters. Its earnings missed estimates in each of the trailing four quarters, delivering a negative average surprise of 36.70%. Viking Therapeutics, Inc. price-eps-surprise | Viking Therapeutics, Inc. Quote Year to date, shares of the company have gained more than 3% compared withthe industry’s nearly 2% growth. Image Source: Zacks Investment Research Per our proven model, companies with the combination of a positive Earnings ESP and a Zacks Rank #1 (Strong Buy), #2 (Buy) or#3 (Hold) have a good chance of delivering an earnings beat. This is not the case here. You can uncover the best stocks to buy or sell before they're reported with our Earnings ESP Filter. Viking has an Earnings ESP of 0.00% and a Zacks Rank #4 (Sell) at present. You can see the complete list of today’s Zacks #1 Rank stocks here. Here are some drug/biotech stocks that have the right combination of elements to beat on earnings this time around: Absci Corporation ABSI has an Earnings ESP of +15.22% and a Zacks Rank #2 at present. Shares of ABSI have skyrocketed about 136% year to date. The company’s earnings beat estimates in one of the trailing four quarters, while missing the mark on the other three occasions. Agenus delivered an average negative surprise of 17.58%. Absci will report second-quarter 2026 earnings on Aug. 11, after market close. Edgewise Therapeutics EWTX has an Earnings ESP of +13.28% and a Zacks Rank #2 at present. Shares of EWTX have surged 54% year to date. The company’s earnings beat estimates in three of the trailing four quarters while missing out on one occasion, delivering an average surprise of 5.06%. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Viking Therapeutics, Inc. (VKTX) : Free Stock Analysis Report Edgewise Therapeutics, Inc. (EWTX) : Free Stock Analysis Report Absci Corporation (ABSI) : Free Stock Analysis Report This article originally published on Zacks Investment Research (zacks.com). Zacks Investment Research

On May 15, 2026, Cormorant Asset Management disclosed a buy of 313,645 shares of Alumis (NASDAQ:ALMS), with the estimated transaction value at $7.84 million based on quarterly average pricing. According to a May 15, 2026 SEC filing, Cormorant Asset Management increased its position in Alumis by 313,645 shares during the first quarter. The firm’s estimated trade size was $7.84 million, calculated using the quarter’s average closing price. The stake’s value at quarter-end rose by $51.52 million, a figure that incorporates both buying activity and market price movements. Cormorant’s buy brings its Alumis stake to 4.37% of 13F assets as of March 31, 2026. Top holdings after the filing: NASDAQ:PRAX: $285.30 million (14.4% of AUM) NASDAQ:BBOT: $160.01 million (8.1% of AUM) NASDAQ:EYPT: $106.54 million (5.4% of AUM) NASDAQ:EWTX: $102.69 million (5.2% of AUM) NASDAQ:ERAS: $93.84 million (4.7% of AUM) As of May 14, 2026, Alumis shares were priced at $24.63, up about 400% over the past year and vastly outperforming the S&P 500’s roughly 25% gain in the same period. Alumis develops clinical-stage biopharmaceutical products targeting autoimmune and neuroinflammatory diseases, including ESK-001 and A-005, with a focus on allosteric TYK2 inhibitors. The company operates a research-driven model, advancing proprietary drug candidates through clinical trials. It targets healthcare providers and pharmaceutical partners addressing autoimmune and neurodegenerative conditions, with a primary focus on the biotechnology and healthcare sectors. Alumis is a clinical-stage biotechnology company specializing in the development of novel therapies for autoimmune and neuroinflammatory disorders. The company leverages its expertise in allosteric TYK2 inhibition to advance a pipeline of differentiated drug candidates. With a research-centric strategy and a focus on high unmet medical needs, Alumis aims to establish a competitive edge in the biopharmaceutical landscape. This is one of several buys Cormorant made last quarter into high-flying biotechs. With shares up roughly 400% over the past year, the fund added even more exposure, signaling confidence that the company’s late-stage autoimmune pipeline could continue driving upside from here.A lot of that optimism centers around envudeucitinib, Alumis’ oral TYK2 inhibitor for plaque psoriasis and lupus. Just last week, the company reported strong Phase 3 psoriasis data showing PASI 90 response rates above 60% and PASI 100 rates ofo about 40% by Week 24, results management said support the drug’s potential as a leading oral psoriasis therapy. Alumis also said it remains on track to submit an NDA in the fourth quarter of 2026, while potentially pivotal lupus data are expected in the third quarter.The balance sheet also gives the company flexibility. Alumis ended March with about $569.5 million in cash, cash equivalents, and marketable securities, which management says should fund operations into late 2027.As with its other buys (in Dianthus and Erasca), the opportunity is clear, but so is the risk. Expectations are now extremely high after the stock’s massive run, meaning future clinical data will matter far more than hype alone. Before you buy stock in Alumis, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the 10 best stocks for investors to buy now… and Alumis wasn’t one of them. The 10 stocks that made the cut could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you’d have $469,293!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you’d have $1,381,332!* Now, it’s worth noting Stock Advisor’s total average return is 993% — a market-crushing outperformance compared to 207% for the S&P 500. Don't miss the latest top 10 list, available with Stock Advisor, and join an investing community built by individual investors for individual investors. See the 10 stocks » *Stock Advisor returns as of May 17, 2026. Jonathan Ponciano has no position in any of the stocks mentioned. The Motley Fool has no position in any of the stocks mentioned. The Motley Fool has a disclosure policy. Alumis Stock Has Soared 400%. Cormorant Bought Another $8 Million Last Quarter was originally published by The Motley Fool

– Announced positive long-term sevasemten data demonstrating sustained functional stabilization in Becker muscular dystrophy (Becker) up to 3.5 years of follow-up – – CIRRUS-HCM 12-week data of EDG-7500 in obstructive and nonobstructive hypertrophic cardiomyopathy (HCM) expected in Q2 2026 – – Pivotal GRAND CANYON results for sevasemten in Becker expected in Q4 2026 – BOULDER, Colo., May 7, 2026 /PRNewswire/ -- Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the first quarter of 2026 and recent business highlights. "For the first time with an investigational agent, we have generated long-term data showing that people with Becker remain stable despite a disease that typically leads to significant functional decline," said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise. "We remain on track to report pivotal top-line data from GRAND CANYON evaluating sevasemten, which has the potential to support our first marketing application in Becker. We also anticipate results this quarter from CIRRUS-HCM 12-week Part D evaluating EDG-7500 in HCM, which will help refine our Phase 3 strategy. With multiple near-term catalysts and a highly focused team, we are well positioned to advance our programs and deliver potentially transformative therapeutic options for patients facing serious muscle diseases facing high unmet need." Recent Highlights Muscular Dystrophy Program MESA open-label extension trial in adults with Becker: MESA is an open-label extension study evaluating sevasemten long-term safety, tolerability, and efficacy in adults and adolescents with Becker previously treated in sevasemten clinical trials. Nearly all eligible participants (99%) from prior sevasemten studies (including ARCH, DUNE and CANYON/GRAND CANYON) chose to enroll in the MESA open-label extension study. At the 2026 MDA Clinical and Scientific Conference, the Company presented long-term data from MESA showing participants on sevasemten experienced stabilization of function up to 3.5 years in marked contrast to the functional decline expected from Becker natural history data. The data reinforce prior clinical findings of sevasemten in Becker, a rare disease with no approved treatments. The results can be found here. The Company also presented a poster with new analysis from the CANYON study showing sevasemten had a well-tolerated cardiac profile over 12 months, with no adverse effects on left ventricular ejection fraction (LVEF) or NT‑proBNP and evidence of LVEF improvement in certain subgroups, with positive trends observed across all adult participants versus placebo. The poster can be viewed here. To learn more about MESA or CANYON, go to clinicaltrials.gov (MESA: NCT06066580, CANYON (NCT05291091). GRAND CANYON, a global pivotal placebo-controlled cohort in Becker: GRAND CANYON is designed to assess the efficacy and safety of sevasemten over an 18-month period, with North Star Ambulatory Assessment (NSAA) as the primary endpoint. The study is highly powered to be able to show a statistically significant difference in NSAA versus placebo over 18 months. The Company expects to report top-line data in the fourth quarter of 2026 and is continuing preparations for a potential marketing application to seek approval as the first targeted therapy for this underserved population, planned for the first half of 2027. To learn more about GRAND CANYON, go to clinicaltrials.gov (NCT05291091). LYNX and FOX Phase 2 placebo-controlled trials in boys with Duchenne: LYNX is designed to evaluate the effect of sevasemten on safety, biomarkers of muscle damage and function in boys with Duchenne in a placebo-controlled dose ranging study, followed by an open-label extension period. FOX is designed to evaluate 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. The Company continues to collect longer-term data in the open-label extension portion of these Phase 2 programs to inform the future clinical studies in Duchenne. For more information, go to clinicaltrials.gov to learn more about LYNX (NCT05540860) and FOX (NCT06100887). Cardiovascular Programs CIRRUS-HCM Phase 2 trial in adults with symptomatic HCM: The Company is advancing CIRRUS-HCM, a multi-part, open-label trial, in participants with HCM at over 20 clinical sites in the U.S. Part A of the trial evaluated the safety and tolerability of a single oral dose of EDG-7500 in participants with obstructive HCM (oHCM). Parts B and C evaluated fixed doses of EDG-7500 over 28 days in oHCM and nonobstructive HCM (nHCM), respectively. The results can be found here. Part D is a 12-week study with an open-label extension including participants with oHCM and nHCM designed to explore dose response and optimization. The Company plans to report 12-week Part D data (oHCM and nHCM) in the second quarter of 2026 and initiate Phase 3 in the second half of 2026. To learn more about CIRRUS-HCM, visit clinicaltrials.gov, NCT06347159. EDG-15400 and heart failure: EDG-15400 is a novel oral, selective, cardiac sarcomere modulator, targeted for the treatment of heart failure and other diseases of diastolic dysfunction. EDG-15400 is currently being evaluated in healthy adults in a Phase 1, randomized, double-blind, placebo-controlled, single and multiple ascending dose study evaluating safety, tolerability, pharmacokinetics and pharmacodynamics. The Company expects to initiate a Phase 2 trial in participants with heart failure with preserved ejection fraction (HFpEF) in the second half of 2026. To learn more about this study, go to clinicaltrials.gov (NCT07177066). First Quarter 2026 Financial Results Cash, cash equivalents and marketable securities were approximately $499.6 million as of March 31, 2026. Research and development (R&D) expenses were $42.7 million for the first quarter of 2026, compared to $36.8 million for the same period in 2025. The increase was primarily driven by increased clinical development activity related to EDG‑7500 and EDG‑15400, as well as higher internal personnel‑related costs to support the advancement of the Company's clinical‑stage programs. These increases were partially offset by lower sevasemten clinical program expenses due to timing of activity in the GRAND CANYON study and patient rollover into the MESA open‑label extension study. General and Administrative (G&A) expenses were $11.5 million for the first quarter of 2026, compared to $9.2 million for the same period in 2025. The increase was primarily due to higher personnel‑related costs, including stock‑based compensation, as the Company expanded its organizational capabilities to support clinical development, as well as increased professional fees and other administrative costs. Net loss and net loss per share were $49.0 million or $0.46 per share for the first quarter of 2026, compared to $40.8 million or $0.43 per share for the same period in 2025. 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 symptomatic hypertrophic cardiomyopathy, currently in Phase 2 clinical development. EDG-15400 is a novel cardiac sarcomere modulator for the treatment of heart failure, currently in Phase 1 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 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, EDG-15400 and its cardiovascular programs; statements regarding Edgewise's expectations relating to its clinical trials, including timing of reporting data (including 12-week data on the CIRRUS-HCM trial, and the data from the GRAND CANYON trial; statements regarding Edgewise's potential marketing application in Becker; statements regarding sevasemten potentially being the first approved therapy for Becker; statements regarding the potential results of Edgewise's GRAND CANYON trial; statements regarding Edgewise's market opportunity; statements regarding Edgewise's plans to continue to collect longer-term open-label extension data for its LYNX and FOX Phase 2 trials; statements regarding timing of Edgewise's initiation of a Phase 3 trial of EDG-7500 in HCM and a Phase 2 trial of EDG-15400 in participants with HFpEF; statements regarding Edgewise's ability to advance its pipeline; 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, EDG-7500 and EDG-15400, and discover, develop and commercialize product candidates in its cardiovascular, cardiometabolic and 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 failure to develop a proprietary drug discovery platform; risks related to exposure to additional risk if we develop sevasemten and potential other programs in connection with other therapies; 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 the regulatory approval processes of domestic and foreign authorities being lengthy, time consuming and inherently unpredictable; risks relating to disruptions at the FDA, the SEC and other government agencies; 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; risks related to future acquisitions or strategic partnerships; risks related to 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. View original content to download multimedia:https://www.prnewswire.com/news-releases/edgewise-therapeutics-reports-first-quarter-2026-financial-results-and-advances-clinical-programs-in-muscular-dystrophy-and-cardiovascular-indications-302764426.html

Get insights on thousands of stocks from the global community of over 7 million individual investors at Simply Wall St. Edgewise Therapeutics (EWTX) just released three and a half year data from its MESA open label extension study of sevasemten in Becker muscular dystrophy, highlighting stable motor function in a setting where decline is typically expected. See our latest analysis for Edgewise Therapeutics. Following the MESA update and recent conference appearances, Edgewise Therapeutics’ share price of $29.59 sits against a 90 day share price return of 29.95% and a 3 year total shareholder return close to 3x. This suggests momentum has been building over the longer term despite some short term volatility. If this kind of clinical news has your attention, it can be useful to see what else is moving in related areas, starting with 34 healthcare AI stocks. After a 3 year total shareholder return near 3x and a share price sitting at $29.59, with analysts’ price targets implying further upside, you have to ask: Is Edgewise Therapeutics still mispriced, or is the market already baking in future growth? At a last close of $29.59, Edgewise Therapeutics trades on a P/B of 6.1x, which sits well above the broader US pharmaceuticals industry average of 2.1x. The P/B ratio compares the company’s market value to its book value, so a higher multiple often reflects expectations for future value creation beyond current net assets. For a business like Edgewise Therapeutics, which is focused on research and development and is currently unprofitable, this usually means investors are paying up for its pipeline potential rather than existing earnings or revenues. What stands out is that while 6.1x looks expensive against the wider industry, it looks much lower against a peer group average of 21.8x. That gap suggests the market is pricing Edgewise Therapeutics at a premium to the typical pharma name, but at a discount to closer peers that investors see as more directly comparable in terms of business model and stage. On balance, the company’s P/B of 6.1x is high versus the broader sector and low versus its peer set. The multiple therefore sits between those two reference points without a clear lean to either extreme. See what the numbers say about this price — find out in our valuation breakdown. Result: Price-to-book of 6.1x (ABOUT RIGHT) However, the story can change quickly if future MESA readouts disappoint or if sevasemten, EDG-7500 or EDG-15400 encounter unexpected safety or regulatory setbacks. Find out about the key risks to this Edgewise Therapeutics narrative. Positive clinical signals and a premium P/B can look reassuring, but investors are clearly watching the risk side too. Weigh the evidence quickly and decide where you stand with 3 important warning signs If Edgewise Therapeutics is on your radar, do not stop there. Widen your options with other ideas that could fit different roles in your portfolio. Target potential bargains by scanning companies that combine quality and attractive pricing through the 49 high quality undervalued stocks. Strengthen your income stream by reviewing companies with higher yields and resilient profiles via the 14 dividend fortresses. Reduce portfolio stress by focusing on companies with steadier profiles using the 74 resilient stocks with low risk scores. This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. Companies discussed in this article include EWTX. Have feedback on this article? Concerned about the content? Get in touch with us directly. Alternatively, email [email protected]

– CIRRUS-HCM 12-week data of EDG-7500 in obstructive and nonobstructive hypertrophic cardiomyopathy (HCM) expected in H1 2026 – – Phase 1 healthy adult trial data of EDG-15400 and plans for heart failure studies expected in H1 2026 – – Pivotal GRAND CANYON results for sevasemten in Becker muscular dystrophy (Becker) expected in Q4 2026 – BOULDER, Colo., Feb. 26, 2026 /PRNewswire/ -- Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the fourth quarter and full year of 2025 and recent business highlights. "Following strong execution in 2025, we have entered a transformative year," said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise. "We remain on track to deliver pivotal GRAND CANYON top-line data for sevasemten, with the potential to support the first marketing application for the treatment of Becker. We also expect CIRRUS-HCM 12-week Part D results for EDG-7500 in HCM in the first half of 2026. Data from Part D will support refinement of our Phase 3 trial strategy in HCM. With multiple near-term catalysts and a high-performing team, we are closer than ever to delivering transformative medicines for patients living with serious conditions that still lack adequate treatment options and represent significant unmet medical needs." Recent Highlights Muscular Dystrophy Program GRAND CANYON, a global pivotal placebo-controlled cohort in Becker: GRAND CANYON is designed to assess the efficacy and safety of sevasemten over an 18-month period, with North Star Ambulatory Assessment (NSAA) as the primary endpoint. The study is highly powered to be able to show a statistically significant difference in NSAA versus placebo over 18 months. The Company expects to report top-line data in the fourth quarter of 2026 and is continuing preparations for a potential NDA submission to the U.S. Food and Drug Administration (FDA) for sevasemten in Becker, planned for the first half of 2027. To learn more about GRAND CANYON, go to clinicaltrials.gov (NCT05291091). MESA open-label extension trial in adults with Becker: The Company continues to advance MESA, an open-label extension trial that collects long-term safety and efficacy data of sevasemten in participants with Becker who were previously enrolled in ARCH, or who completed CANYON, GRAND CANYON, or DUNE. As of December 2025, 99% of eligible participants have enrolled in MESA, with exposures to sevasemten for up to four years. LYNX and FOX Phase 2 placebo-controlled trials in boys with Duchenne: LYNX is designed to evaluate the effect of sevasemten on safety, biomarkers of muscle damage and function in 4- to 9-year-old participants with Duchenne in a placebo-controlled dose ranging study, followed by an open-label extension period. FOX is designed to evaluate the effect of sevasemten on safety, biomarkers of muscle damage and function in 6- to 14-year-old participants with Duchenne who have been previously treated with gene therapy. The Company will continue to collect longer-term safety and functional data in the open-label extensions of LYNX and FOX while it plans for a Phase 3 program. For more information, go to clinicaltrials.gov to learn more about LYNX (NCT05540860) and FOX (NCT06100887). Cardiovascular Programs CIRRUS-HCM Phase 2 trial in adults with symptomatic HCM: The Company is advancing CIRRUS-HCM, a multi-part, open-label trial, in participants with HCM at over 20 clinical sites in the U.S. Part A of the trial evaluated the safety and tolerability of a single oral dose of EDG-7500 in participants with obstructive HCM (oHCM). Parts B and C evaluated fixed doses of EDG-7500 over 28 days in oHCM and nonobstructive HCM (nHCM), respectively. Part D is a 12-week study with an open-label extension including participants with oHCM and nHCM designed to explore dose response and optimization. The Company plans to report 12-week Part D data in oHCM and nHCM in the first half of 2026 and initiate Phase 3 in the second half of 2026. To learn more about CIRRUS-HCM, visit clinicaltrials.gov, NCT06347159. EDG-15400 and heart failure: EDG-15400 is a novel oral, selective, cardiac sarcomere modulator, targeted for the treatment of heart failure and other diseases of diastolic dysfunction. EDG-15400 is currently being evaluated in healthy adults in a Phase 1, randomized, double-blind, placebo-controlled, single and multiple ascending dose study evaluating safety, tolerability, pharmacokinetics and pharmacodynamics. The Company expects top-line results from this study in the first half of 2026 and to initiate a Phase 2 trial in participants with heart failure with preserved ejection fraction (HFpEF) in the second half of 2026. To learn more about this study, go to clinicaltrials.gov (NCT07177066). Fourth Quarter Financial Results Cash, cash equivalents and marketable securities were approximately $530.1 million as of December 31, 2025. Research and development (R&D) expenses were $43.6 million for the fourth quarter of 2025, compared to $37.5 million for the immediately preceding quarter. The increase of $6.1 million was primarily driven by a $3.5 million increase in personnel related costs, $2.2 million increase in EDG-15400 clinical development activities related to Phase 1 trial activity initiated in the third quarter 2025, a $1.9 million increase in EDG-7500 clinical development activities related to continued patient activity in our CIRRUS-HCM trial and other pharmacokinetic studies, partially offset by a $0.8 million decrease in sevasemten clinical development activities related to decreased patient activity in GRAND CANYON as patients transition to the MESA open-label extension trial and a $0.7 million decrease in professional fees, manufacturing, and other research costs. General and administrative (G&A) expenses were $12.4 million for the fourth quarter of 2025, compared to $9.4 million for the immediately preceding quarter. The increase of $3.0 million was primarily driven by a $2.2 million increase in personnel-related costs and a $0.8 million increase in professional fees and other administrative costs. Net loss and net loss per share for the fourth quarter of 2025 was $50.2 million or $0.47 per share, compared to $40.7 million or $0.39 per share for the immediately 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 symptomatic hypertrophic cardiomyopathy, currently in Phase 2 clinical development. EDG-15400 is a novel cardiac sarcomere modulator for the treatment of heart failure, currently in Phase 1 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 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, EDG-15400 and its cardiovascular programs; statements regarding Edgewise's expectations relating to its clinical trials, including timing of reporting data (including 12-week data on the CIRRUS-HCM trial, the data from the GRAND CANYON trial and the data from the Phase 1 healthy adult trial of EDG-15400); statements regarding sevasemten potentially being the first approved therapy for Becker; statements regarding a potential NDA submission to the FDA for sevasemten in Becker and the timing of such submission; statements regarding the potential results of Edgewise's GRAND CANYON trial; statements regarding Edgewise's market opportunity; statements regarding Edgewise's plans to continue to collect longer-term open-label extension data; statements regarding Edgewise's Phase 3 trial design in Duchenne; statements regarding timing of Edgewise's initiation of a Phase 3 trial of EDG-7500 in HCM and a Phase 2 trial of EDG-15400 in participants with HFpEF; statements regarding Edgewise's ability to advance its pipeline; 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, EDG-7500 and EDG-15400, and discover, develop and commercialize product candidates in its cardiovascular, cardiometabolic and 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 failure to develop a proprietary drug discovery platform; risks related to exposure to additional risk if we develop sevasemten and potential other programs in connection with other therapies; 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 the regulatory approval processes of domestic and foreign authorities being lengthy, time consuming and inherently unpredictable; risks relating to disruptions at the FDA, the SEC and other government agencies; 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; risks related to future acquisitions or strategic partnerships; risks related to 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. View original content to download multimedia:https://www.prnewswire.com/news-releases/edgewise-therapeutics-reports-fourth-quarter-and-full-year-2025-financial-results-with-strong-progress-across-muscular-dystrophy-and-cardiovascular-programs-302696484.html

– In the CIRRUS-HCM trial, including interim safety results from Part D, EDG-7500 was generally well tolerated; no clinically meaningful reductions in LVEF or LVEF <50% – – On track to deliver full 12-week Part D readout in 2Q 2026 and Phase 3 start in 4Q 2026 – BOULDER, Colo., Dec. 24, 2025 /PRNewswire/ -- Edgewise Therapeutics, Inc., (Nasdaq: EWTX), today announced positive updates from the ongoing CIRRUS-HCM, Phase 2 clinical trial of EDG-7500, a novel oral, selective, cardiac sarcomere modulator, specifically designed to slow early contraction velocity and address impaired cardiac relaxation associated with hypertrophic cardiomyopathy (HCM) without impacting systolic function, two central clinical goals in the current management of HCM. CIRRUS-HCM is a multi-part, open label trial of EDG-7500 in participants with obstructive and nonobstructive HCM. Earlier this year, the Company reported positive top-line results from the Part B (oHCM, n=17) and Part C (nHCM, n=12) 50 mg and 100 mg fixed dose cohorts, in which EDG-7500 administration led to improvements in key HCM disease markers, including N-terminal pro-B-type natriuretic peptide (NT-proBNP), Kansas City Cardiomyopathy Questionnaire (KCCQ), New York Heart Association (NYHA) class and left ventricular outflow tract gradient (LVOT-G) (in oHCM participants). In both Parts B and C, EDG-7500 administration led to KCCQ score improvements that appeared favorable relative to those reported in other cardiovascular trials, including those with cardiac myosin inhibitors (CMIs). EDG-7500 administration was also associated with measurable improvements in key diastolic parameters, including robust reductions in NT-proBNP, reduced left ventricular filling pressures, and improved relaxation metrics. In addition to the 50 mg and 100 mg fixed doses previously reported, the Company evaluated a lower 25 mg fixed dose in 4 oHCM and 10 nHCM participants in CIRRUS-HCM Parts B and C, respectively. In these cohorts, evidence of clinical activity was observed across key HCM disease markers, including NT-proBNP, KCCQ, NYHA and LVOT-G (in oHCM participants), while maintaining a favorable safety profile, with no clinically meaningful changes in left ventricular ejection fraction (LVEF) or reductions in LVEF to below <50% and no events of atrial fibrillation (AF). The CIRRUS-HCM Part B and C cohorts have completed dosing, with a total of 43 participants enrolled across all dose groups. CIRRUS-HCM Part D is designed to explore exposure-response correlations, assess biomarker-guided dose optimization to inform the design of Phase 3 trials, and support regulatory discussions. EDG-7500 is being evaluated over a 12-week active treatment period; participants will be provided the opportunity to continue into a long-term extension study. Dosing is guided by objective measures—LVOT-G in oHCM and NT-proBNP in nHCM—with participants dosed from 25 mg to 150 mg and eligible for further optimization up to 200 mg in the extension phase. With intra-patient dose optimization results from Part D, the Company aims to build on the robust clinical improvements observed in Parts B and C to further support EDG-7500's best-in-disease potential. Screening at sites involved in Part D is complete, with more than 40 participants enrolled. As of the December 23, 2025 data cutoff, approximately 70% of participants had reached a dose of 100 mg or higher. Overall, patient demographics in Part D are generally consistent with those reported in Phase 3 studies of CMIs. In this interim safety update, for the 20 participants who completed 12 weeks of dosing in Part D as of the data cutoff date (8 with oHCM and 12 with nHCM), EDG-7500 generally had a favorable safety profile and was well tolerated. Consistent with previous observations, no clinically significant changes in LVEF or reductions in LVEF to below 50% were observed, with EDG-7500 continuing to demonstrate a differentiated LVEF profile relative to CMIs. The safety profile observed with EDG-7500 to date, across both healthy volunteers and the CIRRUS-HCM trial, supports the potential use of EDG-7500 in diverse HCM patient populations. Unlike CMIs, which have a mechanism of action that has been associated with a risk of systolic dysfunction and an increased risk of heart failure, EDG-7500 administration continues to present no meaningful effect on LVEF. Preservation of systolic function has the potential to avoid the patient safety monitoring burden that could limit CMI adoption outside of HCM centers of excellence, which could expand prescriber reach and patient access, if approved. Furthermore, in Part D, continuous active cardiac monitoring was conducted for four weeks during screening and for two weeks following each dose escalation. More than 300 ambulatory cardiac monitoring devices have been deployed to participants, capturing over 2,600 patient-days of active cardiac monitoring, during which no clinically detectable AF or atrial flutter has been observed. A single adverse event of new onset AF was reported; this event occurred in an oHCM participant who was not on active cardiac monitoring at the time and who had a history of arrhythmias, including supraventricular tachycardia. The investigator deemed the event as not related to study drug. "I'm excited about the advances we've made in Part D of the CIRRUS-HCM trial, where we've exceeded our year-end enrollment goal, highlighting continued enthusiasm for the program from patients and physicians" said Kevin Koch, Ph.D., President and Chief Executive Officer. "I'm especially pleased with EDG-7500's safety profile to date, and the lack of clinically relevant drops in ejection fraction, or any ejection fraction drops below 50%. A major focus of 2026 will be refining our development strategy to deliver the best-in-disease therapeutic profile that EDG-7500 may offer to obstructive and nonobstructive HCM patients." The Company remains on track to deliver comprehensive efficacy and safety data from Part D of the CIRRUS-HCM trial in the second quarter of 2026 and is advancing Phase 3 trial design in preparation for trial initiation by the end of 2026. About Hypertrophic Cardiomyopathy 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. Individuals with HCM can become extremely limited in their functional capacity and ability to perform the activities of daily living. Commonly experienced symptoms include breathlessness, irregular heartbeats, chest pain, tiredness, dizziness, or even fainting. These symptoms are caused by excessive contraction and thickening (hypertrophy) of the left ventricular wall of the heart. Over time, the thickened muscle becomes stiff, making it difficult for the heart to relax and fill with blood (diastolic dysfunction). There are two major forms of HCM obstructive and nonobstructive. Despite advancements in treatment options for some patients with HCM, there remains a significant unmet need for additional therapeutic approaches for patients. 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. EDG-7500 is a novel cardiac sarcomere modulator for the treatment of hypertrophic cardiomyopathy , currently in Phase 2 clinical development. Sevasemten is an orally administered first-in-class fast skeletal myosin inhibitor in late-stage clinical trials in Becker and Duchenne muscular dystrophies. EDG-15400 is a novel cardiac sarcomere modulator for the treatment of heart failure, currently in Phase 1 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 and X. 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 benefits and potential of, and expectations regarding, EDG-7500; statements regarding the market opportunity for EDG-7500; statements regarding Edgewise's expectations relating to its clinical trials, including timing of reporting comprehensive efficacy and safety data from Part D of the CIRRUS-HCM trial and timing of the initiation of the Phase 3 trials of EDG-7500 ); clinical outcomes from trials of EDG-7500, which may materially change as more patient data become available; statements regarding the best-in-disease therapeutic profile of EDG-7500; 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: the potential for the results of the ongoing or any future clinical trial of EDG-7500 to differ from the results of Parts B and C and the interim results of Part D of the ongoing CIRRUS-HCM trial; the risk of delays in completing the ongoing Part D of the CIRRUS-HCM trial or initiating the planned Phase 3 trials of EDG-7500; risks associated with unexpected events during the remainder of Part D of the ongoing CIRRUS-HCM trial, including serious adverse events, toxicities or other undesirable side effects; the risk of difficulties in enrolling or maintaining patients in the clinical trials of EDG-7500; 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, EDG-7500 and EDG-15400 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 failure to develop a proprietary drug discovery platform; risks related to exposure to additional risk if we develop sevasemten and potential other programs in connection with other therapies; 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 the regulatory approval processes of domestic and foreign authorities being lengthy, time consuming and inherently unpredictable; risks relating to disruptions at the FDA, the SEC and other government agencies; 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; risks related to future acquisitions or strategic partnerships; risks related to 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. View original content to download multimedia:https://www.prnewswire.com/news-releases/edgewise-therapeutics-announces-completion-of-the-edg-7500-cirrus-hcm-phase-2-parts-b-and-c-and-favorable-interim-safety-results-from-the-ongoing-part-d-study-in-hypertrophic-cardiomyopathy-302649113.html

– Pivotal GRAND CANYON cohort of sevasemten in Becker expected to read out in Q4 2026; building infrastructure to support potential commercial launch – – MESA open-label extension trial of sevasemten in Becker muscular dystrophy continues to enroll nearly all eligible participants – – Continuing to advance Phase 2 CIRRUS-HCM trial of EDG-7500 in Hypertrophic Cardiomyopathy; on track to provide program update in Q4 2025 – – Initiated Phase 1 healthy adult trial of EDG-15400 for future studies in Heart Failure – BOULDER, Colo., Nov. 6, 2025 /PRNewswire/ -- Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the third quarter of 2025 and recent business highlights. "With a strong balance sheet, we continue to make great progress on our cardiac and skeletal muscle programs," said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise. "Planning for success in GRAND CANYON, we are building the commercial infrastructure to support a potential launch of sevasemten in Becker. We recently initiated clinical development of EDG-15400, our new clinical candidate targeting heart failure and we're actively developing Phase 3 trial designs in HCM and Duchenne, while continuing to advance our pipeline through innovative R&D." Recent Highlights Muscular Dystrophy Program 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. MESA open-label extension trial in adults with Becker: The Company continues to advance MESA, an open-label extension trial that collects long term safety and efficacy data of sevasemten to participants with Becker who were previously enrolled in ARCH, or completed CANYON, GRAND CANYON, or DUNE. As of September 2025, 99% of eligible participants are enrolled in MESA. 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 cohort is active in 12 countries, and has 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. GRAND CANYON is highly powered to be able to show a statistically significant difference in NSAA versus placebo over 18 months and the Company expects to report topline data in the fourth quarter of 2026. To learn more, go to clinicaltrials.gov (NCT05291091). LYNX and FOX Phase 2 placebo-controlled trials in boys with Duchenne: LYNX is designed to evaluate the effect of sevasemten on safety, biomarkers of muscle damage and function in four- to nine-year-old participants with Duchenne treated with sevasemten in a three-month placebo-controlled dose ranging study, followed by an open-label extension period. In June 2025, the Company announced encouraging observations across functional measures, including Stride Velocity 95th Centile (SV95C), NSAA and 4 stair-climb, while identifying a dose of 10 mg to evaluate in Phase 3. FOX is designed to evaluate the effect of sevasemten on safety, biomarkers of muscle damage and function in six- to 14-year-old participants with Duchenne who have been previously treated with gene therapy. FOX participants are on average over 10 years old and four years out from receiving gene therapy. Initial results from the FOX study support that sevasemten 10 mg has the potential to reduce the rate of functional decline. The Company plans to meet with the FDA to discuss a Phase 3 study design including input on the patient population and endpoints, with plans to initiate a pivotal study in 2026. The Company will continue to collect longer-term safety and functional data in the open-label extensions of LYNX and FOX. For more information, go to clinicaltrials.gov to learn more about LYNX (NCT05540860) and FOX (NCT06100887). Cardiovascular and Cardiometabolic Programs 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. 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 over 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 (oHCM). 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 oHCM (Part B) and in participants with nHCM (Part C). Part D was designed to evaluate the safety and efficacy of EDG-7500 for 12 weeks in participants with oHCM and nHCM and has achieved strong enrollment. The Company is on track to share a program update in the fourth quarter of 2025, with more comprehensive data expected in the first half of 2026. To learn more about CIRRUS-HCM, visit clinicaltrials.gov, NCT06347159 (Phase 2). EDG-15400 and Heart Failure: EDG-15400 is a novel oral, selective, cardiac sarcomere modulator, being developed for the treatment of heart failure with preserved ejection fraction (HFpEF), a very common form of heart failure. EDG-15400 is currently being dosed in healthy adults in a Phase 1, randomized, double-blind, placebo-controlled, single and multiple ascending dose study evaluating safety, tolerability, pharmacokinetics and pharmacodynamics. The Company expects topline results from this study in the first half of 2026. To learn more about this study, go to clinicaltrials.gov (NCT07177066). Cardiometabolic preclinical program: The Company continues to advance its preclinical 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 Annual International Congress of the World Muscle Society, Becker Education and Engagement Day, European Society of Cardiology Congress, Heart Failure Society of America Annual Scientific Meeting, and the International Workshop on Cardiomyopathies. The Company continues to sponsor and participate in numerous other clinician and patient-focused events. Third Quarter Financial Results Cash, cash equivalents and marketable securities were approximately $563.3 million as of September 30, 2025. Research and development (R&D) expenses were $37.5 million for the third quarter of 2025, compared to $33.6 million for the immediately preceding quarter. The increase of $3.9 million was primarily driven by a $1.4 million increase in EDG-15400 clinical development activities related to initiation of a Phase 1 trial in the third quarter 2025, a $0.9 million increase in EDG-7500 clinical development activities related to continued patient activity in our CIRRUS-HCM trial and other pharmacokinetic studies, a $0.5 million increase in sevasemten clinical development activities related to additional costs incurred to support continued patient activity in GRAND CANYON and roll over to the MESA open label extension, a $0.9 million increase in professional fees and other research costs, and a $0.2 million increase in manufacturing expenses to support clinical development across our cardiac programs. General and administrative (G&A) expenses were $9.4 million for the third quarter of 2025, compared to $9.1 million for the immediately preceding quarter. The increase of $0.3 million was primarily driven by additional personnel-related costs. Net loss and net loss per share for the third quarter of 2025 was $40.7 million or $0.39 per share, compared to $36.1 million or $0.34 per share for the immediately 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. EDG-15400 is a novel cardiac sarcomere modulator for the treatment of heart failure, currently in Phase 1 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, EDG-15400 and its cardiovascular and cardiometabolic programs; statements regarding a potential U.S. launch of sevasemten in Becker; statements regarding the market opportunity for Duchenne, Becker and HFpEF; statements regarding Edgewise's expectations relating to its clinical trials, including timing of reporting data (including an update and comprehensive data on the CIRRUS-HCM trial, the presentation of data from the GRAND CANYON trial and the presentation of data from the Phase 1 trial of EDG-15400); timing of the initiation of a pivotal study of sevasemten; statements regarding Edgewise's commercial readiness for a U.S. launch of sevasemten in Becker; statements regarding Edgewise's Phase 3 trial designs in HCM and Duchenne; statements regarding Edgewise's ability to advance its pipeline; statements regarding Edgewise's plans to continue to collect longer-term open label extension data; statements regarding Edgewise's plans to meet with the FDA; statements regarding the potential results of Edgewise's GRAND CANYON trial; 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, EDG-7500 and EDG-15400, and discover, develop and commercialize product candidates in its cardiovascular, cardiometabolic and 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 failure to develop a proprietary drug discovery platform; risks related to exposure to additional risk if we develop sevasemten and potential other programs in connection with other therapies; 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 the regulatory approval processes of domestic and foreign authorities being lengthy, time consuming and inherently unpredictable; risks relating to disruptions at the FDA, the SEC and other government agencies; 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; risks related to future acquisitions or strategic partnerships; risks related to 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. View original content to download multimedia:https://www.prnewswire.com/news-releases/edgewise-therapeutics-reports-third-quarter-2025-financial-results-and-recent-business-highlights-302606313.html

– Announced positive top-line data from the open-label extension MESA trial of sevasemten in participants with Becker muscular dystrophy who previously completed ARCH and CANYON studies – – Announced encouraging top-line data from the Phase 2 LYNX and FOX trials of sevasemten in Duchenne muscular dystrophy – – Advanced the Phase 2 CIRRUS-HCM trial of EDG-7500 in Hypertrophic Cardiomyopathy (HCM) – BOULDER, Colo., Aug. 7, 2025 /PRNewswire/ -- Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the second quarter of 2025 and recent business highlights. "In the first half of 2025, we reached key milestones that bring us closer to delivering on our mission," said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise. "With strong funding in place, we're advancing our skeletal and cardiac muscle programs and building the commercial infrastructure with precision and purpose to support a potential U.S. launch of sevasemten in Becker. We're also actively exploring Phase 3 trial designs in HCM and Duchenne, while continuing to advance our pipeline through bold, innovative R&D." Recent Highlights Muscular Dystrophy Program 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. MESA open-label extension trial in adults with Becker: In June 2025, the Company announced positive data from MESA, an open-label extension trial that provides continued access to sevasemten to participants with Becker who were previously enrolled in ARCH, or completed CANYON, GRAND CANYON, or DUNE. As of the March 2025 data cut, 99% of eligible participants (n=85) are enrolled in MESA, which was designed to collect long-term safety and efficacy data. The MESA data demonstrated sustained disease stabilization in North Star Ambulatory Assessment (NSAA), reinforcing prior ARCH and CANYON findings. Importantly, CANYON participants who rolled over to MESA showed stable NSAA scores over 18 months with a trend toward improvement in placebo participants switching to sevasemten. During the 18 months of sevasemten treatment, participants' NSAA scores continued to diverge relative to the expected functional declines seen in multiple Becker natural history studies. Further, NSAA scores for ARCH participants who rolled over to MESA remained stable after three years of treatment. Sevasemten continues to demonstrate a favorable safety profile after up to three years of treatment. 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 cohort is active in 12 countries, 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. GRAND CANYON is highly powered to be able to show a statistically significant difference in NSAA versus placebo over 18 months and is on track for topline data in the fourth quarter of 2026. In the second quarter of 2025, the Company completed a successful Type C meeting with the FDA, which provided a clear path to registration of sevasemten as the first ever therapy for Becker. To learn more, go to clinicaltrials.gov (NCT05291091). 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 at treating the disease, there remains a high unmet need for additional therapies. LYNX Phase 2 placebo-controlled, dose-finding trial in boys with Duchenne: LYNX is designed to evaluate the effect of sevasemten on safety, biomarkers of muscle damage and function in four- to nine-year-old participants with Duchenne treated with sevasemten in a three-month placebo-controlled dose ranging study, followed by an open-label extension period. In June 2025, the Company announced encouraging observations across functional measures, including Stride Velocity 95th Centile (SV95C), NSAA and 4 stair-climb, while identifying a dose of 10 mg to evaluate in Phase 3. FOX Phase 2 placebo-controlled trial in boys with Duchenne: FOX is designed to evaluate the effect of sevasemten on safety, biomarkers of muscle damage and function in six- to 14-year-old participants with Duchenne who have been previously treated with gene therapy. FOX participants are on average over 10 years old and four years out from receiving gene therapy. Despite the lack of extensive natural history in Duchenne gene therapy treated boys, initial results from the FOX study support that sevasemten 10 mg has the potential to reduce the rate of functional decline. The Company plans to meet with the FDA in the fourth quarter of 2025 to discuss a Phase 3 design including input on the patient population and endpoints, with plans to initiate a pivotal study in 2026. In addition, the Company plans to continue to collect longer-term open label extension data, which will provide further access to the drug to trial participants. 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 (nHCM) 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 over 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 (oHCM). 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 oHCM (Part B) and in participants with nHCM (Part C). Part D of the trial, a 12-week study in both oHCM and nHCM, is actively recruiting participants. The Company expects to provide an update on the CIRRUS-HCM trial in participants with oHCM and nHCM during the fourth quarter of 2025. To learn more about CIRRUS-HCM, visit clinicaltrials.gov, NCT06347159 (Phase 2). Heart failure and preclinical programs: During the third quarter of 2025, the Company expects to begin dosing in a first-in-human Phase 1 trial of EDG-15400, a novel drug candidate intended for the treatment of heart failure. The Company continues to advance its preclinical cardiometabolic programs. 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 Parent Project Muscular Dystrophy annual conference, CureDuchenne Futures conference and the Annual Congress of the Heart Failure Association of the ESC. The Company continues to sponsor and participate in numerous other clinician and patient-focused events. Second Quarter Financial Results Cash, cash equivalents and marketable securities were approximately $594.0 million as of June 30, 2025. Research and development (R&D) expenses were $33.6 million for the second quarter of 2025, compared to $36.8 million for the immediately preceding quarter. The decrease of $3.2 million was primarily driven by a $2.6 million decrease in sevasemten clinical development activities, a $0.9 million decrease in EDG-7500 clinical development activities, a $1.1 million decrease in personnel related costs due to higher stock-based compensation in the prior quarter, and a $0.3 million decrease in professional fees and other research costs, offset by a $1.7 million increase in manufacturing expenses to support clinical development across our programs. The decrease in R&D expense for sevasemten is related to higher costs incurred in the first quarter related to a drug interaction study that was substantially executed in the prior quarter and higher costs related to final enrollment activities of GRAND CANYON. The decrease in R&D expense for EDG-7500 related to the completion of a human pharmacokinetic study which was substantially completed in the prior quarter. General and Administrative (G&A) expenses were $9.1 million for the second quarter of 2025, compared to $9.2 million for the immediately preceding quarter. There were no significant changes between the second quarter of 2025 and the immediately preceding quarter. Net loss and net loss per share for the second quarter of 2025 was $36.1 million or $0.34 per share, compared to $40.8 million or $0.43 per share for the immediately 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, EDG-15400 and its cardiovascular and cardiometabolic programs; statements regarding a potential U.S. launch of sevasemten in Becker; statements regarding the market opportunity for Duchenne and Becker; statements regarding Edgewise's expectations relating to its clinical trials, including timing of reporting data (including an update on the CIRRUS-HCM trial and the presentation of data from the GRAND CANYON trial); timing of beginning dosing in a Phase 1 trial of EDG-15400; timing of the initiation of a pivotal study of sevasemten; statements regarding Edgewise's commercial readiness for a U.S. launch of sevasemten in Becker; statements regarding Edgewise's Phase 3 trial designs in HCM and Duchenne; statements regarding Edgewise's ability to advance its pipeline; statements regarding Edgewise's plans to continue to collect longer-term open label extension data; statements regarding Edgewise's plans to meet with the U.S. FDA; statements regarding the potential results of Edgewise's GRAND CANYON trial; statements regarding Edgewise's ability to register sevasemten as the first ever therapy for Becker; 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, EDG-7500 and EDG-15400, and discover, develop and commercialize product candidates in its cardiovascular, cardiometabolic and 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 failure to develop a proprietary drug discovery platform; risks related to exposure to additional risk if we develop sevasemten and potential other programs in connection with other therapies; 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 the regulatory approval processes of domestic and foreign authorities being lengthy, time consuming and inherently unpredictable; 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; risks related to future acquisitions or strategic partnerships; risks related to 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. View original content to download multimedia:https://www.prnewswire.com/news-releases/edgewise-therapeutics-reports-second-quarter-2025-financial-results-and-recent-business-highlights-302523640.html SOURCE Edgewise Therapeutics

– New open label data in Becker demonstrated sustained disease stabilization up to three years, reinforcing prior clinical findings – – Ongoing pivotal trial and FDA Type C meeting provide clear path to potential sevasemten registration as the first ever therapy for Becker – – Encouraging Phase 2 observations in Duchenne define the dose and inform design for Phase 3 – – Edgewise leadership to discuss these updates on Thursday, June 26 at 8:30 a.m. Eastern Time at a virtual investor event – BOULDER, Colo., June 26, 2025 /PRNewswire/ -- Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today unveiled positive results in its sevasemten program for Becker and Duchenne muscular dystrophies. The Company announced positive data from MESA, an open label extension trial that is providing continued access to sevasemten to participants with Becker who were previously enrolled in ARCH, or completed CANYON, GRAND CANYON, or DUNE. As of the March 2025 data cut, 99% of eligible participants (n=85) are enrolled in MESA. The MESA data demonstrated sustained disease stabilization, reinforcing prior ARCH and CANYON findings. Importantly, CANYON participants who rolled over to MESA showed increased North Star Ambulatory Assessment (NSAA) scores over 18 months (0.8 point improvement from baseline), with a trend toward improvement in placebo participants switching to sevasemten (0.2 point improvement since initiation of sevasemten). During the 18 months of sevasemten treatment, participants' NSAA scores continued to diverge relative to the expected functional declines seen in multiple Becker natural history studies. Further, NSAA scores for ARCH participants who rolled over to MESA remained stable after three years of treatment. Sevasemten continues to demonstrate a favorable safety profile after up to three years of treatment. "We are thrilled with the tremendous excitement from physicians and the patient community around the data on sevasemten to date and their unwavering commitment to our ongoing pivotal program," said Joanne Donovan, Ph.D., M.D., Chief Medical Officer. "We are well positioned to deliver the first ever therapy for individuals with Becker muscular dystrophy." Edgewise recently completed a successful Type C meeting with the U.S. Food and Drug Administration (FDA), which provided a clear path to registration of sevasemten as the first ever therapy for Becker. While the FDA deemed the CANYON data alone insufficient for an accelerated approval, the Agency reiterated that NSAA is a clinically meaningful endpoint for traditional approval. The FDA encouraged Edgewise to continue to share MESA data and natural history prospective modeling ahead of GRAND CANYON completion. Further, the FDA emphasized their support for GRAND CANYON, the ongoing global pivotal placebo-controlled cohort, and its potential as a single adequate well-controlled study to support registration. GRAND CANYON is highly powered to show a statistically significant difference in NSAA versus placebo over 18 months and is on track for topline data in the fourth quarter of 2026. The Company also announced encouraging topline data from its Phase 2 Duchenne trials, LYNX and FOX. The goals were to explore a range of doses to assess safety and identify a potentially beneficial dose for Phase 3. The trial's dose escalation paradigm provided a three-month placebo-controlled period to evaluate biomarkers for dose selection, followed by an open label period. Across both studies, at target doses, sevasemten was well-tolerated. LYNX is an ongoing multi-center, placebo-controlled, dose-finding Phase 2 trial to evaluate the effect of sevasemten on safety, biomarkers of muscle damage and function in four- to nine-year-old participants with Duchenne treated with sevasemten in a three-month placebo-controlled dose ranging study. Consistent observations across functional measures, including Stride Velocity 95th Centile (SV95C), NSAA and 4 stair-climb, identified a dose of 10 mg to evaluate in Phase 3. Similar to the LYNX design, FOX is an ongoing multi-center, placebo-controlled Phase 2 trial to evaluate the effect of sevasemten on safety, biomarkers of muscle damage and function in six- to 14-year-old participants with Duchenne who have been previously treated with gene therapy. FOX participants are on average over 10 years old and four years out from receiving gene therapy. Despite the lack of extensive natural history in Duchenne gene therapy treated boys, initial results from the FOX study indicate that sevasemten 10 mg has the potential to reduce the rate of functional decline. "We have executed an important Phase 2 exploration of sevasemten in Duchenne," said Kevin Koch, Ph.D., President and Chief Executive Officer. "We are encouraged by the functional response seen at the 10 mg dose and look forward to our discussions with the Agency later this year." The Company plans to meet with the FDA in the fourth quarter of 2025 to discuss a Phase 3 design including input on the patient population and endpoints, with plans to initiate the pivotal study in 2026. In addition, the Company plans to continue to collect longer-term open label extension data, which will provide further access to the drug to trial participants. Virtual Investor Event Members of the Edgewise management team will hold a live webcast on Thursday, June 26, at 8:30 a.m. ET to discuss the sevasemten program. An accompanying slide presentation will also be available. To register for the live webcast and replay, please visit the Edgewise events page. About Sevasemten 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. Sevasemten presents a novel mechanism of action designed to selectively limit the exaggerated muscle damage caused by the absence or loss of functional dystrophin. Its unique mechanism of action provides the potential to establish sevasemten as a foundational therapy in dystrophinopathies, either as a single agent therapy or in combination with available therapies and those in development. Sevasemten has achieved notable regulatory milestones by securing FDA Orphan Drug Designation for the treatment of Becker and Duchenne, Rare Pediatric Disease Designation (RPDD) for the treatment of Duchenne, and Fast Track designations for the treatment of Becker and Duchenne. Further, sevasemten secured the EMA Orphan Drug Designations for the treatment of Becker and Duchenne. About Becker 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. About 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. 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 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. References [1] Bello L, et al. Functional changes in Becker muscular dystrophy: implications for clinical trials in dystrophinopathies. Sci Rep. 2016;6:32439. doi:10.1038/srep32439 [2] van de Velde NM, et al. Selection approach to identify the optimal biomarker using quantitative muscle MRI and functional assessments in Becker muscular dystrophy. Neurology. 2021;97(5):e513-e522. doi: 10.1212/WNL.0000000000012233. [3] De Wel B, et al. Lessons for future clinical trials in adults with Becker muscular dystrophy: disease progression detected by muscle magnetic resonance imaging, clinical and patient-reported outcome measures. Eur J Neurol. 2024:e16282. doi:10.1111/ene.16282. Online ahead of print. [4] Muntoni F, et. al., Neuromuscul Disord., 2022 Apr;32(4):271-283. doi: 10.1016/j.nmd.2022.02.009 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 sevasemten, statements regarding the potential market opportunity for sevasemten, statements regarding Edgewise's expectations and milestones relating to its clinical trials and clinical development of sevasemten, including the timing of topline data for the GRAND CANYON trial and the timing of the initiation of a pivotal study of sevasemten, statements regarding the potential outcome of the GRAND CANYON trial, statements regarding the timing and outcome of Edgewise's discussions with the FDA, statements regarding the clear path to potential sevasemten registration as the first ever therapy for Becker, and statements by Edgewise's President and Chief Executive Officer and Chief Medical 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; Edgewise's ability to develop and commercialize sevasemten 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. View original content to download multimedia:https://www.prnewswire.com/news-releases/edgewise-therapeutics-reports-positive-results-on-sevasemten-program-for-becker-and-duchenne-muscular-dystrophies-302491562.html SOURCE Edgewise Therapeutics