PresentationWelcome, everyone, to the 44th Annual JPMorgan Healthcare Conference. My name is Tessa Romero, and I'm one of the senior biotech analysts here at JPMorgan. Our next presenting company is Edgewise Therapeutics. And presenting on behalf of the company, we have President and CEO, Kevin Koch. Kevin, over to you.Kevin KochPresident, CEO & Director Okay. Thanks, Tessa. Always great to thanks for joining today. Let me get organized here. These are the forward-looking statements. Edgewise Therapeutics i ...

Forward Looking Statement This presentation contains 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 presentation 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, ...

Company Overview - Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on precision medicines for rare muscle disorders, particularly dystrophinopathies [5][8] - The company’s lead product candidate, EDG-5506, targets Duchenne and Becker muscular dystrophy [8] - As of the latest period, Edgewise has not generated commercial revenue, relying on a research-driven business model [8] Financial Performance - As of September 30, Edgewise Therapeutics had a market capitalization of $2.61 billion and reported a net income loss of $157.24 million over the trailing twelve months [4] - The stock price was $24.62, reflecting a 14% decline over the past year, underperforming the S&P 500, which increased by approximately 15.5% during the same period [3][4] - The company reported a third-quarter net loss of about $40.7 million, attributed to increased R&D spending as it advanced multiple late-stage and mid-stage programs [10] Investment Activity - Foresite Capital Management VI fully exited its position in Edgewise Therapeutics during the third quarter, selling 585,000 shares valued at approximately $7.67 million [1][2] - Prior to the exit, Edgewise represented about 4.3% of Foresite's assets under management [3] - The exit reflects a recalibration of risk-reward dynamics for Foresite, as the remaining portfolio is heavily concentrated in early-stage biotech [11] Market Context - Edgewise's stock experienced significant volatility, with shares dropping as much as 40% from earlier highs following a $200 million underwritten offering announced in April [9] - By the end of the third quarter, the stock remained down year-over-year despite a strong balance sheet with approximately $563 million in cash and marketable securities [10]

Core Insights - Edgewise Therapeutics, Inc. (NASDAQ:EWTX) is recognized as a promising small-cap biotech stock by analysts [1] Financial Performance - In Q3 2025, Edgewise Therapeutics reported a net loss of $40.7 million, equating to $0.39 per share, with R&D expenses increasing by $3.9 million to $37.5 million compared to the same quarter the previous year [3] - General and administrative (G&A) expenses rose by $0.3 million sequentially [3] Research and Development Progress - The company is advancing its muscular dystrophy and cardiac programs, with the GRAND CANYON cohort for sevasemten in Becker muscular dystrophy set for readout in Q4 2026 [2] - The MESA open-label extension trial is seeing continued enrollment of nearly all eligible participants [2] - The Phase 2 CIRRUS-HCM trial of EDG-7500 in hypertrophic cardiomyopathy is progressing well, with an update expected in Q4 2025 [2] - Phase 1 dosing of EDG-15400 for heart failure has commenced [2] Cash Position - Edgewise Therapeutics closed the quarter with cash balances of $563.3 million, providing sufficient runway for ongoing clinical development and potential future commercial launches [4]

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

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

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

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

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

Becker Muscular Dystrophy (BMD) - Sevasemten shows positive observations in Becker patients with continued dosing[12] - A positive Type C FDA meeting offers a clear path to potential approval for Sevasemten[12] - The GRAND CANYON trial is on track for topline data in Q4 2026[12], with >98% power to deliver a statistically significant difference in NSAA vs placebo[20] - In the CANYON study, the NSAA score between the Sevasemten group and the placebo group showed a difference of +1.12 at month 12[27] - In the MESA open-label study, 99% of eligible participants are currently enrolled[30] - Natural history modeling reveals that 92% of CANYON participants improved vs their predicted scores[39], and 89% of ARCH participants achieved higher NSAA scores vs predicted[42] Duchenne Muscular Dystrophy (DMD) - In the LYNX & FOX trials, Sevasemten treatment reduced the functional decline in Duchenne patients[15] - A path to Phase 3 is open with the selection of 10 mg as the target dose[16] - In Duchenne patients, a -82% change in TNNI2 was observed in the 30mg cohort at Month 3[72] - In the FOX study, participants are an average of 11 years old and 4 years out from receiving gene therapy[99] Financial Status - Edgewise has approximately $624 million in cash, cash equivalents, and marketable securities as of March 31, 2025, providing a cash runway through 2028[112][113]