BOULDER, Colo. / Oct 01, 2024 / Business Wire / Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced its participation at the 29th International Annual Congress of the World Muscle Society (WMS) with an industry-sponsored symposium and the presentation of seven scientific posters. These presentations will highlight the effects of sevasemten in individuals with Becker based on findings from the DUNE and ARCH clinical trials, as well as provide perspectives on biomarker and functional endpoints being studied in the CANYON Phase 2 trial. Sevasemten is an orally administered small molecule designed to prevent contraction-induced muscle damage in muscular dystrophies including Becker and Duchenne muscular dystrophy. The conference will take place at The Prague Congress Centre in Prague, Czechia, October 8-12, 2024.

Details of the Edgewise symposium and scientific posters at WMS:

Edgewise Symposium with Key Opinion Leaders

Date: Tuesday, October 8, 2024, at 4:30 pm CEST

Title: Understanding disease progression and a potential novel agent to protect muscle

Presenters: Luca Bello, M.D., Ph.D, Associate Professor, Department of Neurosciences, DNS, University of Padova and Craig McDonald, M.D., Professor and Chair, Department of Physical Medicine & Rehabilitation, Professor of Pediatrics, Director MDA Neuromuscular Disease Clinics, University of California Davis.

The symposium will also include discussion of the positive two-year topline results from the ARCH trial, an open label, single-center study assessing safety, tolerability, impact on muscle damage biomarkers, pharmacokinetics (PK) and functional measures with sevasemten in adults with Becker. Only registered conference attendees can register for the symposium.

Scientific Posters

Title: Sevasemten, a fast myosin inhibitor, in adults with Becker muscular dystrophy results in reduced muscle damage biomarkers and functional stabilization (351P)

Title: Rasch evaluation of North Star Ambulatory Assessment and North Star Assessment for Limb-Girdle Type Muscular Dystrophies in Becker muscular dystrophy (322P)

Title: Exploring the content validity of patient-reported outcome (PRO) measures to capture the patient experience of Becker Muscular Dystrophy (BMD) (339P)

Title: Comparison of short- and long-term proteomic response to the fast skeletal myosin inhibitor, sevasemten (EDG-5506), in Becker muscular dystrophy (BMD) (349P)

Title: Protein biomarkers of muscle injury exhibit differential reduction with subject age in adults with Becker muscular dystrophy (729LBP)

Title: Post-exercise biomarkers of muscle injury are reduced by sevasemten, a fast myosin inhibitor, in adults with Becker muscular dystrophy (732LBP)

Title: Clinical trial readiness in rare and underserved disease: learnings from community engagement and the lived experience in Becker muscular dystrophy (Becker) (708LPB)

All Edgewise posters are being presented on Friday, October 11, 2024, in Poster Session 4 (Forum Hall) from 3:45 to 4:45 pm CEST. The full WMS Congress program is available here.

The Edgewise symposium presentation and posters will be available on the Edgewise website.

About Sevasemten for Becker and Duchenne Muscular Dystrophies

Sevasemten is an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies 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. By minimizing the progressive muscle damage that leads to functional impairment, sevasemten has the potential to benefit a broad range of patients suffering from debilitating neuromuscular disorders.

In Becker, after completion of the two-year Phase 1 ARCH trial, the Company continues to advance sevasemten through the clinic with the Phase 2 CANYON trial, which is fully enrolled, evaluating safety and effects on function and biomarkers of muscle damage in adult males with Becker. The Company expects to report CANYON data in the fourth quarter of 2024. The CANYON trial has expanded to include an additional 120 adult participants in a pivotal cohort GRAND CANYON, which is currently enrolling in the United States, Europe, Israel, Australia and New Zealand. Data from GRAND CANYON, if positive, could support a marketing application.

In Duchenne, the Company is advancing its Phase 2 clinical trials, LYNX, assessing safety, pharmacokinetics and biomarkers of muscle damage, and FOX, which includes children and adolescents previously treated with gene therapy. The Company expects to report LYNX data in the fourth quarter of 2024. The Company will rely on LYNX data, along with data from the FOX trial of Duchenne children previously treated with gene therapy, to guide the design and powering of a Phase 3 trial in Duchenne, planned to be initiated in the first half of 2025.

For more information on Edgewise’s clinical trials https://edgewisetx.com/science-pipeline/clinical-trials/.

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.

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 timing of reporting data (including the data from the CANYON trial and the LYNX trial); statements regarding Edgewise’s expectations relating to its clinical trials (including the CANYON trial, GRAND CANYON trial, LYNX trial and FOX trial); statements regarding the data from GRAND CANYON being able to support a marketing application; and statements regarding the commencement of trials (including the Phase 3 trial of sevasemten in Duchenne). 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 its 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 being lengthy, time consuming and inherently unpredictable; 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.

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