Edgewise Therapeutics Begins Dosing First-in-Human Phase 1 Trial of EDG-7500, its Lead Clinical Candidate for Hypertrophic Cardiomyopathy (HCM) and Other Serious Diseases of Cardiac Diastolic Dysfunction

– EDG-7500 is a first-in-class oral, selective, cardiac sarcomere modulator that has the potential to benefit a broad spectrum of HCM and other cardiomyopathy patients –

BOULDER, Colo., September 14, 2023--(BUSINESS WIRE)--Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced initial dosing in a Phase 1 trial of EDG-7500. EDG-7500 is a first-in-class 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. The Phase 1 trial will assess the safety, tolerability, pharmacokinetics and pharmacodynamics of EDG-7500 in healthy adults. The Company is also planning to begin a Phase 1b study of EDG-7500 in individuals with obstructive HCM in the first half of 2024.

"Initiation of our Phase 1 study of EDG-7500, our first cardiovascular therapeutic candidate, is an important milestone for our company," said Marc Semigran, M.D., Chief Development Officer, Edgewise Therapeutics. "I have spent over 30 years working to help patients with serious heart disease and am excited about the potential of EDG-7500 to help individuals affected by HCM, where significant unmet needs remain."

Kevin Koch, Ph.D., President and Chief Executive Officer, Edgewise Therapeutics added, "We are pleased to add EDG-7500 to our growing clinical pipeline demonstrating our commitment to developing precision therapies for patients with severe genetic muscle diseases. EDG-7500 is representative of the robust discovery and development capabilities at Edgewise, and our commitment to patients with serious skeletal and cardiac muscle diseases. As we mark this achievement, we look forward to initiating trials next year in HCM, and broadly in disorders of diastolic dysfunction."

About EDG-7500 Phase 1 Clinical Trial

The Phase 1 trial is a randomized, double-blind, placebo-controlled, single and multiple ascending dose study evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of EDG-7500 in healthy adults. Participants enrolled in this study will receive a single oral dose or multiple oral doses of EDG-7500 or a placebo. To learn more about this study (NCT06011317), go to clinicaltrials.gov.

About Hypertrophic Cardiomyopathy

HCM is the most common form of genetic heart disease with an estimated prevalence of as many as 700,000 people in the United States. The molecular underpinnings of HCM lead to abnormal cardiac sarcomeric protein function that eventually results in excessive contraction of the heart muscle, referred to as hypercontractility. Over time, hypercontractility increases the thickness (hypertrophy) of the heart’s left ventricular (LV) walls – the heart’s primary pumping chamber. Progressive hypertrophy eventually leads to a thick and stiff LV thereby reducing its ability to fill with blood during relaxation (diastole) and pump blood during contraction (systole). A decreased volume of blood entering and leaving the LV causes shortness of breath, rapid heartbeat, chest pain, unexplained fatigue, dizziness, and syncope (loss of consciousness), which together cause HCM patients to become limited in their ability to perform activities of daily living. HCM can be diagnosed at any age; disease progression increases the risk of heart failure, stroke and atrial fibrillation. Despite advancements in the development of therapies to treat HCM, there is currently a significant unmet need for additional therapies to safely and more effectively treat the disease.

About EDG-7500 for HCM

EDG-7500 is a first-in-class oral, selective, cardiac sarcomere modulator that is advancing in a Phase 1 trial. The compound is designed to slow early contraction velocity and improve impaired cardiac relaxation. This novel mechanism is anticipated to have a broad therapeutic index that may facilitate fixed patient dosing and minimize monitoring during therapy. Preclinical data of EDG-7500 support beneficial activity in animal models of both obstructive HCM and cardiac diastolic dysfunction with minimal changes in left ventricular systolic performance. The Company plans to present preclinical data of EDG-7500 at a major cardiovascular meeting later this year.

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 first-in-class therapeutics. EDG-5506 is an orally administered skeletal myosin inhibitor in advanced clinical trials in patients with Becker, Duchenne, and Limb-Girdle muscular dystrophies as well as McArdle Disease. EDG-7500, currently in a Phase 1 trial, is a novel cardiac sarcomere modulator for the treatment of HCM and other disorders of cardiac diastolic dysfunction. 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 (formerly Twitter), Facebook, Instagram and Threads.

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 EDG-7500; statements regarding Edgewise’s expectations relating to its preclinical studies and clinical trials, including the commencement of trials (including the Phase 1b study of EDG-7500 in individuals with obstructive HCM); and statements by Edgewise’s president and chief executive officer and chief development 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 the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and operating as an early clinical stage company including the potential for Edgewise’s product candidates to cause serious adverse events; Edgewise’s ability to develop, initiate or complete preclinical studies and clinical trials for, obtain approvals for and commercialize any of its product candidates; the timing, progress and results of preclinical studies and clinical trials for EDG-7500; Edgewise’s ability to raise any additional funding it will need to continue to pursue its business and product development plans; the timing, scope and likelihood of regulatory filings and approvals; the potential for any clinical trial results to differ from preclinical, interim, preliminary, topline or expected results; Edgewise’s ability to develop a proprietary drug discovery platform to build a pipeline of product candidates; Edgewise’s manufacturing, commercialization and marketing capabilities and strategy; the size of the market opportunity for Edgewise’s product candidates; the loss of key scientific or management personnel; competition in the industry in which Edgewise operates; Edgewise’s reliance on third parties; Edgewise’s ability to obtain and maintain intellectual property protection for its product candidates; 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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