New 2-Month Interim Data from the ARCH Open Label Study of EDG-5506 in Individuals with Becker Muscular Dystrophy (BMD) to be Presented at the Upcoming New Directions in Biology and Disease of Skeletal Muscle Conference

Rhea-AI Summary

Edgewise Therapeutics (NASDAQ: EWTX) is set to present new interim data from the ARCH study on EDG-5506, targeting Becker Muscular Dystrophy (BMD), at the 2022 New Directions Conference from June 20-23, 2022. A conference call discussing this data will occur on June 21 at 9 a.m. ET. Additionally, further presentations will be showcased at the 2022 PPMD Annual Conference on June 23-26. EDG-5506 has received Fast Track designation from the FDA, emphasizing its potential in treating severe neuromuscular disorders.

Positive

• EDG-5506 has received Fast Track designation from the FDA for BMD treatment.
• Upcoming presentations at notable conferences enhance visibility and credibility.
• New interim data from the ARCH study may boost investor confidence.

Negative

• None.

- Edgewise management to host webcast conference call on Tuesday, June 21, 2022 at 9 a.m. Eastern Time -

- Additional presentations and posters featuring EDG-5506 to be featured at the New Directions Conference and the upcoming PPMD Annual Conference -

BOULDER, Colo.--(BUSINESS WIRE)-- Edgewise Therapeutics, Inc., (NASDAQ: EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for rare muscle disorders, today announced that new 2-month interim data from the ARCH open label study of EDG-5506 in individuals with BMD will be presented at the 2022 New Directions in Biology and Disease of Skeletal Muscle Conference, being held June 20-23, 2022.

Edgewise ARCH Interim Data Review Conference Call

Members of the Edgewise management team will hold a conference call and webcast on Tuesday, June 21, at 9:00 am ET to discuss the ARCH interim data. To participate, please dial 844-200-6205 (domestic) or 646-904-5544 (international) and refer to access code 260476. Visit the Edgewise events page to access the webcast, including replay and conference call slides.

The Company will have additional presentations and posters featuring EDG-5506 at the New Directions Conference and the 2022 PPMD Annual Conference, being held June 23-26, 2022. The presentations and posters will be available on the Edgewise website after they are presented.

2022 New Directions in Biology and Disease of Skeletal Muscle Conference
Industry Workshop
Presentation: Taking a New Approach to Protect Muscle in Duchenne and Becker Muscular Dystrophy: EDG-5506
Date: Monday, June 20 at 12:10 p.m. ET
Presenter: Joanne Donovan, M.D., Ph.D., Chief Medical Officer, Edgewise Therapeutics

Scientific Posters
The scientific posters listed below will be presented during Poster Session I on Wednesday, June 22, from 2 – 3:15 p.m. ET, and during Poster Session II on Thursday, June 23, from 7:30 – 9:45 a.m. ET.

Title: Skeletal Myosin Inhibitor EDG-5506 Protects Dystrophic Muscle from Contraction-Induced Injury
Title: Use of an Exercise Challenge System to Define a Universal Proteomic Signature of Muscle Injury in a Diverse Set of Adult Individuals with Inherited Myopathy
Title: Short-Term Proteomic Responses to the Fast Skeletal Myosin Inhibitor EDG-5506 in Becker Muscular Dystrophy
Title: EDG-5506 is a Selective Inhibitor of Fast Skeletal Muscle Myosin

2022 PPMD Annual Conference
Session: Emerging Therapies for Becker Muscular Dystrophy
Presentation: A Novel Approach to Protect Muscle in Becker Muscular Dystrophy: EDG-5506
Date: Friday, June 24 at 4:45 p.m. MST
Presenter: Joanne Donovan, M.D., Ph.D., Chief Medical Officer, Edgewise Therapeutics

Session: Novel Approaches for Combatting Duchenne
Presentation: A Novel Approach to Protect Muscle in Duchenne Muscular Dystrophy: EDG-5506
Date: Saturday, June 25 at 11 a.m. MST
Presenter: Joanne Donovan, M.D., Ph.D., Chief Medical Officer, Edgewise Therapeutics

Scientific Posters
The scientific posters listed below will be presented on Thursday, June 23, from 6:15 – 9 p.m. MST.

Title: EDG-5506: A Novel Approach to Protect Muscle in Duchenne and Becker Muscular Dystrophy
Title: Insights into the Becker Muscular Dystrophy Experience from The Duchenne Registry a Comprehensive Registry for Duchenne and Becker Muscular Dystrophy (DMD and BMD)
Title: Understanding the Becker Muscular Dystrophy (BMD) Experience from the Patient Perspective: It’s More Than Mild Duchenne

About the ARCH Open Label Study

The ARCH open label study is evaluating EDG-5506 in 12 adult males with BMD. All those who participated the Phase 1b first-in-human study of EDG-5506 enrolled in the study. The study is evaluating multiple doses of EDG-5506 administered daily over 12 months. Safety, pharmacokinetics (PK), changes in biomarkers of muscle damage such as creatine kinase (CK) and fast skeletal muscle troponin I (TNNI2), measures of function with North Star Ambulatory Assessment / North Star Assessment for Limb Girdle Type Muscular Dystrophies (NSAA/NSAD), time function tests and patient-reported outcomes, will be evaluated. Go to clinicaltrials.gov to learn more about this study (NCT05160415).

About EDG-5506 for DMD and BMD

EDG-5506 is an orally administered small molecule designed to address muscle damage induced by mechanical stress in dystrophinopathies including DMD and BMD. EDG-5506 presents a novel mechanism of action to selectively limit the exaggerated muscle damage caused by the absence of functional dystrophin. EDG-5506 has the potential to benefit a broad range of patients suffering from debilitating rare neuromuscular disorders. It is anticipated to be used as a single agent therapy, but it may also provide a synergistic or additive effect in combination with available therapies and therapies currently in development. In August 2021, the U.S. Food and Drug Administration granted Fast Track designation to EDG-5506 for the treatment of individuals with BMD.

The Company has completed a Phase 1 clinical trial of EDG-5506 designed to evaluate safety, tolerability, PK and PD of EDG-5506 in adult healthy volunteers (Phase 1a) and in adults with BMD (Phase 1b). Go to clinicaltrials.gov to learn more about this clinical trial (NCT04585464). A follow-on open-label, single-center study is assessing the long-term safety and PK of EDG-5506 in adults with BMD (NCT05160415). We anticipate initiation of Phase 2 trials in individuals with BMD in the coming weeks and DMD in the second half of 2022.

About Edgewise Therapeutics

Edgewise Therapeutics is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of innovative treatments for severe, rare neuromuscular and cardiac disorders for which there is significant unmet medical need. Guided by its holistic drug discovery approach to targeting the muscle as an organ, Edgewise has combined its foundational expertise in muscle biology and small molecule engineering to build its proprietary, muscle-focused drug discovery platform. Edgewise’s platform utilizes custom-built high throughput and translatable systems that measure integrated muscle function in whole organ extracts to identify small molecule precision medicines regulating key proteins in muscle tissue. To learn more, go to: www.edgewisetx.com or follow us on LinkedIn, Twitter and Facebook.

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 drug discovery platform, product candidates and programs, including EDG-5506; statements regarding enrollment of individuals in the BMD natural history trial; statements regarding Edgewise’s expectations relating to the results from its preclinical studies, its clinical trials and the BMD natural history trial; statements about the expected timing of Edgewise’s initiation of its preclinical studies and clinical trials including its Phase 2 clinical trials of EDG-5506 in BMD and DMD; and statements regarding Edgewise’s pipeline of product candidates and programs. 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 for muscular dystrophy patients or other patient populations; the timing, progress and results of preclinical studies and clinical trials for EDG-5506 ; Edgewise’s ability to raise any additional funding it will need to continue to pursue its business and product development plans; negative impacts of the COVID-19 pandemic on Edgewise’s operations, including preclinical and clinical trials; 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 ability to enroll and maintain patients in its preclinical studies and its clinical trials; the enrollment of individuals in the BMD natural history trial; 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 Securities and Exchange Commission (the “SEC”). 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.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220616005291/en/

Investors & Media

Michael Carruthers

Chief Financial Officer

ir@edgewisetx.com

Source: Edgewise Therapeutics

FAQ

When is the Edgewise Therapeutics conference call about EDG-5506?

The conference call will be held on June 21, 2022, at 9 a.m. ET.

What data will Edgewise present at the 2022 New Directions Conference?

Edgewise will present new 2-month interim data from the ARCH study of EDG-5506 for Becker Muscular Dystrophy.

What is EDG-5506's significance in treating muscle disorders?

EDG-5506 is designed to address muscle damage in dystrophinopathies such as Duchenne and Becker Muscular Dystrophy.

What conferences is Edgewise participating in for EDG-5506 presentations?

Edgewise will participate in the 2022 New Directions Conference and the 2022 PPMD Annual Conference.

What is the purpose of the ARCH open-label study for EDG-5506?

The ARCH study evaluates the safety, pharmacokinetics, and muscle damage biomarkers associated with EDG-5506 in patients with Becker Muscular Dystrophy.