Edgewise Therapeutics Announces Initiation of LYNX Phase 2 Clinical Trial of EDG-5506 for the Treatment of Duchenne Muscular Dystrophy (DMD)

– Rare Disease Research, LLC first site activated to enroll children with DMD –

– Edgewise to hold virtual webinars with the patient community to discuss EDG-5506 clinical trials in November –

Edgewise Therapeutics, Inc., (NASDAQ: EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for the treatment of rare muscle disorders, today announced that it has initiated the LYNX Phase 2 clinical trial at Rare Disease Research, LLC in Atlanta, Georgia. The trial will examine the safety, pharmacokinetics (PK) and effect on biomarkers of muscle damage of EDG-5506 in children with DMD. The study will also explore changes in functional measures, such as the North Star Ambulatory Assessment (NSAA) and self-reported/caregiver-reported outcomes. EDG-5506 is an investigational orally administered small molecule myosin modulator designed to protect injury-susceptible fast skeletal muscle fibers in dystrophinopathies such as DMD and Becker muscular dystrophy (BMD).

The LYNX Phase 2 trial is a placebo-controlled trial to assess the effect of three doses of EDG-5506 over 12 weeks on safety, PK and biomarkers of muscle damage. After the initial 12 weeks, LYNX participants will then continue on open-label EDG-5506 for an additional 9 months to gain further insights into safety and functional measures. Approximately 27 children with DMD aged 4 to 9 years on stable corticosteroids are expected to be enrolled in this 12-month trial. Importantly, this trial is designed to identify the doses of EDG-5506 that have the potential to reduce biomarkers of muscle damage and provide functional benefit to patients in a Phase 3 trial. The research center in Atlanta is the first site to be activated to enroll children with DMD. Up to 12 investigative sites across the United States are expected to participate in the trial.

“We are very pleased to begin this important clinical trial of EDG-5506,” said Han C. Phan, M.D., Head of Research & Principal Investigator, Pediatric Neurologist, Rare Disease Research, LLC. “Having studied EDG-5506 over the past year in individuals with Becker with promising results, I’m excited to see the potential of EDG-5506 in children with Duchenne.”

“Our team has worked with experts and patient advocacy groups to thoughtfully design our LYNX Phase 2 clinical trial,” said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Edgewise. “We are thrilled to expand our study of EDG-5506 into children with Duchenne, which is supported by the promising safety profile and positive trends that we have seen to date in studies of EDG-5506 in individuals with Becker.”

Edgewise Community Webinars

Edgewise is working with CureDuchenne and Jett Foundation to host free virtual community webinars where Edgewise leadership will discuss and answer questions about the clinical trials of EDG-5506 in DMD and BMD. Registration is required for the events and replays of the webinars will be made available after the events.

Community Webinar with CureDuchenne:

Wednesday, November 2, 2022 at 9 am PT

Register here on the CureDuchenne website.

Community Webinar with Jett Foundation:

Thursday, November 17, 2022 at 11 am ET

Register here on the Jett Foundation website.

Edgewise recently held a community webinar with Parent Project Muscular Dystrophy; a replay of the webinar is available here.

About Duchenne Muscular Dystrophy

DMD is a severe, degenerative genetic disorder characterized by progressive impairment of muscle function. DMD affects an estimated one in every 3,500–5,000 male births, with an estimated 12,000–15,000 patients in the United States and approximately 25,000 patients in Europe. DMD, the most common type of muscular dystrophy, is caused by the absence of dystrophin, a protein that protects muscle from contraction-induced damage. Nearly all boys with DMD require the use of a wheelchair by the time they are young teens. Median life expectancy for a patient with DMD is around 30 years old. There is no cure for DMD and currently limited options are available for treatment of DMD.

About EDG-5506

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 designed to selectively limit the exaggerated muscle damage caused by the absence or loss of functional dystrophin. By impacting the progressive muscle damage that leads to functional impairment, 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 (FDA) 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 pharmacodynamics of EDG-5506 in adult healthy volunteers (Phase 1a) and in adults with BMD (Phase 1b) (NCT04585464). In ARCH, a follow-on open-label, single-center trial (NCT05160415) assessing long-term safety and PK, decreases in biomarkers of muscle damage and improvements in NSAA have been observed. CANYON, a Phase 2 trial (NCT05291091), is assessing safety, PK, biomarkers and functional measures in participants with BMD. LYNX, a Phase 2 trial (NCT05540860), will assess the safety, PK and biomarkers of muscle damage in participants with DMD.

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 the LYNX Phase 2 clinical trial, including with respect to trial design, patient enrollment and investigative sites; and statements by Edgewise’s 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 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 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 clinical trials; 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/20221027006078/en/