Edgewise Receives European Medicines Agency (EMA) Orphan Drug Designations for Sevasemten (EDG-5506) for the Treatment of Becker and Duchenne Muscular Dystrophies

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designations for sevasemten for the treatment of Becker muscular dystrophy (Becker) and for the treatment of Duchenne muscular dystrophy (Duchenne). Sevasemten is an investigational orally administered small molecule designed to prevent contraction-induced muscle damage. Sevasemten is currently in late-stage clinical trials for individuals with Becker and is also being studied in Duchenne.

“Receiving Orphan Drug designation in the U.S. and now in Europe highlights alignment across regions on the urgent and critical need for new and better therapeutic options for people living with Becker or Duchenne,” said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise. “We are focused on advancing these muscle-targeted therapeutics as quickly as possible through clinical development for these communities.”

The EMA grants orphan drug designation to drugs and biologics intended for the treatment, diagnosis or prevention of rare, life-threatening or chronically debilitating diseases or conditions that affect no more than five in 10,000 people in the European Union, among other criteria. Orphan designation allows companies certain benefits, including reduced regulatory fees, clinical protocol assistance, access to centralized authorization procedure and up to 10 years of market exclusivity in the European Union, if approved.

Sevasemten has previously achieved notable milestones by securing U.S. Food and Drug Administration (FDA) Orphan Drug Designation for the treatment of Becker and Duchenne, Rare Pediatric Disease Designation (RPDD) for the treatment of Duchenne, and Fast Track designations for the treatment of Becker and Duchenne.

About Becker Muscular Dystrophy

Becker is a rare, genetic, life-shortening, debilitating and degenerative neuromuscular disorder. The disease predominantly affects males and imposes significant physical, emotional, financial, and social impacts on the individual and their caregivers. Individuals with Becker experience contraction-induced muscle damage, which is the primary driver of muscle loss and impaired motor function in muscular dystrophies. Functional decline can begin at any age, and once that muscle loss occurs, the decline in function is irreversible and continues throughout the individual’s life. Some individuals living with Becker experience heart failure from cardiomyopathy, which may result in heart transplantation or early death. Currently, there is no cure for Becker; early and long-term multidisciplinary care is critical for optimized disease management. There is a great need for more Becker-specific scientific research, clinical programs, and treatment guidelines to improve management of this disease. To learn more about Becker, go to https://beckermusculardystrophy.com/

About Duchenne Muscular Dystrophy

Duchenne is a severe, degenerative muscle disorder with a median life expectancy of around 30 years old. People living with Duchenne begin to lose their ability to walk without assistance by their early teens and nearly all will require the use of a wheelchair by the time they are in their mid-teens. Duchenne is the most common type of muscular dystrophy, and genetic mutations in the dystrophin gene result in contraction-induced muscle damage, which is the primary driver of irreversible muscle loss and impaired motor function. Currently, there is no cure for Duchenne; early, active multidisciplinary care from neuromuscular specialists, cardiologists, physical therapists, and other specialists is critical for optimized disease management. Current therapeutic options for Duchenne are inadequate to prevent significant morbidity and mortality; novel therapies in development for Duchenne, including muscle targeted interventions, aim to positively impact disease trajectory.

About Sevasemten (EDG-5506) 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. Its unique mechanism of action provides the potential to establish sevasemten as a foundational therapy in dystrophinopathies, either as a single agent therapy or in combination with available therapies and those in development.

In Becker, Edgewise is advancing sevasemten in a Phase 2 trial, called CANYON, evaluating safety and effects on function and biomarkers of muscle damage in adult males with Becker. The CANYON trial, which is fully enrolled, has been expanded to include an additional 120 adult participants in a pivotal cohort called GRAND CANYON. This study is currently enrolling at sites in the United States and Europe.

In Duchenne, Edgewise is advancing its LYNX Phase 2 dose-finding clinical trial, assessing safety, PK, biomarkers of muscle damage and functional measures in boys with Duchenne. It is also advancing a second Phase 2 trial, called FOX, assessing safety, PK, biomarkers of muscle damage and functional measures in children and adolescents previously treated with gene therapy.

For more information on Edgewise’s clinical trials https://edgewisetx.com/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 first-in-class 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 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 (formerly Twitter), Facebook, Instagram and Threads.

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