Edgewise Therapeutics Announces Publication of Data Demonstrating the Elevation of Fast but Not Slow Skeletal Muscle Fiber Injury Biomarkers in the Circulation of Patients with Becker and Duchenne Muscular Dystrophy
Edgewise Therapeutics, Inc., (NASDAQ: EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, small molecule therapies for rare muscle disorders, today announced the publication of the first cross-sectional, retrospective study to describe selective elevation of fast but not slow skeletal muscle fiber injury biomarkers in the blood of patients with Becker and Duchenne muscular dystrophy (BMD, DMD) in the journal, Muscle & Nerve.
Human skeletal muscle is composed of fast and slow fibers in roughly equal proportion. Previous studies have demonstrated that DMD patient muscle is more prone to fast fiber injury compared to slow fiber injury. This study extends these findings to examine fiber-type specific biomarkers of muscle injury in patient blood. The results build on previous reports and suggest that slow skeletal muscle fibers do not appear to leak muscle proteins associated with muscle injury and damage in BMD and DMD. Furthermore, the study demonstrated that fast skeletal troponin I (TNNI2) may represent a more sensitive biomarker of muscle injury than creatine kinase (CK), particularly in the setting of BMD or older DMD patients where plasma CK is commonly lower than in young DMD patients. These data further support EDG-5506’s unique mechanism of action aimed at protecting injury-susceptible fast skeletal muscle fibers in BMD and DMD.
“We are pleased to share our findings that elevation of fast skeletal muscle fiber injury biomarkers may be more informative, compared to slow skeletal muscle injury biomarkers, of muscle injury in patients with BMD and DMD,” said Alan Russell, Ph.D., Chief Scientific Officer of Edgewise and senior author of the study. “These data guided the design of our lead clinical candidate, EDG-5506, which selectively protects injury-susceptible fast skeletal muscle fibers for BMD and DMD. Indeed, EDG-5506, in preclinical models, acts as a muscle stabilizer by protecting muscle through limiting excessive contraction. This protection reduces injury and the leak of muscle proteins that in the long term protects against fibrosis and functional declines associated with prolonged inflammation and degradation of the muscle.”
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