Edgewise Therapeutics Initiates Phase 1 Clinical Trial of EDG-5506 in Becker Muscular Dystrophy
- Category: Small Molecules
- Published on Thursday, 12 November 2020 14:56
- Hits: 1260
- EDG-5506 is a novel, oral, small molecule drug candidate with the potential to promote muscle function in patients with muscular dystrophy -
BOULDER, CO, USA I November 11, 2020 I Edgewise Therapeutics, a clinical-stage biopharmaceutical company developing small molecule therapies for musculoskeletal diseases, today announced the initiation of a Phase 1 clinical trial of EDG-5506. The Phase 1 trial is designed to assess the safety, tolerability and pharmacokinetics (PK) of EDG-5506 in adult healthy volunteers and in adults with Becker muscular dystrophy (BMD). The Phase 1 study is being conducted by investigators at Worldwide Clinical Trials in San Antonio, Texas.
“Initiation of our Phase 1 study of EDG-5506 is an important milestone for our company,” said Kevin Koch, Ph.D., President and Chief Executive Officer, Edgewise Therapeutics. “I’m excited about the potential of EDG-5506 for patients affected by BMD as well as Duchenne muscular dystrophy (DMD), who are currently underserved with limited treatment options. We look forward to the results of our Phase 1 trial, which will help us select the optimal dose of EDG-5506 for pivotal clinical studies in these patient populations.”
EDG-5506 was designed to arrest muscle fiber break down, the hallmark of ongoing disease in muscular dystrophy (MD) patients, thus limiting the subsequent inflammatory and fibrotic response while promoting muscle integrity and physical function. This approach has the potential to benefit a broad population of MD patients and represents a new strategy for treating this devastating disease.
About EDG-5506 Phase 1 Clinical Trial
The Phase 1 trial is a randomized, placebo-controlled, double-blind, Single and Multiple Ascending Dose (SAD and MAD) study evaluating the safety, tolerability, PK and pharmacodynamics of EDG-5506 in adult healthy volunteers and adults with BMD. Participants enrolled in this study will receive a single oral dose or multiple oral doses of EDG-5506 or a placebo. The study will enroll adult patients with BMD following appropriate safety, tolerability and PK results of EDG-5506 in healthy volunteers. To learn more about this study (NCT04585464), go to clinicaltrials.gov.
About Muscular Dystrophy
Muscular dystrophies are a group of genetic disorders associated with defects in the critical muscle-associated structural protein dystrophin or the sarcomere complex and are characterized by progressive muscle degeneration and weakness. In individuals with neuromuscular conditions such as Duchenne muscular dystrophy, normal muscle use leads to continued rounds of muscle breakdown that the body struggles to repair. Overtime fibrosis and fatty tissue accumulate in the muscle portending a steep decline and permanent loss of physical function that ends with mortality. There remains an unmet need for treatments that reduce muscle breakdown in patients with neuromuscular conditions. Arresting this amplified muscle response will have a dramatic effect on disease progression.
About Becker Muscular Dystrophy
Becker muscular dystrophy is a rare, genetic, disease that causes progressive muscle degeneration with loss of skeletal and cardiac function. The disease mainly affects boys with symptoms usually beginning in childhood. It is estimated to affect one in 17,000 male births globally. Becker muscular dystrophy is caused by mutations in the gene responsible for the production of dystrophin, a key protein structural component in muscle fibers. As patients do not have enough working dystrophin, muscle fibers gradually break down with everyday activities, leading to permanent muscle weakness. Becker muscular dystrophy causes long-term disability and is life-threatening because of its effects on the heart. No therapies are currently approved for Becker muscular dystrophy in the United States or the European Union.
About Edgewise Therapeutics
Edgewise Therapeutics, founded in 2017 by Alan Russell, Ph.D.; Peter Thompson, M.D. (Orbimed Advisors); and Badreddin Edris, Ph.D., (Springworks Inc.), is dedicated to the development of first-in-class medicines for the treatment of high morbidity musculoskeletal diseases. Skeletal muscle is the largest organ system in the human body, regulating both force production to enable muscle contraction, locomotion, and postural maintenance and the metabolism of glucose, and fatty and amino acids. By modulating these processes in skeletal muscle, Edgewise Therapeutics is aiming to create therapeutic agents that will reduce muscle damage, normalize muscle function, decrease mortality and profoundly benefit patients' quality of life. To learn more, go to: www.edgewisetx.com or follow us on LinkedIn.
SOURCE: Edgewise Therapeutics