Since our inception, our precision medicine muscle platform has generated several programs to address a variety of muscle disorders. We are developing a pipeline of precision medicine product candidates that target key muscle proteins and modulators to address a broad array of genetically defined muscle disorders.
Duchenne - Duchenne muscular dystrophy
Becker - Becker muscular dystrophy
LGMD - Limb-girdle muscular dystrophy
Spasticity Disorders, such as cerebral palsy
Active Clinical Trials
EDG-5506, our lead product candidate for Duchenne and Becker muscular dystrophy (DMD and BMD), is advancing in clinical development. An initial clinical trial is being conducted in healthy volunteers. We also plan to pursue clinical trials in patients with BMD and DMD depending on the results of the clinical trials and after regulatory discussions with the FDA and EMA.
A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults
For more information about this trial, please contact, firstname.lastname@example.org
Our Focus on Muscular Dystrophies
Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a severe, degenerative muscle disease that affects an estimated one in every 3,500 to 5,000 live male births, with an estimated patient population of approximately 12,000 to 15,000 in the United States and approximately 25,000 in Europe. It is the most common type of muscular dystrophy and is caused by absence of dystrophin, a large protein that connects muscle contraction to the supporting basement membrane around muscle fibers. Loss of ambulation often begins by 13 years of age and nearly all boys with DMD will require the use of a wheelchair by the time they are young teens, with mortality generally in the second or third decade of life.
Becker Muscular Dystrophy
Becker muscular dystrophy (BMD) is a milder form of DMD, mostly affecting boys and has a much lower incidence of approximately 1 in every 18,450 live male births. We estimate there are between 4,000 and 5,000 patients with BMD in the United States, with similar numbers estimated in Europe. It is caused by mutation of dystrophin that results in truncated of the protein. The disease is variable and can range from mild to almost as severe as the symptoms of DMD. Patients usually have weakness of the hips and pelvis and may have problems walking by their late teens. Disability and heart problems are common in these patients.
Limb Girdle Muscular Dystrophies
Edgewise is also developing medicines for additional muscle disorders that include slower-onset forms of inherited muscle myopathies such as Limb Girdle Muscular Dystrophies (LGMD) and movement disorders that include spasticity and contractures resulting from multiple sclerosis, spinal cord injury or stroke. Collectively, LGMDs are the fourth most common genetic cause of muscle weakness. The approximate global prevalence of LGMDs as a group is estimated to be from 0.56 to 5.75 per 100,000. Males and females are affected in equal numbers. Onset may occur in childhood, adolescence, young adulthood, or even later. LGMD does not shorten lifespan; however, problems can arise in individuals who have problems in heart muscles and the respiratory system.