Many serious inherited myopathies are marked by an exaggerated injury response with everyday activities.
Amongst these, muscle injury in the context of Duchenne is best understood. Skeletal muscle contraction leads to membrane stress and muscle fiber breakdown through excessive contractures and programmed cell death. We are designing strategies to stabilize patient skeletal muscle so that muscle use is uncoupled from its excessive injury response. Our approaches do not rely on dystrophin replacement so will be applicable for patients with any dystrophin mutation and in combination with all approved and experimental therapies for this disease.
Current Status
EDG-5506, our lead product candidate for Duchenne and Becker muscular dystrophy (DMD and BMD), is advancing in clinical development. Initial clinical studies are being conducted in healthy volunteers, and with appropriate results, will be followed by studies in patients with DMD and BMD.
Areas of Focus
Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a severe, incurable degenerative muscle disease that affects about one in 5,000 males at birth. 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. Young patients are ambulant but become wheelchair bound in their teen years and have a shortened lifespan, generally to the third decade of life with death from either respiratory or cardiac failure.
Becker Muscular Dystrophy
Becker muscular dystrophy (BMD) is a milder form of DMD, mostly affecting boys. 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 common in adults. These 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.
Edgewise Therapeutics Pipeline
Advancing 3 programs in rare myopathies and neuromuscular disorders
program
indications
research
preclinical
POP+
POP+
phase 1
phase 2/3
EDG-5506
EDG-002
EDG-003
Duchenne Muscular Dystrophy (DMD)
Limb Girdle Muscular Dystrophy (LGMD)
Genetically-defined Neuromuscular
and Metabolic Disorders
and Metabolic Disorders
Genetically-defined Cardiac Indications
Current
Year End 2021
+ Proof of Principle, validated in advanced models of disease
Scientific Presentations & Publications
Active Clinical Trials
A Study to Assess Safety, Tolerability, and PK of EDG-5506 in Healthy Volunteers and Becker Muscular Dystrophy Adults
EDG-5506 is an investigational product intended to protect and improve function of dystrophic muscle fibers. This Phase 1 study of EDG-5506 will assess the safety, tolerability, and pharmacokinetics (PK) and of EDG-5506 in adult healthy volunteers and in adults with Becker muscular dystrophy (BMD).
For more information about this trial, please contact, info@edgewisetx.com