No therapies are currently approved for the treatment of DMD. Although corticosteroids have been shown to improve walking capacity and preserve respiratory function, their benefits are temporary and are often accompanied by undesirable side effects including weight gain and progressive bone loss.
There is a tremendous unmet need for therapies that preserve or improve muscle quality, strength and function for boys with DMD. In a mouse model of DMD, treatment with a mouse form of ACE-031 increased muscle mass and strength, reduced intramuscular fat and fibrosis, increased expression of utrophin (a structurally and functionally related protein to dystrophin) and reduced release of creatine kinase (a marker of muscle injury).
Whereas there are various types of mutations in the dystrophin gene that cause DMD, there is a need for therapies that could be broadly applicable for patients. Unlike experimental therapies that target specific dystrophin genetic mutations, ACE-031 targets proteins that regulate muscle growth and thus may be applicable to DMD patients regardless of their dystrophin genotype.
We are designing clinical trials to evaluate the potential for ACE-031 to provide disease-modifying effects on muscle quality that translate into durable benefits on strength and function for boys with DMD. Acceleron is partnering with research and clinical institutions worldwide and has built strong collaborations with the following organizations to advance the development of ACE-031 in DMD: