Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced the publication, “Specific Inhibition of Myostatin Activation is Beneficial in Mouse Models of SMA Therapy” in the peer-reviewed journal Human Molecular Genetics.
The publication details preclinical studies demonstrating that a highly specific inhibitor of myostatin activation, SRK-015, effectively increased muscle mass and function in mouse models of spinal muscular atrophy (SMA).
SRK-015, Scholar Rock's most advanced drug candidate, is a selective and local inhibitor of latent myostatin. Scholar Rock is developing and investigating SRK-015 as a treatment to improve muscle strength and motor function in patients with SMA.
Myostatin is a member of the TGFβ superfamily of growth factors and is expressed primarily in skeletal muscle cells to inhibit muscle growth. In the body, it works in concert with other growth factors and hormones to maintain appropriate muscle mass. There has been an emerging interest in therapeutically targeting myostatin following the discovery of myostatin-deficient animals that have increased muscle mass and strength.
SRK-015 uniquely targets the latent form of myostatin, specifically blocking its activation in muscle. Inhibiting the supracellular activation of myostatin, rather than the traditional approach of blocking already activated, mature myostatin or the myostatin receptor, avoids blocking the activity of other closely-related members of the TGFβ superfamily that may lead to undesirable side effects.