Scholar Rock® discovered SRK-015, a selective and local inhibitor of latent myostatin activation for the treatment of primary myopathies.
Myostatin is a member of the TGFβ superfamily of growth factors that 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 are hypermuscular.
Because there are several growth factors that are very closely related to myostatin, Scholar Rock’s targeting of supracellular activation offers a distinct approach for avoiding cross-talk across the TGFβ superfamily by targeting myostatin activation rather than the traditional approach of targeting mature myostatin. SRK-015 uniquely targets the latent form of myostatin, specifically inhibiting its activation in muscle. SRK-015 has demonstrated reproducible and robust increases in lean mass and muscle weight in preclinical models of muscle disease. Scholar Rock believes SRK-015 has the potential to preserve or restore normal muscle function in patients suffering from a wide range of muscle diseases.
SRK-015 has demonstrated reproducible and robust increases in lean mass and muscle weight in preclinical models of muscle disease.