SRK-015 for Spinal Muscular Atrophy (SMA)

SRK-015, our 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 Spinal Muscular Atrophy (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.

Development Plans

Scholar Rock is advancing SRK-015, a first-in-class selective inhibitor of the activation of myostatin, into clinical development for the treatment of SMA. We have initiated a Phase 1 clinical trial and intend to commence a Phase 2 trial in patients with later-onset SMA in the first quarter of 2019. We believe that SRK-015 has the potential to be the first muscle-directed therapy to reverse or prevent muscle atrophy in SMA patients and could be used both as a monotherapy or together with the current standard of care.

SRK-015 is an investigational drug candidate. The effectiveness and safety of SRK-015 has not been established and SRK-015 has not been approved by the FDA or any other regulatory agency.
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