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 actively working to advance SRK-015 into clinical trials, which are anticipated to commence in mid-2018. We intend to develop SRK-015 in combination with therapies aimed at correcting the underlying genetic defect in SMA and as monotherapy in patients with certain subtypes of SMA.

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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