Investor Overview

Corporate Profile

Scholar Rock is a biopharmaceutical company focused on the discovery and development of innovative medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. Our most advanced product candidate, SRK-015, a selective first-in-class inhibitor of the activation of the growth factor myostatin in skeletal muscle, is currently in development for the treatment of spinal muscular atrophy, or SMA. We have successfully completed a Phase 1 clinical trial in healthy volunteers and initiated a Phase 2 proof-of-concept trial (named TOPAZ) in patients with later-onset (Type 2 and Type 3) SMA in the second quarter of 2019. In addition, we initiated the DRAGON Phase 1 clinical trial for our second product candidate, SRK-181, a highly specific inhibitor of latent TGFβ1 activation, in patients with locally advanced and metastatic solid tumors in the first quarter of 2020. We believe SRK-181 has the potential to render resistant solid tumors vulnerable to immune checkpoint inhibitors such as anti-PD1/PDL1 therapies and drive tumor regression through combination therapy.

Utilizing our proprietary platform, we are creating a pipeline of novel product candidates with the potential to transform the lives of patients suffering from a wide range of serious diseases, including neuromuscular disorders, cancer, fibrosis and anemia.

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