Leonard Zon, Timothy A. Springer, Tony Kingsley, Nagesh K. Mahanthappa
Number of employees
Total funding ($M)
Cormorant Asset Management, EcoR1 Capital Fund, Fidelity Management and Research Company, Invus, Polaris Partners, Redmile Group
Altered intercellular communication
spinal muscular atrophy
Phase 2 Trials
We take risks and challenge the status quo because disease must be fought with unexpected practice.
Scholar Rock is a clinical-stage 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, apitegromab (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 are currently evaluating apitegromab in our TOPAZ Phase 2 proof-of-concept trial in patients with later-onset (Type 2 and Type 3) SMA. Positive six-month interim analysis results were announced in October 2020 and top-line data for the full 12-month treatment period are anticipated in the second quarter of 2021. In addition, we are conducting our DRAGON Phase 1 proof-of-concept trial for SRK-181, a highly specific inhibitor of latent TGFβ1 activation, in patients with locally advanced and metastatic solid tumors. We believe SRK-181 has the potential to overcome resistance and meaningfully increase the number of patients who may benefit from checkpoint inhibitors, such as anti-PD-1 or anti-PD-L1 therapies.