The below statement is a community update from Scholar Rock on SRK-015. 

Dear SMA Community,

Scholar Rock is dedicated to developing novel medicines to improve the lives of individuals who have Spinal Muscular Atrophy (SMA). We want to thank the SMA community for your continued support of our mission.

We are excited to share that we have initiated our first clinical trial (called TOPAZ) to evaluate SRK-015 for the treatment of SMA and have started patient dosing. SRK-015 targets the latent form of myostatin by specifically blocking its activation in the muscle. We believe that SRK-015 has the potential to be the first muscle-directed therapy to address the motor functional challenges that continue to represent a significant unmet need for patients with SMA.

TOPAZ is a Phase 2 proof-of-concept clinical trial to assess the safety and efficacy of SRK-015 in patients with Type 2 or Type 3 SMA. The TOPAZ trial is anticipated to enroll 55 patients between the ages of 2 and 21 across three cohorts, each representing a distinct subpopulation of patients. All patients will receive SRK-015 intravenously once every four weeks either as a monotherapy or in conjunction with an approved SMN upregulator treatment (e.g. Spinraza®) over a 12-month treatment period. The primary efficacy endpoints will measure motor function through clinically meaningful outcome measures validated in SMA, such as the Hammersmith Functional Motor Scale Expanded (HFMSE) in non-ambulatory SMA and the Revised Hammersmith Scale (RHS) in ambulatory SMA. We intend to use results from the TOPAZ trial to help plan additional SRK-015 trials in SMA.

We have started activating clinical trial sites for the TOPAZ trial in the US and will continue to do so throughout 2019. Additional sites in Canada and Europe are also expected to participate. Please refer to for active site status and information regarding the study. We will continue to share additional updates on the SRK-015 program with the SMA community.

Thanks again to everyone across the scientific, medical, and SMA community for your contributions and continued support towards this important mission of advancing the SMA treatment paradigm!

Best regards,

The Scholar Rock Team

Scholar Rock, Inc.
Cambridge, MA 02139