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Scholar Rock Reports Full Year 2019 Financial Results and Highlights Business Progress

- Initiated the Phase 1 proof-of-concept clinical trial of SRK-181, a potent and highly selective inhibitor of TGFβ1 activation, in patients with locally

articleScholar Rock Holding CorporationMarch 12, 20204/company/scholar-rock-holding-corp/news/scholar-rock-reports-full-year-2019-financial-results-and-highlights-business-progress
Scholar Rock Reports Full Year 2019 Financial Results and Highlights Business Progress

About this update from Scholar Rock Holding Corporation

[{"type":"text","content":"\n- Initiated the Phase 1 proof-of-concept clinical trial of SRK-181, a potent and highly selective inhibitor of TGFβ1 activation, in patients with locally advanced or metastatic solid tumors\n\n\n- On track to report interim safety and efficacy data from the TOPAZ Phase 2 clinical trial of SRK-015 in patients with Type 2 and Type 3 Spinal Muscular Atrophy in mid-2020\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nScholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today reported financial results for the full year ended December 31, 2019 and highlighted recent progress and upcoming milestones for its pipeline programs.\n\n\n“The year is off to a strong start with the initiation of the Phase 1 trial of SRK-181 in immuno-oncology and the completion of enrollment in the SRK-015 TOPAZ Phase 2 trial in SMA,” said Nagesh Mahanthappa, Ph.D., President and CEO of Scholar Rock. “We now have two product candidates in the clinic with data expected from both programs later this year that will offer initial insights on their potential to treat patients with SMA and solid tumors, respectively. Scholar Rock is well-positioned to continue to execute and build momentum across its portfolio of product candidates.”\n\n\n\n\nCompany Highlights and Upcoming Milestones\n\n\nSRK-015 Program for Spinal Muscular Atrophy (SMA):\n\n\n\nPublished Structural Insights into the Mechanism by which SRK-015 Targets the Precursor or Latent Form of the Myostatin Growth Factor. In February 2020, the Journal of Biological Chemistry(1) published “Structural basis for specific inhibition of extracellular activation of pro- or latent myostatin by the monoclonal antibody SRK-015” detailing the integrated structural and biochemical approaches used to elucidate the unique molecular mechanism of SRK-015, a highly specific inhibitor of the activation of latent myostatin. The publication offers the first structural insights into the approach of targeting precursor forms to modulate growth factor activation. This approach could be applied to the discovery of specific modulators across members of the TGFβ superfamily or, even more broadly, to other signaling proteins that require precursor processing for activation.\n\n\n\n\nOn Track to Report Interim Safety and...

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