Scholar Rock Holding (SRRK) Company Conference Presentation - Slideshow

SRK-015 Program (SMA) - Scholar Rock's SRK-015 is a muscle-directed therapy aimed at improving motor function in SMA patients, complementing SMN upregulators [12] - The overall prevalence of patients with SMA is estimated to be 30,000-35,000 in the U S and Europe [12] - In a Nusinersen CHERISH trial, less than 15% of patients with later-onset SMA experienced a >3-point increase in motor function [17] - Preliminary Phase 2 TOPAZ data demonstrates target engagement in SMA patients [20] - Phase 2 TOPAZ trial includes three cohorts with interim efficacy and safety results expected in Q4 2020 and topline 12-month data in 1H21 [22, 9] SRK-181 Program (Immuno-Oncology) - The current checkpoint inhibitor market is valued at over $25 billion, but only ~20% of patients respond [26] - SRK-181 is a TGFβ1-selective inhibitor designed to overcome checkpoint inhibitor resistance [32] - Preclinical data shows that SRK-181 combined with CPI led to significant impact on anti-tumor responses and survival rates in preclinical models [36] - DRAGON Phase 1 trial is evaluating SRK-181's ability to overcome primary resistance to checkpoint inhibitors, with dose escalation update expected in Q4 2020 and clinical response/safety data in 2021 [39, 40] - DRAGON Part B initiation is planned for 1Q21, with anti-tumor and safety data expected starting in 2021, focusing on patients with primary resistance to CPI [53]