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Scholar Rock Holding (SRRK) Presents At BMO Capital Markets Healthcare Conference - Slideshow
Scholar RockScholar Rock(US:SRRK)2020-06-24 18:46

Pipeline and Programs - Scholar Rock is developing SRK-015 for Spinal Muscular Atrophy (SMA) and SRK-181 for Immuno-Oncology, targeting latent growth factor activation to optimize potency and selectivity [5] - SRK-015 is in Phase 2 clinical trials, with interim efficacy and safety results expected in 4Q20 and top-line 12-month results in 1H21 [5, 61, 77] - SRK-181 is in a Phase 1 proof-of-concept (POC) trial (DRAGON), with an update on dose escalation expected in 4Q20 and clinical response and safety data in 2021 [5, 8, 40, 81] - The company plans to identify a second indication for SRK-015 in 2020 and nominate a product candidate in the RGMc program for anemias in 2020 [12, 79, 81] SRK-181 and Immuno-Oncology - Approximately 80% of patients with metastatic solid tumors do not respond to Checkpoint Inhibitors (CPIs), highlighting a significant unmet medical need [16, 38, 87] - Preclinical data suggests that TGFβ1 blockade with SRK-181 can render tumors susceptible to anti-PD1 therapy, potentially increasing the response rate to CPIs [20, 24, 33] - In a preclinical bladder cancer model, combination treatment with SRK-181-mIgG1/anti-PD1 led to a significant increase in effector T cells, averaging 34% of the tumor's immune cells, compared to a control average of 35% [29] SRK-015 and Spinal Muscular Atrophy (SMA) - There remains a significant unmet need for muscle-directed therapies to complement the disease-stabilizing benefits of SMN upregulators in SMA [45, 47] - Preclinical studies in SMNΔ7 mouse models showed that treatment with muSRK-015P improved muscle strength, with maximal torque of the plantar flexor muscle group increasing by 44%-51% in a more severe model and 20%-30% in a less severe model [52] - Preliminary TOPAZ Phase 2 biomarker data demonstrated robust target engagement, with ~100-fold increases in serum latent myostatin levels following a single 20 mg/kg dose [55] Gilead Collaboration - Scholar Rock has a fibrosis partnership with Gilead, including an upfront cash and equity investment of $80 million and potential additional milestones of up to $1425 million across three programs [70, 71] - Scholar Rock achieved a $25 million preclinical milestone with Gilead at the end of 2019 [71]