Scholar Rock Holding Corporation · NASDAQ:SRRK
Scholar Rock — Q2 2020 Earnings Presentation
26 slides from Scholar Rock's earnings presentation, Aug 7, 2020
Slide 1: 2Q20 Financial Results and Business Progress
2Q20 Financial Results and Business Progress August 7, 2020
Slide 2: Disclaimers
Disclaimers Various statements in this presentation concerning Scholar Rock’s future expectations, plans and prospects, including without limitation, Scholar Rock’s expectations regarding its strategy, its product candidate selection and development timing, including timing for the initiation of and
Slide 3: Newest Members of Highly Experienced Leadership Team
Newest Members of Highly Experienced Leadership Team 3 TONY KINGSLEY, MBAPresident & CEO TED MYLES, MBACFO & Head of Business Operations Joined Scholar Rock’s Board of Directors in May 2020President & CEO of Taris Bio President & COO of The Medicines CompanyEVP at Biogen, led global commercial opera
Slide 4: Proprietary platform has produced multiple programs with significant therapeutic potential
Proprietary platform has produced multiple programs with significant therapeutic potential Two most advanced programs, SRK-015 and SRK-181, are advancing quickly and address markets that are well developed and growing Multiple programs and multiple anticipated milestones offer near- and long-term va
Slide 5: Differentiated Pipeline with a Series of Anticipated Milestones
5 DRAGON Part A2 Initiation DRAGON Part B Initiation Nominate RGMcProduct Candidate Gilead Fibrosis-focused TGFβ Collaboration DRAGON Clinical Response and Safety Data Across Multiple Tumor TypesPart A2 Combo Efficacy and Safety DataPart B – MelanomaPart B – NSCLCPart B – Urothelial CarcinomaPart B
Slide 6: Review of Clinical Programs: SRK-015 and SRK-181
Review of Clinical Programs: SRK-015 and SRK-181 6 Yung Chyung, MDChief Medical Officer
Slide 7: SMA Treatment Landscape: The Shifting Focus to Muscle-Directed Therapies
SMA Treatment Landscape: The Shifting Focus to Muscle-Directed Therapies †Mean improvement in HFMSE score experienced by patients withlater-onset SMA in the Phase 3 CHERISH clinical trial of nusinersen HFMSE Score at Month 15 Mean baseline score Mean improvement with nusinersen Unmet medical need fo
Slide 8: Later-Onset SMA: High Unmet Need for Muscle-Directed Therapy
†Source: Mercuri E, et.al. Nusinersen versus sham control in later-onset spinal muscular atrophy. N Engl J Med. 2018;378:625-635.† †Efficacy and safety of risdiplam (RG7916) in patients with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA) Roche/PTC Therapeutics Nusinersen CHERISH Trial i
Slide 9: Progress on Path Towards Investigating SRK-015's Therapeutic Potential in SMA
Progress on Path Towards Investigating SRK-015’s Therapeutic Potential in SMA Phase 2 TOPAZ interim analysis to evaluate efficacy and safety in SMA Results anticipated 4Q20 Strong translational rationale for investigating myostatin blockade in SMA Therapeutic effects in SMA preclinical mouse model
Slide 10: SRK-015 Phase 2 TOPAZ Trial Design
SRK-015 Phase 2 Trial Design Cohort 1 Cohort 3 Design Patients N= 23*; ages 5-21Open-label, single-arm20 mg/kg SRK-015 IV Q4W12-month treatment period Ambulatory Type 3 SMAReceiving treatment with approved SMN upregulator or as monotherapy Cohort 2 N= 15; ages 5-21 Open-label, single-arm20 mg/kg SRK
Slide 11: Each Cohort Represents Important POC Opportunity With Significant Potential
Each Cohort Represents Important POC Opportunity With Significant Potential Cohort 3 Efficacy – Therapeutic Goals Safety Goals Efficacy signal enables investigation of SRK-015’s broader potential Cohort 1 RHS: Absolute increase in mean change from baseline RHS: Substantial % of patients attain >3-po
Slide 12: SRK-181 Has Potential to Increase Response and Be Backbone Therapy to All Checkpoint Inhibitors
SRK-181 Has Potential to Increase Response and Be Backbone Therapy to All Checkpoint Inhibitors *Source: Company information, Wall Street research, Evaluate Pharma.†Source: Carretero-Gonzalez A, et al. Oncotarget. 2018;9:8706-8715. Meta-analysis of 12 randomized trials with control arm or adequate s
Slide 13: Significant Interest in Potential Role of TGFβ Inhibition in Immuno-Oncology
Significant Interest in Potential Role of TGFβ Inhibition in Immuno-Oncology Nature (online), Feb. 14, 2018. AuthorsWilly Hugo, Jesse M. Zaretsky, Lu Sun, Douglas B. Johnson, Antoni Ribas, Roger S. Lo Volume 165, Issue 1, 24 March 2016, Pages 35-44 July 24, 2020: https://doi.org/10.1038/ s41571-020-
Slide 14: Broad Potential for TGFβ Blockade Across Many Solid Tumors
Cancer Genome Atlas RNAseq analysis of >10,000 samples spanning 33 tumor types* *Source: National Cancer Institute - Cancer Genome Atlas Program.†Priti H, et al. Top 10 challenges in cancer immunotherapy. Immunity. 2020 Jan 14:52(1):17-35. https://doi.org/10.1016/j.immuni.2019.12.011. Substantial pr
Slide 15: SRK-181: Unique TGFβ1-Selective Approach to Overcoming Checkpoint Inhibitor Resistance
SRK-181: Unique TGFβ1-Selective Approach to Overcoming Checkpoint Inhibitor Resistance 15 SRK-181Latent TGFβ1 Inhibitor Inhibits TGFβ1 pathway - implicated in CPI resistance Highly selective targeting - avoids binding to latent TGFβ2 and TGFβ3 isoforms Aimed at increasing therapeutic window – potent
Slide 16: SRK-181: Therapeutic Rationale Continues to Strengthen
SRK-181: Therapeutic Rationale Continues to Strengthen EVIDENCE TO DATE Increasing evidence implicating TGFβ1’s pivotal role in CPI resistance TGFβ1 implicated in immune excluded tumor phenotype; poor CPI responsesTGFβ1 expression and immune exclusion broadly observed across solid tumorsMerck KGaA/G
Slide 17: DRAGON Phase 1 POC Trial to Evaluate SRK-181
DRAGON Phase 1 POC Trial to Evaluate SRK-181’s Ability to Overcome Primary Resistance to Checkpoint Inhibitors Open-label, dose escalation, and dose expansion clinical trialEvaluate the efficacy, safety/tolerability, and PK/PD of SRK-181 in combination with approved anti-PD-(L)1 therapyPatients with
Slide 18: DRAGON Part A: Progressing Quickly to Evaluation of SRK-181 with Anti-PD-(L)1 Therapy
DRAGON Part A: Progressing Quickly to Evaluation of SRK-181 with Anti-PD-(L)1 Therapy NCT04291079 on www.clinicaltrials.gov. SRK-181 with approved anti-PD-(L)13+3 dose escalation Focus on patients with primary resistance to single-agent anti-PD-1 or anti-PD-L1 Treat with same anti-PD-(L)1 as previou
Slide 19: DRAGON Part B: Multiple Opportunities for Efficacy Signals
DRAGON Part B: Multiple Opportunities for Efficacy Signals * Planning to open eligibility to patients with history of primary resistance to either pembrolizumab or nivolumabNCT04291079 on www.clinicaltrials.gov. Study population focused on patients already shown to have primary resistance to single-
Slide 20: 2Q20 Financial Results
20 2Q20 Financial ResultsTed MylesCFO & Head of Business Operations
Slide 21: 2Q20 Financial Update (GAAP)
21 2Q20 Financial Update (GAAP) Revenues Operating Expenses Net Loss Ended June 30, 2020 with $141M in cash, cash equivalents, and marketable securities Revenues of $3.9 millionRelated to the Gilead fibrosis-focused collaboration R&D expense of $17.0 millionY/Y increase due to acceptance of Specific
Slide 22: Differentiated Pipeline with a Series of Anticipated Milestones
22 DRAGON Part A2 Initiation DRAGON Part B Initiation Nominate RGMcProduct Candidate Gilead Fibrosis-focused TGFβ Collaboration DRAGON Clinical Response and Safety Data Across Multiple Tumor TypesPart A2 Combo Efficacy and Safety DataPart B – MelanomaPart B – NSCLCPart B – Urothelial CarcinomaPart B
Slide 23: Appendix
Appendix % $$ ScholarRock.
Slide 24: Preliminary TOPAZ Biomarker Data Provide First Demonstration of Target Engagement
24 Robust Target Engagement Observed~100-fold increases in serum latent myostatin levels following single 20 mg/kg dose in all cohorts of TOPAZConfirms presence of latent myostatin in patients with SMAWell-Behaved, Linear PK Profile Minimal variability across TOPAZ cohortsDose proportional increase
Slide 25: TGFβ1 Blockade with SRK-181-mIgG1 Rendered Preclinical Tumor Models Susceptible to Anti-PD1 Therapy
25 TGFβ1 Blockade with SRK-181-mIgG1 Rendered Preclinical Tumor Models Susceptible to Anti-PD1 Therapy Preclinical data published in Science Translational Medicine. Martin CJ, et al. Sci Transl Med. 2020 Mar 25;12(536):eaay8456. https://scholarrock.com/platform/publications. Melanoma (Cloudman S91)
Slide 26: TGFβ1 Isoform Specificity of SRK-181 Improved Preclinical Toxicity Profile
TGFβ1 Isoform Specificity of SRK-181 Improved Preclinical Toxicity Profile Preclinical data published in Science Translational Medicine. Martin CJ, et al. Sci Transl Med 2020 Mar 25;12(536): eaay8456. *Source: Anderton MJ, et al. Induction of heart valve lesions by small-molecule ALK5 inhibitors. To