Scholar Rock Holding Corporation · NASDAQ:SRRK
Scholar Rock — Investor Presentation (November 2021)
45 slides from Scholar Rock's investor presentation, Nov 30, 2021
Slide 1: Deep Insights, Impactful Medicines
Deep Insights,Impactful Medicines
Slide 2: Disclaimers
Disclaimers Various statements in this presentation concerning the future expectations. plans and prospects of Scholar Rock. Inc. (‘Scholar Rock’). including without limitation, Scholar Rock's expectations regarding its strategy. its product candidate selection and development timing. including timi
Slide 3: 2021 Momentum to Carry into 2022 Across Portfolio
2021 Momentum to Carry into 2022 Across Portfolio SAPPHIRE Phase 3 Trial ae ne ——_————_;__ => Atrophy (SMA) TOPAZ extension (55 of the 57 patients who completed TOPAZ I2-month pertod continue to participate nthe extension) Apitegromab é Other Indications ‘Muttiple opportunities for additional myosta
Slide 4: Bringing a Revolutionary Approach to Highly Sought-After Growth Factors
Bringing a Revolutionary Approach to Highly Sought-After Growth Factors Implicated in Devastating Diseases Scholar Rock’s R&D Platform Scholar Rock’s Target Transform Medical Practice Growth Factor Precursor (Latent Form) + Pursue important targets with well-validated biology but are difficult to dr
Slide 5: Apitegromab Positioned to be Next Potential Transformative Therapy for Patients with SMA
Apitegromab Positioned to be Next Potential Transformative Therapy for Patients with SMA $8 scholarRock
Slide 6: Spinal Muscular Atrophy Overview
Spinal Muscular Atrophy Overview Global disease with 30,000-35,000 affected in U.S. and Europe alone TOPAZ* 12-month results showed transformative potential in non-ambulatory Type 2 and 3 patients + Significant, progressive motor function ‘ment; many lose ambulation Represents ~2/3 of overall patien
Slide 7: Potential to Pioneer a New Treatment Era: Opportunity for Muscle-Directed Therapy
Potential to Pioneer a New Treatment Era: Opportunity for Muscle-Directed Therapy to Complement SMN Upregulators (nusinersen) Phase 3 Trial Design Indication + >11,000* patient: + $2+ billion in revenu + ~4,000" str Nv + ~CHF243 million in revenues (1H21) 00*** patients treat 2 billion in revenues (
Slide 8: Patients with Type 2 and 3 SMA Continue to Experience Major Functional Deficits
Patients with Type 2 and 3 SMA Continue to Experience Major Functional Deficits Despite Improvement from SMN Therapy*
Slide 9: Non-Ambulatory Type 2/3 SMA: Majority Started on SMN Rx After Age 5
Non-Ambulatory Type 2/3 SMA: Majority of Patients Started on SMN Rx After Age 5 Do Not Experience Motor Function Increases*
Slide 10: TOPAZ Top-Line Data Showed Apitegromab's Transformative Potential
TOPAZ Top-Line Data Showed Apitegromab’s Transformative Potential in Patients with Type 2/3 SMA Majority of non-ambulatory patients* experienced HFMSE increases from apitegromab as add- on during chronic maintenance phase of SMN therapy v_ Apitegromab led to HFMSE improvements in both non-ambulatory
Slide 11: Safety Results from TOPAZ 12-Month Top-Line Analysis
Safety Results from TOPAZ 12-Month Top-Line Analysis Support Evaluation of Apitegromab in Phase 3 Trial
Slide 12: SAPPHIRE Phase 3 Design Optimized by Insights from TOPAZ
SAPPHIRE Phase 3 Design Optimized by Insights from TOPAZ
Slide 13: SAPPHIRE Phase 3 Design Optimized by Insights from TOPAZ
SAPPHIRE Phase 3 Design Optimized by Insights from TOPAZ
Slide 14: TOPAZ Age 2-12 Exploratory Analysis
TOPAZ Age 2-12 Exploratory Analysis(Non-Ambulatory Type 2/3 SMA)
Slide 15: TOPAZ Age 2-12 Analysis in Pooled Non-Ambulatory Cohorts
TOPAZ Age 2-12 Analysis* in Pooled Non-Ambulatory Cohorts Transformative Potential as Add-on for Apitegromab
Slide 16: SAPPHIRE (Phase 3) Trial Overview
SAPPHIRE (Phase 3) Trial Overview
Slide 17: SAPPHIRE Details
SAPPHIRE Details Main population ‘Age 2-12, non-ambulatory Type 2 and Type 3 SMA Chronic maintenance phase of SMN Rx (minimum prior duration of treatment before screening of 10 mo’s for nusinersen or 6 mo’s for risdiplam) Stratified randomization to ensure balanced allocation: 1) age at SMN Rx initi
Slide 18: Additional Therapeutic Opportunities May Be Pursued
Additional Therapeutic Opportunities May Be Pursued With Separate Development Strategies Global disease with 30,000-35,000 affected in U.S. and Europe alone Ambulatory patients + Smaller population but high unmet need as benefits of SMN regulators not well-established + TOPAZ suggests potential clin
Slide 19: SRK-181: Potential Transformative Backbone for a New Era of Cancer Immunotherapy
SRK-181: Potential Transformative Backbone for a New Era of Cancer Immunotherapy DRAGON $$$ ScholarRock. 19
Slide 20: Significant Interest in Potential Role of TGFβ Inhibition in Immuno-Oncology
Significant Interest in Potential Role of TGFβ Inhibition in Immuno-Oncology
Slide 21: Emerging Evidence Implicates TGFβ1 as Driving Primary Resistance to Checkpoint Inhibitors
Emerging Evidence Implicates TGFβ1 as Driving Primary Resistance to Checkpoint Inhibitors
Slide 22: SRK-181: Unique TGFβ1-Selective Approach to Overcoming Checkpoint Inhibitor Resistance
SRK-181: Unique TGFβ1-Selective Approach to Overcoming Checkpoint Inhibitor Resistance
Slide 23: TGFβ1 Blockade with SRK-181-mIgG1 Rendered Preclinical Tumor Models Susceptible to Anti-PD1
TGFβ1 Blockade with SRK-181-mIgG1 Rendered Preclinical Tumor Models Susceptible to Anti-PD1 Therapy
Slide 24: TGFβ1 Isoform Specificity of SRK-181 Improved Preclinical Toxicity Profile
TGFβ1 Isoform Specificity of SRK-181 Improved Preclinical Toxicity Profile
Slide 25: SRK-181 Therapeutic Hypothesis: Potential Advantages of Latent TGFβ1 Inhibitor
SRK-181 Therapeutic Hypothesis: Potential Advantages of Latent TGFβ1 Inhibitor
Slide 26: 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
Slide 27: DRAGON Part A: Dose Escalation Update and Safety Data
DRAGON Part A: Dose Escalation Update and Safety Data
Slide 28: DRAGON Part A: Preliminary Pharmacokinetics Summary of SRK-181
DRAGON Part A: Preliminary Pharmacokinetics (PK) Summary of SRK-181
Slide 29: DRAGON Part A: Preliminary Anti-Tumor Effects
DRAGON Part A: Preliminary Anti-Tumor Effects*
Slide 30: DRAGON Has Advanced to Part B to Test Proof of Concept
DRAGON Has Advanced to Part B to Test Proof of Concept for SRK-181 in Overcoming Anti-PD-(L)1 Resistance
Slide 31: 2021 Momentum to Carry into 2022 Across Portfolio
2021 Momentum to Carry into 2022 Across Portfolio SAPPHIRE Phase 3 Trial ae ne ——_————_;__ => Atrophy (SMA) TOPAZ extension (55 of the 57 patients who completed TOPAZ I2-month pertod continue to participate nthe extension) Apitegromab é Other Indications ‘Muttiple opportunities for additional myosta
Slide 32: Appendix
Appendix 8 ScholarRock
Slide 33: Apitegromab: Pairing the latent form with important translational insights
Apitegromab: Pairing the latent form with important translational insights
Slide 34: Apitegromab: Muscle-Directed Therapy Aimed at Complementing SMN Upregulators
Apitegromab: Muscle-Directed Therapy Aimed at Complementing SMN Upregulators
Slide 35: Apitegromab Phase 2 Trial Design
Apitegromab Phase 2 Trial Design
Slide 36: Baseline Characteristics
Baseline CharacteristicsNusinersen-treated patients well into chronic maintenance phase
Slide 37: Non-Ambulatory Type 2/3 Cohort: Initiated nusinersen age <5
Non-Ambulatory Type 2 Cohort: Initiated nusinersen age <5
Slide 38: Non-Ambulatory Type 2/3 Cohort: Initiated nusinersen age ≥5
Non-Ambulatory Type 2/3 Cohort: Initiated nusinersen age ≥5
Slide 39: Majority of Ambulatory Patients Maintained or Improved in RHS Score
Majority of Ambulatory Patients Maintained or Improved in RHS Score from Baseline
Slide 40: Pharmacokinetic and Pharmacodynamic Data are Supportive of Clinically Observed Effects
Pharmacokinetic and Pharmacodynamic Data are Supportive of Clinically Observed Effects
Slide 41: HFMSE Improvements Observed Across Age Range of Non-Ambulatory Patients
HFMSE Improvements Observed Across Age Range of Non-Ambulatory Patients with Relatively Larger Gains from Earlier Treatment 2fe . 2 16 & 3 3 3 ° 8 E ° 5 ‘° £ - 2 @ 5 : 2 ° . 5 ° : “ 5, ese : = s ° 2 . : . = x= os ° . e ° Youngest ® 0 yan : (19 years) (2 years) Age of patient at baseline in TOPAZ tsw
Slide 42: Increases in HFMSE Not Correlated with Duration of Prior Nusinersen Treatment
Increases in HFMSE Not Correlated with Duration of Prior Nusinersen Treatment
Slide 43: WHO Motor Development Milestone Achievements Further Support Apitegromab's Potential
WHO Motor Development Milestone Achievements Further Support Apitegromab’s Potential to Improve Motor Function
Slide 44: Inhibition of TGFβ1: Multipronged Approach for Immuno-Oncology
Inhibition of TGFβ1: Multipronged Approach for Immuno-Oncology
Slide 45: TGFβ1 Blockade with SRK-181-mIgG1 Rendered Preclinical Tumor Models Susceptible to Anti-PD1
TGFβ1 Blockade with SRK-181-mIgG1 Rendered Preclinical Tumor Models Susceptible to Anti-PD1 Therapy
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