Scholar Rock Holding Corporation · NASDAQ:SRRK

Scholar Rock — 43rd Annual J.P. Morgan Healthcare Conference (January 2025)

35 slides from Scholar Rock's conference presentation, Jan 14, 2025

Slide 1: 43rd Annual J.P. Morgan Healthcare Conference
© 2025 Scholar Rock, Inc. All rights reserved. 43rd Annual J.P. Morgan Healthcare Conference Jay Backstrom, M.D., MPH President and Chief Executive Officer January 14, 2025 1 Exhibit 99.1
Slide 2: Forward-Looking Statements
© 2025 Scholar Rock, Inc. All rights reserved. Forward-Looking Statements Various statements in this presentation concerning the future expectations, plans and prospects of Scholar Rock Holding Corporation and Scholar Rock, Inc. (collectively, “Scholar Rock”), including without limitation, Scholar R
Slide 3: Our Mission
OUR MISSION To discover, develop, and deliver life-changing therapies by harnessing cutting-edge science to create new possibilities for people living with serious diseases Industry-leading technology, life-changing potential 3
Slide 4: Today's Focus: Moving with a Sense of Urgency
© 2025 Scholar Rock, Inc. All rights reserved. TODAY’S FOCUS Scholar Rock is Moving with a Sense of Urgency To Bring Transformative Medicines to Patients 4 *Pending regulatory approval. EXPAND Apitegromab Development Program: Building a Pipeline in a Product COMMERCIALIZE Execute a Successful Commer
Slide 5: Scholar Rock's Target: Latent Growth Factor
© 2025 Scholar Rock, Inc. All rights reserved. Scholar Rock’s Target Latent Growth Factor Traditional Target “Mature” Active Growth Factor 5 Scholar Rock Has Succeeded Where Others Have Failed Selectivity Drives Success Targeting the ‘cage’ before growth factor is released allows for exquisite selec
Slide 6: Growing Pipeline Across High Value Therapeutic Areas
© 2025 Scholar Rock, Inc. All rights reserved. 6 Growing Pipeline Across High Value Therapeutic Areas Industry-leading Anti-myostatin Programs *undisclosed indication **UC=urothelial cell carcinoma; RCC=renal cell carcinoma. THERAPEUTIC AREA PRODUCT (target) Discovery/ Preclinical PHASE 1 PHASE 2 PH
Slide 8: Progressive Muscle Weakness Remains Core Unmet Need in SMA
© 2025 Scholar Rock, Inc. All rights reserved. 8 Progressive Muscle Weakness Remains Core Unmet Need in SMA 1. Finkel RS et al. “Final Safety and Efficacy Data From the SHINE Study in Participants With Infantile-Onset and Later-Onset SMA.” Presented at Cure SMA Annual Conference, July 2024. 2. Cure
Slide 9: The Only Muscle-Targeted Therapy with Clinical Success in SMA
© 2025 Scholar Rock, Inc. All rights reserved. SELECTIVITY DRIVES SUCCESS The Only Muscle-Targeted Therapy with Clinical Success in SMA 9 * Based on apitegromab combined dose (10 mg/kg and 20 mg/kg) + SOC versus placebo + SOC (Hochberg multiplicity adjustment). † 12.5% of patients on placebo + SOC a
Slide 10: Apitegromab: Potential to Transform the Standard of Care in SMA
© 2025 Scholar Rock, Inc. All rights reserved. Apitegromab: Potential to Transform the Standard of Care in SMA Finkel RS et al. “Final Safety and Efficacy Data From the SHINE Study in Participants With Infantile-Onset and Later-Onset SMA.” Presented at Cure SMA Annual Conference, July 2024 *Patient
Slide 11: Apitegromab: Shifting the Treatment Paradigm in SMA
© 2025 Scholar Rock, Inc. All rights reserved. 11 Apitegromab: Shifting the Treatment Paradigm in SMA Transformative Therapeutic Potential • Validated with a rigorous approach: Strong trial design using a gold-standard SMA scale • Potential to transform standard of care in SMA Suitable for a Broad S
Slide 12: Expanding our Impact: Initiating Phase 2 OPAL Trial in mid-2025
© 2025 Scholar Rock, Inc. All rights reserved. 12 Expanding our Impact: Initiating Phase 2 OPAL Trial in mid-2025 Studying apitegromab in patients under 2 years old Potential to alter the course of SMA in a broad population CHANGING More Lives Reaching patients earlier in their treatment journey TIM
Slide 13: Positioned for a Successful Commercial Launch
© 2025 Scholar Rock, Inc. All rights reserved. Scholar Rock is Positioned for a Successful Commercial Launch THE RIGHT... Potential to alter the course of SMA MEDICINE Clear unmet need and first and only muscle-targeted therapy MARKET Engagement, patient focus & execution PLAN 13
Slide 14: Existing Therapies Generate ~$4.5B in Annual Revenues
© 2025 Scholar Rock, Inc. All rights reserved. 14 Existing Therapies Generate ~$4.5B in Annual Revenues Addressing the Motor Neuron 1. Hua Y, et al. Nature. 2011;478(7367):123-6. 2. Figure adapted from: SMA Foundation Overview. http://www.smafoundation.org/wp-content/uploads/2012/03/SMA-Overview.pdf
Slide 15: $2B+ Global Opportunity for Apitegromab in SMA
© 2025 Scholar Rock, Inc. All rights reserved. $2B+ Global Opportunity for Apitegromab in SMA ~$4.5B1 Global revenue for three SMN-targeted therapies Apitegromab global revenue potential >$2.0B2 Apitegromab global revenue potential $2B+2 1st And only muscle-targeted treatment to show clinical benefi
Slide 16: SMA is a Defined Market and Optimal for Apitegromab Launch
© 2025 Scholar Rock, Inc. All rights reserved. SMA is a Defined Market and Optimal For Apitegromab Launch Patients are diagnosed and treated, but still need more Patients Identified and Treated ~25K patients in US and EU 100% US newborn screening 2/3 of US patients on treatment Patient, HCP, and pay
Slide 17: Advantaged by Deep Rare Disease Commercial Experience
© 2025 Scholar Rock, Inc. All rights reserved. 17 Advantaged by Deep Rare Disease Commercial Experience Commercial, medical, and advocacy teams with deep rare disease launch experience Launches Leadership Seasoned executive team with average of 20+ years industry experience
Slide 18: Partnering With the SMA Community and Engaging Top HCPs
© 2025 Scholar Rock, Inc. All rights reserved. Partnering With the SMA Community and Engaging Top HCPs © 2024 Scholar Rock, Inc. All rights reserved. Disease EDUCATION Amplifying the patient voice in first muscle-focused SMA disease education campaign Partnering with US and European patient advocacy
Slide 19: Preparing for a Successful Launch
© 2025 Scholar Rock, Inc. All rights reserved. Preparing for a Successful Launch Delivering Apitegromab Customer Engagement Commercial launch supply secured Rare disease distribution partners selected Home infusion at launch Account team with average 30 years experience engaging with US commercial a
Slide 20: SMA is Only the Beginning: Additional Neuromuscular Diseases
© 2025 Scholar Rock, Inc. All rights reserved. SMA is Only the Beginning: Creating Possibilities with Apitegromab in Additional Neuromuscular Diseases *Numbers represent prevalence in the US and Europe based on internal market research. SM=Spinal Muscular Atrophy; ALS=Amyotrophic Lateral Sclerosis;
Slide 21: Foundation of a Future Multi-Billion Dollar Neuromuscular Franchise
© 2025 Scholar Rock, Inc. All rights reserved. Apitegromab is the Foundation of a Future Multi-Billion Dollar Neuromuscular Franchise* *Subject to regulatory approval. Commercial Launch* Global expansion, starting with Europe SMA expansion with Ph 2 OPAL trial for patients under 2 and subcutaneous f
Slide 23: GLP-1 RAs are Transforming Weight Loss for Millions of People
© 2025 Scholar Rock, Inc. All rights reserved. GLP-1 RAs are Transforming Weight Loss for Millions of People Source: UBS Bank, GLP-1: A medication worth $126 billion in sales by 2029? https://www.ubs.com/global/en/investment-bank/insights-and-data/2024/glp-1-a-medication.html. GLP-1 RA=GLP-1 recepto
Slide 24: However, Patients Want Healthier Weight Loss
© 2025 Scholar Rock, Inc. All rights reserved. However, Patients Want Healthier Weight Loss* *Source: Scholar Rock market research, completed December 2024. GLP-1 RA=GLP-1 receptor agonist. WEAKNESS is a Concern Patients complain of reduced strength after GLP-1 RA treatment Patients hope for a combi
Slide 25: Leveraging Apitegromab's Success to Advance the Obesity Program
© 2025 Scholar Rock, Inc. All rights reserved. 25 Industry-Leading Anti-Myostatin Platform: Leveraging Apitegromab’s Success to Advance the Obesity Program *Expected timelines POC=Proof of Concept; GLP-1 RA=GLP-1 receptor agonist. POC data readout Q2* Ph 2 proof-of-concept trial APITEGROMAB + tirzep
Slide 26: SRK-439: Potential Best in Class
© 2025 Scholar Rock, Inc. All rights reserved. 26 GLP-1 RA=GLP-1 receptor agonist. Preclinical data to date show strong potential to support healthier weight loss in combination with GLP-1 RAs: SRK-439: Potential Best in Class Strong Scientific Validation and Promising Preclinical Evidence Preservat
Slide 27: SRK-439 Protects from Tirzepatide-Induced Muscle Loss in DIO Mice
© 2025 Scholar Rock, Inc. All rights reserved. SRK-439 Protects from Tirzepatide-Induced Muscle Loss in DIO Mice qNMR % Lean Mass Change from Baseline Key Observations • Considerable lean mass loss with tirzepatide treatment • Combination with SRK-439 led to dose-dependent lean mass preservation • L
Slide 28: Potential to Optimize Benefit-Risk with Myostatin Selectivity
© 2025 Scholar Rock, Inc. All rights reserved. Signaling receptor shared by Myostatin, GDF-11 & Activin A 28 Potential to Optimize Benefit-Risk with Myostatin Selectivity Promyostatin Pro-GDF-11 Proactivin GDF-11 Activin A ActRIIA/B Apitegromab SRK-439 Other Approaches • GI problems, e.g., diarrhea,
Slide 29: Phase 2 Proof-of-Concept Study of Apitegromab in Obesity
© 2025 Scholar Rock, Inc. All rights reserved. 29 Data from Phase 2 Proof-of-Concept Study of Apitegromab in Obesity Expected Q2 2025 *Due to expedited enrollment and timing of semaglutide clinical supply, all enrolled patients received tirzepatide. Apitegromab dose regimen will be 10 mg/kg Q4W, bas
Slide 30: Goals of the EMBRAZE Proof-of-Concept Study
© 2025 Scholar Rock, Inc. All rights reserved. Goals of the EMBRAZE Proof-of-Concept Study Initiated trial in May 2024, ahead of target timeline Enrollment completed in September 2024 Topline data expected in Q2 2025 30 INSIGHTS GAINED FROM EMBRAZE STUDY to inform SRK-439 development Study Aims to D
Slide 31: Tirzepatide-Induced Weight Loss Accompanies Significant Muscle Loss
© 2025 Scholar Rock, Inc. All rights reserved. Tirzepatide-Induced Weight Loss Accompanies Significant Muscle Loss Figure from Jastreboff, A.M. et al. N Engl J Med. 2022;387(3):205-216. * Data from Phase 3 clinical trial of tirzepatide in adults with obesity. Tirzepatide DECREASE in lean mass of 10.
Slide 33: Delivering on the Mission of Bringing Transformative Medicines to Patients
© 2025 Scholar Rock, Inc. All rights reserved. 33 Delivering on the Mission of Bringing Transformative Medicines to Patients * Pending approval from regulatory agencies. Apitegromab is an investigational drug candidate under evaluation and has not been approved by any regulatory agency. Leveraging R
Slide 34: 2025 Milestones: A Transformative Year for Scholar Rock
© 2025 Scholar Rock, Inc. All rights reserved. 2025 Milestones: A Transformative Year for Scholar Rock 34 * Pending regulatory approval. Apitegromab is an investigational drug candidate under evaluation and has not been approved by any regulatory agency. • Submit FDA and EMA applications in 1Q 2025

Scholar Rock Holding — 43rd Annual J.P. Morgan Healthcare Conference (January 2025) on 6ix

Scholar Rock — 43rd Annual J.P. Morgan Healthcare Conference (January 2025)

Slide 1: 43rd Annual J.P. Morgan Healthcare Conference

Slide 2: Forward-Looking Statements

Slide 3: Our Mission

Slide 4: Today's Focus: Moving with a Sense of Urgency

Slide 5: Scholar Rock's Target: Latent Growth Factor

Slide 6: Growing Pipeline Across High Value Therapeutic Areas

Slide 7: Transforming the Treatment of SMA: Commercializing Apitegromab

Slide 8: Progressive Muscle Weakness Remains Core Unmet Need in SMA

Slide 9: The Only Muscle-Targeted Therapy with Clinical Success in SMA

Slide 10: Apitegromab: Potential to Transform the Standard of Care in SMA

Slide 11: Apitegromab: Shifting the Treatment Paradigm in SMA

Slide 12: Expanding our Impact: Initiating Phase 2 OPAL Trial in mid-2025

Slide 13: Positioned for a Successful Commercial Launch

Slide 14: Existing Therapies Generate ~$4.5B in Annual Revenues

Slide 15: $2B+ Global Opportunity for Apitegromab in SMA

Slide 16: SMA is a Defined Market and Optimal for Apitegromab Launch

Slide 17: Advantaged by Deep Rare Disease Commercial Experience

Slide 18: Partnering With the SMA Community and Engaging Top HCPs

Slide 19: Preparing for a Successful Launch

Slide 20: SMA is Only the Beginning: Additional Neuromuscular Diseases

Slide 21: Foundation of a Future Multi-Billion Dollar Neuromuscular Franchise

Slide 22: Unlocking Value in Our Anti-Myostatin Platform

Slide 23: GLP-1 RAs are Transforming Weight Loss for Millions of People

Slide 24: However, Patients Want Healthier Weight Loss

Slide 25: Leveraging Apitegromab's Success to Advance the Obesity Program

Slide 26: SRK-439: Potential Best in Class

Slide 27: SRK-439 Protects from Tirzepatide-Induced Muscle Loss in DIO Mice

Slide 28: Potential to Optimize Benefit-Risk with Myostatin Selectivity

Slide 29: Phase 2 Proof-of-Concept Study of Apitegromab in Obesity

Slide 30: Goals of the EMBRAZE Proof-of-Concept Study

Slide 31: Tirzepatide-Induced Weight Loss Accompanies Significant Muscle Loss

Slide 32: Conclusion

Slide 33: Delivering on the Mission of Bringing Transformative Medicines to Patients

Slide 34: 2025 Milestones: A Transformative Year for Scholar Rock

Slide 35: Thank you!