Scholar Rock Holding Corporation · NASDAQ:SRRK
Scholar Rock — Q3 2024 Earnings Presentation
27 slides from Scholar Rock's earnings presentation, Nov 12, 2024
Slide 1: Third Quarter 2024 Business Update
© 2024 Scholar Rock, Inc. All rights reserved. Third Quarter 2024 Business Update November 12, 2024 1 Exhibit 99.2
Slide 2: Forward-Looking Statements
© 2024 Scholar Rock, Inc. All rights reserved. Forward-Looking Statements This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Scholar Rock’s future expectations, plans
Slide 3: Company Overview
© 2024 Scholar Rock, Inc. All rights reserved. Company Overview Jay Backstrom, M.D., MPH President & Chief Executive Officer 3
Slide 4: Company Speakers
© 2024 Scholar Rock, Inc. All rights reserved. 4 Company Speakers Jay Backstrom, M.D., MPH President & Chief Executive Officer Ted Myles, MBA Chief Operating Officer and Chief Financial Officer
Slide 5: Today's Agenda
© 2024 Scholar Rock, Inc. All rights reserved. Today’s Agenda 5 Topic Speaker Company Overview Jay Backstrom, President & Chief Executive Officer Ted Myles, Chief Operating Officer & Chief Financial Officer Financial Update Conclusions Jay Backstrom, President & Chief Executive Officer Q&A Ses
Slide 6: A Successful Q3, Continuing to Build on Strong 2024 Progress
© 2024 Scholar Rock, Inc. All rights reserved. Expanding the Executive Team • Beth Shafer, Ph.D. joined as Chief Business Officer Advancing apitegromab in SMA • Positive Phase 3 SAPPHIRE Study • BLA/MAA submission planned for Q1 2025 • Preparing for commercial launch EMBRAZE Ph 2 Trial continuing mo
Slide 7: Our Purpose: Create Possibilities for Those Living with SMA
© 2024 Scholar Rock, Inc. All rights reserved. Our Purpose: Create Possibilities for Those Living with Spinal Muscular Atrophy (SMA) Muscle is everything. I want to live knowing that I have the strength to take care of myself if left alone. - Lyza 7 © 2024 Scholar Rock, Inc. All rights reserved.
Slide 8: Positive Phase 3 SAPPHIRE Trial: Transformative Benefit in SMA
© 2024 Scholar Rock, Inc. All rights reserved. 8 CONSISTENT clinically meaningful benefit across all age groups (2-21) FAVORABLE SAFETY profile consistent with >48 months experience in Phase 2 TOPAZ trial 30% of apitegromab patients ACHIEVED ≥3 POINT IMPROVEMENT IN HFMSE† 1.8 (p=0.0192) POINT IMPROV
Slide 9: SAPPHIRE – Apitegromab in SMA: HFMSE Change Over Time
© 2024 Scholar Rock, Inc. All rights reserved. 9 SAPPHIRE – APITEGROMAB IN SMA Early-dosed (CHERISH/SHINE) Delayed-dosed (SHINE) Sham control (CHERISH) -6 -4 -2 0 2 4 6 ∆ -1.3 (SD: 9.3) From Baseline ∆ -2.3 (SD: 9.6) From Baseline ∆ -0.4 (SD: 4.9) From Baseline Study Day [Year] Change (SE) in HFMSE
Slide 10: Early and Increasing HFMSE Improvement vs. Placebo
© 2024 Scholar Rock, Inc. All rights reserved. 10 CI=Confidence Interval; EXP=Exploration Subpopulation; HFMSE=Hammersmith Functional Motor Scale Expanded; LS=Least Squares; MEP=Main Efficacy Population; SOC=standard of care. SAPPHIRE – APITEGROMAB IN SMA Early and Increasing HFMSE Improvement vs. P
Slide 11: Improvement in HFMSE Consistent Across Doses and Age Groups
© 2024 Scholar Rock, Inc. All rights reserved. -5 -4 -3 -2 -1 0 1 2 3 4 5 11 Improvement in HFMSE Consistent Across Doses and Age Groups SAPPHIRE – APITEGROMAB IN SMA LS Mean Difference (95% CI) [Active-Placebo] 1.8 (0.30, 3.32) 2-12 years apitegromab combined dose (10 & 20 mg/kg; n=106) vs. placebo
Slide 12: 30% of Apitegromab Patients Achieved ≥3 Points on HFMSE
© 2024 Scholar Rock, Inc. All rights reserved. 30% of Apitegromab Patients Achieved ≥3 Points on HFMSE SA PPH IRE – APITEGR OMA B IN S MA ≥3 Point Improvement in HFMSE 30.4% 16 HFMSE=Hammersmith Functional Motor Scale Expanded; SOC=standard of care. 12.5% 10% 5% 0% 15% 30% 25% 20% 35% Apitegromab Co
Slide 13: Potential to be Suitable for Broad SMA Population
© 2024 Scholar Rock, Inc. All rights reserved. 13 Broadly representative study population Improvement seen across all age groups (2-21) Moving quickly on filing BLA and MAA in Q1 2025 * If approved by regulatory authorities Potential to be Suitable for Broad SMA Population* SPINAL MUSCULAR ATROPHY S
Slide 14: Next Wave of Innovation: Selective Anti-Myostatin for Healthy Weight Management
© 2024 Scholar Rock, Inc. All rights reserved. Next Wave of Innovation: Selective Anti-Myostatin for Healthy Weight Management 14 Tests hypothesis of selective anti-myostatin in obese population POC study start* POC data readout Q2* Ph 2 proof-of-concept trial APITEGROMAB + GLP-1 Agonist 2024 2025 I
Slide 15: Strong Scientific Validation and Promising Preclinical Evidence
© 2024 Scholar Rock, Inc. All rights reserved. 15 OBESITY Preclinical data to date show strong potential to support healthier weight loss in combination with GLP-1 RA: Preservation of lean mass during GLP-1 RA-induced weight loss and improvement in fasting glucose Increase in lean mass and attenuati
Slide 16: Therapeutic Potential of Muscle-Targeted Approach in Obesity
© 2024 Scholar Rock, Inc. All rights reserved. Potential Benefits Our Solution Delivers Attractive Clinical Risk/Benefit Profile 16 • Inhibition of myostatin, a negative regulator of muscle, is known to promote muscle growth and function • Apitegromab, a selective myostatin inhibitor, has been shown
Slide 17: Financial & Business Update
© 2024 Scholar Rock, Inc. All rights reserved. Financial & Business Update Ted Myles Chief Operating Officer & Chief Financial Officer
Slide 18: Financial Update
© 2024 Scholar Rock, Inc. All rights reserved. Commercial Prep Underway • Continuing SMA stakeholder engagement and education • Ensuring an optimized treatment experience • Building team to deliver US & European launch Financial Update 18 High Sense of Urgency to Deliver for Patients Strong Balance
Slide 19: Building to Achieve Commercial Success
© 2024 Scholar Rock, Inc. All rights reserved. Building to Achieve Commercial Success 19 Geographic Expansion • Commercialize in selected European countries* • Remaining Europe and ROW expansion through distributorships & partnerships • Commercialize in selected European countries* • Remaining Europ
Slide 20: Path to Achieving Commercial Success in SMA
© 2024 Scholar Rock, Inc. All rights reserved. Path to Achieving Commercial Success in SMA 20 Clear unmet need and favorable market dynamics Competitive and attractive potential profile Engagement, patient focus & execution The right market The right medicine The right plan Apitegromab global
Slide 21: Upcoming Planned Key Milestones
© 2024 Scholar Rock, Inc. All rights reserved. 21 Upcoming Planned Key Milestones * If approved by relevant health authorities • Submit FDA and EMA applications in Q1 2025 • Request priority review (FDA) and accelerated assessment (EMA) Apitegromab Regulatory Submissions • US launch in Q4 2025 and E
Slide 22: Conclusions
© 2024 Scholar Rock, Inc. All rights reserved. Conclusions Jay Backstrom, M.D., MPH President & Chief Executive Officer 22
Slide 23: Our Mission
© 2024 Scholar Rock, Inc. All rights reserved. TGFβ=Transforming growth factor-beta. 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 We are a global leader in harnessing the l
Slide 24: Potential to Transform Standard of Care in SMA
© 2024 Scholar Rock, Inc. All rights reserved. 18 Potential to Transform Standard of Care in SMA 1.8-point improvement in HFMSE (p=0.0192) compared to placebo Patients improving on apitegromab vs. declining on placebo Favorable safety profile supports durability of treatment >48 months treatment exp
Slide 25: Q&A Session
© 2024 Scholar Rock, Inc. All rights reserved. Q&A Session 25
Slide 26: Company Speakers
© 2024 Scholar Rock, Inc. All rights reserved. 26 Company Speakers Jay Backstrom, M.D., MPH President & Chief Executive Officer Ted Myles, MBA Chief Operating Officer and Chief Financial Officer
Slide 27: Thank you!
© 2023 Scholar Rock, Inc. All rights reserved. 27 Thank you!