Scholar Rock Holding Corporation · NASDAQ:SRRK
Scholar Rock — Q2 2024 Earnings Presentation
31 slides from Scholar Rock's earnings presentation, Aug 8, 2024
Slide 1: Second Quarter 2024 Business Update
© 2024 Scholar Rock, Inc. All rights reserved. Second Quarter 2024 Business Update August 8, 2024 1
Slide 2: Forward-Looking Statements
© 2024 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: Introduction & Business Update
© 2024 Scholar Rock, Inc. All rights reserved. Introduction & Business Update Jay Backstrom, M.D., MPH President & Chief Executive Officer 3
Slide 4: Today's Agenda
© 2024 Scholar Rock, Inc. All rights reserved. Today’s Agenda 4 Topic Speaker Introduction & Business Update Jay Backstrom, President & Chief Executive Officer Development Update Jing Marantz, Chief Medical Officer Pipeline Update Mo Qatanani, Chief Scientific Officer Upcoming Milestones Jay
Slide 5: Selectivity is the Key
© 2024 Scholar Rock, Inc. All rights reserved. Selectivity is the Key The hallmark of our differentiated platform is unparalleled selectivity 1 Productive Platform Strong progress & momentum supports SRRK’s scientific approach, capability to grow pipeline and ability to execute 2 Excellent Opportuni
Slide 6: Our Approach: Selectivity Drives Success
© 2024 Scholar Rock, Inc. All rights reserved. Traditional Target “mature” active growth factor Scholar Rock’s Target L Latent Growth Factor Complex 6 Our Approach S e l e c t i v i t y D r i v e s S u c c e s s Industry-leading antibody design and protein engineering to selectively target latent gr
Slide 7: Scientific Platform Yielding Growing Pipeline Across High Value Therapeutic Areas
© 2024 Scholar Rock, Inc. All rights reserved. Scientific Platform Yielding Growing Pipeline Across High Value Therapeutic Areas 7 *Utilized data from previously completed Ph 1 study in healthy volunteers and initiate a Ph 2 POC trial in 2024. LTBP1=Latent transforming growth factor beta binding pro
Slide 8: High Value Growth Opportunities
Upcoming SAPPHIRE readout - Q4 2024 Initiate study in SMA patients < 2 years old: Planned for 2025 Exploring additional neuromuscular populations Ph 2 POC EMBRAZE study enrolling: Topline data expected in Q2 2025 Advancing SRK-439 to IND Established PoC with SRK-181 in multiple advanced solid tumors
Slide 9: Cutting-Edge Research Recognized by Global Scientific Community
© 2024 Scholar Rock, Inc. All rights reserved. 9 Cutting-Edge Research Recognized by Global Scientific Community Promising objective response rates, safety, and novel biomarker data highlighted in oral presentation at ASCO SRK-181 Featured on the cover of Science Signaling and in Focus article: SRK-
Slide 10: 2024 & 2025 Anticipated Milestones
© 2024 Scholar Rock, Inc. All rights reserved. 10 2024 & 2025 Anticipated Milestones Milestones 2024 2025 SRK-181 data at ASCO • Oral presentation June 3 • Developmental Therapeutics-Immunotherapy SRK-439 data at American Diabetes Association • Oral presentation June 23 • New Insights into Therapeut
Slide 11: Development Update
© 2024 Scholar Rock, Inc. All rights reserved. Development Update Jing Marantz, M.D., Ph.D. Chief Medical Officer
Slide 12: Why We Are Confident: Apitegromab for Spinal Muscular Atrophy
© 2024 Scholar Rock, Inc. All rights reserved. 12 Why We Are Confident A P I T E GR O M A B F O R S P I NA L M U S C U L A R A T RO P H Y Selective muscle-targeting designed to improve motor function while minimizing off-target effects >90% patient retention,1 well tolerated profile supports durabil
Slide 13: CHERISH/SHINE study: Long-Term Results of Nusinersen-Treated Patients
© 2024 Scholar Rock, Inc. All rights reserved. S PI N AL M U S C U LA R A T R O P HY 13 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 CHERISH Study Baseline Nusinerse
Slide 14: TOPAZ: Motor Function Outcomes by HFMSE Over 48 Months
© 2024 Scholar Rock, Inc. All rights reserved. 3.4 4.6 5.2 4.8 6.4 0 1 2 3 4 5 6 7 8 9 Baseline 6 months 12 months 24 months 36 months 48 months Apitegromab TOPAZ Clinical Trial: Motor Function Outcomes by HFMSE Over 48 Months Improvements Were Substantial and Sustained 1. For the 48-month evaluatio
Slide 15: TOPAZ: Motor Function Outcomes by RULM Over 48 Months
© 2024 Scholar Rock, Inc. All rights reserved. 0.2 1.2 2.2 2.8 4.5 0 1 2 3 4 5 6 Baseline 6 Months 12 Months 24 Months 36 Months 48 Months 0.6 1.3 2.3 2.4 3.6 0 1 2 3 4 5 6 Baseline 6 Months 12 Months 24 Months 36 Months 48 Months Apitegromab TOPAZ Clinical Trial: Motor Function Outcomes by RULM Ove
Slide 16: Summary of TOPAZ Data
© 2024 Scholar Rock, Inc. All rights reserved. Substantial and Sustained Improvement over 48 MONTHS1 1- A total of 11 patients in the population had scoliosis surgery during the study and their data was excluded from any HFMSE and RULM assessments at 48 months. PRO=Patient Reported Outcome *Pooled n
Slide 17: SAPPHIRE Trial Designed for Clinical Success
© 2024 Scholar Rock, Inc. All rights reserved. SAPPHIRE Trial Designed for Clinical Success 17 Randomized, double-blind, placebo-controlled, parallel arm design (n=204) Enrolling patients who are on SMN-directed therapy (nusinersen or risdiplam) Completed enrollment in Q3 2023 TREATMENT (52 weeks) A
Slide 18: Goals of the EMBRAZE Proof-of-Concept Study
© 2024 Scholar Rock, Inc. All rights reserved. Goals of the EMBRAZE Proof-of-Concept Study C A R D I O M E T A B O L I C D E V E L O P M E NT P R O G R A M IN SI GHT S GA INE D F R OM E MB R A ZE S T UD Y to inform SRK-439 development Study Aims to Demonstrate O B E S I T Y Effect of apitegromab to
Slide 19: Enrolling Phase 2 Proof-of-Concept Study of Apitegromab in Obesity
© 2024 Scholar Rock, Inc. All rights reserved. Enrolling Phase 2 Proof-of-Concept Study of Apitegromab in Obesity 19 E M B R A Z E TR I A L D E S I G N Randomized, double-blind, placebo-controlled (n=100) Enrolling patients who are overweight or obese Enrollment ahead of schedule; topline data expec
Slide 20: Pipeline Update
© 2024 Scholar Rock, Inc. All rights reserved. Pipeline Update Mo Qatanani, Ph.D. Chief Scientific Officer
Slide 21: Platform and Expertise Drive Success in Clinic
© 2024 Scholar Rock, Inc. All rights reserved. Platform and Expertise Drive Success in Clinic 21 Superb Engineering Clinic SRK-439 Cardiometabolic IND planned for 2025 SRK-181 Immuno-oncology T R A N S L A T IO NA L S U C C ES S TO TH E C L I N I C Apitegromab Spinal Muscular Atrophy Translational M
Slide 22: Preclinical Data to Date Show Strong Potential in Obesity
© 2024 Scholar Rock, Inc. All rights reserved. 22 O B E S I T Y 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 metabolic parameters Increase in lean mass an
Slide 23: SRK-439 Increased Lean Mass and Attenuated Regain of Fat Mass After GLP-1 RA Withdrawal
© 2024 Scholar Rock, Inc. All rights reserved. 23 SRK-439 Increased Lean Mass and Attenuated Regain of Fat Mass After GLP-1 RA Withdrawal in Obesity Mouse Model Study conducted in Diet Induced Obesity (DIO) mouse model utilizing a murine chimera of SRK-439 GLP-1 RA: GLP1 receptor agonist. Day 7 is s
Slide 24: SRK-439 Improved Body Composition After GLP-1 RA Withdrawal
© 2024 Scholar Rock, Inc. All rights reserved. 24 SRK-439 Improved Body Composition After GLP 1-RA Withdrawal Study conducted in Diet Induced Obesity (DIO) mouse model utilizing a murine chimera of SRK-439 GLP-1 RA: GLP1 receptor agonist. HFD: High Fat Diet • SRK-439 attenuates regain of fat mass af
Slide 25: SRK-439 Is More Potent than Anti-ActRII Antibody at Maintaining Lean Mass
© 2024 Scholar Rock, Inc. All rights reserved. SRK-439 Is More Potent than Anti-ActRII Antibody at Maintaining Lean Mass During GLP-1 RA-Induced Weight Loss 25 L O W E FF I C A C I O U S D O S E A N D C O M P E T IT I V E P R O F IL E Head-to-Head Comparison to Non-Selective Myostatin Inhibitor in D
Slide 26: Leveraging Apitegromab to Inform Obesity Program
© 2024 Scholar Rock, Inc. All rights reserved. Leveraging Apitegromab to Inform Obesity Program 26 P IP E L I N E O V E R VI E W 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 IN
Slide 27: Upcoming Milestones
© 2024 Scholar Rock, Inc. All rights reserved. Upcoming Milestones Jay Backstrom Chief Executive Officer
Slide 28: Looking Ahead: Upcoming Milestones
© 2024 Scholar Rock, Inc. All rights reserved. 28 Looking Ahead: Upcoming Milestones IND=Investigational new drug; POC=Proof of concept; Q2 2024 ACCOMPLISHMENTS PRESENTED encouraging SRK-181 data at ASCO INITIATED EMBRAZE trial with apitegromab in obesity 2H 2024 focus SAPPHIRE Readout in Q4 Complet
Slide 29: Q&A Session
© 2024 Scholar Rock, Inc. All rights reserved. Q&A Session 29
Slide 30: Company Speakers
© 2024 Scholar Rock, Inc. All rights reserved. 30 Company Speakers Jay Backstrom, M.D., MPH President & Chief Executive Officer Jing Marantz, M.D., Ph.D. Chief Medical Officer Ted Myles, MBA Chief Operating Officer and Chief Financial Officer Mo Qatanani, Ph.D. Chief Scientific Officer
Slide 31: Thank you!
© 2023 Scho lar Rock, Inc. All rights reserved. 31 Thank you!