Scholar Rock Holding Corporation · NASDAQ:SRRK

Scholar Rock — Technical Presentation (October 2025)

6 slides from Scholar Rock's technical presentation, October 2025

  1. Slide 1: Efficacy and safety of apitegromab in patients aged 13-21 with SMA: SAPPHIRE

    Efficacy and safety of apitegromab in patients aged 13-21 years with type 2 or 3 spinal muscular atrophy: outcomes from the SAPPHIRE phase 3 trial Andreea M. Seferian Institut de Myologie, I-Motion Clinical Trials Platform, Paris, France; Hôpital Armand Trousseau, Paris, France

  2. Slide 2: Disclosure

    Disclosure • I have the following conflict/s of interest to declare: • I am a principal investigator of the Scholar Rock, Inc., – sponsored Phase 3 SAPPHIRE trial, a paid consultant for Scholar Rock, Inc., and have received personal compensation from Audentes and Biogen

  3. Slide 3: Phase 3 SAPPHIRE trial design

    Phase 3 SAPPHIRE trial design Randomized, double-blind, placebo-controlled, parallel-arm design (n = 188) SCREENING Treatment (52 weeks) 2–12 POPULATION (n = 156) n=53 Apitegromab (20 mg/kg IV Q4W) + SMN-targeted treatment Nonambulatory SMA type 2/3 Stratification factors 1. Age at SMN-targeted trea

  4. Slide 4: Primary endpoint met with consistency across doses and age groups

    Primary endpoint met with consistency across doses and age groups LS mean change from baseline in HFMSE total score by visit (2–21 population) 2.0 LS mean (± SE) change from baseline in HFMSE 1.0 1.1 ± 0.40 Change from baseline in HFMSE total score at 0.0 month 12 for predefined populations -0.7 ± 0

  5. Slide 5: Well-tolerated safety consistent with established profile

    Well-tolerated safety consistent with established profile 2–21 pooled population 13–21 population Placebo + Apitegromab + Placebo + Apitegromab 20 mg/kg + SMN-targeted treatment SMN-targeted treatment SMN-targeted treatment SMN-targeted treatment Summary of AEs, n (%) (n = 60) (n = 128) (n = 10) (n

  6. Slide 6: Conclusions

    Conclusions • Apitegromab, an investigational, muscle-targeted treatment, resulted in clinically meaningful improvements1-3 in motor function in the 2–21 pooled and 13–21 SAPPHIRE populations4 − Efficacy results were consistent across outcome measures (ie, HFMSE, RULM, and WHO; shown in poster 426P)

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