Scholar Rock(SRRK) - 2025 Q2 - Earnings Call Presentation

Apitegromab in SMA - Positive Phase 3 trial data showed a 18 point improvement versus placebo in Hammersmith Functional Motor Scale-Expanded (HFMSE) in SMA patients[23] - 30% of apitegromab patients achieved ≥3-point improvement in HFMSE compared to 125% of placebo patients[23] - FDA accepted BLA under Priority Review with a target action date of September 22, 2025[25] - MAA Validated with anticipated EU approval in 2026[14] - The company is initiating Phase 2 OPAL Trial in Q3 to study apitegromab in patients under 2 years old[14, 27] Pipeline Expansion - EMBRAZE trial achieved primary endpoint with positive Phase 2 proof-of-concept in obesity[14] - Apitegromab combined with tirzepatide resulted in a 549% reduction in lean mass loss versus tirzepatide alone[36] - The company is on track to file IND for SRK-439 in 2H 2025[14, 39, 43] Financial Status - The company had $295 million cash as of June 30, 2025[14]