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Press Release

June 11, 2021

Scholar Rock Presents TOPAZ Phase 2 Data Showing the Transformative Potential of Apitegromab in Patients with Type 2 and 3 Spinal Muscular Atrophy (SMA) at the 2021 Virtual SMA Research & Clinical Care Meeting
- Majority (74%) of patients with non-ambulatory Type 2 and Type 3 SMA achieved a clinical improvement in Hammersmith Functional Motor Scale Expanded (HFMSE) after 12 months - New exploratory analysis of TOPAZ data found no correlation between duration of prior nusinersen treatment and increases in
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April 6, 2021

Scholar Rock Announces Positive 12-Month Top-Line Results From the TOPAZ Phase 2 Clinical Trial Evaluating Apitegromab in Patients With Type 2 and Type 3 Spinal Muscular Atrophy (SMA)
- Data further demonstrate proof-of-concept for the therapeutic potential of apitegromab in patients with Type 2 and Type 3 SMA - Phase 3 registrational trial initiation expected by the end of 2021 - European Medicines Agency (EMA) granted Priority Medicines (PRIME) designation to apitegromab,
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