Scholar Rock updates 2023 pipeline priorities; Phase 3 SMA trial enrollment completion expected in 2023

Scholar Rock Holding Corp

Phase 3 SAPPHIRE trial enrollment to complete in 2023; top-line data expected in 2024, potential launch in 2025.
Cash, cash equivalents and marketable securities ~$315M as of Dec 31, 2022; runway into 2025.
36-month TOPAZ extension data expected H1 2023; 90% of patients remain enrolled in long-term extension.
Preclinical fibrosis and iron-restricted anemia programs advancing toward IND-enabling studies.
Jay Backstrom appointed President & CEO (Oct 2022); Jing Marantz appointed CMO (Nov 2022).

This headline and bullets were generated automatically by deepseek-v4-flash:cloud@v2 from the public filing. Read the source on SEC.gov before relying on any specific claim. Not investment advice. See methodology for how this pipeline works.