Quarterly ResultMay 7, 2026, 07:13 AM
Scholar Rock's Apitegromab BLA Accepted by FDA; Q1 Net Loss $105.5M
AI Summary
Scholar Rock announced its Q1 2026 financial results and significant business updates. The FDA accepted the Biologics License Application (BLA) for apitegromab for spinal muscular atrophy (SMA), setting a PDUFA action date of September 30, 2026. The company reported a net loss of $105.5 million for the quarter, with cash, cash equivalents, and marketable securities totaling $479.9 million as of March 31, 2026. Preparations for a U.S. commercial launch are underway, with European regulatory review also progressing.
Key Highlights
- FDA accepted apitegromab BLA for SMA, with September 30, 2026 PDUFA action date.
- Q1 2026 net loss was $105.5 million ($0.83 per share), compared to $74.7 million ($0.67 per share) in Q1 2025.
- Cash, cash equivalents, and marketable securities totaled $479.9 million as of March 31, 2026.
- Research and development expense rose to $51.8 million in Q1 2026 from $48.7 million in Q1 2025.
- General and administrative expense increased to $50.2 million in Q1 2026 from $28.4 million in Q1 2025.
- EMA CHMP opinion for apitegromab anticipated near mid-2026; Europe launch planned H2 2026.
- Phase 2 FORGE trial for FSHD on track to initiate in mid-2026.
- SRK-439 Phase 1 healthy volunteer study topline data expected in H2 2026.