Scholar Rock (SRRK) investor relations material

Executive summary

• FDA accepted the Biologics License Application (BLA) for apitegromab for SMA, with a PDUFA action date of September 30, 2026, and two independent fill-finish facilities included; EMA review is ongoing with a CHMP opinion expected mid-2026.

• Commercial launch preparations are advanced in both the U.S. and Europe, with teams ready to launch immediately upon approval and headquarters established in Switzerland.

• The company is progressing its anti-myostatin pipeline, including phase II and phase I studies for additional indications and formulations, such as the OPAL and FORGE trials and SRK-439.

• Pivotal Phase 3 SAPPHIRE trial for apitegromab met its primary endpoint in SMA patients.

• No revenue recorded for Q1 2026; net loss for Q1 2026 was $105.5 million, with an accumulated deficit of $1.4 billion as of March 31, 2026.

Financial highlights

• Ended Q1 2026 with $480 million in cash, cash equivalents, and marketable securities, expected to fund operations into 2027.

• Q1 operating expenses were $102 million, including $80 million in non-cash stock-based compensation; R&D expense was $51.8 million and G&A expense was $50.2 million.

• Net loss per share was $0.83 for Q1 2026, compared to $0.67 for Q1 2025.

• Raised $100 million from a debt facility and $98 million from an ATM program during Q1 2026.

• Cash flow from financing activities was $193.8 million, primarily from ATM equity sales and new debt facility.

Outlook and guidance

• Anticipates apitegromab approval in the U.S. by or before September 30, 2026, with commercial supply ready in early Q3; EMA decision anticipated mid-2026 and European launch planned for the second half of 2026, starting in Germany.

• Existing cash resources expected to fund operations into 2027; additional capital will be needed for full commercialization and pipeline development.

• Plans to monetize a priority review voucher and may draw an additional $150 million from the debt facility upon FDA approval.

• Continued investment in SMA and FSHD clinical programs, with Phase 2 FORGE trial in FSHD to initiate mid-2026.