Scholar Rock — Third Quarter 2026 Earnings Summary

Financial Performance:

• Reported net loss of $107.2 million for the quarter ended September 30, 2026, compared to a net loss of $102.2 million for the same period in 2025.
• Net loss per common share was $0.81 for the quarter ended September 30, 2026, compared to $0.90 per share in 2025.
• Recorded net product revenue of $0.6 million from U.S. sales of ISEMBYLD, with no revenue recorded in the prior year quarter.
• Cost of revenue was immaterial ($8,000) for the quarter ended September 30, 2026, compared to zero in 2025.
• Research and development expense was $54.1 million for the quarter, an increase from $50.5 million in 2025.
• Selling, general and administrative expense was $52.5 million for the quarter, down from $53.1 million in 2025.
• Cash, cash equivalents, and marketable securities totaled $413.6 million as of September 30, 2026, up from $367.6 million as of December 31, 2025.
• Total current liabilities were $80.1 million as of September 30, 2026, compared to $55.4 million as of December 31, 2025.
• Total liabilities were $277.3 million as of September 30, 2026, compared to $158.8 million as of December 31, 2025.

Guidance and Future Outlook:

• Expects a Committee for Medicinal Products for Human Use (CHMP) opinion on the apitegromab Marketing Authorisation Application (MAA) in Europe in the first half of 2027.
• Anticipates Japan's Pharmaceuticals and Medical Devices Agency (PMDA) to complete priority review of the Japanese New Drug Application (JNDA) in approximately nine months.
• Plans to submit an ISEMBYLD-specific J-code application to CMS effective April 1, 2027.
• Expects to have topline data from the Phase 1 SRK-439 study by the end of 2026, with plans to share data in early 2027.
• Plans to expand access to ISEMBYLD globally in up to 50 countries, subject to regulatory approvals.

Business Segments and Product Lines:

• U.S. launch of ISEMBYLD (apitegromab-mstn), the first muscle-targeted therapy for spinal muscular atrophy (SMA), is underway following FDA approval on September 11, 2026.
• ISEMBYLD vials were shipped to the specialty network prior to quarter-end, with initial patient infusions occurring in October 2026.
• The apitegromab MAA for SMA was resubmitted to the European Medicines Agency (EMA) in October 2026 and validated, utilizing an alternate fill-finish facility.
• The apitegromab JNDA for SMA was submitted to Japan and accepted under priority review, with Orphan Drug Designation granted.
• Phase 2 OPAL trial for apitegromab in infants and toddlers with SMA is advancing with enrollment well advanced and dosing ongoing.
• Phase 2 FORGE trial for apitegromab in Facioscapulohumeral Muscular Dystrophy (FSHD) is ongoing, enrolling approximately 60 patients; FDA granted Fast Track and Orphan Drug designations in September 2026.
• Phase 1 study for SRK-439, a subcutaneous myostatin inhibitor, is progressing in healthy volunteers.

Market and Competitive Landscape:

• ISEMBYLD is the first and only muscle-targeted therapy for SMA indicated for adults and children two years of age and older currently receiving SMN2-targeted treatment.
• Early launch dynamics show strong, broad-based demand across SMA treatment centers, physicians, and patients.
• The company is engaging with national and regional payers regarding reimbursement.

Risks and Challenges:

• Forward-looking statements note risks regarding the sufficiency of clinical data to support regulatory approval or further development.
• Risks include the ability to successfully commercialize ISEMBYLD, achieve market acceptance, and obtain adequate coverage and reimbursement.
• Potential delays or failures in regulatory reviews for apitegromab in the EU and Japan are noted.
• Risks exist regarding the ability to maintain sufficient commercial supply and the satisfaction of closing conditions for the Priority Review Voucher (PRV) sale.
• Dependence on third parties for development and manufacturing of product candidates is highlighted.

Management Commentary and Tone:

• Chairman and CEO David L. Hallal described the quarter as a "defining period" marked by FDA approval and U.S. launch.
• Management expressed encouragement regarding early launch dynamics and reinforced demand across the SMA community.
• Leadership stated the company is building a foundation to redefine neuromuscular disease treatment globally and drive sustainable growth.

Other Key Points:

• In November, the company reached a definitive agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $205 million, with cash to be received upon closing.
• The PRV was awarded following the September 2026 FDA approval of ISEMBYLD.
• The PRV transaction is subject to customary closing conditions, including the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act.
• The company will host a conference call and webcast on November 10 at 8:00 a.m. ET to review results.