Scholar Rock(SRRK) - 2025 Q4 - Annual Results

Financial Position - Scholar Rock has a cash position of $365 million as of December 31, 2025, providing a cash runway into 2027[4] - As of December 31, 2025, Scholar Rock has approximately $365 million in cash and equivalents, providing a cash runway to support operations into 2027[30] Product Development and Launch - The anticipated U.S. launch of apitegromab is expected in 2026, pending FDA approval, with an EMA decision expected in mid-2026[4][9] - The Biologics License Application (BLA) for Apitegromab was submitted to the FDA in January 2025, with acceptance under priority review in March 2025, and a U.S. launch anticipated in 2026[15] - Scholar Rock is preparing for a significant market opportunity in Europe, with a launch readiness for Apitegromab ahead of the mid-2026 EMA decision[16] - The company plans to commercialize apitegromab for both children and adults with SMA, following regulatory approvals[9] - Scholar Rock aims to expand the development of apitegromab for patients with SMA under 2 years of age and for additional rare neuromuscular diseases, with a Phase 2 FORGE study initiation planned for mid-2026[31] - The company is progressing the Phase 2 OPAL study for SMA patients under 2 years of age, with an EMA decision expected in mid-2026 and an initial launch planned in Germany[31] Market Opportunity - Approximately 35,000 patients have received SMN-targeted therapies, indicating a significant market opportunity for apitegromab, which targets muscle atrophy in SMA[6][12] - The global opportunity for apitegromab in SMA alone is estimated to be $2 billion[4] - The estimated global revenue potential for Apitegromab is over $2 billion, with a demand for the first and only muscle-targeted therapy in SMA valued at approximately $5 billion[18] Clinical Trials and Efficacy - Scholar Rock is initiating a Phase 2 study for apitegromab in patients with facioscapulohumeral muscular dystrophy (FSHD) in mid-2026[4][9] - Apitegromab demonstrated a 30.0% improvement in HFMSE for patients compared to 12.5% on SMN2-targeted treatment alone, with a statistically significant mean change of +1.8 HFMSE points (p=0.0192) in a Phase 3 study[14] - Ongoing Phase 2 OPAL study is evaluating Apitegromab in infants and toddlers with SMA, addressing the needs of patients under 2 years of age[20] - The Phase 1 study of subcutaneous Apitegromab showed favorable bioavailability, with pharmacodynamic profiles comparable to intravenous administration[26] - SRK-439, a novel myostatin inhibitor, is in Phase 1 trials, with dosing commenced in December 2025 and topline data expected in H2 2026[28] - Scholar Rock is advancing its anti-myostatin pipeline, with SRK-439 currently in a Phase 1 study[9] - Scholar Rock is advancing its anti-myostatin pipeline, with a completed Phase 1 study for subcutaneous apitegromab and a Phase 1 study for SRK-439, with topline data expected in H2 2026[31] Strategic Focus - The company aims to expand its market presence to 50 countries, focusing on rare neuromuscular diseases[7] - Scholar Rock emphasizes financial discipline and focused execution as key priorities for 2026[9] - The company has established a U.S. commercial field team focused on engagement and education across the SMA stakeholder landscape, with over 2,600 SMA prescribers identified[16] - Scholar Rock is building a world-class team and expanding its physical presence in Zug and Dublin to enhance its market access and engagement with the SMA community[16] - Scholar Rock is expanding its pipeline to include Apitegromab for additional neuromuscular diseases, starting with facioscapulohumeral muscular dystrophy (FSHD)[24]