Scholar Rock (SRRK) Corporate presentation summary

Strategic focus and pipeline advancement

• Preparing for U.S. launch of apitegromab in 2026, with European expansion starting in Germany and plans for global reach across 50 countries.

• Apitegromab is the first and only muscle-targeted therapy to show statistically significant, clinically meaningful benefit in SMA, with a successful Phase 3 study and BLA accepted by FDA; PDUFA action date set for September 30, 2026.

• Ongoing and planned studies include Phase 2 OPAL for infants and toddlers with SMA, and Phase 2 FORGE for facioscapulohumeral muscular dystrophy (FSHD), with additional rare neuromuscular disease indications under evaluation.

• Advancing a world-leading anti-myostatin pipeline, including subcutaneous apitegromab and SRK-439, a novel, highly potent myostatin inhibitor optimized for subcutaneous administration.

• $480M in cash and equivalents as of March 31, 2026, supporting commercial launch readiness, supply chain strengthening, and clinical program advancement.

Clinical and commercial highlights

• Apitegromab demonstrated a statistically significant improvement in motor function for SMA patients in Phase 3, with 30% achieving ≥3-point HFMSE improvement versus 12.5% on standard of care alone.

• Two independent fill-finish facilities included in BLA resubmission provide dual paths to FDA approval and ensure commercial supply readiness.

• U.S. commercial team is prepared for immediate launch upon approval, targeting 140 SMA centers and over 2,600 prescribers.

• European launch readiness includes building a world-class team, engaging the SMA community, and establishing access ahead of mid-2026 CHMP opinion.

• Apitegromab global revenue potential estimated at over $2B, with the SMA market for SMN-targeted therapies exceeding $5B in 2025.

Expansion into additional indications

• Phase 2 OPAL study underway for infants and toddlers with SMA, aiming to address needs of patients under 2 years and those who received SMN1-targeted gene therapy.

• Phase 2 FORGE study in FSHD to initiate mid-2026, targeting a rare disease with over 30,000 diagnosed patients in the U.S. and Europe and no approved therapies.

• Preclinical studies in FSHD models show increased muscle mass, strength, and endurance with apitegromab.

• Broad pipeline-in-a-product strategy includes potential expansion into other rare neuromuscular diseases such as ALS, DMD, LGMD, and metabolic myopathies.

• SRK-439 Phase 1 study in healthy volunteers is ongoing, with topline data expected in H2 2026.