Scholar Rock (SRRK) Goldman Sachs 47th Annual Global Healthcare Conference 2026 summary

Company overview and recent achievements

• Leader in myostatin biology, with a unique platform and the only successful late-stage myostatin inhibitor program for SMA, highlighted by a positive phase III trial for apitegromab.

• Apitegromab demonstrated statistically significant and clinically meaningful motor function improvements in SMA patients, with regulatory filings underway in the US and Europe and anticipated launches in 2026.

• Plans to commercialize apitegromab in up to 50 countries, targeting a global SMA patient population of approximately 35,000.

Pipeline and clinical development

• Phase II trial for apitegromab in FSHD to begin after mid-year, focusing on lean muscle volume and functional outcomes, with results expected in 2027 or early 2028.

• Ongoing OPAL phase II study for SMA patients under age two, aiming to expand the label after approval based on PK/PD and safety data.

• Advancing a subcutaneous version of apitegromab and SRK-439, a new molecular entity, with healthy volunteer data expected by end of 2026.

Market positioning and patient eligibility

• Apitegromab addresses the muscle component of SMA, complementing SMN-targeted therapies, and is positioned for broad eligibility across SMA phenotypes and ages two and above.

• Surveys indicate 95% of SMA patients and three-quarters of physicians see muscle strength as the top unmet need, supporting strong demand.