Scholar Rock (SRRK) Morgan Stanley 22nd Annual Global Healthcare Conference summary

Key clinical milestones and data readouts

• SAPPHIRE pivotal study data for apitegromab in SMA expected in Q4, with filing plans closely following data release.

• TOPAZ phase II extension shows sustained benefit over four years, with over 90% patient retention and meaningful improvements in Hammersmith and upper limb function.

• EMBRACE obesity study with apitegromab and GLP-1 is ahead of schedule, with a 100-patient, six-month readout now expected in Q2 2025.

• IND filing for SRK-439, a purpose-built obesity asset, targeted for mid-2025.

• Expansion plans include studies in SMA patients under two years old and broader neuromuscular indications.

Competitive landscape and differentiation

• SAPPHIRE is the only registrational SMA study informed by successful proof-of-concept data, targeting a broad age range.

• Scholar Rock's approach targets latent myostatin for high selectivity, reducing off-target effects compared to competitors like Biohaven and bimagrumab.

• Head-to-head preclinical data show SRK-439 matches bimagrumab efficacy at one-twentieth the dose.

• SRK-439 preserves lean mass and reduces fat regain after GLP-1 withdrawal, addressing key gaps in current obesity therapies.

Commercial and strategic outlook

• SMA market is valued at $4.5 billion, with significant unmet need for muscle-directed therapies alongside existing neuro-correctors.

• Payers are open to multiple therapies if data support patient value; most patients are already on background therapy.

• Commercialization plans focus on the US and Europe, with SAPPHIRE designed for global regulatory alignment.

• Obesity program may be partnered with a larger company for global reach, while SMA remains a core franchise.