Scholar Rock Holding Corp Q3 FY2024 earnings call

• SAPPHIRE Phase 3 study in spinal muscular atrophy (SMA) met primary endpoints, enabling submission of BLA and MAA in Q1 2025 and planned commercial launch in US Q4 2025.
• EMBRAZE Phase 2 proof-of-concept with apitegromab completed enrollment ahead of schedule, targeting Q2 2025 for top-line results.
• SRK-439 non-clinical data on obesity and cardiometabolic disorders is ongoing.
• Added Beth Shafer as Chief Business Officer to guide investment decisions and partnering.
• Completed an upsized follow-on offering of $345 million, resulting in a cash balance of ~$463 million.
• Plans for OPAL trial in mid-2025 to explore apitegromab in combination with standard of care for patients under two years of age.

No detailed breakdown of product segments by absolute revenue and contribution % provided in the transcript.

• Targeting Q1 2025 for submitting BLA and MAA for apitegromab.
• Planned commercial launch of apitegromab in US Q4 2025 with Europe to follow upon regulatory approval.
• Intends to submit IND for SRK-439 mid-year 2025.
• OPAL trial in mid-2025 to explore apitegromab in combination for under-two SMA patients.

Q: Jenna Lee asked about differences between Lilly's trial and EMBRAZE study, and key parameters for EMBRAZE trial.

A: Jay responded that EMBRAZE is a 24-week proof-of-concept study with lean muscle mass as primary endpoint, using apitegromab, while Lilly's trial is exploring doses against tirzepatide.

Q: Etzer Darout inquired about OPAL study and obesity combination approaches.

A: Jay discussed OPAL study dosing based on SAPPHIRE data and obesity combination approaches focusing on preserving lean muscle mass.

Q: Gary Nachman asked about OPAL study dosing, payer discussions, and labeling.

A: Jay and Ted discussed OPAL study dosing based on PK/PD data, payer interactions being positive, and confidence in broad labeling due to consistent data.

Q: Alex Nugent asked about broader labeling including ambulatory status.

A: Jay stated FDA views SMA as a continuum, anticipating a broad label for apitegromab similar to existing treatments.