Yahoo Finance

• Clinical Success: Apitegromab plus standard of care delivered a 1.8-point improvement compared to placebo in the SMA-specific Hammersmith functional motor scale at week 52.

• Patient Improvement: 30% of patients achieved an additional 3-point or greater improvement in Hammersmith scores compared to 12.5% with placebo plus SMN therapy.

• Safety Profile: Safety consistent with TOPAZ data; over 95% of patients rolled over and remained in long-term follow-up study.

• Market Potential: Approximately two-thirds of 10,000 SMA patients in the US and 35,000 globally have received SMN-targeted therapy.

• Commercial Preparation: Scaling customer-facing team to roughly 50 personnel in late Q2 and early Q3 for potential Q4 launch.

• Cardiometabolic Program: Top-line data from EMBRAZE expected in Q2; IND filing for SRK439 in Q3.

• Lean Muscle Preservation: Aim to reduce lean muscle mass loss by 20% to 40% in EMBRAZE study.

• Regulatory Milestones: BLA for apitegromab submitted in the US in January; MAA submission in the EU on track for March.

• Warning! GuruFocus has detected 2 Warning Sign with SRRK.

Release Date: February 27, 2025

For the complete transcript of the earnings call, please refer to the full earnings call transcript.

Positive Points

• Scholar Rock Holding Corp (NASDAQ:SRRK) is on track to launch apitegromab, a muscle-targeted therapy for SMA, in the US in 2025, with plans to expand to the EU in 2026.

• The SAPPHIRE study showed that apitegromab plus standard of care delivered clinically meaningful and statistically significant improvements in motor function for SMA patients.

• The company is making significant progress in its cardiometabolic program, with plans to share top-line data from the EMBRAZE study in Q2 2025.

• Scholar Rock Holding Corp (NASDAQ:SRRK) has a strong commercial strategy, including the 'Life Takes Muscle' campaign, to prepare the market for the new treatment paradigm in SMA.

• The company is actively engaging with payers and stakeholders to ensure access and coverage for apitegromab, reflecting its value in treating SMA.

Negative Points

• There is still uncertainty around the regulatory acceptance of body composition changes as a primary endpoint for the cardiometabolic program.

• The competitive landscape for weight loss therapies is intensifying, which may impact the commercial success of SRK-439.

• The company has not yet disclosed pricing details for apitegromab, which could affect market acceptance and payer negotiations.

• The development of additional neuromuscular indications for apitegromab is still in the early stages, with no specific timelines provided.

• The potential impact of preserving lean muscle mass on overall weight loss with GLP-1 receptor therapies remains uncertain, which could affect the perceived value of SRK-439.