Scholar Rock Holding Corp's (SRRK) stock soared 5.06% on Friday, following the company's promising fourth-quarter 2024 earnings call providing updates on its lead drug candidate apitegromab for the treatment of spinal muscular atrophy (SMA).

The key highlights that drove the stock's rally include:

• Positive top-line results from the pivotal Phase 3 SAPPHIRE trial evaluating apitegromab, the first muscle-targeted therapy for SMA, in combination with standard-of-care SMN-directed therapies. The trial met its primary endpoint, demonstrating clinically meaningful and statistically significant improvements in motor function compared to placebo.
• Scholar Rock submitted the Biologics License Application (BLA) for apitegromab in the U.S. in January and remains on track to submit the Marketing Authorization Application (MAA) in the European Union (EU) in March, setting the stage for potential commercial launches in late 2025 and 2026, respectively.
• The company is actively preparing for the U.S. launch, including engaging with commercial and federal payers, building out its customer-facing team, and offering patient services like home infusion.

Additionally, Scholar Rock provided updates on its cardiometabolic programs, including the ongoing Phase 2 EMBRAZE trial evaluating apitegromab in combination with a GLP-1 receptor agonist for weight management, and the planned Investigational New Drug (IND) filing for its novel anti-myostatin candidate SRK-439 in the third quarter of 2025.