Regulatory and Commercial Progress for Apitegromab
The BLA for apitegromab has been accepted under priority review with a target action date of September 22. The company is preparing for a U.S. launch for SMA (spinal muscular atrophy) treatment and anticipates a European launch, starting with Germany in 2026.
Positive Clinical Trial Results
The SAPPHIRE trial showed statistically significant improvement in motor function for SMA patients treated with apitegromab. The EMBRAZE study met its primary endpoint, showing that apitegromab increased lean mass preservation by over 54% compared to tirzepatide alone.
Financial Stability and Strategic Investments
The company ended the quarter with $295 million. An additional $50 million is available under a debt facility, with a projected financial runway into 2027. The company is strategically investing in commercial readiness and pipeline expansion.
Global Launch Preparedness
Scholar Rock has fully staffed and deployed a U.S. customer-facing team to engage with SMA treatment centers, key opinion leaders, and payers. The company plans to reach patients globally, with an emphasis on targeting the unmet need for treatments that address muscle degeneration in SMA.