Scholar Rock Sets Sights on $2 Billion Apitegromab Revenue Amid Spinal Muscular Atrophy Market Growth

Core Insights - Scholar Rock Holding Corporation is focusing on the development of apitegromab for spinal muscular atrophy (SMA), with discussions at the J.P. Morgan Healthcare Conference highlighting its potential market and adoption factors [1][4]. Group 1: Product Development and Trials - The company plans to submit U.S. and European marketing applications for apitegromab in Q1 2025, targeting a U.S. launch in Q4 2025 and a subsequent European launch [7]. - Upcoming results from the Phase 2 EMBRAZE trial, evaluating apitegromab in combination with tirzepatide or semaglutide for overweight or obese adults, are expected in Q2 2025 [3]. - Detailed results from the Phase 3 SAPPHIRE trial, involving non-ambulatory type 2/3 SMA patients, will be presented at medical meetings in 2025 [7]. Group 2: Market Potential and Financial Projections - The current SMA market is estimated at approximately $4.5 billion across three SMN-targeted therapies, primarily for the 2-21 age group [7]. - Scholar Rock expects apitegromab to generate over $2 billion in global revenue, an increase from previous estimates of $1 billion, due to strong data and a favorable competitive landscape [7]. - JP Morgan maintains a positive outlook on Scholar Rock, anticipating a strong product launch and further value if cardiometabolic data is favorable, with initial results expected in Q2 2025 [4]. Group 3: Stock Performance - Scholar Rock's stock (SRRK) has seen an increase of 4.89%, reaching $43.56 [5].