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

Scholar Rock Holding Corporation SRRK President & CEO Jay Backstrom presented at the J.P. Morgan Healthcare Conference.

The discussions mainly focused on apitegromab for spinal muscular atrophy (SMA), including its label if approved. They also covered commercial aspects such as the prevalence of SMA, the potential market, and factors influencing its adoption.

Also Read: Scholar Rock’s Muscle Wasting Drug Excels In Pivotal Trial, Plans FDA Submission Next Year

• Regarding apitegromab in SMA, the company plans to submit U.S. and European marketing applications in the first quarter of 2025, aiming for a potential U.S. launch in the fourth quarter of 2025, followed by a European launch.
• Detailed results from the phase 3 SAPPHIRE trial, which involves patients with non-ambulatory type 2/3 SMA treated with Spinraza (nusinersen) or Evrysdi (risdiplam), are expected to be presented at medical meetings in 2025.
• By mid-2025, the company aims to start the phase 2 OPAL trial, focusing on patients under 2 years old to understand how the drug behaves and guide dosing.
• Regarding the addressable market, the company estimates the current SMA market at around $4.5 billion across three SMN-targeted therapies, mainly in the 2-21-year-old age group. They now expect apitegromab to reach over $2 billion in global revenue, an increase from previous estimates of $1 billion, driven by strong data and a favorable competitive landscape.

The analyst also discussed the upcoming results from the Phase 2 EMBRAZE trial, which is evaluating apitegromab combined with tirzepatide or semaglutide in adults who are overweight or obese. The data is expected in the second quarter of 2025.

JP Morgan remains positive on Scholar Rock (Overweight rating), as the recent SAPPHIRE trial results have unlocked strong growth potential for apitegromab in spinal muscular atrophy. The company could enter the commercial stage by 2025, and the analyst expects a strong product launch.

The analyst also sees potential for further value in the stock if the cardiometabolic data shows positive results, with initial data expected in the second quarter of 2025.

Price Action: SRRK stock is up 4.89% at $43.56 at last check Wednesday.

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