Scholar Rock Targets $2 Billion Apitegromab Revenue Amid SMA Market Growth

Scholar Rock Holding Corporation (SRRK) is setting its sights on a significant revenue milestone for its investigational muscle-targeted therapy, apitegromab, as the company anticipates a $2 billion revenue target for the drug in the spinal muscular atrophy (SMA) market. With positive topline data from the Phase 3 SAPPHIRE trial and a strong pipeline, Scholar Rock is poised to capture a substantial share of the growing SMA market.

The $2 billion revenue target for apitegromab is supported by several key factors, including regulatory approval and commercial launch, expansion of the drug's potential benefit beyond SMA, and positive clinical trial results. Scholar Rock is on track to submit a biologics license application (BLA) to the FDA and a marketing authorisation application (MAA) to the European Medicines Agency in 1Q 2025 for apitegromab in SMA. If approved, the U.S. launch is expected in 4Q 2025, with the European launch to follow. This regulatory approval and subsequent commercial launch will significantly contribute to the projected revenue.

Scholar Rock is also exploring the development of apitegromab in other rare neuromuscular disorders, such as Duchenne muscular dystrophy, Becker muscular dystrophy, Facioscapulohumeral muscular dystrophy, and amyotrophic lateral sclerosis, to build a growing neuromuscular franchise. Additionally, the company is planning to initiate a Phase 2 OPAL clinical trial in SMA patients under two years of age in mid-2025, further expanding the drug's potential market. These expansion efforts can potentially increase the addressable market and patient population for apitegromab, driving revenue growth.

Positive clinical trial results, such as the recent topline data from the Phase 3 SAPPHIRE trial, which showed that apitegromab met its primary endpoint and demonstrated a statistically significant and clinically meaningful improvement in motor function over placebo, will likely support the drug's approval and commercial success. The presentation of detailed results from the SAPPHIRE trial at upcoming medical meetings in 2025 can further validate the drug's efficacy and safety, enhancing its market potential.

Scholar Rock believes that its existing cash, cash equivalents, and marketable securities will be sufficient to fund the company's anticipated operating expenses and capital expenditure requirements into the fourth quarter of 2026. This financial stability ensures that the company has the resources to invest in the development and commercialization of apitegromab, supporting its revenue projections.



In conclusion, Scholar Rock's ambitious $2 billion revenue target for apitegromab in the SMA market is supported by a strong pipeline, positive clinical trial results, and a growing addressable market. As the company continues to invest in research and development and execute its commercialization strategy, it is well-positioned to capture a significant share of the SMA market and deliver value to shareholders.