Scholar Rock Holding Corp. (NASDAQ:SRRK) is one of the best multibagger stocks to invest in right now. The company’s stock’s performance was largely supported by optimism around its lead drug candidate, apitegromab. The stock surged nearly sixfold in 2024, but as the company approaches a potential U.S. market launch in Q3 2025, volatility has increased. Shares are down approximately 25% year-to-date, although market sentiment remains highly positive, with a consensus Buy rating.
Why is apitegromab viewed as a potential game changer? Scholar Rock Holding Corp. (NASDAQ:SRRK) highlights that it is the first and only muscle-targeted therapy to show clinically meaningful and statistically significant functional improvement in spinal muscular atrophy (SMA), and the only anti-myostatin therapy to demonstrate such results in a pivotal Phase 3 trial. Scholar Rock Holding Corp. (NASDAQ:SRRK) estimates the global market opportunity for apitegromab in SMA could exceed $2 billion, highlighting its strong commercial potential.
Recent analyst commentary suggests that bullish expectations for Scholar Rock Holding Corp. (NASDAQ:SRRK) are likely to continue. On August 21, Jefferies analyst Amy Li initiated coverage on the stock with a Buy rating and a $50 price target. Li views apitegromab, the company’s lead drug candidate, as a strong commercial opportunity in SMA, arguing that an FDA approval covering patients aged two and older across SMA types 1-4 would substantially expand the drug’s addressable market.
The analyst noted that the September 22 PDUFA date could face a modest delay into the first quarter due to procedural issues flagged during an FDA site inspection. However, she does not believe this will affect the likelihood of approval. Li modeled risk-adjusted peak sales of $1.8 billion for apitegromab, with a 90% probability of success in patients over two years of age and 50% for those under two.
She also highlighted the longer-term potential for apitegromab and pipeline candidate SRK-439 in other neuromuscular conditions, including Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD), as well as possible applications in obesity through external partnerships.
Scholar Rock Holding Corp. (NASDAQ:SRRK) is a late-stage biopharmaceutical company focused on developing therapies for spinal muscular atrophy (SMA) and other severe and debilitating neuromuscular diseases.
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Disclosure: None. This article is originally published at Insider Monkey.
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