We came across a bullish thesis on Sarepta Therapeutics, Inc. (SRPT) on Substack by Two Natural Capital. In this article, we will summarize the bulls’ thesis on SRPT. Sarepta Therapeutics, Inc. (SRPT)’s share was trading at $124.47 as of Dec 12th. SRPT’s trailing and forward P/E were 80.82 and 10.07 respectively according to Yahoo Finance.
A scientist carefully examining a petri dish containing a rare disease’s gene therapy.
Sarepta Therapeutics is a pioneering biotechnology company focused on developing treatments for rare genetic diseases, particularly Duchenne muscular dystrophy (DMD). The company has carved a strong niche in the therapeutic landscape through its innovative approaches, including exon-skipping RNA therapeutics and gene therapies, addressing unmet needs for a devastating condition with limited options. Sarepta’s exon-skipping treatments, EXONDYS 51, VYONDYS 53, and AMONDYS 45, are designed to skip specific exons in the dystrophin gene, enabling the production of a functional, albeit shortened, dystrophin protein. These therapies have been a significant step forward, though their applicability is limited to a subset of patients with specific exon mutations.
Recognizing the limitations of exon-skipping, Sarepta developed ELEVIDYS, a gene therapy approved by the FDA in June 2023, and later expanded in 2024 to treat both ambulatory and non-ambulatory DMD patients aged 4 and above. ELEVIDYS addresses a broader patient population using a truncated dystrophin gene delivered via an AAV vector. This one-time treatment represents a meaningful advance, offering improved functional outcomes in clinical trials despite not achieving statistical significance on certain primary endpoints. The therapy’s premium pricing of $3.2 million per patient positions it among the most expensive treatments in the U.S., yet it is cost-effective over time compared to recurring exon-skipping therapies, which cost up to $1.5 million annually.
The approval of ELEVIDYS marks a pivotal shift in Sarepta’s business model, transitioning from a predominantly recurring revenue structure to a model relying more on high-value, one-time therapies. This shift is strategic, allowing Sarepta to generate substantial cash flow to fuel innovation while maintaining a balanced pipeline. To mitigate risks associated with over-reliance on one-time treatments, Sarepta is prudently sustaining its RNA therapeutic programs and has recently partnered with Arrowhead Pharmaceuticals to explore siRNA-based treatments. This collaboration underscores the company’s commitment to maintaining a diversified revenue base while advancing cutting-edge technologies.
Sarepta’s pipeline extends beyond DMD, with SRP-9003, a gene therapy for limb-girdle muscular dystrophy (LGMD2E), currently in phase 3 trials. Leveraging the same delivery vector as ELEVIDYS, SRP-9003 reduces development risks and exemplifies Sarepta’s effective strategy for rare diseases. By addressing smaller, high-need patient populations with scalable approaches, Sarepta maximizes the impact of its therapies while building a foundation for broader applications.
Despite its leadership position, Sarepta faces competitive risks. Companies like Vertex and CRISPR Therapeutics are exploring CRISPR/Cas9-based therapies, while other gene therapy developers, such as Solid Biosciences and Regenxbio, are advancing candidates in early-stage trials. While competitors are years away from regulatory approval, Sarepta’s ability to maintain its edge will hinge on the continued success of ELEVIDYS, the evolution of its pipeline, and its capacity to innovate.
Sarepta’s balanced approach to gene therapies and recurring RNA-based treatments positions it uniquely in the biotech sector. With a robust pipeline, strategic partnerships, and a clear focus on addressing unmet needs, the company stands well-poised to sustain its leadership in rare disease therapeutics while navigating the challenges of a competitive and rapidly evolving field.
Sarepta Therapeutics, Inc. (SRPT) is not on our list of the 31 Most Popular Stocks Among Hedge Funds. As per our database, 50 hedge fund portfolios held SRPT at the end of the third quarter which was 55 in the previous quarter. While we acknowledge the risk and potential of SRPT as an investment, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns, and doing so within a shorter timeframe. If you are looking for an AI stock that is more promising than SRPT but that trades at less than 5 times its earnings, check out our report about the cheapest AI stock.
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Disclosure: None. This article was originally published at Insider Monkey.