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Sarepta Therapeutics (SRPT - Free Report) is the market leader in the Duchenne muscular dystrophy (DMD) space. It currently markets four therapies, all of which are approved in this space.
The stock has been surging as of late, all thanks to the commercial potential of Elevidys, which is the first and currently the only FDA-approved one-shot gene therapy for DMD.
Despite being initially approved last year with a confined label to treat children aged between four and five years with DMD, Elevidys demonstrated blockbuster potential. Sarepta recorded revenues of more than $200 million from the gene therapy’s sales last year, which is an encouraging figure for a therapy that was commercially launched in the second half of 2023. During the first quarter of 2023, Elevidys contributed $133.9 million to the company’s top line.
Last month, the FDA granted label expansion to Elevidys to treat all DMD patients aged four years and older. While the FDA granted traditional approval for the therapy to treat ambulatory DMD patients (those who can still walk), it has granted accelerated approval for non-ambulatory patients.
This latest approval significantly expands the company’s target market as it can increase its reach to a wider patient group. It also suggests that the company should now be able to generate more revenues from Elevidys sales. This potential surge in revenues could also help the company’s pipeline development, which currently consists of more than 40 programs across various stages of clinical development targeting different muscular dystrophies.
Sarepta’s shares have surged 57.4% year to date against the industry’s 6.9% fall, likely owing to the encouraging sales potential of Elevidys and Sarepta’s pipeline potential.
Image Source: Zacks Investment Research
Another factor that also benefitted the company during this period was the failure of pharma giant Pfizer’s (PFE - Free Report) gene therapy candidate for DMD. Just a week before Elevidys’ label expansion, Pfizer announced that a late-stage study evaluating its DMD gene therapy candidate failed to meet the primary endpoint and key secondary endpoints. Treatment with the Pfizer therapy failed to improve motor function among the DMD boys. Pfizer plans to share more detailed results from the study at an upcoming medical meeting.
Sarepta developed Elevidys in collaboration with Roche (RHHBY - Free Report) . Both companies entered into a licensing agreement in 2019 to develop and market Elevidys. Per the agreement, Sarepta is responsible for marketing the therapy in the United States, while Roche is responsible for marketing the gene therapy outside the country. Sarepta is also eligible to receive collaboration revenues on the ex-U.S. sales made by Roche.
Last month, Roche announced that the EMA had validated a regulatory filing seeking marketing authorization for Elevidys in patients aged 3-7 years with DMD. A potential approval will make Elevidys the first and only gene therapy to address the underlying cause of DMD available in Europe.
However, competition in the DMD space is stiff as companies like Regenxbio (RGNX - Free Report) and Solid Biosciences (SLDB - Free Report) have been developing their respective gene therapy candidates for DMD.
Regenxbio is planning to hold an end-of-phase II (EOP2) meeting with the FDA early in third-quarter 2024 to discuss the study design of a pivotal late-stage study on RGX-202, its DMD gene therapy candidate. Based on the feedback, Regenxbio plans to start this pivotal study before 2024-end. If the outcome of this study is positive, it intends to seek accelerated approval for its therapy from the FDA.
Last year, the FDA cleared Solid Biosciences’ investigational new drug (IND) to start a phase I/II study on SGT-003 in pediatric patients with DMD. This study is expected to begin by the end of this month. SLDB expects to report initial data from this study by this year’s end. The FDA has granted fast-track, orphan drug, and rare pediatric disease designations to Solid Biosciences’ gene therapy in DMD indication.
Image: Bigstock
Here's Why Sarepta (SRPT) Stock Price Surged 57% YTD
Sarepta Therapeutics (SRPT - Free Report) is the market leader in the Duchenne muscular dystrophy (DMD) space. It currently markets four therapies, all of which are approved in this space.
The stock has been surging as of late, all thanks to the commercial potential of Elevidys, which is the first and currently the only FDA-approved one-shot gene therapy for DMD.
Despite being initially approved last year with a confined label to treat children aged between four and five years with DMD, Elevidys demonstrated blockbuster potential. Sarepta recorded revenues of more than $200 million from the gene therapy’s sales last year, which is an encouraging figure for a therapy that was commercially launched in the second half of 2023. During the first quarter of 2023, Elevidys contributed $133.9 million to the company’s top line.
Last month, the FDA granted label expansion to Elevidys to treat all DMD patients aged four years and older. While the FDA granted traditional approval for the therapy to treat ambulatory DMD patients (those who can still walk), it has granted accelerated approval for non-ambulatory patients.
This latest approval significantly expands the company’s target market as it can increase its reach to a wider patient group. It also suggests that the company should now be able to generate more revenues from Elevidys sales. This potential surge in revenues could also help the company’s pipeline development, which currently consists of more than 40 programs across various stages of clinical development targeting different muscular dystrophies.
Sarepta’s shares have surged 57.4% year to date against the industry’s 6.9% fall, likely owing to the encouraging sales potential of Elevidys and Sarepta’s pipeline potential.
Image Source: Zacks Investment Research
Another factor that also benefitted the company during this period was the failure of pharma giant Pfizer’s (PFE - Free Report) gene therapy candidate for DMD. Just a week before Elevidys’ label expansion, Pfizer announced that a late-stage study evaluating its DMD gene therapy candidate failed to meet the primary endpoint and key secondary endpoints. Treatment with the Pfizer therapy failed to improve motor function among the DMD boys. Pfizer plans to share more detailed results from the study at an upcoming medical meeting.
Sarepta developed Elevidys in collaboration with Roche (RHHBY - Free Report) . Both companies entered into a licensing agreement in 2019 to develop and market Elevidys. Per the agreement, Sarepta is responsible for marketing the therapy in the United States, while Roche is responsible for marketing the gene therapy outside the country. Sarepta is also eligible to receive collaboration revenues on the ex-U.S. sales made by Roche.
Last month, Roche announced that the EMA had validated a regulatory filing seeking marketing authorization for Elevidys in patients aged 3-7 years with DMD. A potential approval will make Elevidys the first and only gene therapy to address the underlying cause of DMD available in Europe.
However, competition in the DMD space is stiff as companies like Regenxbio (RGNX - Free Report) and Solid Biosciences (SLDB - Free Report) have been developing their respective gene therapy candidates for DMD.
Regenxbio is planning to hold an end-of-phase II (EOP2) meeting with the FDA early in third-quarter 2024 to discuss the study design of a pivotal late-stage study on RGX-202, its DMD gene therapy candidate. Based on the feedback, Regenxbio plans to start this pivotal study before 2024-end. If the outcome of this study is positive, it intends to seek accelerated approval for its therapy from the FDA.
Last year, the FDA cleared Solid Biosciences’ investigational new drug (IND) to start a phase I/II study on SGT-003 in pediatric patients with DMD. This study is expected to begin by the end of this month. SLDB expects to report initial data from this study by this year’s end. The FDA has granted fast-track, orphan drug, and rare pediatric disease designations to Solid Biosciences’ gene therapy in DMD indication.
Zacks Rank
Sarepta sports a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.