Sarepta Therapeutics SRPT submitted a biologics license application (BLA) to the FDA seeking accelerated approval for its gene therapy candidate, SRP-9001, to treat ambulant patients with Duchenne muscular dystrophy (DMD). The therapy is being developed in partnership with Roche RHHBY.
The BLA filing is supported by data from multiple studies from the clinical development program evaluating SRP-9001 in DMD. Earlier this June, Sarepta and Roche announced new functional data across these studies, demonstrating that treatment with SRP-9001 led to functional improvements in individuals suffering from DMD compared with a propensity-weighted external control group at multiple times. The time points vary from one-, two- and four years post-treatment.
Management also initiated a pivotal phase III study, EMBARK, last year. It will act as a confirmatory study seeking full approval for SRP-9001 in DMD.
Shares of Sarepta have risen 21.7% in the year-to-date period against the industry’s 25.2% decline.
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If approved by the FDA, SRP-9001 will be the first gene therapy available for DMD patients. The therapy is also expected to generate a billion dollars in revenue for Sarepta. The gene therapy has been granted Fast Track, Rare Pediatric Disease (RPD) and orphan drug designations by the FDA.
Sarepta and Roche entered into a licensing agreement in 2019 to develop SRP-9001 for DMD. Per the agreement, Roche has exclusive rights to launch and commercialize SRP-9001 in ex-U.S. markets.
Apart from SRP-9001, the company is also developing SRP-5051 (vesleteplirsen), its next-generation exon-skipping pipeline candidate for treating DMD patients with skipping exon 51.
Earlier this month, the FDA removed a clinical hold on clinical studies evaluating SRP-5051. As part of the condition for removing the clinical hold, Sarepta will modify the global protocols for clinical studies evaluating SRP-5051 to include expanded monitoring of urine biomarkers.
Sarepta’s commercial portfolio consists of three RNA-based PMO therapies, targeting DMD — Exondys 51, Vyondys 53 and Amondys 45. These drugs can potentially address nearly a third of all patients with DMD in the United States.
Sarepta Therapeutics, Inc. Price
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Zacks Rank & Stocks to Consider
Sarepta currently carries a Zacks Rank #4 (Sell). Some better-ranked stocks in the overall healthcare sector include Morphic MORF and Sanofi SNY. While Morphic sports a Zacks Rank #1 (Strong Buy) at present, Sanofi carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, estimates for Morphic’s 2022 loss per share have narrowed from $3.38 to $1.80. Loss estimates for 2023 have narrowed from $3.91 to $3.62 during the same period. Shares of Morphic have lost 40.9% in the year-to-date period.
Earnings of Morphic beat estimates in three of the last four quarters and missed the mark just once, witnessing a surprise of 48.29%, on average. In the last reported quarter, MORF delivered an earnings surprise of 183.95%.
In the past 60 days, estimates for Sanofi’s 2022 earnings per share have increased from $4.08 to $4.14. Earnings estimates for 2023 have increased from $4.25 to $4.29 during the same period. Shares of Sanofi have lost 24.2% in the year-to-date period.
Earnings of Sanofi beat estimates in each of the last four quarters, witnessing a surprise of 9.37%, on average. In the last reported quarter, SNY delivered an earnings surprise of 8.24%.
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