Shares of Sarepta Therapeutics SRPT have surged 76.8% in the past three months compared with the industry’s 10.2% rise.
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Sarepta’s commercial portfolio consists of three RNA-based PMO therapies, targeting Duchenne muscular dystrophy (“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. DMD is a type of muscular dystrophy.
Apart from this, the company is also focused on developing its pipeline. Earlier this June, Sarepta and partner Roche RHHBY announced new functional data across multiple studies from the clinical development program evaluating its investigational gene-therapy candidate SRP-9001 in DMD.
The data from these studies demonstrated that treatment with SRP-9001 led to functional improvements in individuals suffering from DMD compared with a propensity-weighted external control group at multiple time points. The time points vary from one, two and four years post-treatment.
Based on discussions with the FDA, Sarepta is expected to initiate a biologic license application (“BLA”) seeking accelerated approval for the candidate in DMD later this year. Management expects to complete the BLA filing by first-half 2023. If granted approval, the gene therapy could generate a billion dollars in sales. Sarepta 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.
Sarepta is developing SRP-9001 in collaboration with Roche. Sarepta and Roche entered into a licensing agreement to develop SRP-9001 for DMD in 2019. 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.
The FDA placed a clinical hold earlier in June following reports of a serious adverse event of hypomagnesemia in part B of the phase II MOMENTUM study. Hypomagnesemia is when an individual has lower-than-normal levels of magnesium in the body. If left untreated, it can cause life-threatening complications. Based on these reports, the regulatory authority requested information from the company to assess the adequacy of the risk mitigation and safety monitoring plan.
This clinical hold only affected the U.S. portion of the MOMENTUM study, which is being conducted globally. The study continued to enroll and dose participants in ex-U.S. regions. Management expects to complete enrollment in the MOMENTUM study by year-end.
SRP-5051 is being evaluated in a similar patient population targeted by Sarepta’s lead drug Exondys 51. SRP-5051 has the potential to offer DMD patients a better alternative with a much lower dose and a new drug with more extended patent protection.
While the company mainly focuses on therapies targeting DMD, it has recently started developing therapies targeting indications other than DMD. Sarepta is also developing gene therapies in pre-clinical studies for treating central nervous system (CNS) disorders, including Mucopolysaccharidosis type IIIA (MPS IIIA) and Pompe Disease.
Sarepta Therapeutics, Inc. Price
Sarepta Therapeutics, Inc. price | Sarepta Therapeutics, Inc. Quote
Zacks Rank & Stocks to Consider
Sarepta Therapeutics currently carries a Zacks Rank #3 (Hold).
A couple of better-ranked stocks in the overall healthcare sector include Kamada KMDA and Morphic MORF, each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, estimates for Kamada’s 2022 earnings per share have risen from 1 cent to 26 cents. Shares of Kamada have lost 24% in the year-to-date period.
Earnings of Kamada missed estimates in three of the last four quarters and beat the mark just once, witnessing a negative surprise of 212.50%, on average. In the last reported quarter, KMDA’s earnings beat estimates by 450%.
In the past 60 days, estimates for Morphic’s 2022 loss per share have narrowed from $3.47 to $1.80. Loss estimates for 2023 have narrowed from $3.96 to $3.62 during the same period. Shares of Morphic have lost 37.6% 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%.
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