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Sarepta's Casimersen NDA Gets Priority Review From FDA

Sarepta Therapeutics, Inc. SRPT announced that the FDA has accepted its rolling new drug application (“NDA”) seeking accelerated approval for casimersen. The NDA is seeking approval for the candidate as a potential treatment for Duchene muscular dystrophy (“DMD”) patients. The FDA has granted priority review to the NDA and a decision is expected by Feb 25, 2021.

The FDA has conditionally approved the brand name for casimersen — Amondys 45. Meanwhile, the regulatory authority does not plan to hold an advisory committee to discuss the application.

Casimersen has been developed using similar technology used for its marketed DMD drugs — Exondys 51 and Vyondys 53. The candidate will treat DMD patients who have genetic mutations that are amenable to skipping exon 45 of the Duchenne gene. Please note Exondys 51 and Vyondys 53 target exon 51 skipping and exon 53 skipping patients, respectively.

The NDA was based on data from an ongoing phase III study — ESSENCE — evaluating efficacy and safety in patients amenable to skipping exons 45 and 53. Data from an interim analysis of the study announced in March 2019 showed that treatment with casimersen led to statistically significant mean increase of dystrophin protein production (as measured by western blot) compared to baseline and placebo. In the study, 22 patients receiving casimersen displayed an increase in skipping exon 45 over their baseline levels, representing 100% response rate.

DMD is a genetic disorder caused by a mutation that prevents the body from producing dystrophin, a protein that muscles need to function properly. Sarepta’s approved drugs and casimersen works through a method called exon skipping, which helps the body's cellular machinery make functional dystrophin.

Final data from the ESSENCE study will support the continued approval for casimersen, if the NDA is accepted by the FDA and granted approval. The same study is also serving as a post-marketing confirmatory study for Vyondys 53, which was granted accelerated approval in December 2019. The study is expected to conclude by 2024.

So far this year, the company’s shares have increased 12.3% while the industry remained flat.

Sales of Exondys 51 continued its growth momentum in the first half of 2020. However, sales were impacted adversely due to COVID-19. Moreover, patient initiation on Vyondys 53, faced challenges during the quarter due to restricted environment where physicians were facing difficulties in monitoring patients regularly.

The company anticipates total patient population targeted by Exondys 51, Vyondys 53 and Amondys 45 to be almost double of Exondys 51 alone. This is likely to lead to higher revenues for Sarepta going forward.

Please note that Sarepta has about 12 other exon-skipping candidates in its pipeline. The company is also developing a next-generation PPMO candidate, SRP-5051, for the treatment of DMD by skipping exon 51 in a phase II study.

Apart from its exon-skipping candidates, the company is developing several gene therapies targeting muscular dystrophies including DMD and central nervous system disorders. Its lead gene therapy candidate, SRP-9001, an AAV-mediated micro-dystrophin gene therapy candidate, is currently being evaluated in a phase I/II study for treating DMD. The promising candidate has also led Roche RHHBY to sign a collaboration deal with Sarepta related to commercialization of SRP-9001 in ex-U.S. markets.

However, we note that there are there are several other companies developing gene therapies for treating DMD including Solid Biosciences SLDB, Audentes Therapeutics, and Pfizer PFE. Successful development of gene therapies for DMD will increase competition in the field. Gene therapies of Solid Biosciences and Pfizer seem to trail Sarepta’s gene therapy for DMD, based on early-stage study data.

Sarepta Therapeutics, Inc. Price

Sarepta Therapeutics, Inc. Price

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Zacks Rank

Sarepta currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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