Vesleteplirsen is under clinical development by Sarepta Therapeutics and currently in Phase II for Duchenne Muscular Dystrophy. According to GlobalData, Phase II drugs for Duchenne Muscular Dystrophy have a 65% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how Vesleteplirsen’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Vesleteplirsen overview

Vesleteplirsen is under development for the treatment of Duchenne muscular dystrophy. It contains a PMO containing an exon 51 sequence (eteplirsen) with a cell-penetrating peptide. It is administered through intravenous route. The drug candidate is developed based on next-generation peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) technology and exon skipping technology which skips exon 51 in dystrophin gene and restores protein translation and dystrophin production.

Sarepta Therapeutics overview

Sarepta Therapeutics (Sarepta) discovers and develops unique RNA-targeted medicines to treat rare diseases. The company develops its pipeline products using its multi-platform Precision Genetic Medicine Engine in gene therapy, RNA, and gene editing. The company’s platform is based on its pioneering work with phosphorodiamidate morpholino oligomer (PMO) chemistries. Its commercial products include Exondys 51, Vyondys 53 and Amondys 45 indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene. Its pipeline product includes SRP-9001, SRP-5051, SRP-9003 and SRP-5045 indicated for the treatment of DMD, limb-girdle muscular dystrophies (LGDMs) and other neuromuscular and central nervous system disorders. Sarepta is headquartered in Cambridge, Massachusetts, the US.

For a complete picture of Vesleteplirsen’s drug-specific PTSR and LoA scores, buy the report here.

This content was updated on 20 February 2024

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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.