WVEN-531 is under clinical development by Wave Life Sciences 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 WVEN-531’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.

WVEN-531 overview

WVEN-531 is under development for the treatment of Duchenne muscular dystrophy. The drug candidate is developed based on exon skipping technology which skips exon 53 in the dystrophin gene. It is developed based on chemistry platform, which utilizes phosphorothioate (PS) modification of nucleic acid therapeutics. It is administered through intravenous route. It is being developed based on PRISM platform.

Wave Life Sciences overview

Wave Life Sciences discovers, develops, and commercializes medicines for genetic diseases. The company’s pipeline consists of an mRNA transcript candidate: WVE-120101 and WVE-120102 for Huntington’s disease; ATXN3 gene candidate for Spinocerebellar ataxia 3; C9orf72 gene candidate for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD); USH2A and RhoP23H candidate for retinal diseases; and multiple candidates for other central nervous system (CNS) disorders, and hepatic diseases. The company employs its proprietary chemistry platform to design, optimize and manufacture stereopure oligonucleotides with improved safety, tolerability and efficacy compared to that of stereorandom oligonucleotides. Wave Life Sciences is jointly advancing its CNS pipeline candidates in collaboration with Takeda. It has operations in Singapore, the US, Japan, the UK, and Ireland. Wave Life Sciences is headquartered in Singapore city, Singapore.

For a complete picture of WVEN-531’s drug-specific PTSR and LoA scores, buy the report here.

This content was updated on 2 September 2023

Premium Insights


The gold standard of business intelligence.

Blending expert knowledge with cutting-edge technology, GlobalData’s unrivalled proprietary data will enable you to decode what’s happening in your market. You can make better informed decisions and gain a future-proof advantage over your competitors.


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.