Wave Life Sciences has announced that it will discontinue development of its investigational DMD drug, suvodirsen, after interim analysis from a Ph 1 study. Suvodirsen was targeting the same subgroup of Duchenne patients for which Sarepta’s Exondys 51 was approved in 2016.
Wave’s decision followed an open-label extension of a Ph 1 trial, which showed no change in dystrophin levels in patients receiving the drug. A Phase 2/3 trial of suvodirsen that the company commenced in June will consequently be terminated.
Wave has said that it intends to continue using its stereopure oligonucleotide platform (“naked” ASOs which are claimed to be more potent, safer, and longer-lasting) to develop therapies to address a number of other indications, including Retinitis Pigmentosa.
The company’s CEO has stated that, while dystrophin restoration wasn’t achieved in the Phase 1 study, there is “a rising tide in nucleic acid therapeutics”, and the company was still fully committed to advancing genetic medicines in other CNS, liver and eye diseases.
https://xconomy.com/boston/2019/12/...after-decision-to-halt-duchenne-drug-studies/
https://www.fiercebiotech.com/biote...hes-as-it-tosses-out-dmd-drug-amid-trial-flop
https://www.statnews.com/2019/12/16...outcome-of-duchenne-muscular-dystrophy-study/
https://www.wavelifesciences.com/pipeline/
https://hotcopper.com.au/threads/an...4733386/page-82?post_id=39286798#.XfoEOmQzaUk
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