Small-cap biotech Wave Life Sciences Ltd (NASDAQ: WVE) — which fell last week following a delay in the release of top-line data from its Huntington disease candidates — is once again under pressure Tuesday.
Wave Life Sciences released final results Tuesday from a Phase 1 trial of suvodirsen, its investigational candidate for treating boys with Duchenne muscular dystrophy, or DMD, who are amenable to exon 51 skipping; as well as the design of a Phase 2/3 trial dubbed DYSTANCE 51 for suvodirsen in DMD.
Both data sets are to be presented at the 2019 Muscular Dystrophy Association Clinical and Scientific Conference in Orlando, Florida.
The Phase 1 trial evaluated the safety, tolerability and plasma concentrations of single ascending doses of suvodirsen administered intravenously.
The higher doses tested (7mg/kg or 10mg/kg) in two patients in the fifth cohort produced the same adverse events observed at lower doses, but with more severity, the company said.
The Phase 2/3 trial is designed to enroll boys ages 5-12 with a genetically confirmed diagnosis of DMD amenable to exon 51 skipping therapy. The patients will be administered 4.5mg/kg or 3mg/kg of suvodirsen or placebo administered intravenously once weekly for 48 weeks.
The primary endpoints are the change in dystrophin protein level and change in the North Star Ambulatory Assessment score, while multiple functional outcome measures will serve as secondary efficacy endpoints, according to Wave Life Sciences.
Wave Life said it expects to report interim efficacy data from the Phase 1 trial later this year and intends to use it as an important component of a submission for accelerated approval in the United States.
The company expects to initiate the DYSTANCE 51 trial in July and use the results from the trial for regulatory submissions globally.
Wave Life Sciences shares were plunging 30.22 percent to $23.74 at the time of publication Tuesday.
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