The results from the clinical study rendered no change from baseline in dystrophin expression

Clinical stage genetic medicines company- Wave Life Sciences Ltd. has reportedly announced the halt of suvodirsen development for patients suffering from Duchenne Muscular Dystrophy who have been diagnosed with mutations amenable to exon 51 skipping, on the basis of the interim analysis during the Phase 1 open-label extension (OLE) trial.

Apparently, the results recorded no difference in dystrophin expression from baseline, when measured with western blot when given either 5 mg/kg or 3.5 mg/kg weekly doses of suvodirsen. Safety concerns or emerging safety signals were not observed.

Due to the aforementioned observations, the company has decided to discontinue the two suvodirsen trials with immediate effect, the Phase 2/3 DYSTANCE 51 trial and the OLE study. Patients are required to undergo a final follow-up visit; however, no further doses and muscle biopsies will be administered.

The analysis was derived from a multicenter suvodirsen OLE study in volunteers who had previously registered in the Phase 1 tolerability and safety study. Follow-up volunteers had enrolled in the OLE and were for the target doses of either 3.5 mg/kg or 5 mg/kg.

For the record, before the initial dosing in the OLE, a biopsy examination of the deltoid muscle was carried out and according to the data cut-off for the provisional analysis, the follow-up biopsies of the deltoid muscle were available for 27 of the 36 patients.

For the 5 mg/kg weekly infusion period, 10 patients obtained follow-up biopsies at twelve weeks while follow up muscle biopsies at twenty-two weeks was received by 9 patients. Eight patients got the twenty-two week follow up muscle biopsies within the 3.5 mg/kg weekly infusion arm. As a part of the interim analysis, biopsies from both the timepoints were analyzed.

About Suvodirsen

Wave Life Science announced the clinical development of suvodirsen in November 2017 and completed a Phase 1 safety and tolerability study by early 2019. It is an investigational stereopure oligonucleotide initially developed for the treatment of patients suffering from DMD who have genetic mutations amenable to exon 51 skipping.

Source Credits: https://ir.wavelifesciences.com/news-releases/news-release-details/wave-life-sciences-announces-discontinuation-suvodirsen