> Sarepta Therapeutics saw its shares jump 13% in early morning trading on new phase 1/2 data from its next-gen DMD candidate golodirsen. The medication hit its primary and secondary endpoints in 25 boys with confirmed deletions of the DMD gene, amenable to skipping exon 53, by boosting mean dystrophin protein by just over 1%, as well as increasing dystrophin immunofluorescence. It now plots an accelerated review with the FDA. Statement.
> Verastem said its medication duvelisib, bought up from struggling Infinity Pharmaceuticals late last year, has seen some positive data, coming after its previous owner last year posted results showing it had hit its primary endpoint in a phase 2 for indolent non-Hodgkin lymphoma, but fell short of expectations. The latest phase 3 readout from Verastem showed it could boost progression-free survival in chronic lymphocytic leukemia/small lymphocytic lymphoma compared to Novartis’ Arzerra. Its shares shot up by 35% this morning. Release.
> Voyager Therapeutics said its ongoing Phase 1b trial of its gene therapy VY-AADC01 in advanced Parkinson’s disease is showing “improvements across multiple measures of patients’ motor function after a one-time administration of the gene therapy.” It, too, saw its stock rise, up around 20% this morning. Statement.