Dai­ichi Sankyo con­cedes an ear­ly set­back on Duchenne MD drug, but dou­bles down on de­vel­op­ment

Two years ago, when in­ves­ti­ga­tors for Dai­ichi Sankyo gave its ex­per­i­men­tal Duchenne mus­cu­lar dy­s­tro­phy drug to the very first pa­tient in a clin­i­cal tri­al, the com­pa­ny con­fi­dent­ly hailed the move as a big step to­ward a mar­ket­ing ap­proval and launch in 2020. But DMD drug de­vel­op­ment isn’t that sim­ple.

Dai­ichi to­day said that DS-5141 — de­signed to spur the pro­duc­tion of in­com­plete but func­tion­al dy­s­trophin by in­duc­ing ex­on 45 skip­ping through dy­s­trophin mes­sen­ger RNA — failed an ear­ly Phase I/II study. But they’re not about to throw in the tow­el.

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