PTC of­fers its best case for a con­tro­ver­sial Duchenne MD PhI­II. But key fail­ures spark fresh doubts of suc­cess at FDA

PTC Ther­a­peu­tics’ years-long ef­fort to push its con­tro­ver­sial Duchenne MD drug Translar­na in­to the US has just run in­to its lat­est set­back.

The biotech re­port­ed a mixed set of da­ta from its con­fir­ma­to­ry study of the drug, with a list of key fail­ures that at least one high pro­file an­a­lyst be­lieves aren’t like­ly to win over skep­tics at the FDA.

The biotech’s ex­ecs were care­ful to spot­light the up­beat as­pects of the da­ta, which they say lays a straight path to their next FDA pitch for treat­ing non­sense mu­ta­tion DMD. And RBC’s Bri­an Abra­hams be­lieves that should keep it on the Eu­ro­pean mar­ket, where it got an ear­ly launch on failed tri­al da­ta. But the an­a­lyst raised doubts over the bad da­ta PTC di­vulged Tues­day morn­ing.

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