PTC's ataluren flops in an­oth­er PhI­II and an an­a­lyst says it's clear­ly a dud; kill it

PTC Ther­a­peu­tics has whiffed once again on Translar­na (ataluren).

The biotech re­port­ed that ataluren, a re­peat flop in Duchenne mus­cu­lar dy­s­tro­phy, failed a Phase III for non­sense mu­ta­tion cys­tic fi­bro­sis on both the pri­ma­ry and sec­ondary end­points.

In­ves­ti­ga­tors tracked a slight but in­signif­i­cant ad­van­tage for pa­tients in the drug arm of the  CF study, not­ing a bet­ter num­ber for lung ca­pac­i­ty and the rate of pul­monary ex­ac­er­ba­tions. But none of it stacked up in a way worth tak­ing to reg­u­la­tors, and the biotech says it will scrap its work in CF and jerk its ap­pli­ca­tion for ap­proval in Eu­rope.

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