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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