Vertex unveils Phase III details for breakthrough combo

Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) unveiled details late Tuesday for its Phase III program to evaluate cystic fibrosis (CF) drug Kalydeco ivacaftor plus VX-809 in CF patients who are homozygous for the delta F508 mutation in the CF transmembrane conductance regulator ( CFTR) gene. The combination treatment and Kalydeco as monotherapy were the first products to receive breakthrough drug designation from FDA, which Vertex announced in January. The news sent shares of Vertex up $2.79 to $46.97 on Wednesday, which translates to a gain in market cap of $604.9 million for a closing valuation of $10.1 billion.

Breakthrough designation commits FDA to collaborate with a sponsor to enable expedited development and review of compounds for serious or life-threatening diseases that show substantial improvements over existing treatments in early trials. Vertex said it was "happy with the outcome" of its discussions with FDA -- including an agreement that a VX-809 monotherapy arm was not required in the Phase III program -- but the company did not provide details on how breakthrough designation impacted the discussions. The company plans to submit an NDA to FDA for the combination treatment in 2014 (see BioCentury, Dec. 3, 2012). ...