VX-809 + ivacaftor, Vertex Pharmaceuticals

Drug: VX-809 + ivacaftor
Company: Vertex Pharmaceuticals
Phase: Phase III
Class: Cystic fibrosis transmembrane conductance regulator (CFTR) corrector
2020 sales: $1.9 billion
Net present value: $5 billion

Vertex ($VRTX) bet the farm on this combo. A late-stage study that wrapped last month came in with positive results, but some analysts quickly noted that the data also came in at the low end of the range needed to demonstrate success. According to Vertex, all four drug arms for the two late-stage trials of lumacaftor (the CFTR corrector VX-809) and Kalydeco (ivacaftor) met their primary endpoint of mean absolute improvement for a measure of lung function called forced expiratory volume, or FEV. For patients who typically can expect to suffer a decline in FEV of 2% a year until their expected death sometime in their late 20s or 30s, there were mean improvements of 2.6% and 4.0% from baseline compared to a placebo arm. All the patients had two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene.

For more:
Bernstein analyst predicts a likely flop for Vertex's crucial CF combo study
Vertex plots a race through PhIII for 'breakthrough' combo CF therapy
Vertex's new data on CF drug cocktail doesn't disappoint

VX-809 + ivacaftor, Vertex Pharmaceuticals

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