In drug development, everything's a gamble, if you're doing something new and shooting at a big target. But there has to be a reasonable assumption that if safety issues aren't being glossed over and the efficacy data hold up, these top drugs can change standards of care and grab market share. So here's my pick of the likely big winners >>
Vertex Pharmaceuticals has set the stage for an ambitious Phase III program for its combo approach matching the experimental VX-809 and Kalydeco for cystic fibrosis. And if it can stick with the plan, Vertex will cut a short path directly to an approval for the groundbreaking combo.
Vertex Pharmaceuticals ($VRTX) kept hopes alive for treating cystic fibrosis patients with the most common genetic mutation behind the lung disease, with new data from a mid-stage study showing improved lung function in patients on a combination of its approved drug Kalydeco and the experimental compound VX-809.
Vertex shares took a nasty tumble this morning, freefalling 18% on the news that the company was forced to revise the data on its closely watched cystic fibrosis combo therapy after learning that the initial interim data report had been bungled.
The data indicate that Vertex might have a remedy for treating a large number of the 70,000 people worldwide with CF.