What if Kalydeco was dosed together with VX-809 from the beginning
It is difficult to understand why there is not more enthusiasm for Vertex stock given the phase II data for VX-770 (Kalydeco)/VX-809 in F508del patients showing significant improvement in FEV1 that is not dependent on a few super responding patients. There were no serious safety concerns with either medication. The upcoming phase III pivotal trials should show even better FEV1 numbers when all patients are dosed at the higher 600 mg VX-809 or, possibly, 800 mg VX-809 levels and the when both VX-770 and VX-809 are given together starting on day 1 of the trial. And this combination therapy for F508del patients has FDA fast track status which should allow Vertex to bring it to the market that much sooner. There are more than 30,000 patients in the U.S. and europe that can be treated with this regimen and it is their best hope to extend their lifespan by treating the underlying cause of this disease.
At one point during the recent webcast by Vertex, Jeff Leiden said the company was "all in" when referring to their CF franchise commitment. They must be confident about the success of their CF drugs to make this comment. My opinion is that their market cap should be much higher based on Vertex progress in cystic fibrosis alone. Everything else from their hepatitis C drugs to their extensive pipeline of drugs for other diseases is a bonus.
"It is difficult to understand why there is not more enthusiasm for Vertex stock given the phase II data for VX-770 (Kalydeco)/VX-809 in F508del patients showing significant improvement in FEV1 that is not dependent on a few super responding patients."
I think some Investors and analysts wrongly came to the conclusion that since the data for the 200mg group was better than 400mg, that there must be something wrong with the efficacy of the combination of the two drugs. The fact that the 600mg group did very well does not phase these naysayers. These days, many investors, analysts, and hedge funds pound a stock when there is the slighest excuse to do so. They figure they are playing it safe!!. Even though VRTX management did not attach any significace to the difference between the 200 and 400 mg groups and also own board poster, Third, detailed the science of low dosages of drugs and how they could just create "noise," these naysayers have not paid much attention. They will after phase III data shows excellent results but by then they will be too late to scoop up cheap shares.