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Vertex Pharmaceuticals Incorporated Message Board

brokenideas 3 posts  |  Last Activity: Jun 4, 2014 8:30 PM Member since: Aug 27, 2012
  • brokenideas brokenideas Jun 4, 2014 8:30 PM Flag

    Why is the FEV1 effect so small? Perhaps the short duration of the study or perhaps because the residual channels and not potentiated as much with ivacaftor? Still, it is amazing that with less than 3% improvement in FEV1 they got a p=0.0001.

    Clearly ivacaftor works as a drug, the question is if lumacaftor does indeed work. We will know soon, I guess.

    I fear that they released this data on a small phase II study ahead of the phase III data to provide some good news prior to "disappointing news" ... I hope to be wrong.

  • brokenideas brokenideas May 11, 2014 12:30 PM Flag

    Thank you. Still, even when they characterize the placebo response as "modest" why do they refuse to show the individual data for each of the 4 placebo patients?

    Everyone understands that 4 people in the placebo group cannot be used for statistical analysis, but their refusal to provide the data, no matter how many efforts they try to describe it as a "modest" change, does not give a good impression. It taints the credibility of the response on FEV1 changes of the main study group.

    Besides this, why they designed the study with just 4 people in the placebo group? Why not powering the study so that at least they can get a statistical signal over placebo?

    Vertex often messes up with the clinical data release. They have done it badly before and releasing data like this doesn't help to build credibility. If they intended to demonstrate "positive" responses why not using a proper placebo group to power the study. It would have been better to wait before releasing data until when they run a larger study that can be reported with statistics over placebo. It is not very smart from their part.

  • Reply to

    Binary or Quaternary

    by thirdmeinvestor May 3, 2014 1:37 PM
    brokenideas brokenideas May 8, 2014 8:43 PM Flag

    Thank you for the analysis. Do you know if there is a tabulated summary on the 5 phase II trials that the CEO mentions to provide proof of concept for the combination of Lumacafor + Ivacafor so we can see the actual absolute FEV1 values that were achieved in these trials. These may be the best guess to predict the FEv1 outcome from the phase III data. Do you know if the FDA has a minimum aceptable FEV1 for approval? - Thank you.

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