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Vertex Pharmaceuticals Incorporated Message Board

  • thirdmeinvestor thirdmeinvestor Aug 18, 2013 6:22 PM Flag

    Meaning of Breakthru Designation reviewed in congressional briefing

    All people who are interested in investing in Vertex should watch the vidio, whose link is given below.
    It is worthwhile to watch through the end. Allow 1.5 hours of time to watch all. CEO Leiden is impressive, and CFer (551) Bill Elder is a living proof that how Kalydeco and VX-809, 661, others mean to the lives of these patients.


    Replace xs with commas before pasting and opening the link.

    I posted a full link earlier in the Investorvillage's Vertex website; go to the site in the case that the above link does not work.

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    • Thanks, watched the video, what was very impressive to me was the med student who had taken Vertex's CF drug and the same day, he could tell improvement. It looks to me a magic bullet. He also stated how he could run for 2 hours versus 20 minutes.... So, my question is, how confident can we be the drug plus the enhancements will work.. I know they were able to get to work in their models and the phase II test have been very good, but does the initial success with one mutation carry over with a reasonable degree of certainty?

      On the positive news front, saw Oppenheimer came out with an overweight rating and a 105 PT after the close....

      Sentiment: Buy

      • 1 Reply to loblolly7
      • As you may be aware, it has been shown clinically that Kalydeco monotherapy alone can boost the lung function for people with gating mutations just as well as it does for the G551D people. It is most likely that other 10 % of all CF people with 'residual' CFTR functions, and with R117H will benefit from the monotherapy.

        Your question is perhaps more directed at the efficacy of VX-809 + Kalydeco for the homozygous F508del persons. VX-809 is an enhancer of the activity of an enzyme family called cytochrome P 450 (=CYP), which metabolizes drugs including Kalydeco and VX-809 itself. For this reason, VX-809 causes an increased rate for the degradation of Kalydeco in the body, and to compensate for the loss, a greater Kalydeco dose has to be given when it is dosed with VX-809. The resulting efficacy of this drug combination is not as strong as the in vitro experiments suggest, and less so than the Kalydeco activity on gating mutations. Clearly, one way to improve the drug combo containing VX-809 is to add an inhibitor of CYP in a controlled way. Grapefruit juice contains an inhibitor of CYP3a, for example.
        On the other hand, VX-661 is not an enhancer of CYP as VX-809 is. So, there are ways to treat 508 people in the future. When a second generation corrector is added to the mix, 70% of all CF persons with 508 mutation would benefit as much as Bill Elder, Jr. has experienced with Kalydeco.

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