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Vertex Pharmaceuticals Incorporated Message Board

thirdmeinvestor 32 posts  |  Last Activity: Aug 4, 2014 9:29 PM Member since: Feb 25, 2004
  • Reply to

    A post from facebook:

    by gladpick Jun 6, 2014 11:24 AM
    thirdmeinvestor thirdmeinvestor Jun 23, 2014 10:12 PM Flag

    The same person (most likely) posted the following blog on May 30 two weeks after the one you posted appeared. In both blogs she wrote without using punctuation marks.

    [[ Judy Law My daughter took part in this study in england it was double blinded for 6 months but now she is taking the active drug [there has to be a "period" here (my comment)] when she started in september she just managed to get on the trial with a lung function of 40% [another period is missing here] to cut a long story short we dont know any results from the trial but we do from the clinic visits weight is up to 56kg sats stable at 96_98 and her lung function at last visit was a whooping 59 % x x ]]

    It is possible this person manufactured this story to encourage other CF persons, but it is likely that she has a valid story to tell because the blogs appeared in QUEST for KALYDECO.

  • Reply to

    809 combo and responders

    by verityvoila Jun 23, 2014 6:50 AM
    thirdmeinvestor thirdmeinvestor Jun 23, 2014 3:00 PM Flag

    Verity, my response to your post does not show here.

  • Reply to

    809 combo and responders

    by verityvoila Jun 23, 2014 6:50 AM
    thirdmeinvestor thirdmeinvestor Jun 23, 2014 2:58 PM Flag

    Verity, I like the results from JP Morgan models, which give:

    FEV-1 improvement

  • Reply to

    A post from facebook:

    by gladpick Jun 6, 2014 11:24 AM
    thirdmeinvestor thirdmeinvestor Jun 23, 2014 1:29 PM Flag

    Don't get confused about individual's data with group averages. In the 661 Ph 2 trial, one 508 patient who is receiving a placebo had an increase of 26% for FEV1. Does it mean that the patient is faking! All are 508 homozygous patients, but the dispersion is huge.

  • Reply to

    A post from facebook:

    by gladpick Jun 6, 2014 11:24 AM
    thirdmeinvestor thirdmeinvestor Jun 23, 2014 12:00 PM Flag

    Getrich, the story quoted by Gladpick from Facebook sounds so real and internally consistent that it is very unlikely a fabrication. The daughter started the trial in September last year and finished the trial in March, then put on the extension study.

  • Reply to

    what is needed for approval?

    by verityvoila Jun 20, 2014 12:11 PM
    thirdmeinvestor thirdmeinvestor Jun 22, 2014 8:33 PM Flag

    The rollover study is also supposed to be a double-blinded trial, but some participants are finding out good enhancement for FEV1 and weight gains. Getrich, do you have anecdotal stories to tell?

  • thirdmeinvestor thirdmeinvestor Jun 19, 2014 3:55 PM Flag

    Verity, it is very difficult to understand his argument of molecular stability when the 508 CFTR is in a cell culture system. Any thing can happen in cultured cells.
    How can he believe in such results while he does not believe in Cohort 2 and Cohort 3 results of Phase 2.
    Only thing I am concerned is drug fatigue for a long term dosing. But there is no basis for that to occur.

    Good luck to you and to all longs.

  • thirdmeinvestor thirdmeinvestor Jun 18, 2014 10:47 AM Flag

    Verity, you exactly pointed out the very weak point of Porges' pseudo-science. They believe in molecular stability argument but do not believe in in vitro biology of Van Goor. Biology is a better predictor of clinical outcome than molecular argument is.

  • [[ Underscoring the bullish sentiment in Vertex's favor, Cowen's Eric Schmidt noted his bet that the drug will likely succeed. Schmidt wrote: "The imminent release of results from VX-809's Ph. III trials has the potential to be one of the biggest binary events in biotech during 2014. We think that there is a 60% chance the trials succeed, and a 40% chance they fail (or have mixed results). We expect VRTX's stock to go to $100+ on success, but $40 on failure. We continue to think that VRTX is fairly valued, and remain at Market Perform."]]

    Eric Schmidt is a MIT PhD who studied at the Paul Schimmel's lab.
    Geoffrey Porges holds a Harvard Business School MBA.

  • Reply to

    A post from facebook:

    by gladpick Jun 6, 2014 11:24 AM
    thirdmeinvestor thirdmeinvestor Jun 13, 2014 9:03 PM Flag

    Glad, thank you for posting this. The mother's blog is entirely believable. Presumably, the lung function she was referring to was FEV1, and it changed from the prior 38% to 59% at the first several weeks of open-labeled rollover study, whether or not the daughter had received the real drug or not during the double-blinded trial. This is an absolute change of 21% and a relative change of 21/38 = 55%. Astounding. Some placebo effect might be superposed, but it cannot be all placebo effect because it is too large for placebo effect.

  • thirdmeinvestor thirdmeinvestor May 21, 2014 8:07 PM Flag

    Q, I think that the 400mg bid regimen is superior in efficacy based on pharmacokinetic data. But, the side effects may be greater also.

  • thirdmeinvestor thirdmeinvestor May 21, 2014 8:02 PM Flag

    Verity and Q, you asked good questions. I am expecting (and hoping) an FEV1 improvement of 5-6% for the Ph III trials. The results of Ph II trial for 508 homozygotes on 661/Kalydeco appear to level off after 2 weeks of dosing, but Ph II results of the same groups on 809/Kalydeco appear to continue to rise beyond 4 weeks of combo. And I am also expecting for this long term dosing positive feedback effects coming from clearing of lung airways inflammation due to stronger immunity resulting from metabolic improvements.

    Verity, I think that the 4.6% from the latest trial is on rather high side. If you exclude the placebo effect, the number is likely about 3.5%. Kalydeco alone can improve CFTR from a single 551 allele better than corrector+potentiator combo can a single 508 allele. Kalydeco alone would improve 551 of 551/508 hetero by 10%. So, on the average, the participants improved the lung function by a number like 13.5%, not 4.7ish %, when measured from the base line of taking no drug at all.

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