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Sarepta Therapeutics, Inc. Message Board

  • simp08801 simp08801 Aug 20, 2013 12:58 PM Flag

    yo tred

    I think that the AA opportunity for Srpt is now dead, but that the straight approval opportunity off the P2 still exists

    IMO, the RNA data does nothing but further cloud the issue on d as a reliable surrogate marker (perhaps the RNA P3 data in october will show a correlation, but I doubt it)

    As for Srpt, it was always suggested, but generally ignored, that its d data was not totally in order as a marker against the clinical benefit

    while the RNA P2 data may be bad for RNA, it does cast a cloud generally as well over the d issue

    IMO, statements about FDA meetings, and video's are no longer impactful here

    once the FDA started to speak, everything else takes a baaaaaaaaaaaaaack seat

    thus, IMO, only FDA official action matters here now, in terms of searching for positive binary events

    on the negative side, the impending RNA P3 will probably be used as negative no matter what that data is

    if its good, the word will be its bad for srpt because rna is "now in the lead"

    if its bad, the word will be its bad for srpt because the FDA will be loathe to approve srpt off a P2 when rna had a poor P3

    again, its now all about the FDA process


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    • "I think that the AA opportunity for Srpt is now dead, but that the straight approval opportunity off the P2 still exists"

      The primary endpoint for the phase2 trial was dystrophin production.

      It's currently impossible to get approval based on this endpoint.

      You're grasping at straw and not making sense.

    • simp - you missed the point of my post - mau and other posters post about the AA issue as if it has created some kind of delay or changed the odds of approval and there is no evidence of that. The timeline for the NDA submissal and potential approval has not changed and the only practical significance of whether they approve under AA regs or not is whether the Phase III trial (that SRPT will go forward with regardless of the FDA's decision) is deemed a confirmatory trial or not. The surrogate issue also has implications for future exons, but if SRPT's strategy is to pursue class approval for future exons, that might be a short-term issue, as well

    • I disagree. If RNA's P3 is bad, which is likely IMHO, then the only competition that exists for SRPT is effectively eliminated, ONCE AND FOR ALL. This is quite positive, not a negative. Who cares if the FDA punts once again, and screws over the boys who desperately need access to the drug by mandating an actual P3? Even if they do so, SRPT will have already won the exon 51 DMD race, and should then be valued based on dominating the entire exon 51 market except for Europe. If RNA's P3 data is poor, SRPT becomes the clear WINNER. The only question is whether the FDA allows the sick boys rapid access or whether they are cold, heartless hypocrites who just love playing games with the power they possess. Make no mistake though, RNA's P3 is bad = SRPT is the DMD winner.

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