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Sarepta Therapeutics, Inc. Message Board

  • immediatereliefxxx immediatereliefxxx Sep 6, 2013 6:48 AM Flag

    RNA ceo doing damage control in advance of data release

    A week or two ago, I argued extensively that Prosensa-connected scientist Aartsma-Rus's public comments clearly indicated knowledge of mediocre results in the Prosensa Phase III for drisapersen (the potential competitor to SRPT's eteplirsen). Now comes the Prosensa ceo, speaking to Adam Feuerstein, basically trying to do damage control in advance of the data release.

    How best to get these points across? Simp style? I'll try:

    Look at the anecdotal evidence

    Aartsma-Rus, who has access to all of the data, has stated that the dystrophin evidence is spotty and that the clinical results are unclear. She stated this in her letter to ppmd.

    The CEO has now warned AF, and subsequently it would seem other analysts in a private meeting yesterday, that the trial was poorly designed to show efficacy. It's foolish to pretend he doesn't already know the trial outcome. He knows. He's just started his spin early.

    Meanwhile, in Sarepta land, we see videos of Billy and Max running almost daily.

    Now what is it we're supposed to do now? "Connect the dots" or something?

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    • It's even worse than I thought then, and FDA granted them BT designation for nothing.

    • Just read AF's comments from the meeting with RNA execs. They are hedging on whether the trial will generate a statistically significant difference in 6MWT vs placebo because they enrolled patients younger than 7 years of age, who (if included in the placebo arm) might show stabilization or improvement, even though they are untreated. They claim that this is new knowledge - that deterioration in leg muscle capacity and 6MWT performance typically doesn't happen until after age 7. Since when is this new knowledge - all these "natural history" studies that CG references in his presentations weren't around when GSK designed this trial? They didn't even stratify the patients by age to ensure that there was balance between the treated and control arms. Like immediate indicated - sounds like the blew the trial and are pre-spinning the results to minimize the damage.

      • 2 Replies to tredleon
      • I don't believe for a moment that the inclusion criteria was a mistake. Young boys were included to maximize the chances that there would be just enough variability in the data to reject the null hypothesis. Intimate knowledge on the natural history is sufficient to rig a study. If it's obvious from the side-effects you're getting drug, you don't really have a placebo control.

      • Yeah, it's crazy, isn't it? Both on Yahoo and twitter, SRPT longs have been bashing GSK/RNA's trial design for months because it included younger boys. How is it possible that a company specializing in DMD just now figured out that boys don't go into measurable decline until they are older?

        But here's the takeaway, in my opinion: If the trial design screwed up the reading of drisapersen's data as much as I think it did, then GSK/RNA will simply not be able to get their application into the FDA ahead of Sarepta. Rather than a submitting a straightforward application, they will have to slice and dice the data, meet with the FDA, meet with the FDA again, etc.

        Sarepta has just pulled into the lead now to be not merely the best but the FIRST drug on market.

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