Wed, Mar 4, 2015, 10:57 AM EST - U.S. Markets close in 5 hrs 3 mins


% | $
Quotes you view appear here for quick access.

Sarepta Therapeutics, Inc. Message Board

  • bionerd51 bionerd51 Oct 30, 2013 10:10 AM Flag

    Seeking Alpha....Seeking a Gullible Audience....

    The author calls a failed exeprimental drug, Drisa, "promising" but calls Eterplirsen's prospects "dismal" and will "fail" it's PhaseIII trial.I'm wondering if this putative analyst/author is dyslexic. Dyslexic in the way he reverses reality. Eterplirsen is the therapeutic that has produced significant truncated functional dystrophin in ALL patients. In the head-to-head IM study, at 2mg/kg and above, Eterplirsen produced significant dystrophin and Drisa produced nada. Questionable whether they[Drisa] produced higher concentrations of dystrophin at 6mg/kg. The one data point that Drisa proponents point to is the 6MWT at 24 weeks. Classic placebo reaction enhanced by the fact that each patient and parent knew who was in the treatment cohort and who was receiving the placebo. The clearest evidence that the 24 week result was not drug related is the fact that dystrophin levels were low and inconsistent and the 6MWT was not repeated post 24 weeks. It's been postulated that for any AO exon-skipping therapy to produce a therapeutic effect dystrophin would have to reach the 15%-20% total dystrophin levels. Drisa didnot come close so there is no correlation between Drisa and dystrophin and the 6MWT. None. Eterplirsen produced statistically significant dystrophin and the 6MWT versus historical decline. This is not in dispute so the only question left to answer is motivation. Why did the author write a "short" hit piece for an online rag like Seeking Alpha? It's not based on data.

    SortNewest  |  Oldest  |  Most Replied Expand all replies
    • "Why did the author write a "short" hit piece for an online rag like Seeking Alpha? It's not based on data"
      Right you are about the hit piece not being based on data. This is about concerted short sellers, and there are a lot, successfully driving the price down to 1) trigger sell stops, 2) break support levels, 3) shake out weak hands, and 4) cover short positions. The shorts are also capitalizing on the panic driven "risk off" in biotech. It's just shorts doing their work. Those who don't need to sell will just wait for additional positive data to overcome and overwhelm the short thesis. Fear is a much more powerful emotion than greed.

      Sentiment: Strong Buy

    • If you buy that bs go long RNA and short SRPT and shut up. Please!

      Sentiment: Strong Buy

    • Bionerd, I’ve been rereading Steven B’s SA article and am confused by its assertion that E is less robust than Drisa wrt dystrophin production (both time to action and distribution %) as well as his contention that while E's dystrophin increase was insignificant within the first 12 weeks, the boys produced a clinically significant result in the 6-MWT at 12 weeks, decoupling the link between the surrogate and clinical endpoints.

      Are his facts just wrong? or cherry-picked? Your post above states that Drisa did not attain the 15-20% dystrophin levels to be therapeutic and that E. does. Thanks for any response.

      The exact part of his article that confuses me is as follows: "it appears that SRPT's eteplirsen acts more slowly, given the 0.8% increase in dystrophin positive fibers at 12 weeks at the 50 mg/kg dose and 22.4% at 24 weeks at the 30 mg/kg dose vs. Prosensa's drisapersen which appears to work much more quickly; large increases in overall dystrophin positive fibers (57%-75% in 7 weeks and earlier compared to baseline levels of 7% and lower signal intensity). Therefore, it is unlikely that eteplirsen will succeed based on this dystrophin data.

      In addition, in study 201, there was a discrepancy in dystrophin production and its correlation with the 6MWT. Dystrophin production was measured for the 50 mg/kg dose and demonstrated a 0.8% increase in dystrophin positive fibers at 12 weeks (not stat. significant), but demonstrated stat. significance for the 50 mg/kg dose on the 6MWT combined with the significant dystrophin production at the 30 mg/kg (22.5% at 24 weeks) but insignificant benefit on the 6MWT calls into question the biological hypothesis of dystrophin production and its associated clinical benefit."

      • 2 Replies to tuneoutthechatter
      • tune...(cont.)
        Mendell also indicates that they have MRI studies between 24-28wks which, on these boys show 'revealing a severe loss of muscle tissue with replacement of fat andconnective tissue that was much more extensive than in all of the other patients.' Thus, their exclusion was based on statistical evaluation for 'outliers' and wasn't just a 'Oh, these don't help so let's throw them out' kind of reasoning.

        Also interesting (to me, anyway) was there were 5 different genotypes/variants of 51 deletions among the 12 study boys. Haven't seen any data evaluating significance of this, if any, clinically.

        Interesting data. Still a bit of a #$%$-shoot trying to predict what FDA will do. Doesn't matter much over long run as long as etep works--and IMO I don't see how it can't based on data so far. Oh, and as per reg's complaint of single site--the followon study (post 24wk) was at 10 sites.

        Regardless, with all the angst about and the beating the short/sell side has given the price, it may continue for a while absent some positive news in SOME area from the company.


      • tune...
        The 'slower' is accurate with 43, 52, 38% change in dystrophin in 50/30/delayed groups repectively by 48wks. All these were statistically sign. to

    • time for chris to step up to the plate and break their backs, like the Bosox eh,

      Sentiment: Strong Buy

      • 2 Replies to rico22
      • He will first have to figure out if what he did that has "a small number of institutional investors" (AF) saying he's a liar which is breaking the backs of investors who believe in Etep. 1/3 of shares short is a disgrace and it doesn't seem to be just a "small number" but a widespread conviction that there is no upside but plenty of downside risk because of unwise expectation management. Like, FDA does not agree with unblinded trial design already communicated, manufacturing is not ahead of schedule as already communicated, NDA will not be accepted for filing until Ph3 trial has finished.

      • Rico

        all in good time

        CG has a plan

        Sentiment: Strong Buy

13.34-0.02(-0.15%)10:56 AMEST

Trending Tickers

Trending Tickers features significant U.S. stocks showing the most dramatic increase in user interest in Yahoo Finance in the previous hour over historic norms. The list is limited to those equities which trade at least 100,000 shares on an average day and have a market cap of more than $300 million.