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Sarepta Therapeutics, Inc. Message Board

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    • Registration of $100,000,000 in mixed securities is apparently now effective.

      http://www.sec.gov/Archives/edgar/data/873303/000119312512125674/d317440ds3.htm

      • 1 Reply to cotheo
      • I have been holding a reduced position in SRPT because of a fear of a secondary.

        The stock is up 20% in that time.

        If the company would do a secondary, I guess it would be at a 10% discount to market, or less, given the new investors who are now beginning to understand the real story here.

        If you are focusing on a secondary as a negative, you are missing the real important catalysts that are on the horizon that can move the stock much, much higher still.

        I have made that mistake and I'm guessing others have as well.

    • This slide just shows the safety profile for a lifelong chronic disease to be able to administer this and so it’s well tolerated and no safety signals. Again, this is through 24 weeks, we also looked at the profile through 36 weeks now, and again, we are seeing no treatment related adverse events. And all of the bullets on the right-hand side are knowing things to occur with competitive chemistries in the space, the type chemistry where ours is a very different backbone chemistry, and we’re not seeing the dose from toxicities that you see with other all the good chemistries.

      • 3 Replies to long_dndn_avii_gnta
      • This slide just shows the safety profile for a lifelong chronic disease to be able to administer this and so it’s well tolerated and no safety signals. Again, this is through 24 weeks, we also looked at the profile through 36 weeks now, and again, we are seeing no treatment related adverse events. And all of the bullets on the right-hand side are knowing things to occur with competitive chemistries in the space, the type chemistry where ours is a very different backbone chemistry, and we’re not seeing the dose from toxicities that you see with other all the good chemistries.

      • And we got our answer; that the good news we did show a statistically significant increase in dystrophin essential protein at 24 weeks, we saw greater than 20% mean levels, so this was beyond our expectations in terms of the level of the dystrophin, we were hoping for, we did hit our p-value of 0.0002, compared to the placebo group, and again this was the 30mg cohort at 24 weeks, after 24 weeks of treatment. And the reason for 10% or greater was the literature on animal study suggested that should translate to functional improvement and the Becker muscular dystrophy, that’s a naturally occurring again inferring deletion. Many of those patients have 8%, 10%, 15%, 20% dystrophin and have much milder disease and Duchenne. There are also Dystrophin patients who have more than 30% or even more than 50% but all of them are marked by a milder disease and Duchenne.

      • We think our treatment difference that we are seeing here at 36 weeks an relatively early time point, compared to some of these studies, is very meaningful, clinically meaningful and a good marker of showing that we may be slowing or halting the progressive disease. And one is on here as well, it’s the most advanced DMD drug that’s been studied the clinic, and again just to compare in the DMD population with a 365 6-minute walk test baseline over 48 weeks they showed a 30 meter improvement, on 6-minute walk test. So we believe that our data set albeit in a small population holds up and stands up well to other genetic diseases and DMD drugs that have been studied on 6-minute walk.

        This is also encouraging because of the potential that this technology has to go after other exon target in the DMD population. The only difference between this drug that’s in development and future drugs that were target other exons is the sequence that is attached to our backbone chemistry. The backbone chemistry would be the same. The way it’s manufactured would be the same. We have seen historically the same backbone, you see similar pharmacokinetics and it’s a very safe profile, no matter what disease it’s been studied in and the various dosing and dosing regimens that have been used.

        So we think this is highly reproducible and leveraged across other exon targets. And we already have two collaborations ongoing to advance approximately the top five or so exons. And we have collaborations in place to do the IND enabling work on exon 45 and exon 50. We are in discussions with granting authorities to support exon 53 in preclinical development and to initial clinical development and then exon 44, we’ve heard some interest from our foundations to support that work. So again, we are very encouraged by this and (inaudible) but also in terms of the implication it has to treat the broader DMD population.

 
SRPT
22.29-0.20(-0.89%)Oct 22 4:00 PMEDT

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