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Sarepta Therapeutics, Inc. Message Board

themicrokid 4 posts  |  Last Activity: 4 hours ago Member since: Jul 10, 2001
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  • themicrokid themicrokid 4 hours ago Flag

    There was one dris data set, which showed a boy on placebo with increased dystrophin. Recall at one time GSK used the standard on an increase of dystrophin by any of 3 methods counted as an increase in dystrophin. I am not interested enough to trace back to where I saw this.

    Sentiment: Buy

  • Reply to

    RNA readthrough to SRPT

    by jnajay Dec 27, 2014 7:12 PM
    themicrokid themicrokid Dec 28, 2014 3:55 PM Flag

    I have no idea on the timeline. And recall, we have clocks running in both US and the EU.

    Sentiment: Buy

  • Reply to

    RNA readthrough to SRPT

    by jnajay Dec 27, 2014 7:12 PM
    themicrokid themicrokid Dec 28, 2014 12:37 PM Flag

    It is really almost impossible short of being a patent attorney highly skilled in drug cases to value the RNA or the SRPT patent portfolios in exon skipping. I have seen lots of arguments, made lots of arguments. But in the end it comes down to the specific claims allowed when all the hoopla about date of invention is decided. And the applicable invention in any case isn't a drug backbone, but an application of a drug to exon skipping for the dsytrophin gene.

    One thing BMRN has the rest of us don't is access to those attorneys, who can make a more informed judgement. And if RNA wins IP battle, it may even be possible for them to base an exon skipper on a morpholino backbone, if need be. Sarepta's morpholino backbone patents on the basic backbone have long expired. Of course an RNA IP win blocking Eteplirsen in some or all meaningful locations might allow BMRN to seek a royalty that would be unreal.

    The bothersome thing in either party really winning, is it is the universities that hold the IP that could prevent boys from life saving drugs. It is clear universities around the world should develop licensing agreements which up front establish a royalty structure for a second party and limit exclusivity. Because otherwise they could really be parties to watching innocent boys die needlessly. Just as the FDA is a party to such.

    Would one call the actions of either manslaughter? Some would argue the same about a drug company not pushing as hard as it could to get such a drug approved. A big differentiator is that the FDA and possibly the unviersities are supported by tax dollars from somewhere in the world and due to their nature should be held to a higher standard than for profit companies.

    Sentiment: Buy

  • Reply to

    Jrrt

    by thigrlsrk Dec 25, 2014 5:53 PM
    themicrokid themicrokid Dec 26, 2014 3:35 PM Flag

    Logic points us in the direction Eteplirsen will be effective or somewhat effective, if there is enough muscle to restore in any region it penetrates, IF it is started early enough to achieve sufficient penetration and time to build dystrophin. The non ambulant boys in the new trial may give some indications of this.

    Sentiment: Buy

SRPT
12.32-0.12(-0.96%)Jan 27 4:00 PMEST

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