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Vertex Pharmaceuticals Incorporated Message Board

themicrokid 6 posts  |  Last Activity: Feb 17, 2015 9:37 PM Member since: Jul 10, 2001
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  • Reply to

    srpt ebola opp is dead

    by simp08801 Feb 6, 2015 8:27 AM
    themicrokid themicrokid Feb 6, 2015 8:45 AM Flag

    Only slightly possibly, if the ebola drug mutated, the Sarepta drug might come into play. If you recall one of the antiviral approaches Iversen took in antivirals was to target conserved regions where mutation was unlikely. Though I think at least one other drug may also do this in the ebola arena.

    Also, the response time, if there were a bio engineered virus based on ebola by some terrorist group, the time to respond, with an antiviral, might go to Sarepta, but at this time the limited production availability would be an issue.

    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

    Efficacy Matters

    by kgrichard5 Feb 17, 2015 7:24 PM
    themicrokid themicrokid Feb 17, 2015 9:25 PM Flag

    The problem is "placebo effect" especially in a trial, where there are early results strongly reporting positive effects. I am very certain the drug works, but expect early reports if any from the new trial subjects will be enhanced by the placebo effect, unless there is real disappointment.

    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

  • themicrokid themicrokid Jan 28, 2015 12:12 AM 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

    BOA Report part 1

    by likeafox7210 Feb 17, 2015 8:05 AM
    themicrokid themicrokid Feb 17, 2015 9:37 PM Flag

    I think another key reason Mendell and Sarepta should want the 4th biopsy beyond the FDA reasons is to better understand the drug and method of action. People have speculated about all kinds of immune reactions, etc. The real question is does dystrophin continue to increase or did it for some reason decrease? Is the dropping in 6 MWT scores in due to dystrophin issues or is it strictly a matter that the boys are becoming taller and heavier. Are they really following a becker model or something else? One of the real issues is that when you think is a statistical manner, the tails steroid treated boys for duchenne and even strictly an exon 51 skip appear to be extremely wide.

    Sentiment: Buy

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