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Vertex Pharmaceuticals Incorporated Message Board

themicrokid 2008 posts  |  Last Activity: Jul 10, 2015 8:42 AM Member since: Jul 10, 2001
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  • Reply to

    It's working in the new trials!

    by immediatereliefxxx Jul 9, 2015 11:23 AM
    themicrokid themicrokid Jul 10, 2015 8:42 AM Flag

    1. The US trial showed no consistent production of dystrophin till 24 weeks. If I recall the 12 wk 50 m, gave hints of some dystrophin increases, but nothing approaching certainty.
    2. The UK trial showed some very inconsistent dystrophin increases at 12 weeks at much smaller dosages.
    3. Dystrophin measurements are only noted where the biopsies are taken, It is quite possible the dystrophin may increase faster or slower in other parts of the body.
    4. It is not obvious to know how much dystrophin is really meaningful.
    5. Isn't it likely dystrophin may increase in a body in a very different way, if that particular body has had large muscle masses atrophy due to being wheelchair bound?
    6. And last not being least, it is quite likely some small portion of the gains are due to placebo effect.

    Hopefully the FDA is ready to step up and do their job as they have done on previous rare diseases. It is a shame it took them so long to get there.

    Sentiment: Strong Buy

  • Reply to

    Patent Law 2015….

    by bionerd51 Jul 3, 2015 9:28 AM
    themicrokid themicrokid Jul 4, 2015 11:38 AM Flag

    Going from memory the A.Rus patent didn't state the Kole patent as prior art. Even if it had expired it is still prior art. Therefore they really didn't address the test as to whether the A.Rus patent was a non obvious step over Kole and other prior art combined. Furthermore there was an A.Rus publication out of the window for time till a patent would have to be filed and they appeared to have ignored whether the patented material was a non obvious step over this earlier A.Rus disclosure. Furthermore the A.Rus patent is very broad and many claims should be narrowed to assure they are 1) non obvious over prior art and 2) They are enabled by the disclosures in the patent.

    On a bigger topic, I have a real issue with universities granting exclusive licences to firms on IP that drives life and death issues. I would have no problem, if they granted a license with some signficant first licensee advantage, Possibly involving lower fees, some short exclusivity, but not to the point where it should be restrictive. Now, if it was a truly private university, I might be more inclined to allow the present system, Thouh I question whether there is really such a thing as a truly private university, since most depening on some government funding and tax benefits.
    Feels to me like a European University got a big home field advantage in deciding to allow the A.Rus patents. I also am not a patent lawyer.

    Sentiment: Strong Buy

  • Reply to

    GSK???

    by pearsby09 Jun 12, 2015 5:17 PM
    themicrokid themicrokid Jun 13, 2015 1:57 PM Flag

    We really have no idea whether the doors were being pounded on at Sarepta. We know Sarepta publically stated they weren't interested in a partnership. One big question with Sarepta's technology, if it will be possible to manufacture PMO's in volume at a viable price, the answer appears to becoming clearer that it will be. The next question is will it be reasonable enough to attack non rare disease markets? If so, and if the testing continues to be good Sarepta may have an incalculable value! It just needs a cash generator to fund going forward.

    It looked clear that based on precedent in rare diseases that the cash generator was there until the FDA set aside science and went off on the not ready tangent based on the screwed up Prosensa/GSK results.

    Now 2 years later, there is a lot more data and the FDA appears ready to do what it should have been ready to do long ago. Never mind the innocent kids that died and lost the ability to walk because of its sad faulty logic.

    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

  • 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

    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

  • 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

    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

  • Reply to

    Settlement discussions on patent inteference

    by rstos14 Oct 22, 2014 10:18 AM
    themicrokid themicrokid Oct 22, 2014 11:57 AM Flag

    My read is that UWA does not agree that settlement discussions are underway, but does agree they are discussing starting settlement agreement discussions.

    UWA --
    "more correct characterization of the status of settlement discussions is:
    24 “In accordance with SO ¶126.4, Senior Party Academisch Ziekenhuis Leiden
    25 hereby indicates that the parties are in contact with each other concerning
    26 initiating discussions directed to settling the interferences. The parties believe
    27 that a teleconference with the Administrative Patent Judge is not necessary at this
    28 time, but they will make themselves available if a conference call is requested by
    29 the APJ.” "

    Leiden --

    "“In accordance with SO ¶126.4, Senior Party Academisch Ziekenhuis Leiden
    15 hereby indicates that the parties are in contact with each other and that initial
    16 settlement discussions as provided in SO ¶ 126.2 have begun between the
    17 parties.” "

    Sentiment: Buy

  • Reply to

    WHY WHY WHY

    by simp08801 Oct 5, 2014 4:18 PM
    themicrokid themicrokid Oct 5, 2014 10:41 PM Flag

    Never underestimate the incompetence and irrationality of burecrats and politicians.

    Sarepta at one time had it's supporters in DOD, they should understand the issue.

    But they also know leaders are the one with arrows in their backs! And if the crisis does get worse in US, CDC and USAMRIID will probably come out of this with better funding than they have had for years.

    ***** Changing gears*****
    Ebola in Africa - crisis level
    Marburg in Africa
    Entrovirus in US

    Is there another shoe to drop? Is it just coincidence viral outbreaks on the ....?

    Sentiment: Buy

  • themicrokid by themicrokid Oct 1, 2014 9:52 AM Flag

    1. This does not cost the company $13 million dollars. It cost the company $1,771.00 plus some legal fees.
    2. Unless the stock is above $21.85 when these are exercised, they won't be exercised.
    3. Hopefully the majority of these are to incentivize new employess for joining company or to reward and encourage employees to stay with the company.
    4. Yes, if exercised they are dilutive. But they also could add $13 million dollars to cash position someday.

    All that said, I agree CG personally gets way to many options for his performance.

    Sentiment: Buy

  • Reply to

    CAN'T DO A EBOLA PRESS RELEASE UNTIL

    by usagary1 Sep 26, 2014 7:58 AM
    themicrokid themicrokid Sep 29, 2014 8:18 AM Flag

    There were past indications that the PMO+ drugs were contracted out. As I recall there may have been a PR stating an issue at a subcontractor with the PMO+ at one time or possibly it was in a transcript.

    Sentiment: Buy

  • Reply to

    CEO disclosure behavior should be duly noted

    by simp08801 Sep 20, 2014 10:18 AM
    themicrokid themicrokid Sep 20, 2014 5:33 PM Flag

    I agree with you also. The bothersome thing is another trial should have been planned and started to increase the population once it was clear the drug worked. And that was almost 2 years ago!

    Sentiment: Buy

  • Reply to

    CEO disclosure behavior should be duly noted

    by simp08801 Sep 20, 2014 10:18 AM
    themicrokid themicrokid Sep 20, 2014 5:31 PM Flag

    I agree with you! It is why I made a graphic showing the slip and posted it. Maybe I should have played nice and also mailed it to Sarepta. The repeated revising and slipping schedules is getting very tiring. I am sure many of these things are beyond Sarepta's control, but still some delays are pure decisions.

    Sentiment: Buy

  • themicrokid themicrokid Sep 15, 2014 12:54 PM Flag

    ROFLMAO -- There are many more folks at Sarepta than Garabedian! Jayant Aphale sounds like he has the general experience to lead up the manufacturing.

    Sentiment: Buy

  • themicrokid themicrokid Sep 14, 2014 7:05 PM Flag

    I agree with you they need alignment with the FDA, but even a listing on clinicaltrials gov would give a slight smoke signal of progress and many companies list and then tweak. What concerns me, is it seems there is a never ending pattern of slipping.

    Hopefully the BOD gave Chris the signal that he has to stop slippage.

    The EU 4053 trial also has slipped from the schedule, though the slippage appears to be only a few months over a couple years.

    Sentiment: Buy

  • Looking at the difference between the July/August presentationa and the September presentation, it is alarming that it appears the start of the open label ambulant trial has been slipping about at a month per month rate.

    See the PDF I posted at IV!

    Why isn't there at least 1 trial listed at clinicaltrials? There really should be at least two! The Eteplirsen in ambulant boys and the EU's 4053 open label study! Originally they were supposed to be dosing now.

    Terrifying number of events Sarepta has to finish in the last 3.5 months of the year!

    Sentiment: Buy

  • Reply to

    ZMapp new study....

    by jrrt1 Sep 6, 2014 7:54 AM
    themicrokid themicrokid Sep 7, 2014 10:59 PM Flag

    we only have a very good idea Sarepta's ebola treatment safe in humans at higher doses, based on the combo ebola trial & marburg similiarity plus the primate studies. Hard to imagine there is not a dose response curve until some saturation point is reached. The half life of the drug will remain unchanged with the dose, BUT if you double the dose, at 1 half life, you will still have twice as much drug in the patients system. obviously there are limits to where you can go in increasing dose till you lose effectiveness in increased amounts or ... Recall Iversen who did initial ebola work was at Nebraska before AVII

    Sentiment: Buy

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