For all we know the FDA is now looking at those "patient reported effects".... Let's hold our fire, not assume or prejudge, and hope they've been moved into a new posture after the last few month's developments. I think it's all got to have had a positive effect.
Remember also, that Chris said months ago that at THAT time he thought they (the FDA) had been given data equivalent to an NDA filing from us. Since then he's given them (and they've asked for) more and more again. So we may be pleasantly surprised with how far in our direction they MAY be willing to move by now.
(Someone needs to at least lay out the positive side of things, right? - once in a while, anyway!)
According to Chris's March 4 presentation we start a new trial in Europe (England?) on our exon 53 drug sometime in the second quarter. If this fast track thing in Britain is for real (FDA take note), if safety is same as Eteplirsen and the drug is shown to be creating dystrophin at 24 weeks or 36 or whenever, then we could have that drug for sale there by early next summer or so. And you'd have to believe that our exon 51 will have found it's way to being made available there too at the same time - if not before. Synergy being what it is.
But I guess you weren't paying attention the other night when you, Jay and Simphero posted the same message over and over between the three of you. With out any comments upon such madness. What was that akll about? Give it up. Others are paying attention too.
That WAS a strage sentence - especially since it was preceeded by the word "Therefor" - yet the sentence had no logical connection to the previous sentences!!!
Additionally, the thought is really unconnected to the key themes of the whole paper. Perhaps it was thrown in (almost randomly, as I say) to placate the hurt feelings or ruffled feathers of the FDA "folks" who are reading it today. The whole point of the paper is to make the case for future accelerted approvals based on dystrophin levels and distribution, and so to throw in a prescription for a non-accelerated approval type trial coming up - the perfect trial in a perfect (albeit unethical or amoral) world - I think was just throwing them a bone. It's pointless as far as the real intentions and ideas of the paper go.
The paper had two themes. The one I thought was most important ultimately (for the future) had to do with what the FDA should be looking for in the next trials if they want to grant an accelerated approval. They are saying "Yes, dystrophin level (and even dystribution) is indeed what counts and here's how to best measure it (with current tech) to predict efficacy".
They got to that important conclusion by way of disputing and correcting the FDA's Nov. 12th misinterpretting of the Drisapersen and Ataluren trial results.
Really couldn't be a better timed paper for our current and future purposes. (And, as I said, could explain the delay Chris may have had a hand in.....waiting upon this publication)
I just finished reading it. Agree that everything that was said was "Eteplirsen positive".
The key take-away seems to be they recommend that in a good determinative trial Western blot must show the intensity of dystrophin increasing from the initial biopsy (before treatment) and the biopsy taken "after" treatment. They at one point seem to say it should show 10% or more dystrophin levels. (more on that below)...Then the immunophloesense test should indicate even distribution of dystrophin over a good percentage of fibers.
I was sad to see our Western blot numbers looked pretty low - but they allow that they may not be seeing all that data. (It seems to me Chris had just recently said that western blot data looked very good but I'd have to review his last presentation to be sure).
But the authors kind of skip over that issue as they wrap up and point to the clinical results that look to be vey good in our kids, seeming thus to leave the question of how much dystrophin a Western blot test should show to indicate efficacy hanging - to be answered in the future perhaps..
This multiple id act - with your whole cast of screwballs and cornballs and neurotics and kooks - makes Sarepta look like a joke company that for some reason attracts screwballs, cornballs, neurotics and kooks. Your act - intended to be educational, Socratic, and liven things up - has at this point resulted in laying a bad stink on the company in many serious investors minds. Turning them off. Maybe it gives even the FDA pause. Why don't you just give it up now and see if Sarepta can get by on it's own merits - without all your "help". Perhaps your time has come and gone. Perhaps you've served your purpose.
Gave yourself away with that one, Grey. Not your style. Whoops. Have a left shoe I could "capture" as a momento of our frienship?
...and may possibly explain the delay in our meeting.....(Chris may have been waiting for this to publish).....
CRN is not UNA (unlocked nucleic acid) .....Additionally we sold the UNA to Arcturus. Not sure if we sold these Tekmira royalty rights from their use of the UNA to Arcturus as well. Maybe Newby can answer that for us if he's listening. (And wants to!)
Seems the way things go around here: - As late as possible for everything. (Except getting the drug manufactured).
Also, big news on April Fool's Day is our norm as well. Usually it's been bad news. Time for something completely different this time? Only seems fair.
Why were you, he and herosimp posting identical messages the other night, then? I've seen that happening on other boards that are filled with "multi-ids"....It seems like it's being done by Yahoo to perhaps begin exposing the annoying game a few choice losers indulge in. An indulgence that ultimately turns real people off and causes them to stop posting here.
Right. But why were you and jay and Grey posting identical messages the other night? Without commenting on it? Give it up. It's becomming embarrassing.... and counter-productive. And insulting to the real posters here. Not to mention a bore.
I noticed you and two or three other ids here were posting the same exact message aver and over a few nights ago. Didn't seem to bother or even interest any of you, though. That was odd. I suppose you missed that? but I think many here didn't. Whoops.
Seems like it.
And you are missing the fact that that's not the story of today. You know, the one we are talking about today?'s Pat "news". You must try to stop living in your past, Starfe. Try and stay current. Try and stay focussed on the present topic. Get off your tried and true hobby-horse. Hard at your old age?
Or are you just excited someone's talking to you? And trying desperately to keep it going? (I think that's probably it)
She did nothing. except explain why she's done nothing for eteplirsen. (While not explaining why she's done everything for drisapersen!)....She said, "I'll push for eteplirsen once it's filed an NDA". Is that supposed to be something unexpected of an organization such as hers? Another point: If the FDA says to Sarepta "go ahead and file it," well, that pretty much means they plan on accepting it. We need Pat's help at that point? Probably not very much at all.
Now if Prosensa files an NDA without being asked, then yes, Pat's help wouldf be VERY important in pushing Prosensa's (rather poor) case with the FDA - who never asked for the submission. Getting it yet?. And so now she is set nicely up to justify her doing just that, isn't she?.