peer review is not without its problems
remember that Korean cloning scandal? They had their stuff published but it was fabricated -- false.
Also, peer review does not mean peer tested and approved nor does it mean they look at other reviewed articles, study it, factor it into a decision whether X is effective or not.
Mistakes Are Made... and let me tell you they are made A LOT more than you'd think.
It is quite common for scientists to take peer reviewed publications and NOT implement it but to try to duplicate it. More times than you'd think, the results can't be duplicated. It is a bit of a natural thing to discuss new research and ask to first try to replicate it. The inside joke is if the paper came from the "journal of irreproducible results" (google it. it's like the onion for peer review science) So, it's literally that common to find such mystery works get published and it is TOTALLY not surprising to find stuff that just can't be replicated.
block trades move the market, but that does not mean much long-term.
I go back to the run from the 30s to the 50s and back. I was seeing block buys on the way up but it did not hold for long.
So, block trades don't indicate for long at all.
the person people hate on here thinks that any good news on SRPT means EVERYTHING to the market... at least for a short while (remember the recent run up from the 30s to the 50s and back to the 30s?)
Well, I personally say the market, if only for today, buy into the Barron's article despite my opinions that the important numbers are flawed ( 1)how long the cash lasts, 2) it is 50%, and 3) it should trade near cash value to discount the risk of 50% and to account for a burn rate that will sooner or later require a secondary or partership( give up part of profits for help) ... see why they hate me?)
So, if I had to pick up, down or flat for today? Up.
I got thousands without a issue.
now up 0.13 at 14.50 on 4,640
we will see how the market interprets the Barron's article today.
I have my opinion, we'll see how happy the market is with it.
(failure to account for higher burn rate from phase III means that they do not have the cash to last through phase III and into approval plus manufacturing. Also the chances are only 50% because the FDA clearly stated he provided data was within expectations of "natural history." Thus, one can't depend on the 12 kid study for anything... thus it's a coin flip right now... 50%. The analys on that story really blew the easy part -- the cash burn rate increasing. He just took the current burn rate to keep going.)
The FDA just looked at their data and essentially said: "this does not ave scientific merit"
this is at best 3 1/2 to 4 years from approval and all of SRP cash will e burned just testing, then on top of that they will need more to manufacture the drug.
So, this stock will have EXCESS $0.00 cash value, and in fact a future cash cost ( a future cash need). Sure, you buy the csh now dollar for dollar, but it will all be spent on development. This is not like buyig apple and haveing excess cash and positive cash flow.
So, I guess what I am getting at is there is substantial risk buying here. The cash will be gone before the testing is over and there is no proof yet that the drug absolutely works. So what would they get after 3 years? It's unknown.
The routes from here are a secondary or a partnership with big pharma. I EXPECT one or the other to be announced soon.
Congress may have some MDs and even do consults before making inquiries, but they are not the ones in the data trenches.
Congress making a politically based pressure call on the FDA would set a dangerous precedent.
If you want to argue, you better have proof positive yourself of FDA incompetence otherwise you have what will be the equivalent of the baseball steroid inquiry. maybe you get some players heads, but the problem just still continues. Why? Who is Congress will be bold enough to hold a vote on whether eteplirsen should or should not be approved? We both know that won't happen. If the FDA bows to pressure an approves the FDA, then all the big pharma will have pharmaco-lobbyists pushing Congress for compassionate approval instead of the FDA because Congress will be the cheaper route to approval.
as mentioned in many other threads, the AA process is not solely based off the safety profile. There is also efficacy
Let's both say this is safer than water.
Well, let's say it is water.
Just because it is safe does that mean anything towards being a drug?
The big point is that by the FDA approving something into a drug status, they are also telling the public "this works."
The fault here lies with SRPT providing no scientifically analyzed data to prove efficacy.
Further, as pointed out by pasteur, SRPT did not address the 2 kids that went non-ambulatory.
Everyone is assuming it was "too late." However, saying it and proving it are different.
I actually taught a 101 lab class as a TA. literally 101, not 102, not 202, a rue to life 101 class. Those students did what they were supposed to do in the discussion of the data -- they dealt with the elephants in the room: why did 2 kids go non-ambulatory? Prove it wasn't eteplirsen. Prove it was. and most importantly... if you are trying a new molecle in the body ... LOOK INTO IT JUST INN CASE. There is a moral obligation to do that.
the number of exon 51 patients is in the low thousands.
this is approximately the size of a typical phase III study these days.
It is not unrealistic to say that all exon 51 patients could be in the trial... well, those that are so low on their abilities that testing along would harm them could be put under compassionate use. But these would be the very, very oldesof the kids.
The rest of the population can be in the phase III.
It's odd that on a thread of hope you are arguing against a hopeful assertion... and from someone who is a SRPT believer, too.
Well, there are plenty of other threads to beat me up about the "ALL" part on. I welcome arguing it with you on them, not here.
I maintain my assertion about ""ALL"" the exon 51 kids being put in the phase III study.
prediction? I was in here telling people it was overbought and would sell.
It certainly can't be ALL wrong as there is right in there,
currently, MRI is not a sole diagnostic tool for muscular disorders. Clinical and laboratory tests are still required.
It is still and interesting idea that would bolster the clinical and laboratory results, not make them useless. So, don't get too excited about the MRI idea.
I want to add that I chose 4/5 and 10/3/12 as reference points because they are exactly 26 weeks apart both in measurement and in release dates.
I am thinking that the December 2012 data was out early... possibly due to the upcoming holidays.
considering week 120 is in February, there are no major holidays then, that I do not expect a pre-release. Although if December is any indication, they had a PR out 3 weeks early and that is still in late January.
There still could be an impending data release though.
108 weeks is the week of Thanksgiving. If they get it out early it could be this week.
recent PR history on X-week results
10/3/12 - 48 weeks
Dec/7/12 - 62 weeks (~9 weeks from 10/3/12)
4/5 - 74 weeks (~17 weeks from 12/7/12)
6/19 - 84 weeks (~11 weeks from 4/5)
9/26 - 96 weeks (~25 weeks from 4/5)
120 weeks - 74 weeks = 46 weeks.
46 - 25 = 21 weeks from 9/26
this is week 3 of Feb 2014
120 week - 48 weeks = 72 weeks from 10/3/12
this is also week 3 of Feb 2014
__EXPECT__ SRPT to continue to release 6MWT data on the 10 kids left to test.
The 120 week results are not due for a couple of months,
What to try again?
exactly what I have been saying thigrlrk.
a phase III is typically 2,000 patients.
I posted that months ago and you exclaimed... that's the ENTIRE population.
Then I wavered and said, ok maybe not everyone but most.. perhaps hundreds.
I changed my mind.
Yes,..... YES open a phase III to ALL exon 51 patients.
and they will have a range of abilities.
-nonabulatory but with upper body
-those non ambulatory and non upper body who get tested just for cardiopulmonary response.
the bad points that I pointed out then still apply now...
everyone is in the trial, BUT some are placebo AND who pays for the trial? The shareholders via SRPT.
everyone can be in this and NO compassionate use because of that. Or few if they are felt to have too low caridopulmonary response and further testing may not be medically advisable. Those would be the much much older patients. This would be solely an attempt to prolong life and no testing would be performed.
I'm taking the message to the "streets" by talking openly in other threads.
I would hope that compassionate use would never come up with SRPT.
I would hope that SRPT open eteplirsen trials to ALL exon 51 DMD patients.
I think that is the only way to go.
test can be designed for patients with varying ability states. It will be hard, though.
The belief of fraud is not well placed.
This mother is a mother of a trial patient.
IF there is a fraud, it is being performed on them making them the defrauded, not the fraudsters.
But I don't think it is fraud, just bad scientific testing so far. I do not see any conclusive evidence that this mother is complicit in anything. Yo have provided none, just insults.