if you think the data shows nothing but stability, on an aggregate basis, I agree
if you are saying the data shows nothing but stability, on an individual basis, I can only conclude you are looking at the wrong data
since I am nuts, I suggest you ask jrrt or micro about what the individual 120 week data suggests
all I want to know is if you are going to call them names when they answer ...
my dear tred:
you just said "the 144 week data almost needs to be flawless or will be subject to attack by the shorts and/or uneducated"
um, that sounds vaguely familiar to me ...
but the real question is, do you remember that when I said it the other day, you immediately told me I was a nut and that the forward data was less important?
I am just so confused by your postings ...
I think we need to assume possibility of rocky road going forward on the data
the combined 120 week data did the trick, as there were some very strong performers, but the individual data suggested some others were on the slide
thus, with each ensuing 24 week data set, the chances for a slip of a few or even one, increases
and with an N of 10 (and individual cohorts of 6 and 4, more importantly), its heightened
again, I was nervous at 120, and am more nervous at 144
Are you suggesting tired is as smart as you are?
He is smart
But I would not go that far
Most of the anecdotal evidence I have comes from your posts
No, I mean it
tred, I am going out on a limb here and inferring that your opinion differs from mine
accordingly, and ergo, I am hereby calling you a butt sniffer who did not cry when old yeller died
you are the spawn of the devil and I will pray for your lost soul
like you, I have now spent quite a bit of time reviewing all available 120 week data
I concluded that the SS status of the data was particularly fragile, given the N, as we saw in the original placebo cohort when one boy broke his ankle
I understand but do not espouse to the stated views that the most important SS data points were already proven and that forward data is not as important any more
having read the quotes from the FDA srpt letter and also the entire FDA rna letter (which I believe has relevance to the srpt letter), I have concluded there essentially are no rigid rules at the FDA, and that in fact, the forward data could influence the FDA as much if not more than the prior data
so IMO, the 144 wk data matters, and remains "binary" to me
the FDA dystrophin analysis remains just as key, in terms of what the FDA views on current d data are, after communicating with those who actually did that work, and whether the 4th biop will be needed, or even required by the FDA in final analysis
as far as "had your opinion changed", the totality of my opinion changes to some degree (big or small), every time I update my diligence
I was mildly worried about the 120 week data, and I believe that data, behind the curtain, did show the fragility of the depicted bullish nature of the results
I am more worried about the 144 week data, as a result of my latest round of diligence
PS: I did not use the word "anecdotal" out of respect for my friend zoooey, but anecdotal evidence does have its role in all of my thinking
your ferocious attacks on my person are excruciating, yet heart warming
but I do feel compelled to remind you that I am now under piney's protective shroud
I have assured her we are close friends but she can be very over protective
maybe you could say it like this: "I say this with a kind heart, but simp is a moron, wrong and a waste"
I continue to believe the long investment community needs to insert a greater sense of realism into future etep data
the n is 12
with regard to ambulation, its really only 10
do the math
we saw what the walk test of the ONE boy with the broken ankle did to the original placebo group aggregate results: SS became CM
all the boys are getting older, and heavier, not not necessarily having a longer stride
IMO, the idea that they are all remaining stable, and even improving, is remote
the drug is not a cure
it has shown, within wide ranges of vagary, to slow the progression vs. NH
the perceived shift in FDA and EMA views on this disease seems to suggest a drug which slows deterioration vs. NH should be approved in this instance (i.e., the patients can deteriorate but still be better off than without the drug)
there are dozens of cancer drugs which were believed to extend life by under a year, when FDA approved
just look at what RNA is doing, OFF ITS RESULTS TO DATE
my point is SIMPle
measure the results against a reasonable realistic standard, and against the improved FDA view, NOT against a standard, this, and no other current drug, can meet
I believe the shorts are now, at least in part, counting on long investor misguided disappointment to provide cover
Thank you to the responders
I assume you can figure out why I asked the question
I think it time to realistically assess the real world prospects on 144 and 168 week results
The boys are getting older and heavier
The drug is not a cure
Stability and/or slower decline is what is more real
Eventually just slower decline
Better than natural history should justify approval
IMO the bar may be set too high by investors
The shorts know this
what is your reaction to the below quote about what individual 120 week data showed:
"Curious on the 144 wk. IMO several boys below 300m at 120wk. Think overall likely stable, but maybe individual a concern to market."
this relates directly to the investigation of Eric Hoffman's bathroom (as opposed to fiduciary) duties being currently undertaken by Simp08801 PDQ, as previously announced