I do not suggest that srpt overstated the drisa tox issues
rather, I state that srpt analyst and investor commentary did
this is not BioMarin's first ride in the orphan drug rodeo
to assume BioMarin has a less informed view of the issue than analysts and investors seems quite unreasonable to me
as for showing SS, there are many approved drugs that never showed that
all the data on all sides is, IMO, noisy to some degree
the one thing I fee quite sure of is that Srpt cannot afford ANY further timetable delays, as the chances of further RNA delays in its schedule are greatly reduced now that BioMarin is involved
as an srpt long investor, I fear any adcom for RNA in an environment where Srpt is a continuing laggard on FDA requirements
so when I hear that all 10 ambulant boys are currently stable through 168, I will find that literally true when and if the eventual 168 data proves it out
until then, I remind all that fuzzy and literal are spelled very differently
I do not know if its true
but there was a suggestion that all 6 boys in the RNA x43 trial have dropped out
but further, enough time has passed that if this was literally true, it would leak out further
and it has not
and those suggestions of this came from somebody the srpt followers generally assume speaks gospel
my current view is that nobody speaks gospel here
almost everything you hear is to some degree, either overstated or understated to fit the bent
The BioMarin purchase of RNA suggests that the RNA toxicity issues have been to some degree, overstated in the Srpt mass media circles
We must assume that BioMarin particularly diligenced this issue and determined that the RNA safety issues did not rise to the point of overcoming the potential clinical value of Drisa to dmd x 51 patients
frankly, I find it impossible to reasonably assume otherwise
I think the "only to find out later" language was specifically written to imply that he was hood winking the community and investors
I am not saying I agree with such assessment, but I take that phrase to clearly insinuate it
I believe those exact words were by design
Here is exactly what AF said when nominating RNA CEO:
Hans Schikan, Prosensa
Let's compare two chief executives, both running companies developing new drugs to treat Duchenne muscular dystrophy, a rare, muscle-wasting disease. One CEO is gregarious in public, eager to court the support of patients and their families. He's a bit promotional and prone to making big promises. If he angers or offends anyone, it's only because he's working so hard to cross the finish line.
The other CEO is quiet, reserved. He cares just as deeply about patients and their families, but is also worried about making promises he can't deliver. He'd rather work behind the scenes to advance his company's goals instead of negotiating in public. Politeness is a virtue.
Who's been more effective in developing a new DMD drug so far? The first CEO's gabby nature and penchant for promise-making appears to give him the edge. Only later do we find out the CEO's feel-good story is a facade covering up fractious corporate infighting, poor decision-making and alienation of regulatory authorities. Patients and investors are punished.
Meanwhile, the second CEO deals diligently with setbacks, working to extricate his company from a foundering Big Pharma partnership in a position to still achieve its goals. He listens to regulatory authorities and quietly builds a case to support his drug's approval. There's nothing flashy about his approach, but it's effective. When the first CEO blows up and his drug is delayed, the second CEO is still standing. His company will be the first to submit for regulatory approval of a DMD drug.
And in the second CEO's "spare" time, he sells his company for $840 million, rewarding shareholders for their patience and bringing in a more experienced partner to increase the odds his drug is approved.
Congratulations, Prosensa's Hans Schikan. Your nomination for Best Biotech CEO of 2014 is well-deserved.
NOW THAT IS SOME CHARACTER ASSASINATION ...
has anyone read the 12/12/14 AF article on his 2014 Biotech CEO of the year nominations?
I knew he kinda pulled back on srpt, BUT HOLY COW!
he completely skewered Garabedian, and was one step away from calling him a complete fraud!
AF clearly insinuated with thin veil, that the Srpt CEO duped dmd community and investors
"I think you're a jackhole but more important to this discussion, you're a horrible clinician"
I was reading holiday cards in Hallmark and I came across this
Happy Holidays to each and every jackhole on this thread!
you are stating the obvious
the FDA has been less than thrilled with the N=12 no matter what lipstick is used
the conversion to a MITT n=10 just made it worse with the FDA
there is lotsa noise from both sides, but of the few things that seem clear, the n=12 was always a problem no what was said by either side to suggest otherwise
2015 should tell the tale, one way or another
the company's repeated delays and not living to the exact letter of the prior FDA guidance was the perfect set up for the 2014 wash out
frankly, the company'd delays literally washed away much of the confusion caused by the FDA, and once the FDA figured this out, it went on the offensive this fall
as they say, the best defense is a good offense
even in the regulatory process
the company literally handed the FDA the ball when it tried to change all its own timelines but somehow the FDA was supposed to stick to its timelines
the FDA got smart and took the ball and ran with it
now its all on the company, and the FDA is just patiently waiting for the company to actually finally honor its promises to the FDA
by the time we would know if the drug is a bust, the company will have burned another $75 to $100 million
worse yet, I have been in two investments that traded below cash value on failed technology
13 could become 6 on failed 166 and data reread bust
unfortunately, I wish I saw it otherwise
I am not expecting, but I believe the wrong data could result in it
wait, we made it already!
I knew I was right
my allocation such price is as follows
1. maptuit: .0001
2. dr. first .0001
3. escrip: .0000000000000000000001
4. DLPeepee: .00000000001
5. BYOB (or is it D?): .0000000001
email: all the rest
did I miss anything?
It was IMO highly unlikely there would be any news this week, but it was not impossible
but after today, we surely graduate into impossible from now to year end
the stock found its balance in the 13s
if you are a long term investor, 2015 may finally be the year where you will find out if srpt will make it, or not
personally, I think it makes it with a very very bumpy road, where things could get even worse before they get better
168 wk data and dyst reread are so key
if they are at least decent, even the fda will eventually settle on a path
if they aint good, look out way way way way way below
do not forsake me
I could be your only true friend here
I consider you my only true friend, since star threw me under the bus
piney does not count, cuz she is more than a friend
I was friends with johnny87778 until his dog bit me
assuming this is literally accurate, this is spectacular news for the entire dmd community, and also for long-term srpt investors
but for now, I am certain that a large percentage of the srpt stock sellers actually already believe this to be true
but they also believe the company will NOT be issuing any scientific evidence to validate this believe any time soon, especially not be 12/31/14
again, the stock is detached from the company for now
its a year-end book marking vehicle for large institutional investors, long and short, and the clear bent is long exit and short topping off into book marking
I am certain the srpt long institutional base has taken a gigantic whack
the BioMarin/RNA deal has proven to be a quadruple negative in perception, for now
the issues may be bigger and deeper
the company had board members who were willing to make open stock market purchases in the $30s
Now, there has not been a single insider buy, even all the way down to the $12s
What is the difference now vs. then?
IMO, its all about confidence in BOD members regarding the issue of whether the CEO is properly reading the FDA signals
previously, I suspect the BOD members assumed what they heard back from the CEO on the FDA vibe was essentially accurate
Now, I suspect they wonder whether he is capable of accurately reading the FDA signals he is receiving
supporting a CEO as a BOD member should never be confused with spending your own money on the stock
THEY ARE VERY VERY VERY DIFFERENT
regarding BOD wallets, I believe the CEO has lost the confidence of BOD members
this does not mean the FDA process will not ultimately result in some sort of drug approval
but I do think it means that pre FDA approval, the BOD members are no longer blind believers of what the CEO says when they decide to invest their own money in srpt stock
none this matters as to stock trading from now til 12/31
the trading is DETACHED from the company
it will matter max mucho and a ton, when reality returns after 12/31
the stock is "trading vehicle mode" and what the company does is 100% irrelevant to the trading right now
take the stock price and subtract the cash
ask yourself if intelligent investors are trading in the "company"
they are not
the trading is in the stock vehicle, ONLY, from now til 12/31
institutional shorts can prepare to mark their year end books with 100% comfort of zero surprises
you just perfectly defined every biotech start up investment I have ever been in
you should start your own dictionary!