now I am thinking the afternoon surge in Srpt had to do with the RNA long-term placebo data
that data is very supportive of the Srpt view of natural history
its all incremental, and IMO, further increases chances for FDA to have enough new data to "save face" even when reversing itself
I think the FDA group who SRPT is dealing with may be concerned about a class action lawsuit by SRPT shareholders who were killed by their ill advised "concerns". I've seen the FDA say the press misconstrued their statements but all the selling shareholders couldn't be misconstruing anything...the information was in a company press release. I was on margin and out for a walk when my broker called one half hour before the market opened to tell me I was sold out and owed them $13,800 more. I would love to join a lawsuit. All the law firms that said they were "investigating" SRPT are presumably too chicken to take on the FDA when it's negligent.
Yesterday Chris said they haven't received minutes from the November and December meetings. I think the FDA is going to their attorneys to figure out how to get around a potential lawsuit. They're not supposed to be gunslingers shooting from the hip using information that is not comparable. Prosensa said in their IPO offering that their chemistry was so different from SRPT and they thought they could sell Drisapersen in the US. If the patents issued by one government agency were based on a new and unexpected chemistry, I don't know why the FDA can be permitted to treat the drugs the same. It's called negligence. Yesterday Chris had to resort to a special slide to show the difference in performance of their chemistries.
The FDA remains a cloud over SRPT. It's arrogant even in the face of new laws and facts. I don't think they are trying to save face. They're trying to save butt.
On the brighter side I bought 265 shares of SRPT late yesterday because I smelled a rose. I didn't buy more because I'm splitting my bets now. Also my broker increased the margin requirement for SRPT from 50 percent to 75 percent. I can make up for a lower performing stock by using it's 30 percent margin requirement to bolster my results compared to SRPT.
Sentiment: Strong Buy
Not sure that FDA would be subject to a lawsuit, but possible congressional hearings. The problem is that some in FDA reason by analogy with regard to drugs targeting same disease when the
PK and MOA are different (witness all the holding back on HepC from one company's problems) If they don't have a good understanding, they lump everybody under one concept, when they are in fact not comparable as to SAEs, pharmacology, etc. But the patients who are the ones harmed by unjustified denial can keep the pressure on the FDA. Patients and efficacy for them are what its really all about, not shareholders.
Good to hear from you again.
I remember that you, like me, bought some shares of SRPT at 50+. Sorry about the brokerage issues.
The good news is we're going to ultimately get more than well. I've sold not one share.
FDA is circling the wagons to figure out how to get out of their mess. Good news will be forthcoming, IMO.
Sentiment: Strong Buy
ariad had iclusig approved simply for the significant patient need even in the face of serious side effects
in this case its not side effects but low statistical power which has now been slanted in their favor by their competitor providing them with all the evidence they need to show their small patient group is very highly significant and merits a second look imo
This is what the FDA is faced with, and here's what C.G. had to say yesterday at JPM14: "Importantly also we have the best control group that we can think of which is the recent GSK study which had the same amenable population that we’re enrolling with eteplirsen in this study followed for 48 weeks had an 83 meter decline if they were over seven years of age. So we have a great example of what would typically happen to an exon 51 amenable population over 48 weeks. Well here we now have over two years where we’re not seeing that decline in any of the patients that we’ve been following here."
This can do nothing but support the case for AA, and I think it's in the works.
Sentiment: Strong Buy
The RNA press release spoke to a treatment benefit in the younger boys (less than 7 years of age) - that goes against any logic - those boys are expected to show stability, so how do you show a treatment effect, unless the placebo boys under 7 years of age also declined over the 48 weeks, which would further taint the FDA's stance on the natural history of Exon 51 boys.
No reason for that. The data at RNA that was good was the greater than 7 yr old, 48 week placebo group (-83meters). This was not good for RNA it was great however for SRPT as it provides the exon 51 natural history that FDA needs for a proper look at what decline would be expected. The rate of functional decline was very near the same rate of decline seen for the small placebo/delayed tx group in the SRPT trial. The natural history data FDA used back in November included Beckers and pts that were not exon 51. This was huge for SRPT today and going foward as AA is reconsidered.
The short thesis of 'natural history' is toast. So is RNA