yesterday, WHILE SPEAKING TO PARENTS, the Srpt CEO described a P3 trial design that would not include a placebo arm. If you do not understand why he would do this, then you are a galactic imbecile times infinity.
for all others, surely the CEO wants the FDA to take "the blame" for any placebo, and this needs no further explanation.
in the world of reality, all sane people (this excludes certain analysts bashing Srpt today), know that Srpt will make the P3 trial design be EXACTLY what the FDA says it wants to see.
the above is apparently so impossible to comprehend that the stock has sold off 7% for bad P3 trial design (there is NO finalized trial design yet!)
I understand why he said what he said, but in reality, it is a non-issue when you consider the timing. Assuming treatment for the Phase III trial starts in Q1 and that any placebo arm would be at worse the same placebo-delayed design as the first trial, then the latest a placebo-delayed boy would have access to the drug would be the end of Q3. Approval for Etep is likely to be around the same time or later, so I can't imagine that a parent would put their son in the trial, then after finding they were on placebo, pull them out with the risk of Etep not getting approved and losing access to the drug through the trial? That being said, I think forcing a placebo arm into this trial would be highly unethical, as it is clear that once these boys lose ambulation, they are not going to get it back.
"That being said" it goes without saying. Hi Tred! What do you think of the Prosensa 168 plan Chris laid out? Did it sound familiar to you in any way, big guy? See you on Monday, Mr. Know-it-all. No computer on the week-end for me. But I'll expect to see your response first thing Monday morning when I check the posts I missed. Courage!
Chris finds a way to keep everybody happy. PPMD. Us. The shorts. A genius.
He knew that placebo ploy would be smiled upon by the shorts - as well as the institutional investors looking for their chance to jump in. Foolls rush out and Angels jump right on in. Not the first time he's made a tricky little move like this, is it?