I think the best way the stock is to go long with the understanding that if the FDA requires completion of Phase 3, you'll have to take a big hit. I plan on playing some puts as a hedge before briefing docs are released. Keeping my shares though at this point. No competition now and eteplirsen will make it to market at some point.
Hypothetically speaking, if that happens, we wait until after P3 has been completed. Then resubmit and get approved then.
Um, it's the exact opposite of that. I have no idea how any reasonable person could draw that conclusion. Leaving the question off the vote makes it 10x easier for the FDA to do what it wants. Had the question been included, there's a good chance plenty of yes votes would have occurred simply due to the advocacy response and the need for some form of treatment. Leaving the question off keeps the FDA free of succumbing to advocacy pressure. The inclusion or lack of of this question in SRPT docs will be a guide to the way in which the FDA is leaning toward eteplirsen.
The FDA is the FDA. That's why, Grey. Time and again they have shown themselves to be a completely unpredictable wildcard. It is not at all uncommon or unusual for the FDA to give guidance on a subject and then do an about face. Happens all the time with companies that have done SPAs with them. The FDA defines how to design the trial and when the review rolls around, the FDA says the trial was junk. It happened to AMRN recently, and it's happened to a few othres.
Consider that people like Farkas will be reviewing eteplirsen. The guy has never shown even an ounce of love for the drug, and he doesn't seem to be much of an expert on DMD. That is dangerous. I think the odds of approval are better than not, but I still think it will be close. (I'd say 55/45.) Expect anything, even if it defies logic.
Your logic is awful. Given that the topline data from the P3 trial will not even be available by the time of the Adcom, the confirmatory trial will not be used as support for efficacy. The patients will not have been on the drug long enough for the endpoints to be met through a statistical analysis. The P3 can and will be used as support for safety, but barring some last-minute data crunching (which would have needed to begin no later than right now), that's it.
Logic. Objectivity. They are your friends.
[And high enough to make FDA happy and show drug is converting DMD patients into less severe BMD patients.]
Conjecture. I wouldn't be so certain that the FDA will be happy about it. They've shown time and again that they aren't really sure how to deal with dystrophin.
Additionally, based on the FDA slides yesterday, the amount of dystrophin the FDA seems to believe is needed to create a Becker's-like MD is well beyond what eteplirsen is known to produce.
Disagree. We have the upper hand now. There is no reason at all to agree to a royalty. Let 'em try to win in court. BMRN's odds of success in court just went down considerably. If they're willing to take a single digit royalty for EU only and nothing for United States, then maybe. But that's it.
Drisa will never make it to market. BMRN won't be running a new trial after the CRL. Their only hope is royalties at this point.
I would add that although I think the briefing docs will have a lot of harsh language, I think there will also be a lot of positive language which is something the BMRN docs completely lacked. So I won't be surprised if SRPT initially sells off on briefing docs and quickly rebounds thereafter. All depends on the tone and the questions asked of the panel, particularly a question about risk-benefit. Including such a question will be hugely bullish. The risk-benefit is a no brainer.
Why? Because they do so without using any logic? Kind of like Donald Trump at the debates. That is entertaining!
Also, I added shares today for anyone who thinks I'm simply trying to sow doubt. I'm not. I'm trying to sow objectivity.
My bet is that etep gets approved. My point was simply that it's not the slam dunk that everyone thinks it will be.
The FDA is going to include the two boys who became non-ambulant in their analysis, and that will raise doubts about statistical significance. So the discussion of small n will be a big barrier. It is, for the record, one that I think SRPT will overcome.
I don't think the emotional component is going to work here. The panel clearly believes there is no reliable evidence of efficacy. In other cases with emotional components for unmet needs with regards to controversial drugs, there was at least some efficacy.
Of course there will be an SRPT Adcom. The only people who don't believe that are retail investors on this board. The circle jerk is a bit much.
I'm amazed anyone would pay to have you medically examine them. You can't even write a grammatically correct t sentence.