So the boys will be effed regardless if etep isn't approved. I'm not implying that means the FDA will suddenly approve etep, but this doesn't really help boys with DMD. How many families can afford the $100k/year at cost price tag for eteplirsen?
If not, no way eteplirsen is approved.
If yes, eteplirsen odds of approval are exponentially higher. The FDA isn't going to ask to see P3 dystrophin data somehow expecting it will greatly exceed P2. That's common sense. If they were going to deny because 0.9% is too low, they'd have already CRL'ed eteplirsen.
Place your bets. I say odds now favor approval.
That's a fair question, but one could also argue why would the FDA ask for the new data if they are beholden to 10%? They know that isn't going to happen. So does the company and so do the parents. So why prolong the CRL in that case? I mean, I wouldn't put it past the FDA to simply stick to their bureaucracy, but that seems unnecessarily cruel given all of the letdowns up to this point.
I'm not convinced AA is in the cards, but I do believe odds have substantially increased.
Yes, they would have released the PR regardless because THEY WERE LEGALLY OBLIGATED TO. It's material info. Nothing can be gleaned solely based on its release.
I doubt winter is short. I think he just likes to consider all sides of an argument. Which in my opinion is wise. Just saying.
No offense, but if you've been investing in biotech for the past three years, you should know that such a highly volatile stock has good odds of tanking before such a major event. I'm dumbfounded as to why you didn't seek counsel before investing in this stock.
Also, I do not intend to be mean when I say this, but you are an absolute fool if this is your largest holding. How are you going to feel if the FDA shoots down eteplirsen and the stock ends up at $5? Just not worth it man. In the very least, I suggest you hedge with puts.
For the record, I think it's a 50/50 coin toss on the FDA's decision, which is why I haven't played SRPT in quite some time. I've been biotech investing for 7 years, and that experience has taught me to stay away. There are so many better opportunities. (Most of which are not in biotech.)
Unfortunately, a lot of the shorts are probably naked and will never unwind their positions. Then they'll get nothing more than a slap on the wrist for failing to do so.
The company hasn't even submitted the new data yet. The FDA will then likely take at least a few weeks to review it and discuss amongst themselves. Where do you come up with this stuff?
I don't agree that if D was bad, it would be pr'ed right away. I think the company could easily say that the quiet period interaction with the FDA provides them with a shield from pr'ing all info. This is not at all unusual with pharmas that are interacting with the FDA. That's why there are always so many surprises in Adcom briefing docs, for example. Most of them include things that investors were never aware of--in SRPT's case, that dystrophin was only 0.9%. They never pr'ed that, so I disagree that they'd suddenly pr the new numbers. I think no news is simply no news in this instance. I do, however, lean toward eteplirsen being given AA.
Another good point that kills this theory.
One more thing to add: How many biotechs can anyone name that have released news related to additional data for an NDA after a delay has occurred due to the FDA requesting new data? My guess is none, which further cements the likelihood that SRPT has no intention of pr'ing anything about dystrophin, regardless of its numbers.
Anyone with a brain knows that ESSENCE was going to be a placebo-controlled trial no matter what the outcome of eteplirsen's NDA is. The FDA wants a placebo controlled trial, and it behooves SRPT to get one started immediately. I don't think it's some sort of handshake agreement that it start now, and I don't think the FDA's decision is being held until ESSENCE starts. They'll decide independently of that.
I don't think SRPT gets a CRL, but I also don't believe lack of news indicates the odds of that lowering each day. I think the quiet period and the fact that the company is interacting with the FDA is a shield from releasing dystrophin data immediately. Because the data will be made public when the FDA makes its decision. Why else have a quiet period? It makes no sense to impose a quiet period and then as soon as data comes, end the quiet period. That's just normal business that wouldn't require a quiet period. I also can't think of a single example dating back to 2009 where the FDA has requested new data from a company and then that company releases said data publicly. If anything, they simply send out a PR that the new data has been submitted per the FDA's specifications. But they don't include the data.
I also don't think you can make the argument that they'd only release data if it was bad. Materially speaking, reasonable investors realize that good dystrophin data would also impact the share price, so if the data is going to be released, I'm of the opinion it will be released no matter what. But the quiet period tells me that it won't.
I agree that if the dyst data is bad, the FDA would waste no time issuing a CRL as soon as they see it.
The world clearly does not know it. If it did, the FDA wouldn't have slammed the drug, and we wouldn't be in the midst of a delay. Hyperbole such as yours has never helped the stock.
Thig, the CDER made its decision based on what it believed; not based on spite. I get that it's hard to accept that, but it's the truth. Thankfully, Janet Woodcock has the ultimate say, and so long as dystrophin is present, it's a fair bet that she'll approve eteplirsen with AA.