"For the first time, with this article, I feel all the pressure being put on politically may do more good than harm with the FDA--wasn't confident of that before. Well done Mr. Astrue!"
Agreed! So many parties are now watching this story unfold, including people with significant
competence in the subject matter. More near-term coverage with increasingly more visible print or TV media is virtually guaranteed raising pressure even more. Etep is moving away from a scientific/medical story to a political one. Assuming this is all because of FDA inflexibility what is their reward? To maintain that expression of modified dystrophin has poor correlation with clinical benefit and is currently not likely to predict clinical outcome? Protecting patients? Really?? Removing this concern will require an unacceptable and totally unwarranted multi-year effort while you have 12 kids that have stable disease after almost 2.5 years on treatment. Or is it s.th. else that is holding things up and we are just not being told about it?
If you're correct with your probability reasoning a better strategy IMO would have been selling a smaller number of contracts for a higher premium. Under your scenario there is just about as little chance for the stock to hit $26 but you have limited your exposure to 20-25% of your trade.
Must have been a gut wrenching decision 4U. Agree that failure to provide timely and meaningful updates drives retail investors away from the company. Tutes possibly as well, or stock would not be down more than 25% in just 5 weeks. I'm sure their angry phone calls are responded to while the likes of you and I are left wondering what's been happening in the last 6 months. Embargoing investors does not do them any good and stock suffers from uncertainty created by news vacuum. Way better to inform those who own the company about the status and if there is a sore spot please tell us what you are doing to fix it.
Equally important question: Will there be an ethics board anywhere that will approve a protocol that includes children who, based on all available information so far, have a high chance of obtaining significant benefit from the treatment but are condemned to be randomized and may end up not getting the drug? Think about it. FDA wants placebo group but Sarepta is unable to find trial sites that will agree with the protocol. What now??
First, it is not yet approved. Second, the advisory panel consists of outside scientists. Third, if FDA denies approval despite the highly favorable advisory votes I would share your concern.
It's not so much cynicism but conspiracy theories and suggestions of criminal behavior. While it is tempting for outsiders to embrace that kind of speculations I cannot fathom there is any truth to them. Mendell has been quoted saying FDA wants data that are not yet available. Clearly, with all the public and political pressure FDA must have a very solid and scientifically defensible reason for stepping on the brakes. It would seem that concerns have been shared with Sarepta but company hasn't shared them with investors.
Your optimism is fine. I agree with you on points 1 and 2. But let's be real. It is naive to suggest somebody at FDA got fired because of Eteplirsen. Also, to suggest that declining stock price is bullish is total bull. Perhaps you want to revisit a 6 month chart and take a good look at Oct/Nov.
Your opinion sucks. The point here is the unacceptable delay in regulatory guidance. With just 12 kids in Ph 2 trial the amount of data to review is quite limited and should have allowed FDA to tell Sarepta in a much more timely manner what is needed to move the program along. Sarepta has plenty to worry about because the delays are costing real money, plenty of it. Much worse, those waiting for a safe and what looks to be an amazingly effective therapy are kicked to the curb.
No doubt GILD has used sound economic reasoning when they priced their product. In that context I can find no fault with the price. However, they were oblivious about the negative sentiment that could hit the company and the stock. You don't win in that department when the headlines just keep on coming about the $1000 pill while the CEO just became a billionaire. IMO, they have flexed the rubber band too far on Sovaldi pricing and that is sinking the stock. Over $10 billion off so far. Did nobody of the high paid guys at Gilead think about that possibility at all when they set pricing? Moderation would have far better, with potential to raise price in subsequent years. Instead they may be forced to lower price in future years. Wanna guess what that announcement will do to the stock?
Many years of additional rigorous testing are needed, with at least one third of trial participants expected to let their natural disease progression play out, because stuff like sure happens in 12 year old DMD boys all the time. Or perhaps this is all fabricated.
"Upcoming placebo controlled phase III studies will be able to show if Eteplirsen can significantly delay the long-term disease progression."
Sure, if the trial is designed to last for 10 or 15 years and denies a great number of kids the benefits that have been plain to see for everybody already. What a ridiculous, heartless statement. Like who cares about some human guinea pigs, meticulous science just requires some sacrifice, get used to it you whiny families. Seriously, is anybody up for this moronic suggestion? Why would they ignore results from 12 kids that have been on this stuff for almost 2.5 years already that, based on plenty of available data covering the progression of the disease in the relevant age range, should have declined significantly in walking and breathing ability? It takes a ton of ignorance, and a total lack of sensitivity, to write stuff like that. Shame on you authors.
I am concerned that FDA is increasingly defensive and rigid in light of the unrelenting efforts by the DMD community to influence their stance. After DMD research leaders and Congress the lobbying effort has now escalated to include the POTUS. I completely understand and support these actions (I have signed the petition) because the FDA has recklessly turned a blind eye on a product that, by all measures available to date, whether objective and controlled science, or individual outcomes that FDA itself had said will be part of regulatory path forward, has demonstrated promise like nothing else. Promise is actually an understatement but let's leave it with that ahead of an "official" conclusion about eteplirsen's performance.
Affected families cannot be expected to keep mum. They are in a fight they cannot afford to lose. I only hope the FDA is understanding of the ongoing activism and will not be defiant and stick to their guns so as to show everybody that David has no business challenging Goliath.
If this even had a remote chance of being a placebo effect I suggest we start infusing all DMD kids immediately with weekly isotonic salt solution....