It is strange - you would think the direct family members alone of DMD boys would be enough to get 100,000 signatures. Obviously, Exon 51 boys are not that numerous, but you would think families with a boy with any Exon would be paying attention and pushing to get the FDA to move on this. Are people just too lazy to spend the few minutes it takes to get through the petition process or are they paranoid about giving their personal and e-mail info to a govt-sponsored website?
I think the FDA is taking their time because they know that regardless of how quickly they make up their minds, SRPT won't be in a position to meet the manufacturing needs until the end of the year. You would have to believe if they are digging into the data as deeply as people assume, when the finally give the nod to file for AA, there won't be much more to look at, so the decision (which even under a priority review is 6months, instead of the regular review of 10 months) won't take more than a few months. That has been my argument all along - why not tell SRPT to submit for AA - by the time the FDA has to make a decision, they will have another 6 months of 6MWT data - if it is stable, it is a no-brainer, if there are signs of deterioration, then they have an out to ask for a Phase III first - what's wrong with that game plan?
thig - I had not heard of this before - where did you hear that the parents sent videos to the FDA and when did this supposedly take place?
The other interesting aspect of the interview was how leading up to the start of the trial, Max used to have to use the lift to get on the school bus, because he was starting to stumble on the stairs - now he gets on the bus with a loaded backpack on his own. She referred to a number of "improvements" in Max's health or strength that don't necessarily translate into an improvement in 6MWT - remember, it is a walk test, it is not a how fast/far can you run test, so if the boys could walk at a normal pace before, walking at a normal pace is as good as it is going to get in a 6MWT. That is what is so frustrating here - I'm sure every parent has a story of how their boy's life has IMPROVED on Etep and these "patient reported outcomes" were supposed to be an important part of the review process in a rare, fatal disease setting. The FDA can't even remove their heads from their @sses for a few minutes and look at the big picture here - 10 boys all stable/improving over 2 years!
There is a general ignorance out there with a lot of the short comments I have seen - they believe if Etep really worked, the boys' 6MWT performance would be improving, not just stable, or that it would have prevented the twins from losing ambulation. It is essentially the belief that if the drug was working, it would restore muscle function, as opposed to preventing the muscle from deteriorating. This distinction is also important to the urgency behind AA - any delay will simply be more boys losing function that cannot be recovered - the moral and intelligent response to that threat is to grant AA as quickly as possible.
overall results in what "space" - the DMD space or the RNAi space? IMO, it is absolutely #$%$ to think that it will take 3-4 years for Etep to hit the market if you assume the 10 boys in the Phase II trial will continue to be stable over the next year - every 6 months of stability in those boys will bring further wrath down on the FDA.
Is the FDA inept and in some cases corrupt, no doubt. But in Etep's case, it boils down to inept. There are no competing drugs to push (if Drisa wasn't dead before GSK bailed, it certainly is now), so even if the FDA was under the influence of GSK, that has evaporated and there certainly are no DMD doctors on the take. This is a simple case of ineptness - as Tracy Seckler's story in HuffPo pointed out, the experts that they brought to meet with the FDA were shocked at how little they knew about the disease. It is easy to imagine that they were taking their cues and education from GSK on Drisa and when that failed, they (without researching it) assumed that Etep was the same drug, generating the same results and it would also fail and looked for reasons on the surface to doubt SRPT's results. Hopefully, they have listened to the experts, looked at the right data and have the intellectual integrity to reverse course.
You are wrong due to the simple fact that they can't "save face" by continuing to deny the evidence. Where is the evidence to support their position - there is none. All the 6MWT natural history data for 7+ year old boys show substantial declines, while 100% of the Etep boys are stable; all the surrogate measures show exon skipping and dystrophin production in 100% of the Etep boys; all the comparative analysis shows that Drisa is nowhere near equivalent to Etep in efficacy and dosing levels; etc., etc. How do they save face by denying AA and then watch the 10 boys continue to be stable every 6 months going forward? The longer they deny the evidence, the less face they have.
They did an offering in Dec of 4M+ shares and when you account for dilution from options, etc. they are probably closer to 25M shares outstanding, so roughly $650M at the current share price.
Depressing presentation - whipped through it in a sad monotone in about 20 minutes as if nobody was there and since there were no questions (with plenty of time left), that is probably the case. The only new twist I noticed was that they had some trials testing for certain tumor markers that will narrow the targeted patient groups.
The issue these "idiots" face is that they have a spotlight on them on how they handle the AA prospects for Etep. Anyone paying attention knows that SRPT management has quietly dismantled the issues the FDA raised in their Nov letter - the key ones being any meaningful comparison between Drisa and Etep in terms of their ability to generate dystrophin (there is none - the drugs are not comparable at all) and the "variability" of the natural history data raising doubts about the stability of Etep's patients - it is clear from the natural history data that without Etep, most of these boys would have had experienced substantial declines in ambulation, if not lost ambulation completely by 120 weeks. It is not CG's job to shove this down the FDA's throat or rant and rave about how clueless they have been in interpreting the data, but if the FDA's dismisses this data and has no conflicting data of their own to back them up, there will be plenty of howling from the parent groups and, hopefully, legislators. Idiots don't like being called out, so expect a complete reversal of their current AA stance.
endo - That is the one thing that has confused me about drugs vs "devices" - obviously, NVDQ's technology has been approved and available to docs for some time in many indications. I understand why hospitals would wait to see certain studies before making a commitment to purchase the devices, but who is in control when it comes to making it a "standard of care"? I assume the docs have the right to use it whether the hospital has it or not, so the docs have to be the driving force of adoption. Are Phase III-type trials what drives docs to make the commitment - I wouldn't think that would be necessary if the leading centers/docs are adopting it and there are solid mid-level trials?
Exactly - the evidence keeps piling up - it is somewhat surprising that this technology has not become the "standard of care" in certain indications by now. Ultimately, it will and this will be a $100 stock, but it gets frustrating sometimes how slow the adoption rate is?
Is that by the time the Phase III confirmatory trial is done (assuming it is a 48-wek read on dystrophin and 6MWT, so not until YE2015), the boys in the Phase II trial will be over 13 years old, on average. It is already clear to anyone with a brain that 120 week stability for boys over 11 years old is clinically meaningful - assuming they show the same stability over the next 18 months, it will be crystal clear that Etep works, even before the Phase III results are revealed. So, if they don't allow SRPT to file for AA this year, the wrath of the parents and DMD community will just grow stronger as the Phase III trial progresses. It is not just the morally proper move to grant AA, it is politically the safest thing for the FDA to do, as well, which is why odds are they will grant AA.
I am with Fidelity and my margin rate on TKMR is 65% - they have that scheduled out as 35% base +10% industry +20% risk premium - not sure if the latter is company specific or related to my portfolio holdings?
CG has never "commented" or speculated on what the FDA will do - he has only reported what information they have given to them and when and whatever formal responses they have received in return - that is the way it should be. If you ask him the question whether Etep should qualify for AA he will give you a resounding yes with all the reasons why it should - he truly believes the drug is working and has met the standard of providing surrogate & clinical evidence that there is a "reasonable likelihood" that Etep will provide a clinical benefit. If you ask him what will the FDA do, what he thinks they will do, or odds that they will allow an AA, he can't answer that, nor should he.
Exactly what they should have done before releasing the minutes back in Nov, before they had all the data and before they had done the appropriate analysis. As CG stated in today's call, they have no obligation to release minutes or provide guidance - the fact that they did that in Nov without having the data analyzed properly shows their bureaucratic incompetence. Now that they have the data, are they competent and honest enough to do the right thing?
A worthy concept, but nothing that will change the fortunes of SRPT. Patients might have a "right" to get the drug, but without insurance companies paying for it, very few patients will be able to afford Etep. Without a defined payment system, they would be forced to manufacture on demand/payment, which would make the manufacturing costs that much greater. In the end, it would probably be a distraction that would cost the company money - how would they price the drug in the face of dying children - even trying to recoup their costs, they would end up looking like an evil corporation?