winter - don't kid yourself. The list of posters you cite have never dismissed the risk the FDA has posed in this saga - we all have been living with their capricious changes in this story for the past two years. We have argued that the clinical results to-date are sufficient for most objective, sensible people to conclude that there is more than a "reasonable likelihood" that Etep is providing a clinical benefit and if you accept the mandate that the FDA has been given in providing accelerated approval for drugs that treat deadly, rare diseases. then it SHOULD be a slam dunk that Etep gets accelerated approval. Despite the FDA's contentions that they are meeting that mandate and being "flexible" with SRPT in assessing their potential AA, the facts do not support that. I am not cynical enough to believe that SRPT is getting the runaround due to political issues - it seems clear to me that they are just inept and behind the curve in understanding this disease and how Etep works.
Your analysis has always been off base - today is no exception - you cite "the additional and unexpected requirements and studies for the NDA filing" - please point those out. Other than bringing the MRI data into the picture, every other data point/study/analysis was something the company was already planning to do and submit during the review process. And this call for the CEO's resignation is equally off-base - the company has provided detailed quotes and analysis from every encounter they have had with FDA - it is clear that the FDA has blindsided the company twice with their changes to what they want to see and when. The company has data on 12 boys and has provided every nugget of that data to the FDA on a timely basis and answered all their inept questions and analysis - what more exactly could the CEO have done to change how the FDA has handled this?
system - when did you speak to him - you do understand that they would not have gotten this letter until after the market closed last Friday. Up until then the company had no clue the FDA was changing the gameplan again.
But then you have to remember -
(1) RNA doesn't need to submit additional safety data - they have plenty of data showing the toxicity of their drug and the dose-limiting issues.
(2) RNA doesn't need to submit additional efficacy data - their data clearly showed there was no benefit vs placebo in the overall population. The fact that they are trying to manufacture some sort of efficacy signal in boys under the age of 7 is laughable, given the natural history data.
(3) RNA doesn't need an independent review of its dystrophin data - the initial results were spotty and inconclusive, so you don't need an objective/independent source to come to the same conclusion.
All the additional data the FDA is now requiring to be formally in the NDA was already in the works (other than the MRI data) and was going to be submitted to the FDA during the review period, so the net effect of this action by the FDA is just another 6 months for them to make a decision. If I was a parent of a DMD boy, I would be #$%$ - this is not the "flexibility" that they supposedly have been working under. Think about it - the 168-week data, the 4th biopsy data, the independent review of earlier biopsy data is still for just 12 boys - why should it take 6 months for them to review 10 or so data sets on 12 boys?
No doubt - with primates dosed at same level having no issues and clinical dosage a fraction of the rat/primate dosage, this issue will reverse itself soon enough. I've seen stocks take lesser hits on suspensions from clinical safety issues - there are none here.
What are we missing here - if you go to Genervon's website, there is no new press release - the data that is in the SA article seems to be coming from a press release on 6/30/14?
endo - I am baffled by your perception that there have been 17 months of positive accomplishments and everything else is right on course. SPY system sales through LifeCell are stalling and with it the expected hypergrowth in breast reconstruction. Firefly is holding its own, but ISRG is having its own growth issues. Seventeen months ago we weren't counting on Luna and PinPoint sales to bail us out of missing what everyone assumed was a conservative revenue growth target. My guess is that Luna and Pinpoint are doing just fine, but the market is discounting any other growth as marginal. I'll say it again - Arun is dropping the ball on exploiting the full potential of the Spy technology - he needs to sell to a company that has the marketing resources to make things happen.
I don't think the recent insider transactions are significant - mostly small share amounts from mid-level managers as part of 105b1 plans (pre-specified, scheduled sales), but if you look at the detail of the transactions in the SEC filings, the options that were exercised were nowhere near expiring - 2020-2023. Also, moving shares into a trust would not cause a change in ownership that would be reflected as a disposition to the SEC - if the owner is the beneficiary of the trust, there is no effective change in ownership.
Doesn't matter what the retail players on this board believe - the big money moving this stock over the past few months is skeptical (and IMO wrong) about what the data is revealing about Etep. Unfortunately, we will probably have to wait until the expert panel weighs in before the "market" takes any notice.
Doubtful - it has become evident that the market just doesn't understand this disease or how Etep works. Between the shorts who believe that if Etep worked, the twins wouldn't have lost ambulation, to the analysts grasping for "correlations" between the dystrophin and 6MWT data and a rationalization of the small declines in 6MWT seen at 144 weeks. Frankly, I don't think CG has done a very good job explaining the results - his explanation at the Leerink presentation focused on the Beckers analogy - he started to go down the right road, but then got lost explaining how Beckers boys experience a slower rate of decline. He needs to frame the analysis/conversation around a few key points -
- DMD is a disease that begins at birth - these boys don't produce dystrophin and their muscles begin deteriorating from day 1. However, the loss of functional muscle is so slow that the boys appear normal until 2-3 years old and will typically continue to show "clinical" improvement on a 6MWT until they are 7+.
- Once the muscle fiber has been lost (scarred, fibrotic), it cannot be recovered - Etep will not restore function in wasted muscle tissue - Etep can only help maintain function or slow the progression in the remaining functional tissue.
- As these boys age, there is a constant battle between the demands their growing bodies place on their muscles and the continued loss of functional muscle mass. Clinically, that battle is lost when their leg muscles are no longer strong enough to support their weight.
- The boys enrolled in the SRPT trial had already lost a substantial level of functional muscle mass - even if Etep is successful in maintaining all of their remaining functional tissue, their ability to maintain ambulation as they grow to adulthood is questionable. The fact that they have all stayed ambulant three years into the trial is borderline miraculous.
Get a clue - the troops are being sent to build infrastructure that will hopefully allow the Liberians to control the disease better - they will not have anything close to direct contact with any patients.
There hasn't been a presentation on Etep at WMS, yet. This is a combination of Ebola fallback (see TKMR) and a setback for another RNAi firm (see ARWR) - SRPT getting dragged down along with them.
No - I didn't see any links on the WMS website for any webcasts. I'm sure if it was available, the company would have posted something by now. The best we can hope for is a press release detailing any new data they present shortly after any official presentation?
They are hosting a 2-hour symposium on the 8th between 6-8PM Berlin time - not sure if that is where they disclose any new data. Other than that, I couldn't find any detail on abstract/poster presentations or lectures?
Wrong - the contract with the DoD has been terminated and SRPT owns the Ebola stock they have on hand. The FDA has given clearance for various drugs, including Sareptas, to be used/tested in the current outbreak, but it is up to the doctors/researchers to make a request for the drug. Regardless of what happens with SRPT's current inventory of Ebola drug, any "stockpile" order would likely involve significant testing to match/test against existing Ebola strains and a manufacturing ramp-up that would take a year+.
I missed that - AVI-7537 is their currently designated Ebola drug and was a component in AVI 6002 (a combination of AVI-7537 and AVI-7539) , which did make it through a single-ascending dose study. The stock they have on hand was probably manufactured to take AVI-7537 through a similar study, before it got axed in Oct 2012. I would have to believe the company has enough safety data on all their drugs to give the doctors comfort (at least put it ahead of TKMR's drug from a safety standpoint), but they probably don't get comfort from the fact that it is not part of any active clinical program - IMO that is still a better position than TKMR having their drug under clinical hold before this outbreak escalated?
The "plan" can't come from the company - the doctors/patients have to request the drug. Despite all the rational evidence that SRPT's Ebola drug (I don't think it was ever dosed in humans) should be as safe as their Marburg drug, it is still a leap of faith for any doctor to use it, especially since it has been on a shelf for a few years and may not be effective against the current strain?
likeafox - You're missing the boat here - I'm quite sure the company is ready and willing to provide the dozen or so doses they have on hand at little to no cost, but in order to build a stockpile of "thousands" of doses, the company would require a contract in the $500M to $1B range to make it feasible. Ultimately, that is the value equation for any company providing an Ebola drug - it is not the ad hoc emergency treatments coming out of this current outbreak, it is the contract to provide a stockpile that is available for the next outbreak. As CG stated in previous discussions, to the extent they "give away" their current drug supply, they at least want it utilized in a fashion that may actually validate its effectiveness - i.e. have it dosed to patients that aren't too far gone and in a medical setting that will give them a chance to survive.
I just have to say how nauseating this country has become by blaming every unpopular govt action on the POTUS - as if Obama had direct input on deciding which DoD programs were funded/dropped - give me a break. I don't care who is in office - 90% of the actions by the govt are taken by unelected civil servants who have no stake in the POTUS. If there is any corruption at hand, it is probably from the revolving door of civil servants that move from govt to industry/lobbying or possibly congressmen who work the levers to repay some big donor. I don't care for Obama, but thinking he has even an indirect link in what goes on at all these govt agencies is just ridiculous.
simp - are you really that clueless? "Recruiting" patients is not the same as enrolling them in the trial - there is patient communications, pre-screening, etc. that the clinics can do before formal enrollment (signing paperwork, formal screening, baseline testing, etc.) takes place. Like most issues you dig into, you are imposing your paranoia and ignorance on the situation and coming up with a non-existent scandal. As CG stated in the Leerink presentation, the prediction/projection in April was that they could start the confirmatory trial in Sep - whether you deem that prediction to have been enrolling patients or dosing patients, they are 1-2 months behind that projection. Is it possible the delay was caused by the FDA dragging their feet on confirming the structure of the trial, instead of the incompetence of SRPT - you have no evidence to conclude one way or the other, just your paranoia. Either way, is two months really worth one of your tiresome rants?