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?
This is the most volatile/schizophrenic stock I have ever owned - no rhyme or reason to its daily moves, which are typically 4%+ in either direction. I keep telling myself they have a great and unique platform (targeting stem cells) and big money backing them, but these swings can leave you a little disoriented at times.
Not bashing the validity of their treatment for BTD, but has the company disclosed whether they have applied for BTD - it is not something you get granted, unless you formally apply for it.
winter - I am totally disagreeing with your assessment. You state the fact that they are still working toward full-scale production as if that is a delay/change in timeline. Wrong - they never said they would have validated full-scale production by now - they knew it would run into 2015 and that depending on how long the FDA wanted stability testing, full-scale may not be validated until after approval. You speak as if full-scale isn't ready that they can't be approved to market production from the mid-scale facilities - again, you are wrong.
CG has never "blamed" CMO's for delays in the past - manufacturing has never been identified as a source of delay in any of their clinical progress - again, you are wrong in your assessment that it has.
He's not going to invest in Ebola production or shift DMD production to cover potential Ebola work until there is a contract in place, which my guess will not happen any time soon. Barring any kind of outbreak in the US, there is no govt agency that is going to contract w SRPT, unless some of these "trials" that might be run by the charitable organization prove out SRPT's drug as the preferred treatment. Alot of ifs in that equation and it certainly won't happen overnight, if it does at all.
The only thing troubling is how clueless you are. As CG has discussed at length in the past and described again this morning at Leerink, they are using CMO's as the primary source of production for the DMD programs. They have already succeeding in generating "mid-scale" production with these firms and are working towards full-scale production over the next year. There is nothing that has changed with this story over the past two years. The fact that they could meet the initial US demand for Etep with the mid-scale production is positive and mitigates the risk if "full-scale" production is not achieved by any of the CMO's. The in-house manufacturing plant they are developing is designed to give them the flexibility to meet gaps in commercial production, as well as provide drug for clinical trials for the other Exons and programs. There is no issue with manufacturing and the fact that you have read between the lines and conjured one up puts you in the same paranoid delusional category as simp.