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.
Conclusion: You're an Idiot - the idea that Etep (or any effective DMD) drug would be restricted to a certain age group is just plain stupid.
grey - The 6MWT water is so muddy right now that you can't rationalize it anymore. Of course the results are "remarkable". I think he also said that there are 3 patients that have improved over the last two years - based on the stories coming from Jen McNary, Max is probably one of them - a boy on the verge of having to use a scooter to get around and 3 years later he is walking farther than ever. This is never seen in the natural history, but once the FDA erroneously called out the natural history data as "variable" and possibly consistent with the results being seen by the Etep patients, the whole context of how to interpret the results was distorted. CG can explain it a hundred times that these boys were enrolled because they were on the verge of beginning to lose ambulation, but it is lost in the haze of the clueless who believe if the drug worked, the twins would be walking. The sad fact is that until you get to the AdCom meeting and have real experts pounding home the point that these results are borderline miraculous, nobody will listen.
McNary Jen found a twitter post she made in Aug 2010 (a year before Max started in the trial) lamenting the fact that she had to pull out one of Austin's old mobility scooters, because Max was having trouble keeping up with the other kids. Four years later and after 3 years on Etep she reports that Max hasn't used a "mobility aid" in two years. I'm sure it's just a placebo effect!
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.
Was posted on Jenn's FB showing Max walking off to catch the bus for his first day of 6th grade - looks pretty normal, even burdened by a full backpack. This is not a kid who is struggling to stay ambulant, even though before starting the trial 3 years ago his mom was pulling his brother's scooter out for him to use. Sure, Etep doesn't work!
My take on the FDA Guidance issue is that the PPMD made it crystal clear in their proposed guidelines that the risk profile of DMD is such that there is no risk that is not worth taking, in light of the 100% certainty of progression (with no potential reversal - once muscle is destroyed, there is no restoring it) toward a gruesome life and certain early death if this disease is not stopped in its tracks at an early age. It seems the FDA is finally getting the message in their expression to be more "flexible". Combine that with the fact that FDASIA does not require certain documented clinical benefit, but only the reasonable likelihood that the treatment provide benefit, and we are starting to tilt toward AA being a slam dunk. A successful 4th biopsy (presumably with newly approved measurement protocols) would certainly put AA over the top, but even if that does not happen, it is hard to imagine any "expert" looking at the lack of progression/improvment in Max, Billy, etc. and concluding that there isn't a reasonable likelihood that Etep is providing them clinical benefit. The idea that 100% of the treated boys have to show the same level of benefit (per the 6MWT) for AA to be likely is simply ridiculous - the experts will understand that issue and drive it home, if need be.
CG "may be too confrontational" in the FDA meetings? How else are you supposed to respond when the FDA, after months of review, comes back with glaring errors in assessing the natural history of the disease and the comparability of Etep to Drisa and throwing the prospects of AA into the trash bin. The fact that he has not publicly called out the FDA for incompetence is a sign of restraint and political maturity.
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?
They have back to back robo-reports, the first saying the stock is a strong sell, because of earnings estimate changes (for a development stage biotech w no product revenues) and the next one hi-liting that the stock just jumped 10% in one day. I suppose they make money off of these worthless "reports" because there are ads tied to them, but they can't be attracting any clients with this kind of nonsense analysis.
No, they won't reveal it if it is not achieved, because the company won't know - unless it is achieved, the company is blinded from the data. The CEO keeps downplaying the odds of getting the 45%, while trumpeting the Phase II data that achieved a 68% increase vs historical (21.5 vs 12.8), so it is not out of the realm of possibility. No doubt, Phase II benefits typically narrow in the stricter constructs of a controlled Phase III trial, but you would think that if the control group survival is 12 months, then 17.5 months seems plausible?
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.
What's "more better" than a moronic short who not only doesn't understand how the IDMC review works (the company never has access to the data), but uses infantile phrases like "more better"?
Between the company's enthusiasm for this drug and its prospects and the market's response to its progress/prospects. It seems every trial provides nuggets of hope for future trials to potentially validate, but the completed trials have issues which leave the results inconclusive - with H&N it was the early termination of patient dosing due to fever, with pancreatic it was wild-type KRAS patients distorting the results - and since the remaining trials are not under the company's control, you can probably guess that there will be issues with those, as well. In the long run, Reo may find a niche in many different cancer treatments, but that is years away from validation, assuming they can find the money (or buyer) to carry on.
On a side note, all the remaining active trials are investigator-sponsored and considering that every question raised by the analysts was met with "we're not sure, because it is not our trial", what exactly do these guys do all day?
Whether DOD changes their mind or not is meaningless - by the time they shift gears on a DoD contract, you'll be two years down the road and this Ebola scare will be a distant memory. Show me the slide that says who is going to pay for the drug and at what price!
ml - again, he was hired to expand the platform, not work on Etep. The rumor on twitter is that he made a power play for CG's job and lost - that has more credibility than him sabotaging or dragging down the Etep clinical path. There was never any indication that he was directly involved in Etep at all, so the speculation that it has anything to do with Etep is off base, IMO.
dawg - exactly, that is one of the things they spoke about in the July 2013 presentation - when asked by an analyst about the enrollment rate (did they add sites, competing trials, etc.), the CEO mentioned that the their VP of Clinical Operations (who was on the call) was grinning ear-to-ear about how strong the enrollment was. The issue of docs seeing meaningful benefit is even more relevant in this trial, because after the patients receive the max 6 cycles of dox (over the first four months), then the TH-302 arm has the option of staying on maintenance therapy with TH-302 alone - in the Phase II trial, this extension period generated additional responses with much less toxicity than the combo therapy. So, no doubt docs are getting a clear picture of the benefits of TH-302 and that influenced the enrollment rate toward the back half of the trial.
"weren't that conclusive" - positive results for both endpoints required a 50% overlap of the confidence intervals between the two groups - both endpoints achieved 100% overlap of the confidence intervals. 46 of the 48 patients that completed the trial elected to stay on the drug during the voluntary extension phase. What exactly is inconclusive, or is it that you don't understand the meaning of the word? Idiot!
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.