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.
Nobody is arguing that two drugs are "worse" - the argument is that the market has written off Vinta and regardless of whether they generate better data when the Phase II lung cancer trial is fully mature, the ovarian failure will taint those results and the "market" will yawn and say, show me final Phase III data and then I'll believe it. The fact that the tubulysin drugs are moving ahead nicely doesn't change the equation on Vinta.
I thought it was a little fishty that the PR didn't mention the number of units covered by the agreement or why the description of the Serena Group didn't indicate how many locations they have. Their website is equally fishy - they don't have a list of locations, which any "multi-center" organization would. When you click on "Contact Us", there is only one address and the picture of Thomas Serena's trophy Exec Assistant, which notes that she is also a "junior staff accountant" for the firm. The other fishy thing about the PR is how it described the agreement with Serena as a "partnership agreement". My sense is that they are probably giving them some machines (or selling them at a huge discount) in exchange for the "registry study" that Serena will perform at no cost to NVDQ.
Since when does the start of a Phase III trial cause a stock to "go nuts"? You are looking at 2016+ before any pivotal data will be released. The only thing that can drive the share price over the next year is if the tubulysin-based drugs show blockbuster potential in the Phase I/II trials. Regardless of how good the final Vintafolide data is, it will still be tainted by the Phase III failure in ovarian and the "stratification" of the Phase II results in lung cancer. Regardless of how rational their assessment of the Phase II data is for Vintafolide, the market has written it off and won't reward the share price for anything but final Phase III data.
The thorny issue they are dealing with is that the tubulysin drug (EC1456) is much more potent than Vintafolide and uses the same folate linker/target, so even though results from the Vintafolide trial may show some proof-of-concept in targeting folate, spending any money on Vintafolide to get it onto the market a year or two before the tubulysin drug would just be a waste. I think part of the reason Merck gave it back to them was they realized the tubulysin drug would take away any market that Vintafolide was able to realize.
paris - your description of the "platform" would indicate that there is significant flexibility and speed in developing drugs for various "targets", but we've been waiting forever for SRPT to disclose just a single new target that they are pursuing. What do you suppose the hold-up is in the process of getting pre-clinical proof of concept for any new targets?
If you are looking to validate the technology, then why wouldn't you spend the time/money/people on a flu treatment, where a real market exists? You are getting caught up in the fear factor of this disease. The prospects that some $5M charity-sponsored "trial" is going to validate SRPT's platform is a fantasy. As others have pointed out, SRPT's drug may not even work against the current strain, the ability to dose patients in a timely manner (i.e. within a week of exposure) is highly doubtful, etc., etc. - nothing will be validated, no revenue will be earned and resources and attention will be taken from programs that are meaningful. Give it a rest!
mauouo - The ONLY way Ebola is a viable program is if the govt comes back and resurrects a contract with SRPT. There is no other market for an Ebola drug than the govt. Speculatively spending shareholder money on Ebola with no prospects for any meaningful future revenue would be "irrational" and "nuts".
"something real" happened on Ebola? A $5M grant from some charity to explore the possibility of testing several companies compounds is "real"? You're the one who is nuts, simp. $5M for an Ebola study is a joke - maybe if SRPT's drug got "prioritised" (as the article points out, "independent experts" will be recommending which company's drug should be pursued first) and the company actually was contracted to provide the drug by whatever agency is going to handle these studies, then you might have something real. Until then, it is like everything else posted on this board about Ebola - pure speculation about a drug/indication that will never make a meaningful difference to SRPT's long-term value.
Try again bubba - today's presentation is the only listing under the Upcoming Events calendar. DUH!
How stupid of the company to post the "Event" and the streaming link on their website when nobody can get access. It is unusual for these scientific presentations to be available to investors - that is why most companies don't schedule them in the Investor section of their website.
maddog - The STS trial began in Sep 2011 (and finished enrollment in Dec 2013), so the longest a patient could have survived in the trial is roughly 36 months, which is not dramatically outside the "normal" range for an expected median of 22 months? In any case, as I indicated before, I don't think the survivors come into play at all in the interim look - I think the IDMC can only assess the results based on those that have passed. If a larger portion of the patient deaths are in the control group, I'm sure they have the ability to assess enrollment/randomization stats and verify there are no imbalances in how each arm has enrolled. Either way, if TH-302 is benefiting the majority of patients, the assessment of median survival for those that have passed will reveal that. If TH-302 is somehow only benefiting a sub-set of patients, that may only be revealed in the final analysis.
dtic - no doubt, this has taken some patience. My hope was that some of the earlier trials in GBM, MM, etc. might draw some attention to how "broad" the potential for TH-302 is outside pancreatic and STS, but it has been a slog. If this interim look at STS isn't a big win, we are looking at another year of waiting for the payoff.
The flaw in your thinking is that treating sick patients will "stop the outbreak" - the outbreak can only be stopped by carefully managing those exposed as quickly as possible, so it does not spread. Treating them after they have potentially exposed other people to the virus won't change the spread equation.
I get your point, and I'm sure if the IDMC saw that there were no patient deaths in the treatment arm, they would have stopped the trial even before the targeted interim look at 224 "events". Theoretically, if the randomization/enrollment was done evenly from the get go, there should be an imbalance in the # of deaths in the TH-302 arm vs the control arm - e.g. the control arm could have 120 deaths and the treated arm could have 104 deaths - but the median survival computed on those 104 patients should reveal that differential, if they are truly dying slower. The fact that most cancer trials involve marginal improvements (20% or less) in survival dictate that you can't "extrapolate" a median by including survivors at an interim look - whatever imbalance that may be there at the interim may not hold up at the end or there may be just as many "long-tail" survivors in the control arm?
Only those that have passed are included in the computation of the interim median. That is why it is usually very difficult to generate a positive result at an interim look (besides the higher hazard ratio required), as the interim results can be dominated by the sickest patients that pass the quickest. If there is a "long tail" of patients in the TH-302 group (i.e. patients that live well beyond the median), their impact on the median may not be revealed until the final data is out.