I was mistaken about who is in control of the trial but the point is still valid - the June 2014 update call (just listened to again) indicated that they expected the data to be unblinded before year-end, but they had to wait for Pfizer and "their careful attorneys" to work through the data and how any press release would be worded, which they expected would be a couple of months, so both LPTN and Pfizer have likely been sitting on the data for at least a month.
There is an obvious natural history to this disease and the patient pool is from those that are not initially responding to standard of care (luventis, etc.), so there could be a positive buzz coming from the clinicians about seeing unusually positive results and/or Pfizer could have been sitting on the results for the past couple of weeks - it is not "material" information to their shareholders, so they had no duty to announce anything?
The only thing giving me hope is that there has been a substantial increase in volume tied to the recent share price increase and given that the pending trial results are through a trial under Pfizer's control, so there are likely numerous people within that organization that have a hint of the results, which could easily have been leaked recently. The fact that Pfizer chose to hold on to this program, while they were divesting all their other ophthalmology assets is a sign that they thought the pending trial results would be worth holding on for?
All these price targets have some level of discount applied to them for the risk of regulatory rejection/delay, so upon approval of Migalistat, FOLD is a $20+ stock. From there, their ERT formulations for Fabry and Pompe will double the value over the next 2-3 years. IOW, the ride has just begun - hang on for greater reward.
jk - an analyst price target is typically a 1-year outlook, so he obviously believes that Etep will secure AA - if it does, $45 will be cheap - you are clueless!
ru - way to step up and mis-state the situation again. SRPT never tried to correlate dystrophin levels with clinical results. In fact, when the analysts tried to press them on the idea that they should be able to correlate X% dystrophin positive fibers with Y% change in 6MWT, CG ran through a host of reasons why the correlation would not exist and any attempt to do so would cloud the picture. Pushing the idea of "refined" data on a rare disease with an endpoint as messy as 6MWT is just ridiculous - it is stunted thinking like that that has held up the approval process - maybe you should work for the FDA?
The only rational part of your latest rant is when you admit you have "no clue". When you say the FDA has a problem with dystrophin as a "marker", you have to distinguish between the validity of the marker and the validity of the measurement - it is clear the FDA has issues with how dystrophin is measured (IMO because they don't understand it), which is the focus of this panel, but not dystrophin's validity as a marker. The only hope of DMD drugs being developed for the lesser populated exons is if they resolve this measurement issue.
In the end, there are more measurement issues inherent in the 6MWT than there are with dystrophin measurement and the idea that BMRN has a drug that is by therapeutic design supposed to produce dystrophin and part of their strategy is to discount the importance of measuring dystrophin is clueless.
johnny - does that mean you are on board now, too - you said you weren't impressed with the CC. I'll admit it wasn't anything to get the juices flowing and waiting another quarter to get any real data on how the launch of Omidria will go is frustrating, but there were positive comments/vibes for much of the pipeline. I think this is one you keep for the long haul, rather than bail out on any positive Omidria traction. Something tells me the value of the rest of the pipeline will eclipse Omidria's value by multiples?
likeafox - I'm struggling with the concept of "rescued", but given the nature of the disease, there is no doubt that muscle tissue is destroyed little-by-little and that at some point it becomes irreversibly damaged. It is hard to imagine any of their diseased muscle tissue is truly healthy, so the idea that Etep can improve the health of all but the tissue that is past the point of rescue is a valid one. Your idea that fatty infiltration and scarring being more advanced in the legs also seems rational, given the burden the leg muscles incur with simple walking vs the rest of the body.
As I stated before, I believe the deterioration of the 6MWT scores is not so much of a story of limited effectiveness of Etep, but more the inability of the remaining healthy leg muscle tissue to keep up with the increasing height and weight burdens of these growing boys. I would bet that just like Christine McSherry, all the parents have seen subtle improvements in the muscle function of their boys outside of their ability to walk. The FDA is supposed to take these "patient reported outcomes" into account as part of the approval process for rare, deadly diseases, but so far they have shown no signs of doing anything but getting caught up in the complications presented by the clinical data.
Another fantasy post - if only big pharma was involved, blah, blah, blah. Yeah, it would have made a world of difference for a company that was not familiar with SRPT's PMO technology and/or DMD to be "educating" the FDA grunts, who have proven that they have absolutely no clue about the disease, dystrophin measurement, etc., etc.
I think it is more than "interesting" - Jett is in college, so hasn't used a pencil in 4+ years and after 4 months on Etep he suddenly feels like writing again? I'm sure it will be viewed as just another placebo effect by the twits at the FDA - they probably believe that if he doesn't get out of the chair, the drug isn't working.
You could tell there was a lot of analyst support on the call - many softball questions. No doubt, the launch has dragged out beyond my expectations, but there is something to be said for their strategy. The purpose of the pre-launch was to make sure the reimbursement systems were in place and working seamlessly, so that when new docs start ordering Omidria, there are no annoying complications - when you are dealing with a $500 product that theoretically can be substituted with a cheaper, compounded product, any problems with getting paid may turn off potential new docs indefinitely.
One of the softball questions that was interesting is the fact that the cost of the compounded product comes out of the doctors pocket, while they will get reimbursed in full plus a handling charge for using Omidria, so this is a money maker for the docs - the per procedure numbers are not huge, but if you are doing hundreds of procedures a year, it can add up to some meaningful dollars?
The flip side of his twisted conclusion that the non-responders declined more rapidly than normal is that the responder group reflected a natural slope/path to their disease progression. The clinicians are stating that the stability reflected in the responder group is not normal - it is an encouraging sign that the treatment works. Who should you believe, the clinicians that work with ALS patients every day or a hack "analyst" who tweets as much about his passion for drinking as he does about biotech stocks?
I have some shares I purchased in an IRA about 3 years ago and haven't paid much attention. I know it is a good growth story, but it is trading 100+X next years EPS - does the future growth support that?
If the "responder" group supposedly had greater arm and leg strength going into the study, then why are their baseline ALSFRS scores comparable to the non-responder group?
As Feldman has characterized it, these results are "encouraging", but not definitive, so you see the weak hands and momentum players bailing out, no doubt exacerbated by additional short sellers hoping to weaken the hands of the undecided. Will they be able to identify potential responders, what portion of the market will that be, how long will the cells provide benefit (will periodic injections be required to sustain the benefit), etc., etc. all need to be answered, which will take time and money. Most biotech "investors" shy away from things that take time and money and move on to the next shiny object. The real value of CUR over the next year will most likely be driven by NSI189 - if it really performs as it has shown in the pre-clinical models, it is more of a game-changer than NSI156.
Kynamro, like RNA's Prosensa, is an oglio-backboned, toxic injection that has a "black box" warning for liver toxicities. Why would ISIS waste shareholder money to pursue additional chronic indications like cholesterol - somebody is bound to come up with a safer means?
winter - don't try to salvage yourself here - the whole point of your initial post was that SRPT may have an issue meeting the mid-year filing target because of recruiting issues in the new trials. The fact is that the data REQUIRED to be in the filing from the new trials is not at risk and any additional safety data, while meaningful in the sense that all data is valuable, has absolutely no bearing on whether Etep gets filed on time or approved. Your initial post was intended to raise an issue with the filing that does not exist and is wrong factually, so write it off as another one of your useless posts and move on!
winter - you can't even get the point when it is spelled out for you. More safety data saying there are no safety issues is meaningless - it is a non-factor in the AA process. Do you really think the FDA is going to conclude that they believe in the potential efficacy, but are going to hold up approval until more safety data is available?