ru - you are not "right" about anything. You incorrectly characterize the basis of your whole argument when you say data was thrown out because the twins "progressed quickly". The basis for removing the twins from the 6MWT results is that it was made perfectly clear through the dystrophin measures that it took longer than 12 weeks for meaningful levels of dystrophin to be produced and by that time they had lost the ability to walk, so the clinical effect of dystrophin production on the 6MWT for those two boys was meaningless. If somebody is enrolled in a cancer drug trial and they get hit by a bus on the way home from their first infusion, should their death influence the clinical benefit results of the trial? Again, this is not a large-scale trial, where the noise from individual anomalies will be eliminated by sheer numbers. The fact that you even speak of statistical significance with respect to this trial (a measure targeted at 48 weeks, when the more meaningful data is the 144 week+ data) shows you have no clue.
"unquestioned gospel truth"? I'm fascinated by the ability of some posters to accurately assess the inner workings of the FDA, as if they have keen insight into this govt bureaucracy as if it is some finely tuned machine. Most govt agencies are looked upon as capricious and inept bodies, motivated more by inertia and political forces than any designated goals or the benefit of the taxpayers - why would the FDA be any different. So, the FDA inspects the dystrophin measurement facilities and records in May and it takes them four months to communicate yet-to-be-defined discrepancies in how the measurements were done, but you take it as the "gospel truth" that because SRPT didn't dose the first patient in the confirmatory trial in Sep, the FDA turned on a dime and pushed on the company a half a dozen additional requirements for the AA filing? The gibberish that gets posted on this board about the relationship between the FDA, SRPT and CG are not "speculation" - they are fantasies, and deranged ones at that.
You have to wonder how they come up with $22. Last year, many analysts had higher targets, even if they thought AA was unlikely. A $22 PT has to assume that AA is not granted and it will be 2017+ before Etep is approved.
likeadolt - it is not name calling when someone makes inane statements not consistent with the facts, which you have just done again. "What hurdle did they change in Sept" - look at the press release - the FDA changed half a dozen things they wanted the company to do before or include in the filing, which led to the filing timeline being shifted from Dec to mid-2015. It baffles me when people make statements that are so completely inconsistent with the facts and get upset when people call them on it. Again, get a clue before posting!
likeafox - CG has "failed to meet timelines that were written in black and white" - that has to be one of the most clueless statements I've seen on this board in months. Every time the FDA has established a hurdle and the company has established a timeline to meet that hurdle, the FDA has changed the hurdle at the 11th hour. There has been nothing black and white from the FDA - they have wavered on every issue of what they want to see and what will be required for an AA filing. Change your board name to likeadolt!
You are blatantly wrong again - the 6MWT wasn't the designated surrogate measure in this trial - it was one of many designated clinical endpoints. The surrogate measure is dystrophin and there has to be a reasonable likelihood that the surrogate measure will predict clinical benefit - not statistically significant proof of clinical benefit. AA only requires that there is a reasonable likelihood that the drug is providing clinical benefit. The FDA can't seem to to get a grasp on the dystrophin measurement issues, so they have indicated that they will look at all the clinical data to determine whether AA is feasible. There is plenty of objective clinical data and plenty of subjective assessments by the parents and doctors of these boys to show that Etep is providing a clinical benefit - anybody that denies that and hides behind the issues of a mITT analysis is just clueless.
ru - You're the moron here. You keep interpreting the trial through the lens of a pivotal trial in the non-rare disease space. Obviously, using a mITT analysis determined on a post-hoc basis would be ridiculous in that context, but in an accelrated approval context (where the drug is approved conditionally pending the outcome of a larger trial) in a rare, deadly disease, it is perfectly rational to exclude the twins from the 6MWT. They aren't being excluded from the dystrophin measures, they aren't being excluded from the pulmonary or arm strength tests - all of which are showing that they are "responding" to the treatment. Nodody is calling the twins outliers - it is perfectly "normal" for some boys with DMD to lose ambulation over a short period of time. There is significant variability in the natural history for 6MWT, which why it is such a troublesome clinical endpoint. The significance of the 6MWT results of the 10 remaining boys is that given the historical variability of the disease, you would have expected at least 3 or 4 more of the boys to have lost ambulation during the 3 years - the fact that none have is what is significant.
This really is a bi-polar market - you have so many stocks trading near 52-week lows, while the indices are near all-time highs. I suppose it makes sense that if most of the money going into the market is in the form of index funds/ETFs, the only action will be in the big-cap names that drive the indices?
You are reinforcing my whole point - you can't make precise assessments on what the change in 6MWT means in terms of overall efficacy. I agree that the "market" may react to further 6MWT declines as if they are meaningful, but do you really believe you can be that precise in assessing an average decrease? If one boy becomes non-ambulant, the average is sure to be decline by more than 30 meters, even if two or three remain stable on 6MWT - should the average 6MWT score be the "be-all, end-all" yardstick, even if they all remain stable on pulmonary and arm strength tests?
mauouo - you remind me of the analyst on the call that was pressing the idea that the company would need to "correlate" 6MWT scores to the dystrophin production numbers. As CG has pointed out many times, the twins who lost ambulation also have robust dystrophin measurements from the biopsy of their ARM muscles - that has little to do with how much functional LEG muscle remained at they time they started treatment and how quickly they will lose ambulation.
The enrollment criteria of baseline 6MWT and age is intended to isolate boys that have a certain level of functional muscle that can be salvaged, but that is an inexact methodology. IOW, the boys' post-treatment 6MWT performance is more a measure of how bad their legs really were at baseline than how well Etep is "working". If five years from now all twelve of the boys are in wheelchairs but their pulmonary function is stable and their arm muscles are still functional, did Etep "work"?
Your commentary is reinforcing a false premise/storyline - i.e. if the boys in this trial lose ambulation as they age, they have not been "cured". For DMD, a "cure" means salvaging the remaining viable muscle tissue, not restoring or regrowing muscle tissue that has been destroyed. The issue of how much functionality can be retained by the remaining viable muscle tissue has many factors at play - in the case of the boys in this trial, one of the most significant factors is how much height & weight they put on as they age. For some boys a cure may mean only retaining their pulmonary function, for others it may mean staying out of a wheelchair until their late teens, and hopefully for boys treated from a very early age it will mean a relatively normal life. The bottom line is that for a group of boys that have already lost a substantial portion of their leg muscles and are hitting a period of rapid growth in height and weight, the 6MWT data cannot expected to be stable. Anyone looking at the data and thinking "if the drug worked, their 6MWT scores should be stable or improving" doesn't understand the disease or how Etep works.
Wrong - the market is finally catching up to value of this company - Migalistat alone is worth the current market cap and it will be on the market in 2016, but the value of Migalistat pales in comparison to the value of their next generation ERT's. The share price will be $15+ upon approval of Migalistat and $50+ within 3-4 years.
So the basic message is you had no clue what was going on with the company, so you bash news because it goes against what you thought you knew, and now you are moving on to another "bet" where I'm guessing you are equally clueless? Thanks for sharing!
The "horse" in the case of Ebola has to be a govt contract - pursuing a pivotal trial/approval without a govt contract would be a waste of resources.
"Encouraging" because these patients were progressing on every other available treatment. I doubt partial responses and stable disease is going to create any buzz, but nothing else has accomplished this.
Nothing is going to change the filing time dramatically. Regardless of how positive the upcoming data may be , the FDA is not going to accelerate anything - their MO has been to stall until they figure out what they are doing. Do you think by some miracle they will see the light over the next two months and say don't wait until June, file it in April?
I knew that, but sometimes the market reaction to a giveback dismisses the nuances. I didn't think it was an antitrust issue facing NVS, but rather the simple conflict of having two potentially competing drugs being sold by one company?