The clinical "significance" achieved was a comparison between the treated group and the placebo-delayed group at wk 48. Since the placebo-delayed group has now been on drug for 120+wks vs 144+wks for the treated group, the relevance of that comparison has lost its meaning - both groups are fully "on treatment" at this stage. Whether the differential in 6MWT changes remains statistically significant at this stage of the extension trial is meaningless - it s more meaningful for both groups to remain as stable as possible.
Beyond the error rate is the risk of an error - the overdosing risk is minimal w Zalviso, while the risk of an error (mechanical, nurse programming, dosage, etc.) w morphine is much greater. A few lost pills is not going to put patients' lives at risk.
simp - the premise of your paranoia is that there is some confidential secrets that would be detrimental to the share price. Saying he had "serious disagreements" with the guy that just fired him is hardly a revelation. If some short comes forward with some "confidential" dirt over the next few weeks, do you really think it won't be hard to attribute it to Krieg. Either way, your belief that there is some dark secrets at the company that the shorts will be trying to squeeze out of Krieg is just another one of your self-induced fantasies.
What "public quotes" are you referring to? Did these quotes include confidential information? You're on the wrong side of the coin on this one simp - if you really think AK is going to give or sell confidential information to short sellers, you're more paranoid than I thought.
Are you implying that the reason the walk test scores are fading is because the "down time" created by the dosing schedule leaves dystrophin production gaps, where muscle is continuing to be destroyed? My knee-jerk reaction was that it didn't make sense, given how long it took for dystrophin to build up (24+weeks). Who knows - unless they build an oral or IM formulation, we may never know. They must have some idea from the animal studies how durable dystrophin is in the body (once produced) - i.e. if Etep allows dystrophin to be produced for 24 hours, but the produced protein only survives for 24 hours and you have 5+days of no dystrophin in the body, then that would be a problem, but I don't think it plays out that way?
Whether they have started recruiting/screening kids, yet is highly contingent on whether the FDA has figured out their position on dystrophin measurement - until they sign off on methodology (that will have to be used for baseline measures, as well as post-treatment), they cannot start the process. Not sure how many sites they were targeting, but regardless, with parents falling over themselves to get their boys in this trial, it is not a question of "recruiting", but a question of screening to get the right profile for the trial. Either way, SRPT has established enough connections in the DMD, community to get this trial enrolled quickly, so if there is any delay here in getting the Phase III trial started, it is most likely due to the FDA, once again.
if they get a complete response letter due to some minor issue - IOW, the FDA signs off on efficacy and safety, but wants some additional information on the dispenser, manufacturing, labeling, etc.??
Part of the reason that nobody cares is that the company does not have the capital to take Asonep into a "pivotal" trial, so the dilution overhang will limit any interest, even if investors found the early results "compelling". If they partnered it with some upfront money, that would change the equation, but for the forseeable future, the Asonep story will not change the equation. Isonep is a different story, since the pending Phase II results involve a potential buy-in by Pfizer (or whoever buys their interest) and provide the company the capital to avoid dilution at the same time Isonep is validated as a blockbuster. Unfortunately, it seems as if we will have to wait until Q1 of 2015 for that story to play out.
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.
The company didn't "point" to anything and he was hired specifically to expand the platform, not work on Etep. What a moronic comment.
It would be a bigger deal if he left - that would mean he got a deep look into the technology and lost faith. You don't get fired these days unless you do something borderline criminal or unethical and it usually has little to do with the company's business.
grey - I think the point of the statement is that they were not going to start the trial until they get the FDA to sign off on the endpoints. Given the FDA's flakiness in dealing with both dystrophin and 6MWT measures, it would be too risky to start a trial until they signed off on the endpoints.
kay - you may have raised a legitimate comparison to the efficacy demonstrated in Prosensa's 2b trial, but when when you say "nothing is tracked" when it comes to safety in the SRPT's trial, you just portray what a moron you are. All the liver tests that Prosensa failed are part of SRPT's trial - to say otherwise is just idiotic.
Not sure if SRPT provided individual 6MWT figures at baseline, but the averages for the different cohorts are in their presentation slides - for the treatment group, the average was ~366 meters and then the average for the placebo delayed group at wk36 was 328 meters. They also provide a range for all 12 with the lowest being 259 meters and the highest being 437.
I've commented on the fact that the parents of boys that have lost the majority of their physical capacity will have a tough time deciding whether to treat their boys, but are you saying someone like Austin McNary, who has been in wheelchair for several years and is now losing the ability to use his arms shouldn't get the drug? Maybe 10%-15% of the boys are past the point where treatment might be hopeless, but for the remaining population, you have to be kidding if you think the "penetration" within the first year is less than 90% - unless there are insurance/payment issues, what parent or doctor is going to say no to this drug?
The "subset of treatable age ranges"? Where do you come up with that - what doctor or parent is going to determine that their boy is too young or old to be getting the one treatment that could benefit them?
She is such a dim bulb, I'm surprised anybody pays attention to her. She has made it clear that they are not going to hike rates until the labor market is fully recovered (not just the unemployment rate, but some sustainable wage inflation) and that is not going to happen for several years, if ever? Not that I believe analyst price targets are any more meaningful, but the majority of my biotech holdings are trading at less than half of what the analysts' median price target is, so how much overvalued can the small cap biotech market be?
What's your point - everybody already knew these stats. The existing population of exon 51 boys in the US was estimated in the 1,500+ range , so assuming Etep helps these boys live longer, the market should slowly grow from 1,500. At the bottom end of the pricing range, Etep would generate 1,500 X $200,000 = $300M annually beginning next year. The remaining exons in SRPT's portfolio would push revenue to over $500M within another 5 years. Are you saying the current share price over-values that revenue opportunity?
The key to a 4th biopsy would be if it is done in accordance with whatever protocol the FDA has deemed appropriate for the upcoming Phase III trial. The FDA has done a lot of hand wringing over dystrophin measurement (what the best test is, how the muscle is sampled, how the labwork is done, etc.), but it is not yet clear whether they have decided the Phase II dystrophin data from the Etep boys is lacking in some manner. A 4th biopsy that showed consistent or improved dystrophin measures (vs 48 weeks) would help prove the durability of the treatment effect and may help interpret the 6MWT and pulmonary data.