What exactly are you looking at - the 96 week data showed every boy except the one with the broken ankle, at less than a 10% decline and the 120 week data showed the boy with the broken ankle improving (from -20% at 96 weeks -15% at 120 weeks) with one boy falling slightly out of the 10% decline category (he was -7.2% at week 96 and fell to -12.7% at week 120). Are you really going to nitpick the one boy that lost 5% over 6 months as something that taints the conclusion that all the boys showed stability at week 120?
Don't kid yourself simp - your post, which I replied to in detail, was that there 96 week and 120 data were "fragile" when it came to statistical significance, as reflected by the impact of the boy that broke his ankle. The 96 and 120 week data show nothing but stability and the fact that a boy breaking his ankle distorted the SS data is meaningless - the fact that he recovered and improved by the next test is much more meaningful. My comment about the data needing to be flawless or subject to attack the uneducated is exactly what your post was about - you don't understand the data, so you think a change in statistical significance (which can happen even if all the boys improve) or a modest decline by one or two the boys would be more meaningful than the remaining data. I was pointing out the flaw in the short thesis and you were promoting the short thesis - two different concepts.
zwerp - your 2nd point is the sad position we are in currently, in which the 144 week data almost needs to be flawless or will be subject to attack by the shorts and/or uneducated. As pointed out before, the significance in the results to date is not in the mean changes from baseline in 6MWT (measured several different ways), but the fact that all ten of the boys have been stable after 24 weeks of treatment. The fact that the FDA could write off ambulatory stability in 10 out of 10 boys over 96 weeks as being due to variability in the natural history of the disease is scary enough, but it erroneously opens the door for the shorts to point to any modest decline by any of the boys as sign that the drug is not working. Given some of the parent stories posted recently, I have hope that some of the boys will actually improve, thereby muting any potential damage by a boy or two showing modest decline. As you point out, there are so many variables (dosage, age, etc.) that will dictate the long-term benefit from Etep - allowing it to get shelved or delayed because the results may not be pristine would be a crime.
BTW, I noticed BLUE today, as well. It presents an interesting valuation puzzle, as the patient population for their drug is 15,000 (much higher than than the Exon 51 population), but it appears their drug effectively cures the patients with a single treatment? While it is only been tested on two patients, the clinical outcome for BLUE's drug is black and white after a few weeks, while the ultimate clinical outcome for DMD boys on Etep won't be known for years, perhaps decades.
simp - you don't have "opinions" - you have delusions. Your MO is to create irrelevant issues because you don't understand the basic facts or concepts. From that platform of cluelessness, you then try to read between the lines of press releases and other documents to conjure up some fantasy about what is going on "behind the scenes" The end result is mental diarrhea, which you then freely deposit on this MB. Many rational people have tried to convince you how worthless your posts are, but you continue on undeterred - another sign of mental health issues. Please seek help for all the sake of us all.
simp - you don't understand anything and you "espouse" nonsense. You should spend less time on "diligence" and more time seeking treatment for whatever blunt force trauma has left you so clueless.
Bill - didn't you read, pete has "done weight training" - he knows everything there is to know about adrenalin and neurological diseases!
Anybody that is still discussing SS doesn't get it. Statistical significance in this trial was defined as the difference in the change of 6MWT distance between the treated group and the placebo delayed group at 48 weeks - that difference of 61 meters was statistically significant. The fact that this differential has held up over 120 weeks (it was 58 meters) is not important - what is important is that both groups continue to be stable. Would the results at 144 weeks be more meaningful to you if the placebo delayed group dropped by 30 meters, while the treated group remained stable (thereby increasing the differential and statistical significance)? Of course not - what is important is their 6MWT scores continue to NOT DECLINE. The other side of this hypothetical coin is what if the placebo delayed group increased their 6MWT at 144 weeks by 30M, while the treated group remained stable, thereby cutting the differential in half and losing SS - would you consider that a bad thing? SS between the treated and placebo delayed groups is meaningless at this stage of the trial - they should be considered one group of treated boys and as long as they continue to be stable or improve, that is what is significant.
jrrt1 - your comment on "redeemable muscle" is key. I think this is where some of the ignorance on the short side lies - Etep does not allow previously damaged/destroyed muscle to be restored - it only prevents further damage to the existing functional muscle tissue. If the Becker's theory holds, it is still unclear what the long-term prognosis is for some of these boys that have already started to decline. It is obvious that a boy that has already lost ambulation will not regain ambulation and the hope is that boys treated early in life will never lose ambulation, but these boys that begin treatment when on the cusp of beginning to lose ambulation (7-9 years old) are in a gray zone - will their existing functional muscle grow with them as they age and allow them gain strength and avoid the wheelchair? I don't think that question will be answered for decades and it won't be absolute for every boy. Some of the recent anecdotes from the parents about their boys gaining strength or "improving" in functional tests offer some hope that they can gain further muscle function as they age, but only time will tell. Either way, Etep is the best hope they have for dealing with DMD.
The "flexibility" from the FDA you are looking for will probably have to be rammed down the FDA's throat in the form of the advisory panel - when the experts get up and (by that time) speak to 168 week results being unprecedented and "bloody obvious" that the drug works and denying conditional approval would be a crime against the boys and families, then the FDA will have not have the flexibility to deny approval.
Idiot - his options have exercise prices of $8.73 and $9.86 - they are out of the money - no "stockholder money gone". Any other clueless observations to share?
What "projections given before" - I don't recall the company giving any projections - only estimates of the potential market in terms of # of procedures. They have never given guidance or estimates on $ revenue potential, although several analysts have. Until they get CMS and other pricing resolved, how can they give projections?
No, I think they trust the CEO, who said yesterday that there will basically be no significant news until q1 of 2015 - they will have data by the end of the year from the MD trial, but any news release has to get approved by Pfizer, which can take some time. The short-term players are moving on.
Omidria is not a "sexy" cancer or rare disease treatment that is going to generate big money quickly from a huge price per patient - this will likely be a slow, multi-year ramp-up to a couple hundred million of revenue annually. That is not enough of a story to grab institutional attention, but it will help fund their pipeline, which is where the real value in OMER is hiding out. Their PDE10 inhibitor (OMS824) is worth more than the current market cap alone, if the early clinical results bear out. The issue here is that nobody believes the pipeline has any value - until they do a major licensing/joint venture deal, nobody will believe in or even bother to look at the pipeline. Is the $60 WBB target reasonable - definitely, but it will most likely take more than a year to get to half that value - they have some early clinical data coming up later in the year on OMS824 and OMS721 - those might move the needle a little bit, but it will take an accompanying venture deal to get past $30/share.
A lot of questions left unanswered - my sense is that they are delaying the launch until they get their CMS status confirmed, so that doctors are assured of a reimbursement for using Omidria. They bragged about all the marketing people being in place already and no issues with manufacturing, so why would they delay the launch until late summer? I can't blame them for not providing pricing info until they get CMS status confirmed.
He wants to be a "headline" presenter at a conference in Oct, which usually means they have to limit the information publicly disclosed beforehand. The headline data will be available over the next 30 days - my sense is that the data will be too good to hold back - we've already heard from a few of the parents speaking to "improvements" that their sons are showing in their latest tests.
The efficacy and safety results from the two clinical trials were so overwhelmingly positive that the only way Omidra was not going to get approved is if the company messed up with the filing or didn't have the manufacturing tied down. Having said that, I thought a 10%+ rise upon approval was in order as a de-risk on the filing/manufacturing issues. The sell-off today is most likely from the short-term traders that were looking for a larger pop and when it didn't materialize are now moving on to greener pastures. Some comments in the PR may have tempered any enthusiasm, as well - I thought they had guided that they were ready to market/ship Omidra almost immediately after approval (Wedbush was assuming Jul) and now they are saying "late summer/early fall". Also, they don't yet have Medicare/Medicaid reimbursement tied down, which is a big part of the revenue profile. Maybe today's call will provide some comfort around the launch and revenue potential, but this may be one of those "show me the money" stocks that doesn't move until the company proves the revenue potential is there?
If SRPT's issue is a lack of "n", RNA's issue is too much "n" - i.e. too many patients with injection reactions and kidney problems and more than enough "n" to produce a statistically significant efficacy result if it was there. That is what seems to be lost on most bashers - you could dismiss an n of 12 if only half of the boys were stabilized, but when 10 out of 10 ambulatory boys are stable after 2 years and 12 of 12 show stable/improving pulmonary function, it is clueless to talk about statistical significance.