I agree that their labeling of slides is confusing, but the "change from baseline" means the absolute difference between two percentages - e.g.. 45% of normal less 5% of normal (at baseline) = 40% of normal change from baseline. Given the vagaries of measuring dystrophin and the low levels of the boys at baseline, the percentage change from baseline would be a strange number to report - if a boy went from 1% at baseline to 4%, that would be a 400% increase - not very meaningful.
new_hunter - where are you getting this #$%$. Every press release and presentation of dystrophin results have been expressed in "% of normal", not a percentage increase from baseline. Look at the Oct 3, 2012 press release - the results were an increase in dystrophin to "47% of normal" - I believe the baseline values were 0%-5% of normal, so you are way off.
I agree that patience will be rewarded, but this stock is not going to move until they resolve the funding issue - they need to raise money and until the level of dilution required to get them to breakeven is covered, there won't be much that can move this stock price.
simp - I thought on the call that CG said the FDA had been provided some dystrophin data by RNA, but they did not have the detailed 6MWT data - either way, you can't make an assessment like that with half the data. IMO, stats get thrown out the window - i.e. the fact that some percentage of the Drisa patients may have produced some level of dystrophin is meaningless - you need to look at each boy individually and make an assessment on how much dystrophin was produced, when it was measured, how it was measured and what relation (if any) that had to their 6MWT performance. The impression from the call is that the FDA did not have the data to do any detailed analysis on an individual patient basis.
Doing a trial with multiple exons is not feasible at this stage - they haven't even filed an IND for any of the other exons, let alone have any manufacturing capacity in place for anything other then exon 51.
Who cares about the shorts, but where do you come up with this 30% dilution in the near future garbage?
msmorris - please refrain from your idiotic babblings - only a complete moron would suggest a company should attempt to do a trial on "ALL" exon 51 patients. You have surpassed pasteur in the idiot contest, so you can comfortably retire - I doubt anyone could surpass in a lifetime the mental turds you have deposited on this site the past few days.
Again, pasteur, your comments just shine a light on your ignorance. DMD is a disease that destroys muscle due to a lack of dystrophin - once the muscle tissue is destroyed, it cannot recover. Etep is a drug that allows dystrophin to be produced, delaying/preventing the muscle from being destroyed - it will not generate an "improvement in strength and motor function" - as evidenced by the clinical results, it prevents the decline. Your comments about IHC and dystrophin as a surrogate are equally ignorant - time for you to move on.
Slide 11 from the Oct 17 presentation has the individual changes in 6MWT, but not baseline figures - it also has ages at baseline. This idea that "children who are stable at 350M generally remain stable" is just idiotic. Many of the natural history studies are 6 months in time, a few go out a year, but there aren't any that are two years in length. Drisa's trial used comparable enrollment criteria as SRPT's trial and their boys aged 7+ declined by 60+ meters over 48 weeks. The twins in SRPT's trial (who were placebo-delayed, so did not begin getting Etep until 24 weeks) lost ambulation within 36 weeks, while the other 4 placebo-delayed boys lost 15%-18% of 6MWT by week 36, so how does anybody come to the conclusion that this patient population is "stable"?
pasteur - you should be working for the FDA - while you dismiss as improbable/irrational that 10 out of 10 boys showing stability in the 6MWT is evidence of clinical benefit, you promote the possibility that Etep sped the decline of the two boys that became non-ambulatory. That kind of reasoning fits well with bureaucratic, brain-dead analysts that decided the Drisa data has some relevance to SRPT's drug/trial.
pasteur - I'm not celebrating anything - I'm simply pointing out the ignorance of those claiming to understand the inner workings of the FDA or a superior understanding of the clinical trial process. You can celebrate all you want thinking your views have been validated by the senseless position taken by the FDA with respect to AA of Etep - none of your arguments make any sense in the context of the AA rules/regulations and none of the "concerns" recently raised by the FDA make any sense, given the data that has been generated by SRPT - why Drisa data is meaningful at all in that assessment is even more senseless. If you take pride in being validated by senseless decisions ignorant bureaucrats make, that is even sadder.
Wow - I sensed you were a complete moron from your earlier postings, but you just sealed the crown of king of the morons - I've never seen such a diatribe of gibberish before. You and pasteur should become fast friends.
What exactly do you mean when you say the "variability amongst the patients were not controlled"? What variability needs to be controlled? This is not a flu treatment, where a patient's response to a drug could be influenced by a number of factors - age, race, sex, etc. This is a genetic disease where the patient population is by definition strictly defined and restricted even further by trial design (intended to measure clinical benefit by decline in 6MWT) - the whole purpose of the enrollment criteria was to isolate a very homogenous patient population. Your comment on variability is senseless.
pasteur - you are the one that is ignorant - you keep pounding "issues" as if they are somehow newly discovered by the FDA. Up until a month ago, the FDA seemed perfectly fine with data that was generated by SRPT's trial - is it your ignorant assessment that they suddenly realized after over a year of communicating with the company that the iTT data excluded the twins? Good analysis!
grey - the most glaring example of the FDA's ignorance/confusion is with respect to the 6MWT data from the Drisa trial and the implications for SRPT's data. They claim that they cannot accept SRPT's 6MWT data as clinically meaningful because the "variability" of 6MWT for SRPT's patient population could explain the stability the boys are experiencing. First off, if you are saying that the natural history is highly variable for the given patient population, then that is in direct conflict with SRPT's data - the 10 boys show remarkably consistent stability. Second, they use the Drisa data to conclude that dystrophin may not be a valid surrogate because the boys in the Drisa trial produced dystrophin (I haven't seen the data, but from what I have read their dystrophin production data was sketchy, at best) but their 6MWT performance declined (by an average of over 60+ meters in both the placebo and treated arms over 48 weeks for boys aged 7+). So, they are telling SRPT your patient population should be stable (over 96 weeks) given the natural history data (which they mis-interpreted), while pointing to the Drisa data in the exact same patient population (showing substantial decline over 48 weeks) to argue against dystrophin. I haven't seen this ignorant of a position in a long time.
You're an idiot - it is clear from the history here that any "expectations" that were communicated from management came directly from conversations with the FDA. It is also clear that the FDA blindsided them with their 180 degree stance on AA over the past month. It is also clear from the communications received by SRPT (i.e. direct quotes from the letter that was sent to SRPT by the FDA last week) that they are clueless as to the natural history of DMD and have completely bailed out on dictating what would be an acceptable method of dsytrophin measurement. Anybody that reads the quotes from that letter understands that the FDA is basically saying "we do not understand the natural history of this disease, we do not understand the various ways of measuring dystrophin, and therefore we cannot provide you guidance on how to set up a clinically meaningful trial". It is also clear from the comments that CG passed on from the meeting that most of the confusion the FDA is dealing with has come from their "interpretation" of the failed Drisa trial, which they admitted they don't have all the data for, yet.
I agree with the "monster" potential - cancer stem cells are the key to any hope for a "cure" and this company is at the forefront. The messy part is not knowing how Wall St is going to manipulate this stock over the short term - stocks coming out of an IPO tend to be subject to some wild swings. I would hope that $13 is as low as they can push it, but who knows?
Pressure will build as time progresses and the boys remain stable - if the 120 week data is as good as the 96 week data, then the parent groups will push some congress member to have a hearing. The dystrophin measurement/surrogate issue is more important for future exons - continuing stability on the 6MWT will push the AA agenda for Etep over the next 6-12 months.