Be serious - these are share purchases under the company's Employee Stock Purchase Plan - CG & Kaye purchased 800 shares each - I didn't check the others - hardly worth celebrating as an insider commitment.
You are going to be disappointed - the CEO stated in the presentation yesterday that they will not be getting access to any data with the upcoming DMC recommendation - they want to stay blinded. The DMC will either advise to carry the trial on with the same targeted enrollment (140 patients) or advise the company to increase the enrollment if the data to-date s not solid enough.
The thing that worries me about this Phase II trial is that the primary endpoint is not an objective measure (e.g. blood ammonia levels or reductions), it is how quickly a patient moves on the subjective HE scale, as interpreted by the doctors. As the CEO explained yesterday, the enrolled patients are essentially in two groups - stage 3 & 4 HE patients (the worst) and stage 2 patients (moderate HE symptoms) and the endpoint is how quickly the 3/4 patients are reduced to stage 2 or the stage 2 patients are reduced to stage 1 (normal). Trials with these subjective observations of efficacy always scare me - unless there is a clear difference in the treated group, the results tend to be flaky, even if the drug is providing an objective benefit. They will be taking blood measurements as a secondary endpoint, so hopefully at least they will get confirmation of efficacy in that respect?
Canaccord believes that Etep is a "very effective drug" and the approval issue is "not a question of if, but when" and can only conjure up a $16 price target? I read their methodology of discounting back fully taxed and diluted 2022 EPS by 40% (discount rate seems high if you firmly believe "if" is not an issue), but does that mean they have given no value to the other exons or the platform?
Surprised the stock is in the green today. I may have to reassess my opinion on whether there are still buyers of the story. Either way, I hope this means that a solid floor is in place and we won't be threatening single digits, again, like we did a few months ago?
big - the operative word here being "preliminary" - when BTD was given to RNA in 2013, they certainly didn't have any of the pivotal trial data. If you are implying that RNA's phase II dataset has been scrutinized to the level that SRPT's has been in determining whether to give Prosensa BTD, you must be joking.
neo - BTD is not a validation of the efficacy, safety or approvability of a treatment, simply a recognition that IF the drug is effective, it would be a new/unique method of treating the disease. If you believe that BTD somehow implies the FDA believes the drug has already met some threshold of efficacy, you are clueless. BTD is typically granted well before any pivotal trials are held, and we all know that RNA's pivotal trial failed miserably.
A valid question, but I'm confident that if he wasn't attending these conferences, you would have as much or more negative speculation as to what is going on. After listening to today's call, I don't know who is worse - the analysts or some of the more paranoid posters on this board. He has clearly stated since the Oct bomb dropped by the FDA what the strategy and timeline is and that they won't be reporting any new data until after meeting with the FDA, yet there is still a scary level of hand-wringing over what exactly is going to happen over the next 6 months. IMO, it sounds like they have been successful in obtaining much of the natural history and MRI data that they were requested to obtain, the dystrophin re-reads are moving ahead and with 11 of 12 boys agreeing to the 4th biopsy, what else could you ask for? The story is the same as it has always been - the company has responded to the hoops the FDA has placed in front of them, but there is no way to predict how the FDA will react to the performance or if more hoops are to follow. I've always argued that the FDA will ultimately be backed into a corner by the Advisory Committee and the parent groups, whereby the data clearly shows that efficacy is "reasonably likely", which is all that is technically required for AA. But given how capricious the FDA has been, it is still a crapshoot. Either way, all this hand-wringing over how this plays out before the AdCom is just senseless.
I can't believe anyone considers BMRN a competitive threat. Their Phase III study failed on 6MWT and the idea that they can somehow extrapolate data on younger boys that proves any level of efficacy is laughable - the natural history clearly shows that these boys are just as likely to increase their 6MWT performance. Even if they could sell the FDA on some level of efficacy, their safety profile is a disaster, especially for a drug that needs to be taken for life. Beyond the human clinical data, the animal models all show Etep to be the superior drug. The fact that BMRN thinks they have a chance at approval with a clearly inferior drug should tell you that SRPT's chances of approval and ultimate success in the market are much greater.
The fact that in five hours you received no responses is very telling. I think a lot of investors have moved on and taken the "I'll believe it when I see it" philosophy. The "story" has always been compelling, because the technology is so impressive, but NVDQ's execution has not kept pace with the story. No doubt the LifeCell takeback was a bump in the road and the early growth from Pinpoint and Luna looks solid, but there have been a number of early growth buds that have failed to blossom. If the real revenue growth rate in 2015 exceeds their targeted 40% (which it should, given the tainted base and all the irons in the fire), then you might bring back some investors that are willing to bet on the story. Otherwise, this is a "show me the money" stock, that will not attract much attention until they hit that standard of care/hypergrowth stage, which won't be for a few years, IMO.
You're equating share price movements to the probability of an approval? Beyond the issue of whether the "market" is always correct in assessing these issues, your commentary dismisses any and all data, other than if a "boy sits down for the last time". So, the dystrophin re-reads verify the initial assessments, the 4th biopsy verifies stable, long-term dystrophin production, pulmonary measures show continued stability, but you've decided that if one boy ends up in the chair by the end of the year, Etep is done. Sound assessment!
What a ridiculous statement - "one of the boys sits down for the last time before approval". You clearly don't understand what is going on with these boys - their ability to continue walking is just one aspect of the clinical benefit that Etep is providing, and given that they are growing every day, asking leg muscles that have been wasted for 9+ years with this disease before they started treatment to keep them on their feet as they grow is ridiculous. If you really think the prospect of Etep being approved hinges on all ten of the boys remaining ambulant before the FDA makes a decision, you are clueless.
grey - it is my sense that the reason CG has committed to withhold disclosure of the new biopsy data (re-reads and the 4th) until they are able to review it with the FDA and get a feel for how it will be used (or not used) in determining AA is that CG does not want to publicly embarrass them and risk the petty backlash that may result?
Where does this fantasy that "large pharma" would have made a difference in the clinical path of Etep come from? The delays that taken place so far have "objectively" been caused by inept decisions or actions by the FDA. Many have claimed the problem is that CG tried to cram an AA down the FDA's throat without the necessary data, so is the argument that a larger company has some sort of political (or corrupt economic) power over FDA officials that would have allowed them to more effectively cram down the AA process?
Like most of your opinions, simp, this one is just speculative nonsense that adds nothing to equation on whether Etep will realize an AA and/or if SRPT is a good investment.
I think they are targeting to complete enrollment by "mid-2015". Given that it is an acute indication (i.e. clinical response is measured in hours/days, rather than weeks/months), they should have Phase IIb results my Q3?
As you mentioned in your initial post, the rate of reduction in the treated group was 4-times that as the placebo - this seems impressive, but it would be nice to get the company to comment on how clinically meaningful this is.
I think the variability issue is something that a larger trial would eliminate or minimize. Besides that, as others pointed out, the dose used in this trial was half the dose being used in the other Phase II trial. The Phase IIb trial is targeted to enroll 200 patients, versus 38 in this current trial, so between the higher dosage and the larger population, the clinical outcomes should be cleaner.
My interpretation, which may not be correct, is that there were several plasma clinical endpoints - ammonia levels (absolute measure); reduction in ammonia levels (again, absolute) and relative reduction in ammonia (% change). Presumably, all patients were considered to have abnormally high ammonia levels at baseline and that they would theoretically be reduced to a more normal level after 12 to 24 hours. Given the high variability in the absolute levels, the difference between placebo and treated at the 12 or 24 hour mark was not statistically significant, even though there were marked differences in the absolute and relative reductions observed. So, for example, if someone in the treated group went from 10 to 5 (a 50% reduction), they were considered equivalent to a placebo patient who went from 6 to 5 in the absolute measurement endpoint.
A similar situation took place with FOLD last year, whereby a clinical endpoint did not take into account the variability at baseline, so even thought the absolute differences observed in the treated group were significantly higher, the trial was deemed a failure. When the dust settled and everyone figured out the drug was working, but the trail just had a poorly defined clinical endpoint, the stock quadrupled. Hopefully, that is the case here, as well.
The lack of statistical significance in a 38-patient trial (19 in each arm) is not unusual - if, as they indicate, the plasma ammonia readings were highly variable among patients, attaining SS with 38 patients is almost impossible. The fact that they did reach SS on urinary levels with such a small population is more reflective of the efficacy impact than the plasma readings.
What does "early efficacy responders" even mean? If there is any lesson to have been learned over the past 3years, it is that the 6MWT is a very flakey clinical endpoint - there are way too many variables at play, besides whether Etep is working - the two key variables being how much damage has already been done before treatment kicks in (impossible to measure accurately) and how quickly the boy is growing (easy to measure, but hard to interpolate how it will impact clinical measures). As has been discussed before, it will be decades before the ultimate clinical impact of Etep is understood - that, however, is not an excuse to withhold approval from boys that would benefit during that time.