CG's mistake was being perfectly honest and open about the company's interactions with the FDA. Why would this cast him in a bad light at the FDA? Because the FDA's actions are often capricious, illogical, and delayed beyond reason and expose them for the lazy, entrenched bureaucrats that they are - accountable to nobody, certainly not to any executive that has the gall to shine a light on their incompetence.
I think it is more than "interesting" - Jett is in college, so hasn't used a pencil in 4+ years and after 4 months on Etep he suddenly feels like writing again? I'm sure it will be viewed as just another placebo effect by the twits at the FDA - they probably believe that if he doesn't get out of the chair, the drug isn't working.
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?
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.
The most interesting, but not surprising, point to come out of the interview is that he confirmed that for the 5 months before the FDA sandbagged SRPT with their Nov 2013 letter putting AA back on the shelf, the FDA had no communications with the company. The FDA reps speak to how they are "working closely" and "collaborating" with SRPT and other companies to accelerate the approval process, but the reality is not even close. Most of the issues the FDA brought up in the Nov 2013 letter could have been addressed in a couple of phone calls, but that is not how they operate - they work in the dark, drag their feet and generate irrational and ignorant formal directives.
The same thing happened last year, when they took 5 months to respond to the dystrophin lab review (done in May) and without any interim communications with the company or the lab came out with a formal conclusion (in Oct) that there were unspecified "marked disparities" in the methodology used for IHC measures. Were any of these "disparities" revealed in the dystrophin measurement conference, or can we conclude the FDA didn't understand dystrophin measurement, took 5 months trying to figure it out on their own and then punted by blaming the lab for something they didn't understand? Is that what they call collaboration?
It will be interesting to see if the parents have the right to know the 4th biopsy results before SRPT may want to disclose them. On the one hand, it would be hard to put your kid through that procedure without having the right to see the results, but I'm guessing the company may have conditioned that with a non-disclosure agreement - i.e. you can see it, but not report/tweet/post/etc. to the outside world?
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.
The only thing you need to know about the 4th biopsy is that 11 out of the 12 boys parents agreed to have it done - the only one declining was because the boy had a reaction to the anesthesia during an earlier biopsy. The 11 boys that agreed included both twins who lost ambulation early in the trial. So, you have to answer this question - why would a parent allow their son to continue on a drug that requires weekly IV infusions, let alone agree to a painful and scarring biopsy, if they did not believe the drug was working? Do you think the parents can't recognize whether the drug is providing a "clinical benefit" after 3+ years?
bf - I'll admit that the BBJ article was void of any "antagonism", but CG has never been publicly antagonistic toward the FDA - he may have expressed frustration, but never disparaged them and is too smart to start doing it now, while the NDA is still in process. But beyond that, your "perspective" on the FDA is far too kind. To couch the FDA's approach to Etep as wanting to "do the right thing and avoid introducing another thalidomide" is just ridiculous - thalidomide was a widely distributed drug to otherwise healthy people for minor symptoms (nausea, anxiety, etc.), while Etep is targeted at a narrow population of dying boys. The mode of action for Etep is well defined and the safety profile is more than acceptable in the context of a rare, deadly disease. Beyond that, the FDA has been mandated by legislation to alter their "process" when it comes to rare, deadly diseases and it is clear that they have not. How you could come to "appreciate" anything the FDA has done with respect to Etep is beyond me?
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.
Get a clue winter - provide us one concrete example where CG disrespected the FDA in public. His policy was full disclosure to investors, analysts and parents about the FDA communications, because that is what they were clamoring for - he never spoke out against the FDA, because he was smart enough to know the parents would do it for him, and rightfully so. Any doubts/uncertainty about what they would do next was sincere, because the FDA changed their minds several times based on faulty assessments or assumptions. The FDA wasn't on "the playing field of science" - they were clueless in their analysis and took months to come to the conclusion that they didn't understand the data they had in hand and wanted to see more. You probably come from a world where politics and #$%$ are meaningful "skill sets" - get lost!
The only rational part of your latest rant is when you admit you have "no clue". When you say the FDA has a problem with dystrophin as a "marker", you have to distinguish between the validity of the marker and the validity of the measurement - it is clear the FDA has issues with how dystrophin is measured (IMO because they don't understand it), which is the focus of this panel, but not dystrophin's validity as a marker. The only hope of DMD drugs being developed for the lesser populated exons is if they resolve this measurement issue.
In the end, there are more measurement issues inherent in the 6MWT than there are with dystrophin measurement and the idea that BMRN has a drug that is by therapeutic design supposed to produce dystrophin and part of their strategy is to discount the importance of measuring dystrophin is clueless.
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.
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.
Another fantasy post - if only big pharma was involved, blah, blah, blah. Yeah, it would have made a world of difference for a company that was not familiar with SRPT's PMO technology and/or DMD to be "educating" the FDA grunts, who have proven that they have absolutely no clue about the disease, dystrophin measurement, etc., etc.
likeafox - I'm struggling with the concept of "rescued", but given the nature of the disease, there is no doubt that muscle tissue is destroyed little-by-little and that at some point it becomes irreversibly damaged. It is hard to imagine any of their diseased muscle tissue is truly healthy, so the idea that Etep can improve the health of all but the tissue that is past the point of rescue is a valid one. Your idea that fatty infiltration and scarring being more advanced in the legs also seems rational, given the burden the leg muscles incur with simple walking vs the rest of the body.
As I stated before, I believe the deterioration of the 6MWT scores is not so much of a story of limited effectiveness of Etep, but more the inability of the remaining healthy leg muscle tissue to keep up with the increasing height and weight burdens of these growing boys. I would bet that just like Christine McSherry, all the parents have seen subtle improvements in the muscle function of their boys outside of their ability to walk. The FDA is supposed to take these "patient reported outcomes" into account as part of the approval process for rare, deadly diseases, but so far they have shown no signs of doing anything but getting caught up in the complications presented by the clinical data.
The thumbs down are because you add no value to the discussion or analysis - saying "bad things have happened" and there is "tremendous risk" are vacuous statements. Do you believe the risk lies in the fact that Etep may not really work or in the fact that the FDA doesn't seem to have a clue on how to define/assess the clinical data? Are the "bad things" the result of management incompetence or capricious actions by the FDA? If I had 20% of my net worth in this stock, I'd hope to bring a better understanding of the risk/reward profile than it "is fantastic given the odds of a likely NDA filing". The filing of the NDA is not a value driver - it is a given that they are going to file the NDA at some point. Unless the company divulges the results of the biopsy re-reads or 4th test first, the next meaningful event that will clarify the risk/reward is the Advisory Committee.
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.
I don't love him - I just don't understand your incessant need to bash him with rants that are either false or make no sense. The fact that you can't understand the truth doesn't make the things he says wrong. The ONLY market that exists for an Ebola treatment is the US Govt (or some other western govt), but because you don't understand that concept, CG is wrong for stating it? What a tool!
They don't need the cash - I'm guessing they are pushing some cheap shares to some "preferred" institutions and insiders. The warrant price speaks volumes - $10.86 - 3-times the offering price. When was the last time you saw a warrant price that high relative to the offering? I suppose if the Phase III trials get dragged out (they are waiting on a certain number of "events", so timing is uncertain), they may start to run low on cash, but they could have waited until the end of this year, when presumably they would have more mature data from the various Phase II trials - between that and the anticipation of the release of the Phase III results, the share price would surely have been higher toward the end of the year.