bionerd - do you think that dystrophin biopsy data from the new trials will be part of the rolling submission or just safety data? I believe the FDA only requested safety data from a certain number of patients as part of the submission and frankly the safety data, while important, is not a game changer. Assuming the biopsy re-reads and 4th biopsy data is clean and shows meaningful dystrophin levels in all of the boys, then you wouldn't think they would need dystrophin confirmation from the new trials and obviously there won't be any meaningful 6MWT data by mid-year from the new trials. Assuming the formal filing does not include any dystrophin or efficacy data from the new trial, but the AdCom is scheduled for Q4, does that open the door for additional data to be part of the AdCom discussion or is it limited to the data from the formal midyear filing?
Thanks, left_e - I went back and looked at the clinical trial description and the primary endpoint is the change in VA scores from baseline, so if Isonep does stabilize vs continued deterioration in the control arm, that will be a success. I seem to recall an endpoint that required an improvement in the # of letters read off some chart, but that is a secondary endpoint.
This is an interesting point - if Asonep is proven to reduce lesion size, but misses on improving VA, is the drug a bust? Like most degenerative diseases, I would think it would be meaningful to provide stability/lack of progression, while the hope for reversal or improvement might be a pipe dream. Did Lucentis and Avastin actually improve VA in their trials - if so, I assume that the improvement was only temporary, as most patients ultimately progress, right?
They seem to be looking to validate whether Omidria is going to sell as well as the positive tone management is setting. I doubt any of them will go out on a limb at this stage - they will likely want to see a couple quarters of real sales figures before they put a stake in the ground. Therefore, we will likely need the rest of the pipeline to drive any appreciation for the rest of the year. OMS721 is looking to be a big winner and if they can just get OMS824 restarted, that may reveal some promise before the end of the year?
What I would like to see more than anything is for them to partner something else in the GPCR pipeline - they obviously won't have the capital to push anything hard until Omidria comes through and I think they need external validation of some value in all these GPCR "targets" they have been tying up for the past decade?
Unless the AdCom panel is staffed with hacks and/or experts with compromised interests, it will be supportive of approval. The idea that the dataset is "thin and inconclusive" is laughable - 3+ years of safety and stability is thin and inconclusive? The fact that they continue to tie Etep's fate to Drisa is also laughable - Drisa has a thick and conclusive dataset - one that proves it is not safe and not effective. Assuming Etep is approved, how do you make an assumption of a 50% market share - are they assuming parents and doctors will choose Drisa over Etep? Practically every comment in their analysis is refutable!
I believe Kaye said they were using some arm strength measurements in the trial for nonambulatory boys, so maybe that trial will produce some unexpected efficacy results. I'm sure the FDA would look at the video and say that Austin and Jenn are probably lying because of their desire to get the drug approved.
Did you hear the moronic analyst who questioned what the company has done to improve relations with the FDA during the 5 weeks since CG has been gone. As if the company is in constant communication with reps at the FDA - what an iditiot. The FDA reps operate in a black hole and no communications are made unless in formal documents or meetings - that is how it is done with all companies. The reason it is an issue with SRPT is the FDA is claiming close collaboration and continuous communication under the AA guidelines, which we all know is BS.
The good news is that they seem to be accelerating the path to commercialization on all fronts - the bad news is that this will greatly increase their cash spend, so they will have to raise money sooner than later. Theoretically, they have plenty of time (through mid-2016) to allow further good news and milestones to lift the share price before raising more cash, but my sense is that this company is too conservative to wait very long?
It is clear why the FDA has done what it has - because the people making the decision have financial ties to the companies that don't want to see the status quo changed. Companies are making hundreds of millions off of the morphine pump system and they don't want that gravy train being disturbed, so they convince their cronies in the FDA to stonewall a company that has a better, safer technology.
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?
Now we know why the big selloff over the past week - Arun tipped off the institutions on the soft quarter in the private "conferences" earlier this month.
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?
They had two private investor "conferences" on March 31st and April 1st. Perhaps they hinted at weak revenue growth numbers in the near-term, prompting some institutional sell-off? If that is the case, then this is just another example of how management has failed investors.
CNBC is a joke - I used to watch it all the time (the Mark Haynes days), then I used to have it on with the sound off just to see the tickers, but now I can't even stand to have it on. There is very little information/discussion/analysis that is useful to investors - as you said, day traders, volatility, option activity and market "action" is the focus. It is a microcosm of what is wrong with our financial/banking system today.
You seem to be confused - the filing of the NDA is expected "mid-year" and the FDA's decision on whether to grant AA won't take place until late 2015 or early 2016. In between that time (most likely in Sep/Oct), the Advisory Committee (which is a public event) will take place. The "filing" is a non-event - whether you don't understand the process or just are careless with the terms you use, you are the one who sounds dumb.
simp - do you really believe "who's in the lead" has any substance to the long-term value of SRPT's DMD franchise? All the data (preclinical, clinical, parent disclosures, etc.) point to Etep being clearly superior from a safety standpoint and more than likely superior from a clinical efficacy standpoint, so bringing Drisa into any discussion of SRPT's long-term value is senseless. As always, the "market" (momentum players, shorts, hedge funds, etc.) will create volatility out of any nugget of FDA action, regardless of how meaningful, but IMO that is just noise. Unfortunately, all the noise the FDA has created over the AA process has trashed the share price, but for those that believe Etep works (including all the parents), it will all flush out in the end.
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.
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?
bionerd - Why would CG have to "plant" Hoffman's conflict of interest - the whole world new it - many on this board have posted about it for years?
You never laid the responsibility equally - I don't recall any of your posts taking issue with what the FDA has done during this process, but you have continually bashed CG without providing any concrete statements of what specifically he did wrong or how you would have handled it differently. I have no doubt that he rubbed people at the FDA the wrong way, because I'm sure he pushed them to provide specific answers to the issues the company was facing in its effort to get Etep approved as quickly as possible. The idea that if he had somehow "played" the Etep data hand differently it would have been approved by now is ridiculous, just as most of your posts are.