I listened - he was never questioned directly about the recounts - he was questioned about the 192 week data and when/would it be released. He indicated that because they would presumably be in a review period with the FDA, they would work with the FDA on whether it was appropriate to disclose it and when and carried that concept into how they were going to deal with the dystrophin re-counts. He stated that they were not sure how the FDA would treat the re-counts - i.e. as verification of the original readings, as new readings that would become part of the filing, etc., etc., . The bottom line was that they were going to be very sensitive to how the FDA was going to interpret/use the data, before they disclosed it. Given that the FDA took five months to get back to them on the lab site review, don't expect a timely response from the FDA or release from the company. The fact that simp finds this "stupefying" is his typical paranoia.
Many posters here have bashed CG for not "reading" the FDA properly and trying to force his will on them, when every disclosure I have heard has been that the company has been ultra sensitive and accommodative to the FDA, despite how capricious they have been in return. He has gone out of his way not to make them look bad. In this case I don't agree with it - he should disclose the re-counts as soon as they have them, because the investors should have access to this "material" information, regardless of how the FDA will deal with it.
"Rich with potentially useful discussion ideas"? Whether the company gets AA or not has been hashed and rehashed on this board numerous times - everyone has an opinion, so what? What I focus on is facts and when posters make statements about facts that don't match reality, I call them on it. You premised your opinion on the timing of potential approval with a mis-statement of fact and I called you on it.
winter - where do you come up with this garbage - a "significant history of delays in its clinical development timelines". Up until this past quarter, the company has had only one active clinical program - the Phase II trial that started 3 years ago. They finally got guidance from the FDA on the confirmatory trials earlier this year and have started 3 new trials in the past three months - the confirmatory trial was started a couple months later than originally projected (not clear whether this was the company's fault an FDA delay on signing off on design issues) and the the other two trials were started either on time or earlier than projected and you concluded that they have a "significant history of delays"? You have a significant history of making clueless statements.
1) Will be confirmed by the independent re-reads of the Phase II biopsies and the 4th biopsy
2) Assuming 1 is taken care of, the clinical data doesn't need to be overwhelming, just confirm that there is a reasonable likelihood of clinical benefit - the AdCom will answer that question in a convincing fashion.
You miss the point - along with most shorts and the "market" in general. The whole purpose behind the AA regulations are that in certain diseases, long-term clinical benefit will be almost impossible to predict in a short-term trial or a trial with very few patients, in the case of rare diseases - DMD falls under both categories. In these cases, the regs allow you to look to a surrogate endpoint in order to get the drug approved, with the prospect of long-term clinical benefit being assessed in the confirmatory and extension trials.
I've said it a hundred times on this MB - what you call the "maximum clinical benefit" won't be defined from the Phase II boys or even the confirmatory trial - it won't be fully understood for decades, but that is no reason to deny these boys the prospect of the benefits this drug can offer during that time.
Regardless, the idea that the "real competition" for Etep is defined by the 6MWT results of 10 boys is, again, clueless!
What a clueless post - your defining the "maximum clinical benefit" Etep based on the loss of ambulation delay in a group of 12 boys that were on the verge of being in a wheelchair? If these boys lose ambulation, but live in into their 30's or 40's being wheelchair dependent, is the "maximum clinical benefit" the two years extra they stayed on their feet? What about boys that will be treated soon after diagnosis and hopefully will remain ambulant into adulthood? The fact that you wasted two full postings with this ridiculous analysis is unbelievable.
jrt1 - It wasn't "prudent caution" that made the FDA change the requirement to have more safety data before the filing, it was bureaucratic stall tactics. The data would have been available during their review period, - if they had let the company file at the end of 2014 they would have until mid-2015 to review additional safety data. How long does it take to analyze safety data on another 24 boys, particularly when there will be little to no adverse events to make sense of? There was plenty of preclinical and clinical data to support the safety profile of Etep - they were just making excuses in order to give themselves more time to pull their heads out of their @sses and make sense of the dystrophin and efficacy data.
SRPT had already committed to provide safety data from the confirmatory trial during the review process, so they FDA is not asking for anything they weren't already going to see. The fact that the FDA made the formal change to force the company to include the safety data in the filing was just part of their effort to stall the filing, because they were still not up to speed on the dystrophin measurement issues or the natural history. It took the FDA 5 months to provide a formal response to their lab visit/review and that response was just a vague dismissal of the measurement methodology, but no clearly specified issues. The point is that the FDA is used to moving at govt speed and that is when they have a clue about the issues at hand. They have shown they have no clue in how to deal with the dystrophin and natural history data/issues, so they are stalling the filing in the hope that the air clears and the issues become more black and white with time.
mc - I'm still here - just frustrated with the lack of near-term catalysts for THLD. I hoped the interim data on some of the Phase II trials might provide a mid-year spark, but I think we will be waiting until 2016 to get anything that will drive it above $5?
kg - you just contradicted yourself - if the market is already pricing it as a safe drug, why would a confirmation of that safety impact the share price? I agree if some unexpected safety issues cropped up, the share price would get trashed, but the upside impact of confirmed safety would be minimal. Under "normal" circumstances, confirmed safety might provide some upside, but the safety history of Etep and the platform are pristine - there have been absolutely no hints of any safety issues, so the market should not react to the same old news.
Safety data will be meaningless - everyone expects it to be clean. The prospect of safety issues is not holding this share price down.
No way CG is going to sell out at this level - a partnership is a long shot, as well - maybe outside the US? The only thing that will change the market perception at this point is the Advisory Committee meeting results. Assuming it is staffed with experts that truly understand the disease, they will set the record straight on how the clinical results to-date warrant AA. As CG said, it doesn't matter if the parents know Etep works, it doesn't matter whether the doctors that are treating the boys know it works - the only thing that matters is what the FDA believes. If the AdCom panel says Etep works and votes in favor of approval, the FDA will not be able to dismiss that conclusion and draw the wrath of the parent groups.
simp - for you, pondering is a dangerous thing. You take a simple premise (SRPT's current EV less than RNA's buyout price) and dredge up "warning signals" that have no basis in reality. Ebola was a shiny, scary distraction for the biotech market for a few weeks, but anyone with half a brain knew it presented no value to the company, given the status of its Ebola program (i.e. canceled by the DoD), let alone was any indication of the value of SRPT's platform. The idea that a "grade A CSO" would impress the market is another ridiculous conclusion - the "market" wouldn't know a grade A CSO from a grade C CSO.
SRPT's EV is simply a reflection of the fact that the FDA's stop-and-go dealings with SRPT has cast a significant shadow of doubt over the prospects of an AA from the Phase II data set and assuming AA is not successful it will be another 2+ years before pivotal data will be available. Very few biotech companies get any credit for their "platform" before they have proof-of-concept clinical data - i.e. data from real patients in a disease that has real market potential (Ebola and Marburg do not) - besides DMD, SRPT does not have anything in the clinic, yet.
You've proven over and over that you do not have an "objective" mind - you have a paranoid, conspiratorial mind that dredges up issues that have no merit - give it a rest!
Marathon's drug is just another steroid, designed to help minimize the damage the immune system causes on the deteriorating muscle. By definition, you can't "cure" DMD without creating dystrophin. Etep allows these boys to create dystrophin - whether it will allow enough dystrophin to functionally cure them is yet to be determined.
Well stated, speedbll. The enrollment criteria that SRPT used in its Phase II were designed to target patients that were on the verge of losing ambulation. The RNA Phase II trial obviously had less stringent enrollment criteria and presents a wide range of functional status when treatment started. The fact that a number of boys showed 6MWT improvement in the short term (before dsytrophin could have been produced) speaks volumes - these boys were not at the same progression level as SRPT's boys. If you want to characterize SRPT's Phase II 6MWT data as "noisy", then how do you characterize RNA's?
The fact that the FDA asked for MRI data provides hope that they at least might understand the issue at hand and be trying to better classify the functional status of the SRPT boys at enrollment. Either way, any rationalization of the variability in 6MWT scores is going to be more speculation than science.
A year ago I thought we could write off the FDA's questioning of the dystrophin data and measurement methodologies, since the 6MWT data looked like it would carry the load - now we need the dystrophin results to be solid and accepted by the FDA. From there, the 6MWT and pulmonary data are sufficient to confirm that a clinical benefit is reasonably likely.
It's not a trade show - I don't think drug companies set up booths at investor conferences. It is by invitation only and I'm quite sure everyone that is invited is expected to give a presentation.
FOLD will be bought out in the next year - the current ERT players have to be looking at them and know they won't have any revenues in a few years. Spend $1B+ now or $3B+ later!
He clearly stated the partnering strategy today - they are going to pursue 566 in the US on their own and only partner it outside the US, while they plan on partnering 189 in the US, but only after "turning over the card", by which he meant showing the results from the Phase II trial. While I agree with that strategy (assuming they can replicate the Phase 1b data), he indicated that the Phase II trial won't be completed for 18-24 months.
GSA - Given the revenue implications of your point #5, don't you think the 40% overall revenue growth is weak? I haven't gone through the numbers in detail, but I would think converting the LifeCell business (where they were splitting revenues) to direct sales would have added 15%-20% to the 2014 numbers all by itself? Beyond that, I can't imagine the 40% includes much of anything from the "18 countries". Last year we were thinking the 40% revenue growth was a slam dunk and didn't make it, which is why I pray he is being conservative in targeting 40% as his "baseline". With all the irons in the fire, the conversion of the LifeCell business and the fact that they missed 2014 (so are growing from a smaller base), I would hope that 60% is the "median" target for 2015?