How do you know whether the 31.8% is an important or meaningful number? That is what is so frustrating about this company's press releases - they use obscure stats that other cancer biotech companies do not and then provide no context about the clinical relevance. The traditional stat used is median PFS - with 32% over 6 months, it is clearly less than 6 months, but is it 5 months or 3 months. Also, what is the patient population here - is this a front-line setting or are these patients that have already failed front-line treatment? On the surface, these stats look impressive (90%+ tumor shrinkage), but with no control arm or even a historical comparison, how can you make any kind of judgement?
But you cannot also do an accelerated approval with a required confirmatory trial, without starting the confirmatory trial first - they would never be able to recruit patients. This is one reason CG is pushing ahead to start the confirmatory trial in Q1. Beyond the Etep approval, this may be the best chance to recruit a large number of patients to generate a solid dystrophin database, which will help the cause with future Exons.
I would think a triplet trial would be one of those combo 2a/2b trials, where they test a few cohorts to get to a MTD and then expand it from there. I haven't been able to get a handle on the antiangiogenic side of it - the preclinical data they presented in earlier investor presentations seem to show a 1+1=3 result in terms of tumor response, but the data they just presented seems a little underwhelming in terms of response rates. Then again, they seem to be getting responses from patients that have already failed monotherapy with the antiangiogenic, so maybe it is better than it appears?
beachbum - the details were in the abstract that you can get if you go to the AACR conference website that they referenced in the Oct 7 press release. I'm not sure why they downplayed it in today's press release - they simply state that the "triplet" showed "greater anti-tumor activity" than Gem+Abraxane - you would think a 100% complete response rate would be trumpeted a little more? I don't have a guess on what their next targeted indication may be - I assume it will be in one of the larger markets, maybe NSCLC - didn't they have some earlier clinical work in that indication that Merck took over?
You seem to be missing the obvious point - the reason it is unethical to have a placebo arm in the confirmatory trial is that the evidence from the Phase II trial is overwhelming that Etep works and that delaying treatment by even 6 months can be the difference between a boy losing ambulation for life. The CEO has hinted on more than one occasion that the purpose of the confirmatory trial is more to provide additional dystrophin data to support future Exons and potential class approval, rather than proving that Etep works - that has already been accomplished. Where do you come up with the company "stirring up the parents" - to the contrary, all the communications/interactions with the parents/DMD community have been very reserved - the parents are "stirred up" because they know that Etep works and they want to get it approved as quickly as possible.
I have stated this before and I will state it again, the most important statement that has come from the FDA was at a presentation to the PPMD about a year ago regarding the accelerated approval process, where the FDA official stated that they have significant flexibility in applying approval standards in the realm of orphan/rare/fatal diseases and when they are faced with clinical data that is limited to a small population, they exercise that flexibility by relying more and more on "patient reported outcomes" - i.e. how has the drug impacted the day-to-day lives of the patients. You can obsess all you want about the limitations of the clinical trial design/data, but if you think all the communications the parents have had with the FDA about how Etep has changed their boys lives will be meaningless to the approval process, you are clueless.
Where do you come up with this garbage. The "Quantum" study is testimony to the flexibility the FDA has and has exercised in approving Orphan Drugs - many with trial design and execution issues much greater than SRPT's Phase II trial. You seem obsessed with this single site issue, as if the site would have an influence on dsytrophin production? All this hand-wringing over a potential placebo effect in a 6MWT is just laughable - as if these boys have the ability to overcome the loss of muscle function - it might be an issue in a 12-24 week trial, but with 48-96 week results it is absurd to even talk about. You've wasted a lot of message board space raising issues that you have created in your own mind, as if nobody at the FDA has even considered these "issues" to date, but will suddenly take prominence when it comes to reviewing the NDA. Your postings smell of desperation brought upon by a bad short position - too bad for you, as it will only get worse for you.
The more interesting presentation is the pre-clinical data of TH-302 in pancreatic cancer when combined with Gem & Abraxane. Abraxane was just approved after a Phase-III trial showing a 1.8 month improvement in OS when combined with Gem vs Gem alone (OS improved from 6.7 months to 8.5 months). In the xenograft/mouse studies, Gem+Abraxane showed a complete response rate of 50%, while the Gem+Abraxane+TH-302 generated a 100% complete rate. Obviously, these xenograft study results don't translate directly to the clinic (otherwise the 50% of the patients in the Abraxane+Gem Phase III trial would have had complete responses), but if TH-302 can double the OS impact, it would still be meaningful. I assume there will be a Phase II study of TH-302+Gem+Abraxane done over the next year, so that by the time the Gem+TH-302 Phase III study is done, they will have a clinical dosing regime in place allowing doctors to add Abraxane "off label", so they don't have to choose which combo to use.
What a ridiculous argument - for every biotech stock that has traded at lofty heights only to fail later, there are numerous stocks that have been dismissed/ignored by Wall St only to turn into multi-billion $ companies. Assessing a company's potential by it's current stock price or market cap has to be the most superficial means possible. Do you have any insights into the pipeline/science or the particular prospects of Sapacitabine or just meaningless observations about unrelated companies that have crashed and burned?
Actually, if GALT can avoid major dilution (perhaps by partnering another indication, like lung fibrosis), then $30 is just a start. Fatty liver alone would be a $1B+ revenue opportunity, so the company's value would be some multiple of that. As we sit right now, the fully diluted market cap is about $370M (17M common shares outstanding plus another 13M of warrants/options/convertible preferred), so $30 per share is roughly a $1B market cap. If we get proof of concept in fatty liver before the next capital raise, then look out above!
copp - your suggestion was for SRPT to recruit the Prosensa boys as part of the confrimatory trial - I also believe you advocated SRPT taking over clinical sites, etc. That is not what is happening here - CG is throwing a bone to the Prosensa families by potentially establishing an additional "cohort" that is separate and apart from the 60 boys that they will recruit to confirm the efficacy of Etep. I stand by my statement - the inclusion of Prosensa patients as part of the confirmatory trial makes no sense - the confirmatory trial patient population needs to be as targeted as the Phase II trial - i.e. patients that are in that "narrow window" that would put them on the verge of losing ambulation, so that if they stabilize on Etep it proves efficacy. Whether any of the Prosensa boys fall into this category is unclear, but to make the blanket assumption that they should be included in the confirmatory trial is sensless on many levels - besides the enrollment criteria, you would not want the efficacy of Etep "tainted" by any questions of lingering impact of Prosensa, let alone any potential safety conflicts. The fact that CG is doing a charity cohort for the Prosensa boys is meaningless to the design and execution of the confirmatory trial. Get over yourself.
I understand why he said what he said, but in reality, it is a non-issue when you consider the timing. Assuming treatment for the Phase III trial starts in Q1 and that any placebo arm would be at worse the same placebo-delayed design as the first trial, then the latest a placebo-delayed boy would have access to the drug would be the end of Q3. Approval for Etep is likely to be around the same time or later, so I can't imagine that a parent would put their son in the trial, then after finding they were on placebo, pull them out with the risk of Etep not getting approved and losing access to the drug through the trial? That being said, I think forcing a placebo arm into this trial would be highly unethical, as it is clear that once these boys lose ambulation, they are not going to get it back.
You should pay attention to your own posts - you say that they "don't become un-ambulatory at a narrow window" and then follow up with "two boys in the treated group became un-ambulatory". The two boys that lost ambulation did so within 6 months of entering the trial, after meeting the same enrollement criteria as the rest of the boys in the study. The study was designed to enroll boys that were on the verge of losing ambulation - i.e. in a "narrow window". Based on the other posts I have read of yours, you have no knowledge of the disease, trial design or other pertinent issues. Combine that with the fact that you refute yourself in your own posts makes it unlikely that you are going to post anything intelligent - give it a rest and move on.
5 months of additional stabilization/safety is nice, but by the time the FDA will be making a decision on whether to approve Etep, there will be an additional year of data/results. Assuming all 10 boys are ambulatory and stable after 3 years on the treatment, there is absolutely no way anyone can dismiss the efficacy of Etep. Frankly, I can't believe anyone is clueless enough to question the efficacy at 96 weeks?
So, your key valuation metric is a single revenue estimate from Yahoo? To start with, your "valuation" of SVNT is flawed because you don't account for their debt - they have $230M of debt - hard to imagine how a company with one product for gout floated that much debt? Second, the revenue estimate for SRPT for 2014 is an average of analysts, some assuming they get approval in late 2014, and some not. The question isn't what 2014 revenue will be, but what revenues will be for 2015 and beyond. There is a reasonable chance that SRPT will be generating $1B of annual revenues 3-5 years down the road - I doubt that there is much chance SVNT will be generating $100M of revenues in that time frame.
The lung cancer indication is not the lead indication - they have a Phase III trial underway in sarcoma, which is also a combination trial. You are correct that going forward most new cancer drugs are going to be add-ons/used in combination, so these results are not surprising. While Vintafolide is the value driver over the next couple years, their drugs using tubulysin will be the big driver over the 3-5 year timeframe - it is 100-times more powerful than Vintafolide.
simp - how did you get access to the call - is there a link to a replay out there somewhere?
starf - I don't need to see the Favus report to know what the truth is. The two boys were not "dropped" from the trial - they lost ambulation prior to Etep having an effect. To say that dystrophin is "non-functional" is akin to saying that if you give it to a patient already in a wheelchair and they don't get up and walk, then the drug doesn't work. The fact that there are only 12 patients in the trial has been known and addressed. There is nothing new here - same old short BS. To suggest that an analyst report that is clearly a hack job will impact the FDA's approval decision or the company's clinical timeline is ridiculous. Whether the report affected the stock price, who knows and who cares, except the short-term momentum players. I have been invested since early 2012 and don't plan on selling until Etep is approved, if then.
Why would misrepresentations effect approval, timeline, etc. You suggesting it would indicate your brain is non-functional.
The fact that Pfizer is selling out, especially before the Phase II readout, sends the message that they don't believe in the drug - whether that is true or not. If LPTN is successful in getting it back, it will force them to raise money sooner (hopefully not until after the Phase II readout) diluting the shares. The best thing that could happen in the short run is a quick sale by Pfizer to a major player who believes in the drug, but in the long run if the drug is worth what management is alluding to, it would be better if the LPTN gets it back?
This could go either way - with topline data pending from their STS trial (due in Q4), either they smelled bad results and pushed an offering to avoid worse dilution later or the results are good and wanted to give cheap shares to crony brokers/institutions. If they are trying to push cheap shares to insiders and existing institutional shareholders, they would typically do a PIPE, but since this seems like a general offering through some second-tier brokers, I'm afraid the pending data is going to be bad?