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?
kart - a couple of thoughts after listening to their presentation. The HCT trial for ASP0113 has a survival endpoint with an initial read on 100 patients in early 2015 - I'm not sure whether the vaccine will necessarily beat anti-viral therapy in a survival test, so it seems like a more risky trial? With respect to the HSV-2 program, I previously thought that VICL had the lead in this space, but AGEN is already in the middle of their Phase II trial. The other issue is that as much as the CEO touts the value of their adjuvant, I would be wary of any program that is tied to this. DVAX just got burned in their HepB vaccine program, where they had stellar results in what I thought were large enough trials - close to 5,000 patients were dosed over the years - but the FDA's panel was not comfortable with the safety of the adjuvant and wants them to do larger studies. The VICL CEO has been pumping the potential value of their adjuvant for as long as I can remember, but they have only a couple of partnerships in the preclinical stage - compare that with AGEN, who has its own adjuvant that is partnered in some huge clinical trials with Phase III readouts coming over the next year. I don't have a position in AGEN - potential dilution coming? - but it seems like a better bet than VICL over the next couple of years?
I have a small position in VICL and haven't dug into the details on whether it makes sense to add at this stage. Glancing through their latest presentation, I don't see any major milestones coming for at least another year, so I will probably let it sit until the middle of next year?
What an #$%$ - these kids have "given" plenty - 2 or 3 biopsies a piece,weekly infusions, countless blood tests, poked and prodded for two years with all the time away from their families. The trial design was approved by the FDA and the boys have met the trial requirements. Even IF another biopsy would provide some critical information, it would still be wrong for the FDA to request it. The fact that it is not likely to provide any meaningful data than already exists makes it unethical. Maybe if you had fallen down the stairs it might have knocked some sense into you!
The point isn't whether how difficult the biopsy will be for the boys or they don't have the courage to deal with it - the point is what will be derived from the test and what impact will it have. I think we can all throw out the possibility that a biopsy will show little to no dystrophin. So, if the biopsies show that the levels of dystrophin have declined marginally or increased marginally from the last readings, how will that change anything? It won't - the 6MWT data at this stage trumps anything that a single dysrophin reading is going to provide. Is there a need for future studies to understand whether dystrophin production is influenced by dosage, age that treatment is started, length of treatment, etc. Of course, but none of those answers are going to come from these boys - they've paid their dues - leave them alone.
IMO the 4th biopsy request is a travesty. The FDA will have plenty of data points on dystrophin from the confirmatory trial - they do not need to put these boys through another round of surgery. What do they expect to find - the idea that the boys could be performing as well as they are clinically without dystrophin is simply absurd and does it really matter whether a boy shows 30% or 50% dystrophin levels at this stage? As discussed on this board previously, the boy's 6MWT performance is contingent on the level of deterioration in their leg muscles - getting a biopsy from the arm may not be indicative of what is going on in their leg muscles, so what is the point?
grey - the one piece of "news" that I was waiting on, somebody posted earlier has already been leaked by CG - i.e. that the NDA filing would take place in Q1, rather than the "first half" guidance that CG has consistently spoken to. I'm not sure how he could speak to a Q1 filing without first having the CMC meeting with the FDA and know specifically what stability testing will be required for the filing? In any event, I don't expect CG to do anything but follow the "conservative enthusiasm" that he has worked with for the past two years. Beyond the clinical/approval nuances of Etep, we have clinical guidelines on future exons (which I don't think the FDA will take a stance on until the Etep confirmatory trial data is out), the patent resolution in Europe (which may be resolved by a marketing agreement w GSK - SRPT obviously doesn't need anyone to "sell" Etep, but distribution logistics in Europe will probably require a partner?), and news on other disease/pipeline programs (which given the performance of the PMO platform in DMD, will probably only require some exciting pre-clinical data to drive value). It is hard to imagine that by this time next year we aren't at least at $100 per share, and unless the govt trashes everything or the biotech bubble bursts, with any "news" we could be double that number?
Just curious as to how do you know she has not "uttered a single word" and why do you care? It's not as if her word carries any weight with the FDA - why would it?
Yes - I responded to his trash article. He was claiming that his experts in the eye surgery field were saying that there was no need/market for OS302 - that existing generic compunds handled the mydriasis issue just fine. When I challenged him with the idea of why would a company waste millions of dollars pushing a drug through the clinic that there was no need/demand for, he spouted off about that's what many small biotechs are doing - lying to the world about the potential market for their drugs in the pipeline in order to keep the share price aloft so they can continue to raise money. No doubt there is some truth to that, but when you have half a dozen legit analysts with target prices in the $15+ range and the stock was trading below $5, something add up?
Your "in depth" research is not even close - what were you reading? First off, they only have $133M in cash as of their last quarterly report (6/30). Second, Sop was not put on hold because of "high liver enzymes" - it was put on hold because there was an interaction with a commonly prescribed HIV drug in a DDI trial that caused the blood levels of both drugs to rise astronomically and they haven't figured out why it happened, yet. Yes, there is hope for Sop if they can figure out and control the interaction with the HIV drug (or possibly even label it as not to be prescribed together), but the larger issue is whether the SVR results due out later this year will be strong enough to continue moving ahead with Sop. If not, they may scrap it for their next generation drug (ACH2684), which will add another year+ to the clinical timeline, while competitive drugs are moving closer to the market. There may be value here in the long run, but until the hold is lifted and the efficacy profile (i.e. SVR) of Sop is clarified, expect this stock to flounder.
Why would individual data be a "bombshell" - they've provided that data in recent investor presentations already?