The 8K filed on 12/17 indicated that ~3,740,0000 shares were issued on exercise and the current share count is ~8,885,000. I assume all of those new 3.7M shares are registered and available to be sold - I'm not sure why you would think that would be bullish - I assume those that choose to exercise are looking to cash out quickly - why exchange a preferred share earning a stated dividend for a common share, unless you are looking to sell it?
Yeah - "medical tourism" will support orphan drug pricing of $300K+ per year - think again. The only reason the drugs for these rare diseases have a chance at development is because of the insurance systems we have in this country for supporting the ridiculous prices it takes to justify the development cost - that price support system requires FDA approval. Most DMD families, already burdened by unimaginable medical costs of having a DMD boy, don't have the money to cover an overseas trip, let alone the targeted price of one dose of Etep. Beyond that, this drug requires weekly infusions - what are they supposed to do - move overseas?
I don't get it - what is the point - who cares about your definition of a startup? The funniest thing is you advise people who doubt you to investigate on-line and they'll "probably" find a similar definition - does this mean you didn't bother to investigate yourself, instead you took the time to post your definition? Who would care enough to doubt you?
On what basis do you have the ability to assess the "professionals" at the FDA? While it is for the most part a black box when it comes to how they have come to decisions, we have a number of facts that have come to light showing them to be less than professional in their decision to curtail the AA path for Etep. Look at the excerpts from the meeting minutes that were quoted in SRPT's 11/12 press release - they cite the Drisa and PTC studies as evidence that increased dystrophin does not lead to clinical benefit. They later admit that they did not have detailed data on dystrophin production or 6MWT results from that trial when they sent that letter and the the PTC study does not relate to Exon 51 patients or an exon-skipping therapy, so how is that relevant? They cite "recent natural history data" that shows patients included in SRPT's trial are expected to show stability in 6MWT over 96 weeks, while a recognized expert in the field (McDonald) is saying the exact opposite and while the placebo patients in the Drisa trial (who were enrolled with criteria similar to the Etep trial) showed significant deterioration in 6MWT over 48 weeks. These are facts and your assessment is these are "professionals" to be respected?
another "commentator" that doesn't understand the situation. The anger coming from parents, investors and I assume SRPT management is due to the fact that there are clear rules in place for the "disciplined gatekeeper" to allow conditional approval of drugs in cases of rare, deadly diseases. By any rational account, SRPT's Phase II Etep data has provided the necessary proof to allow that conditional approval. Up until two months ago, the FDA agreed with that preliminary assessment, but then changed their stance based on irrelevant or misconstrued data coming from trials/research that had no bearing on the effectiveness of Etep. Patience, as an investor, is one thing, but asking parents of all DMD boys to be patient while the FDA comes to its senses is another.
starf - I had you on ignore, but curious as to why you would reply 3 times to a simple question - each answer showed you to be progressively a bigger #$%$ with each response - now I remember why I put you on ignore. Thanks!
Finally got around to listening to the conference call. While they mentioned the US launch of their FHACT test for cervical cancer, it was not until an analyst posed the question that they spoke to the size of the potential market. They indicated that there are potentially 500,000 tests to be covered by their direct marketing efforts with a sale price in the $400-$500 range and another 2 million potential tests to be sold through partners at $150 per test - the CEO stated that the revenue can get into the "hundreds of millions fairly quickly" as they focus on this market over the next several years. Combine that with targeted gross margins in the 60%+ range and a current enterprise value of less than $100M and you have one very undervalued stock. If they hit $100M in revenue over the next 3-5 years, you have a $50+ share price to look forward to.
Simple - the accelerated approval regs require only that a clinical benefit be "reasonably likely" - anyone that looks at the 2-year data from Etep and concludes that a clinical benefit is not reasonably likely is just plain clueless. The whole purpose of the AA regs is to provide the FDA the flexibility to look at all available data in determining whether patients with rare, deadly diseases can be helped. There is plenty of data - clinical and anecdotal - to conclude that Etep works, but instead the FDA looks at a failed Drisa trial and irrelevant natural history data to conclude otherwise? They follow the same, stunted logic that you do and end up looking just as stupid.
I've never seen someone so obsessed with useless information/assessments before - pasteur believes the trial was originally designed to be 24 weeks, so any data that comes after that doesn't count; the primary endpoint was dystrophin, so 6MWT results don't count, etc., etc. In pasteur's world, any data derived from the extension of an existing trial is meaningless - I suppose you are supposed to scrap the existing trial, stop treating the existing patients and start from scratch with new patients in order to test whether treatment over 48 weeks adds any benefit over 24 weeks. Then if that trial shows potential and you want to extend to 96 weeks, you scrap that patient population and start anew - 7 years after starting with the original trial, you have safety and efficacy data over 2 years. Good plan - good use of company resources and more boys deteriorating and dying. I'd say you are #$%$, but that would be an insult to the mentally challenged
Assuming you have pre-determined the number of "events" (n) and have a reasonable estimate of what the control arm number will be, then the differential is driven by those numbers. IOW, given that they decided to do an interim analysis based on 67 events and they new that the median survival for the control arm was going to come in around historical averages (I think that is ~18 months), then in order to generate a statistically significant resutl (p
jrrt - your point about "looking to the individual" for evidence of efficacy, so that the drug may provide benefits to the group is spot-on. There is too much noise in group data, precisely because no boy will be exactly the same at enrollment in terms of disease progression and there are not enough boys to enroll in order to eliminate this noise. In an earlier presentation/discussion with PPMD, the FDA (I think it was Temple) discussed how they supposedly used "patient reported outcomes" in small trials of rare diseases to provide evidence of efficacy - I heard nothing about that it in the recent PPMD conference - it was all about technical/clinical/statistical issues. Instead of having a 2 hour discussion about the issues surrounding dystrophin measurement, how about having a 2 hour discussion about how Etep has changed the lives of the boys in the trial. Isn't as simple of having the parents and doctors of these boys providing testimony about how their boys have stabilized or improved while on Etep? I'm guessing at least half of the doctors would testify that they have never seen a DMD patient over age 7 stabilize or improve over a 2-year period like the boys in the trial have? You would think that type of "evidence" would trump any noise surrounding clinical measurements and natural history?
EDIT - I am already in 2014 mode - I meant hopefully they will "comfortably make it into 2015" and get to breakeven by "early/mid 2015".
As of 9/30 they actually had $11M in cash and they have been burning about $3M per quarter, so hopefully with the increased pricing on their test, they will comfortably make it into 2014. When I said bad financial management, I wasn't referring to their operating budget/cash controls, I was referring to the fact that they didn't raise enough money when they had the chance, so now they have to cut investment/research and may not have the funds to aggressively expand the marketing program. Their IPO was in Feb, 2011 , where they raised ~$40M at $7.00 per share. I'm sure at that time, they had an operating plan that showed that was enough to get to break even - either way, they should have doubled the raise to make sure, because things don't always go as planned. Instead, they were forced to raise money at $2.00 per share back in Jan, 2013 and may have to go to the well, again at an even lower price? The only thing that can save them from another dilutive offering is if they develop enough momentum on the marketing side to get close to a breakeven by early/mid next year - then, they may have a chance of borrowing the money, instead?
Not much brewing here - why is the market ignoring this company? I tried to listen to the Investor Day presentation, but it cut off halfway through. The presentation was very technical with respect to the AML/MDS trials - the treatment regimes in this area are so complex and changing rapidly, so it is hard to get a read on how revolutionary their drug might be? I didn't get the sense their were any significant milestones in the next 6 months - might have to wait until ASH in December, 2014 - is that your read on it?
kart - I wouldn't say I'm positive about IMUC - I do think that there is a possibility that the OS results from the trial could improve and be statisitically significant when they get more "events" - why they would get a data read after only 67 patient deaths (out of 124 enrolled) is beyond me. The article today noting the listing/funding issues facing the company is spot on - if they don't get any better updated OS results over the next year, then it could be ugly. If you believe that the Phase I results (50% of patients living 5+ years) is no fluke and that the Phase II trial will show a "long tail" of survival in the KM curve, then it might be worth placing a small bet on it?
The primary reason is they are running out of money and will likely have to do a very dilutive offering. The current price doesn't reflect the future value of the technology - it reflects the bad financial management.
owtside - you are incorrect - read the CYTR press release announcing the results - they state it is "first line" therapy. However, that is one of the problems with the way the trial was conducted - the enrollment criteria allowed prior "adjuvant" chemotherapy ( i.e. used in conjunction with surgery), including three cycles of Dox. Dox has a lifetime dose cap of 6 cycles, so I'm not sure how they could enroll any patient that had prior Dox therapy into a controlled trial where they would potentially be targeted to receive 6 cycles of Dox, if randomized into the placebo group. Combine this pseudo-front line issue with the trial sites in eastern Europe and Russia and it starts to seem a little flaky - why would you need to go outside the US to enroll a 105 patient trial?
That's the fallacy in the assessment they gave to SRPT last month - i.e. that the "disease is variable", so the stability seen in the Etep boys can be written off as random chance. It isn't the disease that is variable, it is the patients that are in various stages of disease and trying to enroll a homogeneous patient population (one where every boy is at the same stage of progression) is almost impossible. SRPT tried to do it (via age and 6MWT distance) and got two boys that were within 6 months of losing ambulation. But that is why the Etep data is so compelling - the idea that they somehow were lucky and enrolled 10 Exon 51 boys over age 7 that wouldn't progress over two years is almost impossible, if you believe the natural history data. If McDonald's research/testimony and GSK's placebo group data don't convince the FDA of that, then they are hopeless. Beyond the lack of "clinical" progression, you have the parents' testimony that their boys are improving on Etep - improvement is never part of the "variability" of the disease.