simp - you badger me about 'WHAT YOU ACTUALLY KNOW" and then follow up with nothing but speculation. You have absolutely no knowledge about what the FDA is going to want to review before providing the company a formal communication about an AA filing. Whether they want to review the manufacturing plan/issues or not, they are still very unlikely to get a response before early Q3. Why you feel compelled to speculate that it will happen sooner is beyond me - does a 60-90 day difference in timing really matter to your valuation expectations - unless you have some options expiring, why would it? Regardless, nobody takes your ramblings seriously anyway, so if you think you are influencing the stock price in the near term with all your gibberish, think again.
Give it a rest, simp - they are not going to hear anything "ANY DAY" - it won't be until Q3 and most likely not until they get through the manufacturing meeting. I don't understand this need to beat the drum on some fantasy about the FDA surprising the company with some ultra-quick response?
Thanks for the update - make sure you keep us informed of other important aspects of your life - e.g. bowel movements, nose hair issues, etc. - we're all on the edge of our seats.
The point is whether they announce it or not, it is a non-event - it is not "news". "News" would be if they didn't submit it. In any event, the idea that the FDA will "quickly" get back to them on whether they should file an AA is absurd. The company already stated they will be requesting another meeting, hopefully before the end of Q2, and there is also the manufacturing meeting in Q3 to get by - odds are that the FDA is not going to give them guidance on an AA submission before they understand the company's status on the manufacturing front.
Thanks for the insight, and while it would be interesting to see the specific stats on the SD patients, ultimately the only stat that matters is whether OS is extended. Given that metastatic disease is what typically kills most cancer victims, you would hope that if Reo is delaying/stopping the progression of metastatic tumors, there should be a meaningful impact on OS. If that concept plays out in the H&N trial and these various Phase II trials show impressive PR/SD stats in late stage patients like this trial did, then we have a blockbuster.
Sorry - caught me at a bad moment - I just get frustrated when people post things that are clearly contradictory to what has been discussed by management. The pumpers on this board that hype the possibility of "approval" over the next few months are just as nauseating as the bashers who post ridiculous issues, comparisons, and lies about why Eteplirsen or the prospects for AA are a failure. There has never been a time where more evidence is at the fingertips of investors, but these boards get monopolized by mis-information and worthless banter.
Where do you come up with these crazy thoughts? "Accelerated" means that it gets approved based on Phase II data and before the confirmatory Phase III trial is completed, which won't be until 2015. Accelerated does not mean that the FDA throws out all the normal review policies/procedures/timelines. The CEO has been clear about the timeline for the past year - get FDA guidance in Q2 (now may be Q3 due to their request for summary on dystrophin as a surrogate) and file in Q3/Q4, so approval would not come until Q1/Q2 of 2014. It amazes me how many morons can listen to him lay out this timeline over and over and then post messages on this board that AA will come by July or Oct or Dec - what is wrong with you people?
"... preparing a profit and loss statement is quite easy if your books are in order"? If you know the history of the company, then you know the books aren't "in order". My recollection was that they acquired a number of different retail stores/outlets and had no control over the inventory - if you can't validate what your inventory is on a given date (i.e. end of each quarter), it is impossible to put together legitimate financial statements. I assumed that they would fix the inventory control system, validate the inventory with a physical count on a given year-end date and move forward, while negotiating with the SEC that inventory levels for prior quarters were subject to estimation. Given the total lack of communication over the past few years, it is difficult to speculate on what is going on, but I would be surprised if they resurrect their listing, let alone provide some basis for valuing the shares?
I used "single agent" when I meant to say non-randomized - I realize the panc trial is a combo trial. They say the first stage was targeted to be 17 patients, but since it met the goal at 6, the implication was that they wouldn't enroll the full 17. To add to the confusion, the Oct 2012 press release indicates the trial completed enrollment, but then instead of confirming the number that had been enrolled, they simply recapped the target of 33 (17 in the first stage and 16 in the second) and that the first stage met its goal after 6 patients. Does this mean the full enrollment was limited to 22 patients (6 + 16) and will all the patients be evaluated on PFS or only the 16 targeted in the second stage? I understand that the trial is not formally "linked" to the NCI randomized study, but why point to the intention of cross referencing the results with a trial that won't be completed for another couple of years, if the intention was to disclose the PFS data as it became available? It has to be one of the more confusing/least informative press releases I've read.
I think there is some confusion about the randomized PII trials (which are all investigator sponsored, so the company has no control over them or the data) and the earlier single agent trials. According to their pipeline timelines in the recent presentations, none of the randomized PII trials are complete - they all seem to be about halfway home. The single agent pancreatic trial that was referenced earlier completed the initial "stage" in Dec 2011, which was the 13 patients, with 8 achieving stable disease or better. The secondary stage, which was targeted to enroll an additional 16 patients completed enrollment in Oct 2012 - this stage was supposed to assess PFS and they indicate in the Oct 2012 press release that the pending results will be "cross referenced" with the results of the randomized trial. I've never seen this kind of disclosure before, so I'm not sure if that means no results from these patients will be disclosed until the randomized trial is complete? Given that the historical PFS for pancreatic cancer is less than 4 months, if enrollment was completed in Oct, they should have the data, already, although getting confirmed progression analyses can sometimes take a couple of months? Unfortunately, I think I've raised more questions than I answered - I may try to contact IR to get some answers.
The key issue is the funding and if they get FDA approval, whether that will allow them to secure some debt financing. Have they spoken about partnering in the US or do they plan on marketing themselves - I would guess building a marketing platform for the US would be extremely expensive upfront?
I have not seen one analyst comment or write-up on why this company is valued at $1B - they won't even have controlled study data until the middle of 2014. For a while, it was trading at twice the average analyst price target (per Yahoo) but that has seemed to caught up recently - it is a mystery?
It is as impressive as any of the clinical data. Brady is on the verge of turning 12, the time when he would normally be losing the ability to walk, and performing a test (getting to his feet from sitting on the floor) twice as quickly as he did when he was 6 years old. People getting caught up in the "n" or dose dependency or linear relationships between dystrophin and 6MWT just don't get it - the impact this drug has had on these boys is borderline miraculous, something that I'm sure is being impressed upon the FDA by the families and will carry as much weight as any of the clinical data.
Do you have a sense of what the annual revenue potential is 2-3 years out? The CEO spoke to a 40% operating margin (I assume pre-tax), so assuming a 25% net profit margin is achievable, is that on a $100M revenue base, or is it higher than that?
A lot of analysts asking inane questions, as if they are trying to uncover the undisclosed nugget that will blow up the AA path. Like the guy who asked how they were dealing with trying to establish the dose response relationship to dystrophin production - give me a break. As if the FDA is going to say, well the data supports meaningful dystrophin production in both dose cohorts, but since there is no linear relationship that can be established, you'll have to do another trial in order to establish a dose-dependent response, no matter how many patients or however long it takes?
$4 for the platform is way off. After it becomes clear that TH-302 will be standard of care with any anti-angiogenic, the platform will be worth $1B+ ($15+ per share). By the end of of 2014, this will be a $20+ stock.
I listened to one of the recent investor AVEO conferences and that one is a tough call. There seems to be a lot of merit to their arguments about comparable or better PFS, better safety and meaningless OS data due to the lack of 2nd line treatment in regions where they held the trials, but it sounds like the RCC indication is a crowded field, so there is no urgency for the FDA to approve a similar drug. The CFO made a comment at the end of the presentation about how if they got a bad ODAC decision they may have to go through a major restructuring - they certainly burn through a lot of cash? I like being able to pick up bargains after minor clinical setbacks, but I think AVEO is one you have to wait for the dust to settle on - it didn't sound like there was anything else in the pipeline beyond TIVO to drive any value before next year, so if they have to do another trial, that might require another capital raise?
kart - two of my more recent picks acquisitions include ONCY and FOLD. ONCY is more on the speculative side, with a single drug, but it is a fascinating one - a virus that causes no problems to healthy cells, but attacks cancer cells and in particular has an affinity for metastatic tumors.
They are waiting on survival data that should come in Q3. I don't normally ascribe a lot of value to enthusiasm displayed by execs in conference calls, but these guys seem genuine and were positively giddy about the early response (i.e. tumor shrinkage) results from the current trial. The virus is being tested on a wide range of cancers, so if it does work, the market potential is huge - worth putting some speculative money on.
FOLD recently took a beat-down due to some sketchy results from their current trial in Fabry disease, but IMO this is a clear case of a drug that works caught in a bad trial design. The issue is that the clinical response measurement requires an invasive kidney biopsy, and the enrollment criteria did not allow them to exclude patients with low baseline biopsy readings once they were enrolled - it was an issue the company discussed with the FDA in the trial design and knew upfront it would result in inconclusive data, because the measurement noise from biopsies on placebo patients with low baseline readings might put them in the "responder" category, which is exactly what happened. The analogy is testing a flu drug for its effect on reducing fever and including patients with a 0.5 degree fever - a 0.2 reduction from that low baseline could be due to a number of factors outside the drug's influence. In any event, this drug has a clinical history that includes the majority of patients from earlier trials staying on the drug through extension phases that have lasted in excess of 5 years. In the current pivotal trial of 60 patients, 57 of them have elected to continue on the drug into the extension phase - why would they do that if it wasn't working?
kart - I actually got out of ZIOP after reading an extensive write-up on issues with one of their earlier trials, so I minimized the damage there, but OMER has left a mark. While I have doubts about the market potential of OMER's surgery drugs, I thought they would generate enough value/cash flow to support the rest of the pipeline, which I think has huge potential. There is definitely some pressure on OMER from the threat of dilution - I thought they would partner OMS-302, instead of building their own marketing, which will be costly in the short-run and exacerbate the dilution. I will wait until they get the funding issue resolved and may double down. I am not familiar with the others, but will take a look over the next week - I need to diversify some of my gains on SRPT & NVDQ, which #$%$ me off with that latest share offering - they are on the verge of generating positive cash flow and have plenty of cash in the bank and they do an offering - only 3M shares, but #$%$?
What a moron - The Street is a quantitative analysis rating that shouldn't even be used on biotechs - they haven't "reviewed" anything about the company, its pipeline or market potential. The fact that you would spout off about this downgrade proves you haven't either.