I agree with most of your points, but you can't be serious that there are "chinese walls" in investment banking offices. While the price targets aren't meant to be what the stock is currently worth, the underwriters in the investment banking side are there typically to line the pockets of their customers with what they believe to be cheap shares. Most companies, especially development-stage, small cap biotechs, don't have the leverage to demand a higher share price than the underwriters want to provide to their clients. I guess I am arguing that the idea of a Chinese Wall is irrelevant - they obviously want the analyst side to have a healthy target above what they are selling the shares at, but beyond that, they are working for their buyers, while pretending to be working for the company floating the shares.
Thanks - just so I understand, the key point here is that they are not comparing median survival to another 2nd line treatment (that presumably, on average, would start about the same time after front-line treatment as SAP is under this trial), and the BSC stats literally start as soon as they fail on front-line treatment?
L2L - you have mentioned the treatment gap as a potential driver from the ASH presentation. Wouldn't the treatment gap issue be common to most 2nd line studies and are the gaps typically measured and reported? I don't recall seeing this measurement reported in any of the other companies/studies I have followed?
alan - I've had you on ignore for as long as I can remember and now I know why - you babble a bunch of gibberish, which you can't bother to explain, and reiterate pointless comments about TH-302 being a "pro-drug". I've been an investor in THLD since 2009 because I know TH-302 "works" and I understood that it had the potential to treat numerous cancer types. The fact that it works doesn't make a single drug a "deep pipeline" or does it make the pancreatic cancer market opportunity worth $14B a year - both clueless comments only an idiot would try to back up. Back on ignore.
alan - why so defensive - perhaps you are the clueless idiot who wrote the article? THLD has a single drug with potentially broad application in many cancer indications - if that single drug fails to show clinical benefit, there is nothing behind it - that is not a "deep pipeline". Saying it is a deep pipeline is the same as saying you have a deep understanding of TH-302, which is obvious that you don't.
grey - the other aspect of this idea that the FDA using Drisa data to argue against Etep is you have to question how the FDA would have gotten access to any detailed data from GSK. The comments by CG and RNA were that the FDA does not have data beyond what has been reported publicly, which makes sense - why would they otherwise? What purpose would GSK have submitting data to the FDA from the Phase III Drisa trial - they supposedly aren't done analyzing the data themselves and have no reason to give it to the FDA - there is no pending filing or future trial issues to hammer out. Bottom line - I don't think the FDA has any detailed data, but for some reason they concluded the overall failure of the trial is meaningful in assessing Etep's efficacy, which it clearly doesn't, except for someone as brainless as pasteur.
pasteur - you have cornered the market on feeble arguments. It has been confirmed by both SRPT's conversation with the FDA and RNA's disclosure that the FDA does not have the detailed data from the phase III Drisa trial. Quit polluting this board with your false premises and twisted logic.
He knows the FDA will get enough venom from the parents - better at this stage to play nice with the FDA and educate them on the errors in their analysis. One of the fascinating aspects of the call was his breakdown of the Exon 51 population in the US and how difficult it would be to enroll a trial - there are barely enough patients to do the trial that SRPT proposed and the FDA wants to double the size and include a placebo arm - idiots. The rational thing to do at this stage is grant AA and do a 3-year confirmatory trial with no placebo arm. The trial would start 9-12 months prior to the drug being available on the market, which is enough time to incentivize parents to get their boys into the trial - waiting a year can be the difference in maintaining ambulation. If the FDA can't accept a natural history comparison over 3 years in 7+ year old DMD boys, they are clueless.
The twins were in the placebo-delayed group, so did not begin to receive Etep until week 25. Based on the 12wk/24wk dystrophin measures in the treated groups, meaningful levels of dystrophin were not present at week 12 but were at week 24. Therefore, it can be assumed that the twins did not begin to produce meaningful levels of dystrophin until after week 40+, but by that time they had already lost ambulation.
Yes - it was good to hear that CG is looking toward the 120 week data as a potential pivot point with the FDA. Between the political pressure that the parent groups will be hammering the FDA/Congress with over the next few months and the additional analysis of Drisa and natural history data that will leave the FDA's current AA position shaky at best, solid 120 week data could be the nail in their coffin of ignorance and force them to flip-flop on their AA stance.
Focus on on the issues the FDA brought up and why the data does not support their position.
BMO raised to $43 (from $32) and Leerink raised to $37 (from $27). So, with a consensus target that will probably settle in the low $40's, with $300M+ in the bank (and more milestone money coming within the next 12 months) and Celgene (among others) backing/validating the technology/pipeline, there is no reason this stock can't trade in the $35+ range.
wggm - Your "recollection" is that they are now "13 to 15 years old and still going strong". Where is the data on this - where has the individual age, dystrophin, and 6MWT change data from this Drisa trial been disclosed? Have you seen the individual data or are you going by a statement by the company?
I am long and have been a fan of THLD for years, but the guy who wrote this article is clueless. He lists the "deep pipeline with the following candidates", as if they are different drugs - they are just different trials with the same drug - TH-302. He then goes on to estimate the market activity for pancreatic cancer at $14B per year - what an idiot.
,,, in yesterday's presentation. CG stated that a number of Drisa patients (treated and placebo) lost ambulation over the 48 week Phase III trial. They have not yet provided details on these patients, but if you assume all of them were in the 7+ year age group, which is likely, then 20% of this 7+ patient population lost ambulation over 48 weeks. Beyond that, he reiterated the earlier disclosed fact that the Drisa 7+ placebo patient population lost 83 meters over 48 weeks - based on enrollment criteria, this is probably a 20%-25% decline from baseline. So, given these two facts, combined with all the other natural history data that McDonald has disclosed, how does the FDA conclude that SRPT's enrolled patient population of 7+ aged boys should be stable over 96 weeks?
helm - I agree that CG is a little "scared" of the FDA, but up until now I don't see that as a problem. As he has spoken to in the past, the FDA has been very responsive and given SRPT significant and timely access - 3 meetings in Nov-Dec. I think he is correct to continue to talk up the positives about the FDA's response to date, although if they don't change their tune about AA and/or cram down some ridiculous confirmatory trial requirements, then I think he needs to take the gloves off and join with the Jett/Abigail letter approach and call these people out for their ignorance in interpreting the data and their inconsistency in applying the AA regulations. With the parent/advocacy groups applying the political pressure, I think it is appropriate to appease the FDA up to a point, but that point is coming soon.
The dimwit asking the questions just wasted valuable time asking about where overseas the company can run trials and whether $280M of cash is enough to complete the trial, etc. How about asking what data the FDA claims to have in its possession from the Drisa Phase III trial, whether the SRPT also has access to the same data, and where the company sees issues with the FDA's interpretation of either the Drisa data or the natural history data and how it relates to SRPT's trial results. Since the company did not have the minutes from the meetings, it was clear CG was not able to provide any substantive insight into the trial design or how long it will take to run, so lets waste the 30 minutes speculating around the edges of what those issues are - a complete waste of time.
smith - I think it has to do with the dosing schedule. I looked up a couple of mono trials that seemed to hit MTD in the 450-540 range, but those had a dosing schedule of TH-302 for the first 5 days of a 21 day cycle. In the glio trial, they are only getting a single dose of TH-302 every 2 weeks. No doubt that in most of the combo trials, the MTD is driven more by the side effects of the combined chemo agent, rather than any side effects from TH-302, so it wouldn't surprise me if they can push the MTD on this trial up even further than the 670 if it is just one dose every 2 weeks.
The abstract is already available, so is there any additional data coming at the Sunday presentation? Data looks good - median OS in the best arm of 9.7 months is about double that of historical norm and no major side effects. I assume this data has been driving the recent price increase?
No - the 100-150 per quarter is their running placement number for equipment (including Firefly, Pinpoint and Luna). They haven't been disclosing kit sale numbers, although on the latest conference call they mentioned they did ~5,900 kits in the most recent quarter. The revenue per kit number is difficult to nail down, because there are different price points for the different indications and whether the kit is being used on a leased vs owned machine. They did mention on the latest earnings call that hospitals are moving more and more toward purchasing the machines, which reduces the cost of the kits - the "lease" structure is set up as a payment per use built into the kit price.