Not less bullish - just resigned that it will take patience to realize the payoff. I hope 2014 will bring additional news on TH-302's value in a variety of cancers, but there won't be any pivotal Phase III data until probably 2015 - there will be an interim analysis in STS in the middle of 2014, but assuming that data isn't blockbuster, we'll have to wait until 2015 for final STS or pancreatic results..
smith - I'm not sure what to make of the data. The hope is that like any cancer with significant hypoxia, TH-302 can play a role, but the treatment regimes in these blood cancers are so complex, it is hard to see at this stage what that role will be. The press release didn't seem to provide any sense on what direction they will take clinically in this space. If the theory is that the hypoxic tumor regions are the source of relapse/refractory/metastatic disease, then you would like to see TH-302 make its way into some kind of front-line role, but that seems a long way off from where they are now? I suppose the key is that they just keep staying active in the clinic in blood cancers - my premise for the ultimate value of TH-302 was that by the time they received approval in pancreatic or STS, there would be numerous Phase II or investigator sponsored trials validating TH-302 in other indications, leading to significant off-label use, so maybe they'll find that niche in blood cancers over the next couple of years?
Between those that bought the first week of trading (when it opened at ~$27), who are looking to recoup and profit-taking from those who bought below $15, there are a bunch of eager sellers. The partnership w Celgerne will put a bottom here around $25, but it may take awhile to drift back up into the $30's.
Is between the short-term players that were playing for a spike, didn't get it, and are currently bailing out vs. investors who recognize that Sapa is potentially a drug worth $1B and the company has an enterprise value of $50M - that imbalance won't last forever.
As long as they have the data on all 21 patients, they know well before the 21st patient has died what the median is - once the patient living the 11th longest has died, the remaining 10 could live forever and that wouldn't change the median. That is one of the issues with median survival analyses for some of these new "personalized" cancer treatments - if only 1/3rd of the patients have a certain marker or sub-type, they need to be able to weed the rest out of the enrollment or it may not impact the median. If 1/3rd of the patients live 5-times as long as the rest, but 2/3rds get no benefit from the drug, the median may be the same as SOC. I'm not saying that is what is happening here - the median is clearly better than SOC, but it doesn't matter how well the top half of the survivors do.
Unfortunately, that is probably not the case - with median survival numbers, it doesn't matter how long the best patients do - only the patient that lives the mid-point in length, so for this trial with 21 patients in each arm, it is the patient that lives the 11th longest. If that patient has already been determined, it does not matter how long the 12th-21st longest patients live.
I agree that the mets, panc and ovarian data could provide a bump, but the dilution issue will keep a cap on this stock. One of the key comments coming out of the PJ conference was his statements on partnering - i.e. they never expected to go it alone with this drug and that partnering is on the agenda in 2014, pending the mets, panc, ovarian and other randomized trials.
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?