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.
Stats actually come out twice a month now - the NASDAQ site has a history of short interest positions. It will be interesting to see how many of the 10M+ shares short as of the end of Oct close out - will they take the risk waiting until 2014 to defer any tax gains?
jalexander - at $2, the company is valued at $60M - I think in the long run it is worth more than that. If their galectin heart risk testing becomes part of the standard risk assessment for heart disease, it could be worth multiples more. I think there is a chance for that, since many doctors are beginning to question the value of cholesterol/lipids screening as a predictor of heart issues.
The trial recap on clinicaltrials.gov shows final data due at the end of next year, but the company has indicated preliminary data may come out soon. When challenged on the last call about the exact timing, they waffled, saying they are not in control of the trial and it is not up to them to decide. Note that the trial has a targeted enrollment of 70 patients (first line, recurrent or metastatic) and the primary endpoint is PFS.
I looked up Mayor Brennan's story - he died in January 2012. In July, 2011 there was a story about his cancer fight, including comments about how Reo had shrunk his tumors. His initial chemo treatments left him dysfunctional, but seemed to be on the road to "recovery" after starting treatment with Reo. As with most cancer treatments, Reo won't be a cure for most, but it seems to be a valuable part of the treatment options, at least with pancreatic cancer - maybe others?
I hope you are right, but I don't think even a "blowout" quarter can save them from having to raise more money - until the dilution overhang is resolved, the long-term value potential of this company's technology won't be reflected in the stock price. They are burning cash at about $3-$4M per quarter currently and only had $11M in cash at the end of Sep. Hopefully, given that they have an approved product and track record/plan for growth, they will be able to borrow the $15M-$20M they will need to get them to breakeven, but that may be too much to ask for. They only have ~30M fully diluted shares outstanding currently, so you would hate to see them have to float another 30M shares (100% dilution) just to get them to profitability.
This is a classic case of financial mis-management. Nobody can argue that the technology or company's progress to market acceptance/profitability isn't in a better position today than it was 3 years ago, when they did the IPO at $7 (sold 5.75M shares and raised ~$40M). At that time, I'm sure they had some kind of financial projection that showed they only needed $30M or so to get to breakeven - whatever the number was, they should have doubled it and raised twice as much money. Instead, they run out of money in 2 years and are forced to do an offering at $2 in Jan of 2013 (sold 6.9M shares and raised ~$12.8M). Now their stock price is less than 10% of the IPO price and they are between a rock and a hard place. No worries - I'm sure the execs made plenty of money along the way for this train wreck!
If I remember correctly, the Phase III STS and Pancreatic trials won't have initial data until mid-2015 - due to the trial sizes, it won't be until the end of 2015 until they file for approval - if THLD starts a 2nd line glio trial in early 2014, because the progression and survival timelines are so short, they could finish the trial by the end of the year or early 2015, depending on the size? It's a long-shot, but stranger things have happened.
It wasn't just Brad - one of the clinicians said they would not participate in a double blinded trial going forward - beyond the cost and hassle of hospitalizing/monitoring a patient for the threat of sepsis, they were frustrated with having to stop dosing even though the patient's tumor was responding.
The strange thing is how the analysts on the call don't seem to get it - the one focused on the overall results of the trial, as if they were meaningful. As they explained on the call, when the trial began, even though the biomarkers were PRE-specified, they weren't sure what percentage of the patient population would have those biomarkers and if MM-121 would only have benefit to those with the biomarkers, so the endpoints of the study were for overall PFS and OS. With only 30% of the patients having the biomarkers, that may not be enough to move the needle on a small trial to get a statistically significant overall result, but in a pivotal trial where the patients are pre-screened for the biomarker, it will be a slam dunk.