There are 50,000 new patients with metastatic prostate cancer to bone per year. With just prostate cancer bone met indication, this is a billion dollar drug.
Add in breast, ovarian, and other, with bone mets, this is a multibillion dollar drug.
Even just looking at soft tissue response at this early stage, the results are great (compared to other drugs), potentially doubling the market again. I wish EXEL would issue rights or something, because I think a buyout very likely.
Why EXEL will unfortunately probably be bought out? If you realize that this drug has a high likelyhood of being a big drug, then estimates of this companies potential are worth looking at. First it is important to understand that the current price EXEL is at is pretty much where it should be. Historically, companies with a good potential drug, owning half, and decently financed will hover around the 1 billion cap.
But EXEL so far owns close to 100% of the drug. The results are very good, and activity is very broad. There is an unmet medical need for bone mets. The drug is active in the most common cancers making the population huge.
Anyway, the potential is probably 3 billion fairly quickly for cancer metastatic to bone. There is nothing more pleasing than to slay bone mets,,,when nothing else works. Even if EXEL ends up with a 50% partner (it really should be a 30% partner who pays all sales costs), we would see 1.5 billion revenue to EXEL. Because most of the growth would still be ahead, price to sales ought to be 10-12 for a market cap of 15-18 billion,,,so EXEL would be over $120/share.
It might seem this would take years, but it is possible that the MTC indication will be good, and approval could follow in a year. Off label use for bone mets will be reimbursed, especially since there has been a major court decision saying medicare/medicaid must reimburse for reasonable off label uses (the government only wanted to reimburse for "approved" uses, which would limit use). EXEL is also going to try to get expedited study/approval for bone mets/pain indication, but EXEL calls that a long shot (which it is, but maybe not as long as they are saying, I think it has a good chance).
Longer term, if EXEL establishes itself as the first drug with this kind of activity, it would be the standard of care, and 10 billion sales would not surprise me.
Of course anything and everything usually goes wrong, with competitors lurking, delays, and unexpected side effects. On the other hand, management so far seems to understand what they need to do, which is unusual. There are other drugs out there that may show similar activity to XL184, but they are earlier stage, and so far I have not heard of blow out results. Don't underestimate the ability of a major drug company to power through big trials very quickly to close any gap. The complete concentration on XL184 is impressive. EXEL has a number of other drugs which are partnered and continue development (by the partner).
I like the trials they are doing, they seem to understand what the FDA wants to see (and requires). Overall, I think EXEL has a better than 50% chance of getting approval for major indications that in the longer term would put EXEL into the mid-hundreds.
Since the drug companies are not totally ignorant, the chances are that EXEL would be bought out long before then. I look for a major partnership by end of June.
I'm arguing with myself a bit.
To back up a bit, consider the studies with bevacizumab in glioblastoma (GBM). There were responses seen, but the gold standard for responses is using MRI with Gadolinium as a contrast agent. Because bevacizumab interferes with permeability, some argued that rather than actually affecting the brain cancer cells, bevacizumab (Avastin) was affecting the imaging process (interfering with Gad uptake) rather than truly slowing the growth of the tumor cells.
In an analagous manner, some could argue that the observation of bone scan resolution (uptake of Technesium tracer) refelcts a change in tracer distribution and uptake rather than a change in the disease.
I raise the other issues (pain, bone markers, improvement in anemia) as an argument against this being artifact since all the various clinical measures (imperfect as any of them are individually) all appear consistent (excepting PSA which has its own history).
Furthermore, with all these clinical parameters moving in the right direction, it argues that it should be possible to show clinical benefit in a clinical trial in prostate cancer, the questions are design and resultant timing.
1>> If one wants to raise questions about the bone scan effects, the best question I would raise is if the effects represent an effect on tracer distribution with the symtomatic improvement being a result of fluid shifts, etc.
2>> But even beyond that is the associated improvements in pain and bone markers, all conistent.
I am not sure I understand your "best question" in #1. Taken alone, #1 looks like a question of whether Cabo is interfering with the "radiographic picture".
But in #2 you admit that the apparent effects of Cabo go beyond the "radiographic picture". To your list of positive effects, add the hemolytic rebound.
So please help me understand the best question.
15 in June.
Buy the dips.
Please let me be clear, I mentioned fraud only in the generic sense that one always needs to worry about it when there are financial motives. I agree with Ernie's assessment that the liklihood of the bone scan results being fraud are next to nil. I believe that both the CT scans and bone scans have been subject to central review which is done in blinded fashion. The number of prestigious KOLs involved who have gone on the record endorsing the excitement also argues against it and so on. I think my re-do post may not have been as clear as the initial lost one. If one wants to raise questions about the bone scan effects, the best question I would raise is if the effects represent an effect on tracer distribution with the symtomatic improvement being a result of fluid shifts, etc. But even beyond that is the associated improvements in pain and bone markers, all conistent. There has been a prior detailed discussion about possible mechanism of action and I don't want to reopen that (though the speculation is interesting). But I do want to be clear that I wasn't implying fraud nor do I think there is any.
Happy Easter and GLTA,
<<Ernie, "fraudulent data" was never proven at SQNM. Company always maintained the data was mishandled and the only person charged, to date, was the person in charge of Quality Control - the recently deceased Betty Dragon. Dragon was charged because she was the one publicly claiming T21 test was 100% specific.>>
Joe, where we differ is semantics vs substance. SQNM did their analysis in house and announced 100% accurate results. As is turned out their test was not 100% accurate. The subsequent house cleaning took a long time from the time of the infraction and Ms Dragon did not lose her job until her boss, CEO Harry Stylli was fired.
"Elizabeth A. Dragon, a former Sequenom senior vice president for research and development, admitted in an appearance before a federal judge this morning that scientists involved in the clinical studies either knew the outcomes before testing the samples, or had been directed to change their initial results. As part of an agreement with prosecutors, Dragon pled guilty to a single count of conspiracy to commit securities fraud."
"The most notable recent case of fraudulent data happened at Sequenom."
Ernie, "fraudulent data" was never proven at SQNM. Company always maintained the data was mishandled and the only person charged, to date, was the person in charge of Quality Control - the recently deceased Betty Dragon. Dragon was charged because she was the one publicly claiming T21 test was 100% specific. These specificity/precision claims were the Dragon's domain of responsibility. When the rest of management realized Dragon did not have the appropriate controls and procedures in place to substantiate the claims (due, at least in part to Dragon's incompetence) they publicly admitted the problem and launched an external investigation with full cooperation of the SEC.
Results of an independent followup study were published in Feb and Sequenom said:
Sequenom announced publication in the American Journal of Obstetrics and Gynecology of Sequenom CMM's "locked assay" study. The performance of a shotgun sequencing based assay for noninvasive detection of fetal aneuploidy was evaluated on a set of 480 plasma samples from pregnant women at high-risk for fetal chromosomal aneuploidy. Utilizing 449 samples, all 39 trisomy 21 samples were correctly identified, while one of the 410 euploid samples was misclassified as T21. The overall classification showed 100% sensitivity and 99.7% specificity.
The original in-house data may have been just as good but "100% sensitivity" claims should not have been made since the procedures and controls were not in place. Certainly not a scenario that I would characterize as fraudulent - more like outrageous incompetence and arrogance on the part of the Dragon.
Disclosure: I know and respect SQNM's Chief Scientific Officer, Charles Cantor.
>>The odds that the results are fraudulent are nil.
I said as much before.
In fact, the bone met "resolution"* was so striking that researchers questioned the results. At first some thought there MUST be some error. (We heard this on the R&D day webcast.) Once the results were published everyone in the field was looking at that drug-induced bone met "resolution".
There is no fraud here - rather we have Cabo inducing bone met "resolution".
Buy the dips.
15 in June.
* I use quotes around resolution as an admission that we are not certain of the precise nature of this resolution. Because of the correlated factors of pain, hemolytic response and blood markers, I am inclined to think the resolution will prove very significant, prolong life, and improve QOL.
<<...is it possible that this could potentially be the case despite the fact that the results were reported during Phase 2 clinical trials (with notable SME's collaborating on the data) and are there any precedents where the results ultimately were fraudulent with similar attention and progression through the clinical stages?>>
The odds that the results are fraudulent are nil. The most notable recent case of fraudulent data happened at Sequenom. They did everything in house. EXEL has attracted a handful of the most respected third party clinicians available to do their trials and tell the Cabo story.
You are the only one with fraud in your mind. With so many renouned Scientist, with so many Patients testifying and so many Big Pharma's attached to EEXL. It surely smells like fraud to a non-named scientist in your league. When impressive data proves itself like the ones on Nov 2010, many like you even canno't believe what you are hearing and cannot face the facts that EXEL will be blockbuster. Even the non-believers who are desperately trying to mown down EXEL cannot come to fae facts that EXEL is one of a kind and will be a blwo out Blockbuster.
Keep trying to make claims as fraud since you cannot believe these data. I would suspect that you maybe liable in a fraudulent Lawsuit from EXEL to make sure that you can understand that every one of their data are being presented by individual Scientists rather than paid promoters.
Keep on wishing what you accused others off and hope to be a recipient of a Lawsuit.