fish--This is a major point, that is that ET can be a precursor to MF. There was some discussion for "platform approval" for IMET. Considering how closely the blood cancers are medically connected (ET, MF, MDS, AML, etc.) "platform approval" makes sense. I suspect that is what JNJ is after with over 100 worldwide trials. Considering the good safety profile with remission and cures, JNJ will probably get what they want. IMET is a transformative medicine, and the FDA knows it.
JNJ has complete control of IMET and Geron. If the new international trials (JNJ) confirm the medical successes (at Mayo Clinic), Piper Jaffrey's $10 will be realized, and your $25 is possible. JNJ is suppose to publish some interim results soon. JNJ is the "voice" that matters.
It appears that IMET has already succeeded in treating 3 blood cancers (ET, MF, MDS), with minimal safety problems and promise for other cancers. Advanced approvals should have already happened. JNJ and the FDA have all of the data (past at Mayo Clinic, and from new worldwide trials by JNJ as it is being generated) and analysis.
Geron's PPS manipulation would make a suitable add-on chapter to "The Big Short". It appears that IMET is the "transformative" medicine that JNJ and Mayo Clinic claim it to be. No one, with any credibility or expertise, has said otherwise. Piper Jaffrey's $10 still stands, and they have followed Geron for a very long time.
JNJ has already said that IMET is a 'transformative" medicine. I think we have to believe both Mayo Clinic and JNJ/Janssen. They are the only ones that have created all patient data (MF & MDS) and have analyzed the results. Their "word" is above reproach.
At some point in time JNJ/Jansen has to release data comparing the limited Mayo Clinic successes and the interim results concerning the 100+ IMET trial sites worldwide (MF & MDS). The silence is deafening. The Geron PPS is a downer.
How can you separate IMET and Geron? "Chippy's" and Geron's futures both depend on JNJ/Janssen. IMET's successes or failures in the 100 + (MF & MDs) worldwide trials are totally in JNJ,s hands. There is no way that "Chippy" can preempt JNJ's IMET announcements, even if he knows the outcome.
Only JNJ/Janssen has all of the IMET data, and is capable of full and proper analysis, at this point in time. JNJ with all of their expertise (medical, scientific, legal) and their financial depth (almost unlimited) will decide how and when IMET fits into the world of cancer treatment (blood cancers first) and cancer research based upon good medical practices, interactions with the FDA and practical company internals. JNJ fully controls IMET, which is the same as saying JNJ fully controls Geron. We do not know what "deals" are in place between JNJ and Geron, but JNJ seems to have all of the leverage.
There has been some talk of JNJ spinning-off an oncology entity. IMET would be an important part. In any event, Dr. S has no reason to absorb another company, unless JNJ wanted it to happen. Dr. S is no fool.
JNJ would never have risked the money (~one billion dollars), the time and effort to set up 100's of trials (MF, MDS, more) to confirm Mayo Clinic's successes and the medical/scientific talent unless they were 100% sure of IMET and in their ability to control IMET and Geron. Dr. Scarlett and Dr. Tefferi found JNJ (Janssen) as the best available oncology partner to introduce IMET to the world as a cancer platform. Most of all they were able to convince JNJ that the story had gravitas, and was part of the treatment and cure for many cancers.
Janssen (JNJ fully owed subsidiary) is being very conservative on the legal side and the medical side. There are "legal-eagles" looking for "IMET-missteps". With ODD status they are fully protected, and can manufacturer IMET in the proper quantities needed for now. JNJ understands the system, and the workings of the FDA. ODD status serves them very well for now as they get IMET up to speed.
Obviously, IMET and the JNJ control (plus Mayo Clinic research) of the product have received an excellent press lately, with extensive good news. The morning light is here and getting brighter. That cannot be stopped.
My point is (as you say from lack of knowledge) that if there is a reduction in the number of blood transfusions needed, there will be some other improvement noted (perhaps remissions, perhaps a lesser improvement). I understand that a reduction in blood transfusions, in itself, is a useful endpoint that the FDA values for IMET approvals, and is a lesser requirement than remissions. IMET has "a lot" going for it.
So you are saying (I think) that IMET, as it "tames" MDS, has the additional advantage of reducing the number of blood transfusions necessary (perhaps to zero for full remissions or cures). That is very positive. What about other blood cancers (MF, etc.).
I hadn't considered iron excess to be a problem. With remissions or even lesser improvements, I suspect the need for blood transfusions is greatly reduced (as noted by Irish & John).
Since the liver holds (ill-conceived) were lifted the major issue has been dosing. Are transfusions (and iron) an issue for all blood cancers? How about MF, where the studies and trials are now concentrated?
As I have said before, Geron's PPS (market forces, hedge funds, manipulation) do not correlate with IMET's good news (science, medicine, approvals) in the short run.
IMET has passed all tests for advanced approvals. The FDA knows this, and the EU equivalent knows this. They are both very familiar with IMET (ODD status granted). Nothing can stop IMET's good science and good medicine. Logic tells me that advanced approvals are coming very, very soon.
"sure thing" (from Martha)
What could derail IMET?
1. IMET is not safe. ---The very questionable liver holds have been lifted. Considering the early and very successful ET plus MF & MDS trials, many hundreds of patients has tried IMET and have proven it to be safe (as cancer drugs go). No new issues have been raised, with dosing always a factor.
2. IMET does not work.---ET has had nearly 100% successes, and may be a forerunner to MDS. MF and MDS have shown spleen reduction, remissions and perhaps cures (time factor), which never before have been seen. No one questions Mayo Clinic's results. JNJ is now responsible for IMET's future and new worldwide data.
3. IMET is not the Best Therapy Available (BAT). ---All the evidence (NEJM, ASH, conferences) show that nothing compares to IMET, plus IMET works in combinations with other drugs (pending AML studies).