"Orphan Drug Status" indicates that in the case one disease (MF), IMET is safe, effective, unique, and life-saving, according to the FDA. This is also likely for the other blood cancers (MDS, AML), and other cancers whose survival depends on their telomere length. The medical community is now able to offer IMET to MF patients, and beyond.
"9:59 am (6/12/15)---Geron lifting after JNJ's Janssen was granted FDA orphan designation for its imetelstat for the treatment of myelofibrosis (MF) -- GERN is parterned with Janssen for the drug (GERN)."
This is the 1st approval of IMET, and should be very good for the PPS. Few expected such a quick approval for fast track status.
The investing world is finally realizing that JNJ/Janssen, after reviewing all existing IMET data, is now going global with this medicine (1st MF, 2nd MDS, 3rd AML, 4th other cancers). JNJ has considerable resources and talent to focus on IMET and patients survivability, including remissions and cures.
Comment on #8--" Partnership with subsidiary of world's largest pharmaceutical and established collaboration with Mayo Clinic Rochester"
The credibility and competence of these two partners are beyond reproach. Mayo's initial trials for MF, with proven successes, and JNJ's reinforcement of Dr. Tefferi's original work, with more than 30 new global trials, is proof positive that they both believe a "very big" IMET future.
There are no significant safety problems, and IMET offers durable remissions and possible cures. A book needs to be written about the market forces that are in complete control of the PPS. Of course, JNJ/Janssen and medical science will eventually dominate, but when?
Comment on #4---" Minimal quality-of-life side effects (nausea, hair loss) and other suppressive effects easily managed."
The liver-holds were the major safety factors that had damaged the Old-Geron, but after the FDA reviewed the Mayo Clinic data, all liver-holds were removed. Now, it is apparent, that they were ill-conceived in the first place. Cancer drugs, that are effective, are also very powerful. IMET is amazingly safe for a medicine that accomplishes so much. JNJ's new global trials (the New-Geron) will focus on dosage and safety, as well as symptom reduction, curability (unique to IMET), and overall responses.
The results are in and Imetelstat is overwhelming the only choice. The Mayo Clinic successes are a matter of record. JNJ feels the necessity to conduct 30 more worldwide trials to repeat and improve upon the Mayo results. They must be very confident to invest so much time, money and talent in this "building-block" to control all cancers, with the blood cancer cousins coming first.
Why has IMET not received advanced approval yet? There is something wrong with the process, and perhaps with the FDA. There have been many theories, with some blaming management. JNJ/Janssen is now in charge, so the mistakes of the past will likely be corrected very soon, with the needs of the patients finally coming first.
Nobody, that I am aware of, doubts Mayo Clinic's conclusions about IMET. JNJ is in the process of improving on the original study by 30 times with a worldwide agenda. I am following the road that Mayo Clinic started and JNJ/Janssen is taking to the stars.
JNJ knows what they are doing (that is a given). They would not go global with about 30 sites unless they were sure of a very positive outcome. In addition these trials will be the ultimate learning experience about dosage, and helping the sickest of all MF patients.
Comment on #1--" Imetelstat induces unprecedented molecular and morphological remissions and bone marrow clearing in substantial numbers of MF patients; and a substantial overall response rate (80%)."
The key words here are "remissions" and "overall response rate". IMET is in a class by itself. In addition, JNJ is setting up trials for combination drugs (example: Doxorubicin and Imetelstat). In this case, they control both drugs.
There are 2 questions in my mind: (1) Does IMET really work ? Does JNJ/Janssen know what they are doing? My answer to both questions is "Yes".
Comment on #6---"Data reported qualifies for accelerated, fast-track, BTD, and/or orphan drug status based on my personal amateur analysis. Due own due diligence".
JNJ/Janssen creation of 30 or so global trial sites will insure overwhelming data about the properties of IMET, in detail and in a format that will satisfy approving-agencies. This is an extension of the original Mayo Clinic study ("Tantamount to a Cure).
Unless some negatives are found in the new JNJ-MF trials (none are expected) IMET will be great, but perhaps not a miracle, since it does cure everyone that it treats. From what I have read, IMET shows some positive effects in about 80% of those that are treated, with durable remissions in some. I am looking for those numbers.
The list of 10 will prevail as the new JNJ/Janssen global trials fill in the "data-gaps".
(To Black****)---"And GERN is still not a $4 stock!!!!". That is a fact. On the other hand, I have been looking for any information that can undo the list of 10 (Irish list). I repeat: "There is no one that can refute any of the 10 above statements. IMET has succeeded, where other medicines have failed." The conclusion has to be that JNJ Jr. ("New Geron") will go much higher. The nay-sayers have no science or medical results to support their negative outlook, as far as I can tell.
There is no one that can refute any of the 10 above statements. IMET has succeeded, where other medicines have failed. Both the FDA and JNJ know that. The patient's needs will be met, and long term investors will be rewarded. These 30 JNJ global trials will answer all IMET questions about MF and beyond.
The markets will be more informed as the JNJ/Janssen trials progress worldwide, with both JNJ and Mayo Clinic getting the word out on the financial networks, and in conferences plus journals. The volcanic forces are slowly building.
These JNJ global trials will define IMET. The case for IMET seems overwhelming from a medical and science prospective. JAK appears to be a different class of drug that only treats symptoms. In IMET's case we are talking about a basket (blood cancer cousins; MF, MDS, AML), durable remissions, and possible cures, with telomere control feasible in 90% of all cancers. It appears that JNJ/Janssen has decided to take a very conservative approach by setting up 30 or so global trials to reproduce and expand upon Dr,Tefferi's MF successes at Mayo Clinic.
In some MF patients positive IMET results are seen as early as 3 to 6 weeks, it has been said. JNJ has seen all of the data and has drafted Dr. Tefferi to lead these trials, so they know that they will get good, great or miraculous results. The unique part of these 24 week trials, is that these patients are the sickest of MF patient's that have previously used and quit JAK .
fuqww---I saw your comments about IMET response time. I did not realize that we could have positive responses to IMET in as little as 3 to 6 weeks. Do you remember where that was discussed? Apparently, JNJ plans to release good news as the trials (30 or so) progress. That could be very soon.