ASCO told us quite a bit. It told us that the conclusions from the Mayo Clinic (MF) pilot studies are intact, and the information published in NEJM is still valid. JNJ would not be focusing on "unmet medical needs" for IMET unless they had irrefutable trial and patient data in hand to satisfy both the EMA and FDA. JNJ is taking all the risks now from getting approvals, manufacturing, distributing and marketing IMET.
Geron is along for the very profitable ride, as JNJ does the heavy lifting with worldwide trials and seeking worldwide approvals. Geron chose well in finding JNJ to "captain the IMET ship". There is no question that IMET works and is safe. The questions are now about genetics, mutations, and IMET's reach beyond blood cancers as part of the "cancer moonshot" alone and in combinations.
We can all trust JNJ to push IMET to its limits medically. IMET saves lives.
Besides the fact that IMET "works" for "unmet medical needs", it has a very good safety profile. It was seriously challenged by the "liver-holds" (Geron's lack of proper data + Incyte's protection of JAK) but that is all well behind us. Juno's safety problems in some ways help IMET. IMET, with ODD approvals (FDA, EMA), is in line for advanced approvals. JNJ is in control with the data, and the strong medical considerations.
The "data-bank" at Janssen is bulging. When will we get the details? JNJ, by now, has a massive database and sufficient results and analyzes to confirm earlier MF data from Mayo clinic. These new trials were scheduled to last 24 weeks. When JNJ final releases their upgraded MF studies, they will be statistically meaningful (FDA & EMA compliant) and genetically mapped to define the most likely successes. The IMET road is ASCO-2016 to ASH-2016 with some important stops in-between. The focus is unmet medical needs that IMET can fill.
Open Label Study With Imetelstat to Determine Effect of Imetelstat in Patients w/ Previously Treated Multiple Myeloma
This study has been completed.
First received: November 16, 2010
Last updated: April 13, 2016
Last verified: April 2016
This is an open label Phase II study to determine the rate of improvement in response of patients with previously treated multiple myeloma to imetelstat alone or in combination with lenalidomide maintenance therapy. This study will include multiple myeloma patients who either have achieved disease stabilization or who have achieved at least a partial response (PR) but failed to achieve a complete response (CR) after cytoreductive therapy for multiple myeloma; ie, have detectable but non-progressing disease and will most likely relapse.
I continue to believe that JNJ is very serious and successful in conducting Imetelstat (IMET) trials, and that they have both the FDA's and EMA's encouragement and recognition (beyond granted ODD status) in developing IMET. The data is in place for full and advanced approvals to meet "unmet needs" (ASCO-2016) for blood cancer patients.
This is consistent with JNJ wanting to keep a competitive advantage, and maintain silence as long as possible, but it is very difficult for current blood cancer victims. ASCO has told us that everything is going as planned, and the good safety profile is being maintained for IMET (IMET for "unmet needs", with expanding trials and combination drugs). JNJ is being too conservative and too cautious, IMO. Let the "good news" roll.
JNJ is gathering data and results on a continuing basis with ongoing and new trials. JNJ has all of the data, all of the results and all of the conclusions. This part of the "cancer-moonshot" is intact. There is no longer any doubt about the importance of telomere length and the enzyme telomerase in the life of a cancerous cell. Imetelstat controls that relationship in blood cancers, and perhaps all cancers (continuing research). The relative safety of IMET puts it on the A-list for advanced approvals, active use in treating blood cancers (ET, MF, MDS, AML and others).
DHR will be the biotech company, will Pall as a major component. Fortive will be electronics and industrial (old Tektronic). They are following the Agilent (A) model: (A) is biotech and KEYS (original Hewlett Packard) is electronics, etc. A merger between Fortive and KEYS in the future makes some sense.
JNJ (Janssen) controls IMET (trials, distribution, manufacturing, data, patients). Geron still owns IMET, but they are a junior partner. The FDA and the EMA have both granted ODD status to Jannsen for IMET. JNJ is completing, continuing and expanding IMET trials in number and scope worldwide. ASCO featured "unmet needs" (filled by IMET), and the "cancer moonshot". This we know.
JNJ, as the most respected company in the USA, certainly has the deep pockets to see IMET through many worldwide trials, for a variety diseases with and without other medicines. Money and time are no objects to them. IMET trials would only proceed, if they confirmed Mayo Clinic's early successes, and showed additional potential.
Patients' "unmet needs" are focusing and accelerating the process (ASCO-2016), and both the FDA and EMA are fully informed (ODD status). New approvals (beyond ODD) are coming soon (logic and unmet medical needs that saves lives), with new data and reliable statistics
ASCO is the best information available to the general public to date. JNJ has all of the answers. Nothing else matters. very much.
Imetelstat will not cure all cancers alone. Blood cancers are the first successes (ET, MF, MDS) and AML, in combination perhaps, is next in line. IMET's role in some other cancers seems to be an "enhancing effect" for other drugs (combinations, cocktails).
Review of ODD (Orphan Drug Designation) granted to Janssen for Imetelstat by both the FDA and EMA.
"The Orphan Drug Act (ODA) provides for granting special status to a drug or biological product (“drug”) to treat a rare disease or condition upon request of a sponsor. This status is referred to as orphan designation (or sometimes “orphan status”). For a drug to qualify for orphan designation both the drug and the disease or condition must meet certain criteria specified in the ODA and FDA’s implementing regulations at 21 CFR Part 316. Orphan designation qualifies the sponsor of the drug for various development incentives of the ODA, including tax credits for qualified clinical testing. A marketing application for a prescription drug product that has received orphan designation is not subject to a prescription drug user fee unless the application includes an indication for other than the rare disease or condition for which the drug was designated."
This is one level of approval. It is now time for new, advanced and full approvals since Imetelstat fills "unmet medical needs" in a safe and unique manner. Imetelstat should be available to all blood cancer patients, alone on in combination as a life saving medicine.
Dr. Tefferi and his group of world class oncology experts told us at ASCO-2016, that approvals are coming for "unmet needs". Since Imetelstat has met all of the necessary criteria, there is an excellent chance that July 2016 will be the month. Who will be first, the FDA or the EMA? JNJ has accumulated and analyzed a great amount of data since June 2015, and the process is continuing.
***Excerpt from ASCO-2016-----"Ruxolitinib, a JAK1/JAK2 inhibitor, is the only approved therapy for MF, and there are no approved treatment options for pts who fail ruxolitinib. There is a great unmet need for effective therapy for this pt population. A pilot study of imetelstat therapy in MF demonstrated complete and partial remissions, including some molecular remissions (Tefferi et al, N Engl J Med2015). Based on the novel mechanism of action imetelstat may provide clinical benefit to pts with intermediate-2 or high-risk MF with refractory/relapsed disease after JAK inhibitor therapy."***
No safety issues, continuing trials, medical successes, no bad news, unmet needs. Why not fully approve (FDA, EMS Asia)?