Why the AML trial didn't start in 2016

[Fishermangents]

Many of us wondered why the AML trial didn't start end 2016, as was being announced earlier. It is even part of the collaboration agreement. In the meanwhile we know that there will be no new trials before the review end Q2 this year. However, we didn't know why the AML trial also has to wait. So I asked Anna. I also asked if she could tell more about the 'numerous pre-clinical studies' that JnJ is sponsoring. This is her full answer:
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"Indeed, clinical trials in other hematologic myeloid malignancies in addition to MF and MDS, including in AML, are contemplated as part of the potential development plan for imetelstat under the collaboration agreement with Janssen. However, as you mention, we do not expect any new trials to be initiated until more data are available from the current IMbark and IMerge studies. There may be important information and data that may inform aspects of a future AML study, including related to dosing, for example. When we have been asked by analysts on public quarterly conference calls to describe what a future AML study might look like, as late as the third quarter conference call in November 2016, the answer has been that we don't have any specifics or anything to announce. The current clinical focus is on IMbark and IMerge. If and when there are definitive plans for any additional clinical trials of imetelstat in any indication, we would share such information.

With regards to preclinical studies using imetelstat being sponsored by Janssen, by way of example, I can reference the data generated at Janssen as well as by academic investigators that has been presented at the AACR and ASH annual meetings during 2016, which we announced. Beyond that, I can't provide information on any additional projects for competitive reasons, as data have not yet been presented or published."
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So they want the IMbark and IMerge data to contribute to the AML trial design, such as dosing. I think that is totally valid: if those data may improve the design of any new trial then it would be stupid not to wait just a couple of months.
Regarding the pre-clinical studies: her answer tells me that 1) we have to keep an eye on the AML and CML studies that were presented at ASH2016 and 2) there is more going on than we can see.

[biotech_bs]

That's for sharing this Fisher. I really appreciate the effort on your part.
I do recall that as the year went on Chip was saying that the AML study would be a combo study and they weren't sure what that combo would look like. There was more preclinical work to be done. I began getting vibes mid-year that the AML trial would not happen in 2016

[Fishermangents]

I guess you were right, biotech. I think that Anna gave a valid explanation.

[Fishermangents]

It is also good to read that we will be informed when plans are definitive, so not only when the trails actually start.

[biopearl]

Fish, thanks for your excellent post. Anna's explanation is clearer that others I have read. I like many have no problem waiting if positive adjustments to the long term plan are based on as yet unevaluated results. I do object to Scarlett dangling the AML study as planed or even likely for 2016 and stating it multiple times and then silently dropping it from his subsequent prepared remarks. A change in course should be accompanied by an explanation to stockholders as a matter of responsibility. Sure its not required but I don't see it as an unreasonable expectation. bp

[sargasso]

This video is linked on the YMB. https://www.youtube.com/watch?v=Es_gS0XKV3w

It was was published in Sept. 2015 so it is somewhat dated and you may have already viewed it.

Nonetheless, the information presented in this video and others like it is the primary reason I continue to hold GERN. What other drug is capable of this type of response?

I think it unlikely that JNJ is going to throw imetelstat in the trash can or walk away from the drug. Rather, I believe they are going to run with it, explore it, refine and develop it.

[Fishermangents]

bio, a totally agree with you. I don't see any reason why the explanation that Anna just gave, wasn't communicated to shareholders in an earlier stage. There must have been a moment in time (when could that have been? Mid 2016?) that it was decided to wait with the trial in order to include more mature data from IMbark and IMerge in order to take better decisions on combo and dosing. I think that anyone would have understood that. It least I would.

Sargasso: still a great video. If you add the ASH2016 presentation to that, the story gets complete: http://www.geron.com/file.cfm/53/docs/H ... H-2016.pdf

[Ptca]

It looks like your questions are being answered and in less than 3 weeks we should hear very good news. Thanks for all the info spread on your site and good luck to all.

[biopearl]

PTCA thanks for pointing out that the full text of the currently embargoed abstract will be available March 1st at 4:30. And we still have the European heme meetings and ASCO way before ASH to look forward to. Best wishes bp

[irishtrader52]

It does seem that imetelstat marches on behind the scenes and the Imerge and Imbark studies are foundational. Perhaps, in a few weeks, we will finally be beyond imetelstat. Stayin' alive for the cause.