Repost from SA by biopearl re recent events

[biopearl]

I just put down some thoughts that is a summary of some of the important discussion points that have been raised and examined by multiple posters on SA:

First of all I want to start this post by thanking CKTC, HI, AW, Sdraw and especially MTB for helping me have this cathexis (not catharsis). I realize I have been fighting the plausible because a part of me has had trouble accepting that the data could be as good as it is. Let me summarize what seems to be coming together in my head. As is so often the case others have brought forth these ideas and I have thought a lot about them and here is what has come together for me. I know I have pushed back in some places especially to CKTK but I have come to think he and others are right particularly with regard to the median OS not being reached by primary data read out. Here is a distillation of what I think are the most important discussion point that have come to light over the past several weeks: 1. The 4.7 arm has people that remain and has not reached median OS at 19 months. While important for these patients it is even more important for the 9.7 group given that initially the 4.7 group was closed to enrollment on what I presume were initial suboptimal responses. Yet here we are with the low dose group not reaching median OS at 19 months. This suggests a late beneficial response that was not anticipated in this group, especially since it was closed down early (thank you MTB and others for so eloquently making this important point). So those that said median OS might not be reached by primary read out may be quite correct especially for the high dose group which presumably have an even greater effect than the low dose group. John, as we have discussed lived for five unanticipated years and was a CR/PR. Yes we may see some CRs and PRs in a group of previously terminal patients, really. That would close some mouths of the already closed minded who have promulgated FUD for personal gain. 2. The MDS protocol is fully enrolled and only needs 8 weeks of TI to reach an endpoint. The drug works fast in MDS when it works, so Geron/Jand J may know something definitive as they have hinted, by the time of the primary MF read out. This is all coming together as well as anyone could hope. Since both MF and MDS data have been presented (and AML for that matter), major journals could correctly be used to update the data but I think we all just have to wait for this year's ASH for a truly definitive presentation, quite possibly a plenary session. I want to emphasize that these are not original ideas but a distillation of thoughts from some of our best posters. Thank you for helping me understand. bp

[jpheis]

Gratitude BP. I have read through your post multiple times and keep coming back to two questions. 1-what was John’s dose? I can’t remember for the life of me. 2-if only 8 weeks is needed on the MDS TI read out, how soon do we get an update? Theoretically late April the data is complete since the slides show full enrollment sometime in February but perhaps we have to wait for May17 annual meeting?

[biopearl]

Hi jpheis, 1. John’s dose was 7.5. He was not allowed to increase his dose. If I remember correctly it was related to the FDA hold which basically paralyzed Dr. Teferri’s study. John’s amazing wife tried valiantly to get some flexibility in how his treatment was approached but was not successful either in achieving an allowance in dose or treatment venue such that he was required to travel to Mayo from The Southeast for each evaluation/treatment. 2. Eight weeks TI is different from eight weeks and the TI eight weeks has to be consecutive so I guess the best answer is tha t we wait! Regards, bp