Brookings Institute

[biopearl]

Fellow board members, first of all nice article by Hoosier, nicely done, thorough and intelligent (as always). Good research on publicly available data (but buried in different places). Can't wait to see if anything pans out in Feb with pediatric study and with possible MM combo studies. For now I had a few thoughts. Have been listening to the Brookings presentation on Breakthrough therapy designation: http://www.brookings.edu/events/2015/04 ... y-criteria and found some interesting things. I recommend this as a basis for discussion on this board. Re MF study there are several features of this discussion that the Embark study sees to be a tailor fit: molecular markers, unmet needs, objective findings of disease regression (bone marrow, spleen, anemia etc) and several other criterion that would make the Tefferi study presented at ASH one the FDA would look at seriously. Lets assume they are. If so the clock would have started once the data was published in the NEJM in Sept. If the clock for BAT is six months we should be getting close. This to me is why there is no phase III part of this study. Janssen/Geron must be pretty sure they will not need it or it will be post market. Right now the off putting time line for 2017 is based on the situation as it stands today but if BAT were granted this could be a Black Swan. Anyone agree? On the other hand the MDS trial has a phase III component because it is unknown whether transfusion reduction may not represent enough of a prognostic factor compared to the clearing of bone marrow fibrosis in MF. Note there was NO bone marrow data as part of the MDS data presented by Dr. T. That is a mystery and maybe too high a bar. But not too high for the MF study. So while the presentation this week at JPM was NOT helpful to stock price especially given the distressingly long time line presented, I wonder if BAT waits in the wings. On the other hand if it did, why would Janssen/Geron not wait and then use the opportunity to raise funds for the Geron acquisition? If BAT were waiting six months is up at the end of Feb from the NEJM article. Fellow board members, please help me parse this. Regards, bp

[biopearl]

http:// www .brookings.edu/ events/2015/04/24-fda- breakthrough-therapy-criteria Couldn't post link, try it without the spaces. bp

[Fishermangents]

Bio, thanks for your interesting post. I think it is realistic that BAT is around the corner. We only know it when we see it. But taking the anticipated approval of 2017 seriously, would mean that a very fast process towards approval is needed.

btw: here is the link: http://www.brookings.edu/events/2015/04 ... y-criteria

(the link in your first post works normally)