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Re: KMBJN post# 150388

Wednesday, 06/08/2016 5:40:01 PM

Wednesday, June 08, 2016 5:40:01 PM

Post# of 403773
OK. I get that safety needs to be shown if no prev trial of 1) K given 3x week, and 2) K plus chemo agent, and that 1b will be to demonstrate that safety.

You spell out the safety data for apr-246. Of course they need to prove safety, but suppose only 6 or 8 of their trial patients were deemed responders. Can we all agree that there would be no round of fundraising and no moving forward with rest of the trial? They have to show some response at planned levels of dosing. They cannot show just safety. They had to show strong data like 18 of 24 pts improving, otherwise it is hopeless.

In the same way K plus doxorubicin cannot just show safety - it must show some very good response rate . Otherwise, it will be game over, even though initial part of the trial is for safety, or to define dosing.

Can you explain how 1/3 of patients responding- by whatever criteria , CA-125 RECIST - for K/doxorubicin will lead to trial completion? APR-246 would have been halted with such data, and so will be K if that happens.

And yes, of course I am worried that all of the eggs are in the ovarian CA basket and failure with 1b will be the end of K. Leo just announced that no other phase 2's are moving forward and the focus is on ovarian CA. They have to succeed there, or who knows if K will get a chance with other tumors.

With respect to other doses, or more x per week, I would expect that to be part of expanded or planned 1B for ovarian trial - dose escalation