The most important focus of the new MF abstract

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biopearl123
Posts: 235
Joined: Fri Jul 20, 2018 5:13 pm

The most important focus of the new MF abstract

Post by biopearl123 » Mon Nov 11, 2019 6:00 am

To Ryan's point about AML, when the venetoclax/imet AML study was presented, I essentially went all in (not my smartest investment move), when it was stated that "cures" were effected in a number of mice. We all know that this did not translate into a clinical study, at least as yet, for opaque reasons (thanks John). Nonetheless, as Ryan and others have noted this new combination study is important. For one thing synergism with Rux was demonstrated, but more importantly, NO suppression of normal hematopoietic stem cells occurred. This is interesting. As we know cytopenias are the dose limiting factor in the use of Imetelstat. This early data suggests that a periodic drug vacation, while another synergistic drug is administered may not only enhance therapeutic benefit but also minimize untoward side effects. Remember, there are no good treatments for MF that prolong life except perhaps a high risk bone marrow transplant. Geron is making a lot of Imetelstat and hiring for manufacturing. They also dropped a big hint when they said Janssen would supply all that was needed to complete PIII (if I am remembering that correctly). So as speculated earlier, they must be planning (and hoping) for some form of approval post FDA meeting but this new Rux combo study I think leads to a reasonable speculation that an additional clinical study is in the offing, not the AML study that has been alluded to for YEARS but more likely a Rux/Imet MF study as suggested by others and is telegraphed by Geron with this presentation. Perhaps the CRO was tasked with phase I responsibilities in this arena. We really don't know what the CRO has actually be asked to do. Since half of the new hires will be in R and D, that suggests an active search for untapped areas of development in combinations (but we don't know what percent of these hires will be devoted to manufacturing alone if any). It is hard to believe that Janssen didn't fully explore combinations (including Rux combinations which are the most obvious--making the Janssen "walk" all the more curious in light of the current new presentation poster.). Best Wishes, bp

Secret Third Arm
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Joined: Tue Aug 28, 2018 3:26 pm

Re: The most important focus of the new MF abstract

Post by Secret Third Arm » Mon Nov 11, 2019 1:50 pm

Great post BP, thanks.

What makes you think they would need to do a Phase 1 for a Rux combo trial? If Imetelstat is approved (God willing) for r/r MF or MDS then I believe any other studies like combos would be Phase 3 trials. That's what makes this such a compelling drug and investment. Once approved in a single indication the path to additional uses becomes much shorter. Hopefully once the war chest fills up they announce a half dozen combo trials and use those funds to exponentially grow the value.

biopearl123
Posts: 235
Joined: Fri Jul 20, 2018 5:13 pm

Re: The most important focus of the new MF abstract

Post by biopearl123 » Mon Nov 11, 2019 4:38 pm

STA, I don't think a new PIII could possibly start out of the gate until further lab studies, dosing studies, human safety studies etc were done. While synergism has its obvious benefits, the potential for synergistic toxicities also need to be evaluated. I think there is much work required before a PIII combo study. Still exciting, if even just for our grandchildren....bp

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