imetelstat: where do we stand?

[Fishermangents]

A repost by sdrawkcabeman (YMB, 20 April 2016)


Approval in MF is a given. Approval in MDS is very likely. Time to set your sights on AML.

The CRs in MF means certain approval, not just b/c of CRs but also in context of inadequate SOC.

The pilot's MDS cohort showed Imet is very active in MDS, makes perfect sense b/c of locality of MF and MDS to bone marrow; more to the point, 11 short weeks to transfusion independence, and TI as an endpoint is an easy regulatory endpoint to meet. Also in the context of inadequate SOC. And, of course, this is as a single agent. Approval in MF will set a precedent for Imet in heme malignancies, the FDA will account for this when approving in MDS.

Now, on to AML. Imbark and Imerge are SINGLE agent studies, and already Imet has shown unprecedented efficacy. And yet, the few combo studies in AML have shown even MORE DRASTIC efficacy than Imet did in MF or MDS. Again, the target is bone marrow. Makes sense that this family of diseases should respond so ridiculously well. The AACR presentation demonstrates the versatility of Imet, the spectrum of SOC it can be combined with to achieve a multi-front action against AML.

Here's what the next AML trial will look like: it will look a lot like Imbark and Imerge. Expect a very large, multi-center, global trial, with multiple arms for dosing, and possibly even a multi-combo trial, with an initial cohort to be followed by full enrollment, and it will be for registration. It will be a paradigm trial for heme malignancy advancement, and will be a landmark trial not just because Imet is a first-in-class drug, but also because it will proffer Imet as a vanguard agent for combo studies. The AACR study was in cell lines, so I think the FIRST of the AML trials will be in combo w/doxorubicin, with multiple trials to follow. Most importantly, the trials that follow will be Imet paired with SOC for the SPECTRUM of cancers. The other AACR presentation validates yet again Imet's MOA; the research of Imet in cancers is just beginning, and every further discovery of Imet pushes closer to trials in solid tumors. And again... the Yale study.