ASH 2025 | A Phase II decentralized clinical trial of ivosidenib in IDH1-mutated CCUS

So the first trial that we’re going to discuss is the ivosidenib trial, which is a decentralized Phase II clinical trial looking at IDH1 mutant clonal cytopenias of undetermined significance. When patients with clonal hematopoiesis of indeterminate potential acquire a cytopenia or abnormality in their blood count, that situation is called clonal cytopenia of undetermined significance. One such mutation that can lead to those cytopenias is IDH1 mutation and the rate of progression from clonal hematopoiesis of indeterminate potential to an overt myeloneoplasm is variable but once the disease has progressed to clonal cytopenia of undetermined significance, the trajectory to developing a myeloneoplasm is variable but once the disease has progressed to clonal cytopenia of undetermined significance the trajectory to developing a myelodysplasm is faster so it’s a much different disease at that point. So we’re always looking for strategies to kind of do early detection for sure but also strategies of disease prevention or halting progression at earlier stages. And then one such strategy was via this trial where ivosidenib, which is a FDA-approved drug for MDS and AML, was used in CCUS to see if we are able to make our patients’ blood counts better. And then we haven’t looked at the progression endpoint as yet. And then there was a decentralized model, which was different in a way that patients could stay closer to their home, anywhere in the nation. And the trial was conducted centrally at the primary site. And then local care was provided with the local doctors, blood work at local labs, and then EKG monitoring through a device present locally as well so that was a decentralized model which is a unique and a newer strategy of enrolling rare diseases. So that was another unique characteristic of this trial. The results were important because patients with idh1 mutant disease can have neutropenia and thrombocytopenia and there were high high response rates seen in this IDH mutant CCUS population. So most patients, several patients had normalization of their blood counts. And then it was associated with corresponding decrease in the size of the IDH1 mutation as well, which tells you there may be some activity going on that may be modifying the disease even. So thereby halting the disease from progressing but also maybe preventing from happening. So I think that was a that was the hallmark of this study. And then it’s a small study there’s 20 patients in this study but perhaps the next steps would be a larger study to show these results and duplicate these results in a meaningful way to help our patient population. But overall, I think there’s a step in the right direction because typically for our CHIP-CCUS patients, we did not have a lot of strategies to explore, but this is a strategy that has shown to improve blood counts, improve time spent in the transfusion, improve risk of infection through by just saying that hematological responses are there. So I think a very meaningful study for our patients.

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