ASH 2024 | A clinical study investigating oral azacitidine plus venetoclax for newly diagnosed and R/R AML

Yeah, this is a study that we’re very excited to present here. So this is an all oral regimen for AML and for both relapsed/refractory AML and newly diagnosed, and it consists of oral azacitidine or CC-486 and venetoclax. So we have already presented previously the results of our dose escalation phase of this study. So we did a dose escalation phase to find the maximum tolerated dose of CC-486. We found no drug-limiting toxicities for these patients, so the maximum tolerated dose was 300 milligrams of oral azacitidine given on days 1 through 14 of a 28-day cycle, and venetoclax was given per FDA label. 

So in terms of toxicities for this regimen, very similar to what we see with conventional azacitidine and venetoclax. So we definitely see some cytopenias in cycle 1. And then aside from that, just grade one and two, some fatigue, diarrhea, nausea, and we did see 23% of the patients get febrile neutropenia. 

Now in terms of efficacy, we’re really excited about this data. So we’ve enrolled 18 patients on the relapsed/refractory cohort and 11 patients in the newly diagnosed cohort. For the relapsed/refractory cohort, again, 18 patients, and we’re seeing a 33% response rate, 22 of those are CR and CRis. And then for the newly diagnosed cohort, we have 11 patients, six of those have TP53 mutations, and we’re seeing an 82% CR-CRi rate, so excellent responses that we’re seeing on this. We’re still enrolling on the protocol. We’re hoping to finish the trial by next year and report the final results then.

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