Courtney DiNardo, MD, on Need for Improved, Effective Regimens in IDH1-Positive AML

A phase Ib/II trial, presented at the 2020 ASCO Virtual Scientific Program, showed that a combination therapy of ivosenidib (Tibsovo) plus venetoclax (Venclexta) with or without azacytidine (Vidaza) was effective in treating patients with IDH1-mutated acute myeloid leukemia (AML).

In an interview with CancerNetwork, senior study author Courtney DiNardo, MD, associate professor of leukemia at The University of Texas MD Anderson Cancer Center, discussed why it was key to conduct this study.

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IDH1 mutations happen in about 6% to 15% of AML patients. So, it’s a minority, but it’s a sizeable minority. Those patients tend to be older, are usually not appropriate or benefitting from intensive chemotherapy. So, there has been a lot of interest over the last couple of years to find better, tolerable and efficacious regimens for IDH1-mutated AML patients.

There are approvals of the IDH1 inhibitor ivosenidib, and then there are approvals of azacytidine with venetoclax, which is kind of a separate, more broad scope AML treatment regimen that also seems to show good responses with IDH1 mutations. So, the question really was: When you have these different treatment options, is there a way to give either ivosenidib and venetoclax together? Those are 2 different oral therapies that could be a well-tolerated, outpatient regimen. Or is there a way to put all 3 together into a triplet regimen of azacytidine, venetoclax, and ivosenidib to further improve on the outcomes we’re seeing in new therapies?