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SOHO 2021 | Updates on KO-539 as a treatment for AML

Amir Fathi, MD, MPH, Massachusetts General Hospital, Boston, MA, shares updates from the a Phase I/II trial (NCT04067336), which investigated the safety and efficacy of KO-539 in patients with acute myeloid leukemia (AML). KO-539 blocks the interaction of menin and KMT2A/MLL, which together are critical for the survival of leukemia cells. Dr Fathi explains that KO-539 appears to be well tolerated and demonstrated sustained complete remission in two patients but more results are awaited to define the more longer-term efficacy of the drug. This interview took place during the ninth annual meeting of the Society of Hematologic Oncology (SOHO 2021) congress.

Transcript (edited for clarity)

The data that we presented, I think a year ago, a couple of years ago, I forget, the year of the pandemic, everything kind of blends together, showed that among the couple of handful of patients that we treated, the drug was very well tolerated. Two patients had very impressive responses, complete remissions lasting several cycles. One of those patients continues to have a remission. So it’s exciting and we’ll have to see if this drug does continue to show efficacy in a proportion of patients...

The data that we presented, I think a year ago, a couple of years ago, I forget, the year of the pandemic, everything kind of blends together, showed that among the couple of handful of patients that we treated, the drug was very well tolerated. Two patients had very impressive responses, complete remissions lasting several cycles. One of those patients continues to have a remission. So it’s exciting and we’ll have to see if this drug does continue to show efficacy in a proportion of patients. Is that efficacy limited to what it would be pre-clinically determined to have efficacy and namely MPM1 mutated patients or MLL or KMT2A rearranged patients, or whether it will have broader implications in terms of therapeutic efficacy.

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Disclosures

Amir Fathi, MD, MPH, has participated in consultancy work with Pfizer, Trillium, Abbvie, Kura, Blueprint, Genentech, Novartis, Trovagene, Daiichi Sankyo, Novartis, Agios/Servier, BMS, Morphosys, Kite, Foghorn, Takeda, Amgen, Seattle Genetics, NewLink Genetics, Forty Seven and Ipsen; and has received clinical trial support from Agios/Servier, BMS and Abbvie.