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ASH 2020 | BTX-A51 for R/R AML or high-risk MDS

Eytan Stein, MD, Memorial Sloan Kettering Cancer Center, New York, NY, discusses the details of a Phase I trial (NCT04243785) of BTX-A51 safety for use in relapsed/refractory (R/R) acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS). BTX-A51 inhibits CK1α, CDK7 and CDK9, to increase p53 protein levels and decrease key oncogene transcription (Myc and Mcl1), resulting in selective apoptosis of cancer cells. The open-label study will determine the maximum tolerated dose in a dose-escalation phase, followed by a dose-expansion phase to identify dose-limiting toxicities and evidence of efficacy. This interview took place during the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, 2020.

Transcript (edited for clarity)

BTX-A51 is very interesting. It’s a CK1-alpha inhibitor. This is a trial that recently started accrual. We’ve accrued a number of patients to this study so far. We don’t have results yet that we’re presenting. What we’re presenting is, is a trials-in-progress abstract.

So again, it’s a CK1-alpha inhibitor. CK1-alpha has been shown in preclinical models that were published both in Cell, and then there was a nice editorial on this published in the New England Journal of Medicine, showing that by inhibiting CK1-alpha, you can get reactivation of the p53 pathway to cause apoptosis leukemia cells...

BTX-A51 is very interesting. It’s a CK1-alpha inhibitor. This is a trial that recently started accrual. We’ve accrued a number of patients to this study so far. We don’t have results yet that we’re presenting. What we’re presenting is, is a trials-in-progress abstract.

So again, it’s a CK1-alpha inhibitor. CK1-alpha has been shown in preclinical models that were published both in Cell, and then there was a nice editorial on this published in the New England Journal of Medicine, showing that by inhibiting CK1-alpha, you can get reactivation of the p53 pathway to cause apoptosis leukemia cells.

We’re in dose escalation. Today things have been going quite well and we’re hoping that we’ll have data to present on the outcomes of this study in the next 6 to 12 months.

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Disclosures

Dr Eytan Stein, MD, has done consultancy for Amgen, Agios Pharmaceuticals, Celgene Pharmaceuticals, Abbvie, Seattle Genetics, Syndax, Genentech, Biotheryx, Novartis, Astellas Pharmaceuticals and Daiichi-Sankyo; has participated in a board of directors or advisory committee with Agios Pharmaceuticals, Celgene Pharmaceuticals, Genentech, Syros, PTC Therapeutics, Novartis, Astellas Pharmaceuticals and Daiichi-Sankyo; and has received research funding from Celgene Pharmaceuticals, Syndax, Bayer, Novartis and Daiichi-Sankyo.