Dr. Stein on Key Highlights of the AUGMENT-101 Trial in Leukemias

Eytan M. Stein, MD, director, Program for Drug Development in Leukemia, Division of Hematologic Malignancies, Memorial Sloan Kettering Cancer Center, discusses the key highlights of the phase 1/2 AUGMENT-101 trial (NCT04065399) in leukemias.

The phase 1 dose-escalation study is examining the maximum tolerated dose of SNDX-5613 in patients with leukemias harboring acute MLL rearrangements or NPM1-mutations.

Preclinical data suggest that menin, an adapter protein, plays a role in the pathogenesis of KMT2A gene rearrangements and NPM1-mutant leukemia, Stein says. Leukemia is driven by the upregulation of leukemogenic genes, and that occurs when menin binds to the KMT2A complex, Stein explains.

It is hypothesized that blocking the interaction between the menin and the MLL complex, those cells will not be primed to develop leukemia. Additionally, the leukemic cells transform into normal cells, Stein concludes.