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author：focus 2025-10-04 11:50:20 - browse：1

SAN DIEGO — One of the toughest subtypes of acute leukemia involves a genetic alteration in the KMT2A gene. Many cancers with this genetic alteration end up relapsing or don’t respond to treatment, but new data presented at the annual American Society of Hematology meeting offer hope of a new targeted therapy for these patients.

The study, called the Phase 2 Augment-101 trial, tested Syndax’s revumenib in patients with relapsed or refractory leukemia with these KMT2A genetic rearrangements. Overall, about 63% of the patients responded to the treatment, with many able to receive a potentially curative stem cell transplant later on, which is often the ultimate goal for patients with relapsed or refractory patients, said Ibrahim Aldoss, a hematologist-oncologist at City of Hope and the study’s presenter, in an interview.

“KMT2A-rearranged diseases represent 10% of all leukemia. They can be exceptionally hard to treat,” Aldoss said. “The median overall survival remains low with current available treatment. Right now there’s no targeted therapy for KMT2A-rearranged disease. We’re really excited about revumenib as it targets the mutation, and we’re seeing encouraging outcomes.”

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