Michel Zwaan, MD, PhD: 'Clear Medical Need’ in KMT2Ar Pediatric Acute Leukemias

Dr. Zwaan, of the Princess Máxima Center, discusses his study, “Pivotal Phase 2 Results of AUGMENT-101 For Revumenib in KMT2Ar Acute Leukemia: Pediatric Experience” presented at the 2024 American Society of Pediatric Hematology/Oncology Conference.

KMT2A rearrangement (KMT2Ar) occurs in both acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL), according to Dr. Zwaan. Infants with ALL have detrimental outcomes if they relapse, he explained, and their post chemotherapy outcome is “insufficient” with a 50% event-free survival.

“There’s a very clear medical need to change something,” Dr. Zwaan said. “[Revumenib] directly attacks the KMT2Ar, which is the driving abnormality of these leukemias.”

The phase II study looked at historical remission rates, which is around 10%, then recruited 57 patients for an interim analysis. The complete remission rate was 23% for both adult and pediatric patients, which Dr. Zwaan described as a statistically significant difference.

“You clearly see remissions in patients with single agent therapy, which is very encouraging because this was a very refractory, relapsed population,” Dr. Zwaan said. “The final goal is to combine this with chemotherapy upfront in newly diagnosed patients to prevent relapse or reduce the number of patients who need to be transplanted.”