Revumenib Displays Encouraging Anti-Tumor Activity in KMT2Ar Acute Leukemia, AUGMENT-101 Trial Stopped Early

Jul 11, 2024

Photo Credit: Dr Microbe

The AUGMENT-101 trial in patients with in KMT2Ar leukemia has been stopped early because of revumenib encouraging anti-tumor activity.

In a heavily pretreated population of participants with KMT2A rearranged (KMT2Ar) acute leukemia, revumenib displayed encouraging anti-tumor activity. Moreover, the phase 2 study was terminated early for efficacy.

The investigational menin-KMT2A interaction-inhibitor revumenib was tested in the phase 2 AUGMENT-101 trial (NCT04065399) among 94 participants with KMT2Ar acute leukemia, either myeloid leukemia (n=78), or lymphoblastic/mixed phenotype leukemia (n=16). The included participants had received a median of 2 lines of prior therapy. The primary outcomes were safety and the rate of complete remission (CR) plus CR with partial hematologic recovery (CRh). Dr. Ibrahim Aldoss, MD, from the City of Hope National Medical Center, in California, presented the findings of the interim analysis.

After a median follow-up of 6.1 months, 23% of participants in the efficacy population (n=57) reached the primary endpoint of CR plus CRh (95% CI 12.7– 35.8). The median duration of response was 6.4 months. The overall response rate was 63% and responses appeared consistent across participants with common co-mutations. Dr. Aldoss also mentioned that 70% of participants who reached CR plus CRh achieved minimal residual disease negativity. Finally, 39% of the responders proceeded to hematopoietic stem cell transplant.

Grade ≥3 treatment-related AEs were documented in 54% of the participants, with differentiation syndrome (16%), febrile neutropenia (14%), and QTc prolongation (14%) as the most commonly observed events of these higher grades. Dr. Aldoss noted that 6% of the participants had discontinued therapy due to treatment-related AEs.

Overall, revumenib provided high overall response rates, CR plus CRh rates, minimal residual disease negativity rates and the possibility to proceed to hematopoietic stem cell transplant in heavily pretreated participants with KMT2Ar leukemia.

Medical writing support was provided by Robert van den Heuvel.