Revumenib Monotherapy in Patients with Relapsed/Refractory KMT2Ar Acute Leukemia: Topline Efficacy and Safety Results from the Pivotal Augment-101 Phase 2 Study

Background: Most patients (pts) with histone-lysine N-methyltransferase 2A (KMT2A)-rearranged (KMT2Ar) acute leukemia, relapse after conventional chemotherapy and hematopoietic stem cell transplant (HSCT). Remission rates after relapse (complete remission [CR], 5%) and median overall survival (2.4 m...

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Published in:Blood 2023-11, Vol.142 (Supplement 2), p.LBA-5-LBA-5
Main Authors: Aldoss, Ibrahim, Issa, Ghayas C., Thirman, Michael, DiPersio, John, Arellano, Martha, Blachly, James S., Mannis, Gabriel N., Perl, Alexander, Dickens, David S., McMahon, Christine M., Traer, Elie, Zwaan, C. Michel, Grove, Carolyn, Stone, Richard, Shami, Paul J., Mantzaris, Ioannis, Greenwood, Matthew, Shukla, Neerav, Cuglievan, Branko, Gu, Yu, Bagley, Rebecca G., Madigan, Kate, Sunkaraneni, Soujanya, Nguyen, Huy Van, McNeer, Nicole, Stein, Eytan M.
Format: Article
Language:English
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Summary:Background: Most patients (pts) with histone-lysine N-methyltransferase 2A (KMT2A)-rearranged (KMT2Ar) acute leukemia, relapse after conventional chemotherapy and hematopoietic stem cell transplant (HSCT). Remission rates after relapse (complete remission [CR], 5%) and median overall survival (2.4 mo) in ≥2nd salvage therapies in adults remain low (Blood Cancer J. 2021;11[9]:162). In KMT2Ar leukemia, interaction of menin with KMT2A fusion proteins is a key driver of leukemogenesis. However, no therapies targeting the menin-KMT2A interaction have been approved. Revumenib (SNDX-5613; rev), a small-molecule inhibitor of menin-KMT2A interactions, demonstrated preliminary efficacy and safety in a phase 1 study of R/R KMT2Ar and nucleophosmin 1-mutated (NPM1m) acute leukemias. We report topline efficacy and safety for pts with R/R KMT2Ar acute leukemia treated with rev in a pivotal phase 2 study (AUGMENT-101; NCT04065399). Methods: Pts aged ≥30 days with R/R KMT2Ar acute leukemia were enrolled in cohort A (acute lymphoblastic leukemia [ALL]/mixed phenotype acute leukemia [MPAL]) and B (acute myeloid leukemia [AML]); cohort C continues to enroll pts with NPM1m and is not included in this analysis. Pts received rev (163 mg or 95 mg/m2 if body weight
ISSN:0006-4971
1528-0020
DOI:10.1182/blood-2023-192042