Abstract
AML-111: Updated Results and Longer Follow-Up From the AUGMENT-101 Phase 2 Study of Revumenib in All Patients With Relapsed or Refractory (R/R) KMT2Ar Acute Leukemia (AL)
Clinical lymphoma, myeloma and leukemia, Vol.25(Suppl 1), pp.S401-S401
09/2025
DOI: 10.1016/S2152-2650(25)01623-4
Abstract
Revumenib, a first-in-class, potent, selective menin inhibitor, met the prespecified efficacy stopping rules in patients with R/R KMT2A-rearranged (r) AL in the phase 2 interim analysis of AUGMENT-101—data that were the basis for FDA approval (NCT04065399).
Report longer follow-up and a larger dataset of the phase 2 KMT2Ar population.
See Issa, J Clin Oncol. 2025;43(1):75–84; the data cutoff (DCO) for this analysis was February 29, 2024.
Aged ≥30 days with R/R KMT2Ar AL (patient/caregiver consent).
Revumenib 163 mg (95 mg/m2 if body weight <40 kg) q12h + strong CYP3A4 inhibitor was given in 28-day cycles until lack of at least morphological leukemia-free state after 4 cycles, disease progression, or unacceptable adverse events (AEs).
Safety, tolerability, and rate of CR+CRh in the efficacy population (defined as centrally confirmed KMT2Ar AL, ≥5% BM blasts at baseline within 28 days prior to start of the study).
As of the DCO, 116 patients received ≥1 dose of revumenib. Median age was 35.5 (0.6–75.0) years; 28 (24%) were <18 years, and 14 (12%) were ≥65 years. Other patient characteristics: 95 (82%) had AML, 51 (44%) had ≥3 prior therapies, 73 (63%) had prior venetoclax, and 59 (51%) had prior allo-HSCT. In the efficacy population (n = 97), 22 (23% [95% CI, 15%–32%]) achieved CR+CRh, with a median duration of response of 6.4 months (95% CI, 1.9 months–NR). CRc was 42% (95% CI, 32%–53%) and ORR was 64% (95% CI, 54%–73%). A total of 61% (11/18) of MRD-evaluable CR+CRh responders and 58% (21/36) of MRD-evaluable CRc responders achieved MRDnegativity.Of 62 patients who achieved an overall response, 21 (34%) proceeded to allo-HSCT and nine resumed revumenib post allo-HSCT. In the safety population (N = 116), 106 (91%) had a grade ≥3 treatmentemergent AE (TEAE). Most common (>15%) grade ≥3 TEAEs were febrile neutropenia (45 [39%]), anemia (23 [20%]), and decreased platelet count (19 [16%]). Sixteen patients (14%) discontinued treatment due to a TEAE; 19 (16%) had a TEAE leading to death.
Revumenib demonstrated clinically meaningful responses and a manageable safety profile in heavily pretreated patients with R/R KMT2Ar AL. Sponsorship: Syndax Pharmaceuticals.
Details
- Title: Subtitle
- AML-111: Updated Results and Longer Follow-Up From the AUGMENT-101 Phase 2 Study of Revumenib in All Patients With Relapsed or Refractory (R/R) KMT2Ar Acute Leukemia (AL)
- Creators
- Ibrahim Aldoss - City Of Hope National Medical CenterGhayas C. Issa - The University of Texas MD Anderson Cancer CenterJames S. Blachly - The Ohio State UniversityMichael J. Thirman - University of ChicagoGabriel N. Mannis - Stanford University School of MedicineMartha L. Arellano - Winship Cancer InstituteJohn F. DiPersio - Washington University in St. Louis School of MedicineElie Traer - OHSU Knight Cancer InstituteC. Michel Zwaan - Princess Máxima CenterNeerav Shukla - Memorial Sloan Kettering Cancer CenterBranko Cuglievan - The University of Texas MD Anderson Cancer CenterCarolyn S. Grove - Sir Charles Gairdner HospitalMatthew Greenwood - The University of SydneyChristine M. McMahon - University of Colorado Anschutz Medical CampusAlexander E. Perl - University of PennsylvaniaRichard M. Stone - Dana-Farber Cancer InstituteCristina Papayannidis - IRCCS Azienda Ospedliero-Universitaria di Bologna Policlinico di Sant'OrsolaDavid S. Dickens - University of IowaMaël Heiblig - Hôpital Lyon SudAndrius Žucenka - Vilnius UniversityPau Montesinos - Hospital Universitari i Politècnic La FeIoannis Mantzaris - Montefiore Einstein Comprehensive Cancer CenterTibor Kovacsovics - City Of Hope National Medical CenterPaul J. Shami - University of UtahLi Yu - Syndax Pharmaceuticals, Inc., New York, NY, USARebecca G. Bagley - Syndax Pharmaceuticals, Inc., New York, NY, USAAngela R. Smith - Syndax Pharmaceuticals, Inc., New York, NY, USAEytan M. Stein - Memorial Sloan Kettering Cancer Center
- Resource Type
- Abstract
- Publication Details
- Clinical lymphoma, myeloma and leukemia, Vol.25(Suppl 1), pp.S401-S401
- DOI
- 10.1016/S2152-2650(25)01623-4
- ISSN
- 2152-2650
- Publisher
- Elsevier Inc
- Language
- English
- Date published
- 09/2025
- Academic Unit
- Stead Family Department of Pediatrics; Hematology/Oncology
- Record Identifier
- 9984949226702771
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