Revumenib

Three clinical trials from MD Anderson Cancer Center showed significant results with novel triplet therapies for leukemias. The SAVE trial achieved an 82% response rate in advanced AML with KMT2A or NUP98 rearrangements. The IDH1-mutant AML trial showed a 94% overall response rate. The CLL trial achieved 98% undetectable MRD in bone marrow and 100% in blood after 13 cycles.

Three clinical trials from MD Anderson Cancer Center showed significant results with novel triplet therapies for leukemias. The SAVE trial with revumenib, ASTX727, and venetoclax achieved an 82% overall response rate in relapsed/refractory AML patients with KMT2A or NUP98 rearrangements. A triplet regimen of ivosidenib, venetoclax, and azacitidine showed 94% overall response and 93% composite complete remission in IDH1-mutant AML. A pirtobrutinib, obinutuzumab, and venetoclax triplet achieved 98% undetectable MRD in previously untreated CLL patients.

FDA approved revumenib for relapsed/refractory acute leukaemia with KMT2A translocation in patients ≥1 year. Efficacy from AUGMENT-101 study showed 21.2% CR plus CRh rate, median duration 6.4 months. Common adverse reactions included haemorrhage, nausea, and infections. Revumenib available via expanded access for patients <40kg.

Revumenib showed a 63.9% overall response rate in relapsed/refractory KMT2Ar acute leukemia, with manageable safety and no discontinuations due to differentiation syndrome or QTc prolongation. Median duration of response was 6.4 months, and 33.9% of patients proceeded to hematopoietic stem cell transplant.

Revumenib showed continued clinically meaningful responses, including higher MRD-negativity rates and progression to HSCT, in patients with relapsed or refractory KMT2Ar acute leukemia. The safety profile remained manageable, with no discontinuations due to differentiation syndrome or QTc prolongation. The overall response rate was 63.9%, with 22.7% achieving CR or CRh and 42.3% achieving CRc. Median duration of response was 6.4 months, and 33.9% proceeded to HSCT. No new safety signals were reported.

Revumenib (Revuforj) demonstrated clinically meaningful responses in patients with relapsed or refractory acute leukemia with a KMT2Ar translocation in the AUGMENT-101 trial. The drug showed long-term durability in efficacy end points, including MRD-negativity and HSCT procession rates, with a manageable safety profile. The overall response rate was 63.9%, with 22.7% achieving complete remission or CR with partial hematologic recovery, and 42.3% achieving composite CR. No new safety signals were reported, and no patients discontinued due to differentiation syndrome or QTc prolongation.

The SAVE study found that an all-oral revumenib-based combination with decitabine/cedazuridine and venetoclax led to high remission rates in relapsed/refractory acute myeloid leukemia patients, particularly those with KMT2Ar, NPM1mt, and NUP98r genetic alterations, with an overall response rate of 82%. Minimal residual disease negativity was observed in 65% of responders, and the combination showed a stable mutational landscape except for FLT3-ITD expansion and B-ALL plus AML phenotype switch. Common adverse events included febrile neutropenia and lung infection.

Revumenib showed significant responses in relapsed/refractory KMT2Ar acute leukemia patients, with an overall response rate of 63.9% and manageable safety profile, according to the AUGMENT-101 trial.

Revuforj demonstrated effectiveness in treating relapsed/refractory KMT2A acute leukemia, showing high MRD negativity rates and progression to HSCT. The phase 2 AUGMENT-101 trial highlighted manageable safety, with no discontinuations due to differentiation syndrome or QTc prolongation. ORR was 63.9%, with significant CR/CRh rates.