Syndax Launches Revuforj® for Relapsed/Refractory Acute Leukemia Treatment
Syndax Pharmaceuticals has launched Revuforj® (revumenib), the first and only menin inhibitor for treating relapsed or refractory (R/R) acute leukaemia with a lysine methyltransferase 2A (KMT2A) gene translocation in adults and paediatric patients aged one year and older.
Revuforj (revumenib) is an oral, first-in-class menin inhibitor that has received FDA approval for treating R/R acute leukaemia with KMT2A translocation in both adult and paediatric patients aged one year and above.
Revumenib is also being developed for R/R acute myeloid leukaemia (AML) with a nucleophosmin 1 mutation (mNPM1). Positive pivotal results from the AUGMENT-101 trial showed promising outcomes for revumenib as a monotherapy in this patient group.
The efficacy of Revuforj was assessed through an FDA analysis of 104 patients with R/R acute leukaemia and a KMT2A translocation, who participated in the Phase 1/2 AUGMENT-101 trial.
The most common adverse reactions (≥20%), including laboratory abnormalities, were hemorrhage, nausea, increased phosphate levels, musculoskeletal pain, infection, elevated aspartate aminotransferase, febrile neutropenia, increased alanine aminotransferase, increased parathyroid hormone intact, bacterial infection, diarrhoea, differentiation syndrome, prolonged QT on electrocardiograms, decreased phosphate levels, increased triglycerides, decreased potassium, reduced appetite, constipation, oedema, viral infection, fatigue, and increased alkaline phosphatase.
The median duration of CR+CRh was 6.4 months (95% CI: 2.7, not estimable), with a median time to achieve CR or CRh of 1.9 months (range: 0.9–5.6 months). Following treatment with Revuforj, 23% (24 out of 104) of patients proceeded to haematopoietic stem cell transplantation (HSCT).
Additionally, multiple clinical trials are ongoing to evaluate revumenib in combination with standard-of-care agents for mNPM1 AML or KMT2A-rearranged acute leukaemia, including studies involving newly diagnosed patients.
