Revumenib

Revumenib demonstrated clinically meaningful responses in relapsed or refractory KMT2Ar acute leukemia, with 63.9% overall response rate, including 22.7% complete remission (CR) or CR with partial hematologic recovery (CRh), and 42.3% composite CR (CRc). Safety profile remained manageable, with no discontinuations due to differentiation syndrome or QTc prolongation. Median duration of response for CR+CRh was 6.4 months, with 8 patients remaining in follow-up without relapse or death. 33.9% of patients proceeded to hematopoietic stem cell transplant (HSCT), with 42.9% restarting revumenib post-HSCT.

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.

Syndax Pharmaceuticals presented positive data on Revuforj (revumenib) in R/R AML and KMT2Ar acute leukemia at the 66th ASH Annual Meeting, showing high ORR and CR/CRh rates, rapid and durable responses, and favorable tolerability.

FDA approval of revumenib (Revuforj) in Nov 2024 offers hope for patients with KMT2A-rearranged relapsed/refractory acute leukemia, showing a 21.2% CR/CRh rate in the AUGMENT-101 study. This new, well-tolerated oral therapy could significantly improve survival outcomes for this difficult-to-treat group.

Revumenib (Revuforj) received FDA approval on November 15, 2024, for treating relapsed/refractory acute leukemia with KMT2A translocations, offering an oral, targeted therapy that induces durable remission and enables more patients to proceed to allogenic stem cell transplantation. The approval was supported by the AUGMENT-101 study, showing 21.2% of patients achieved complete remission (CR) or CR with partial hematologic recovery (CRh). Key toxicities include QTc prolongation and differentiation syndrome, managed with regular monitoring and supportive measures. Revumenib's potential future roles include integration into frontline treatment and combination strategies.

Florida Cancer Specialists & Research Institute, LLC (FCS) presents breakthrough research on blood cancers at the 2024 American Society of Hematology Annual Meeting. FCS Director of Drug Development Manish Patel, MD, highlights transformative research advancing next-gen therapies, with over 130 clinical trials available. Co-authored abstracts by FCS hematologists and medical oncologists will be presented, including studies on PRT543, AC676, and belantamab mafodotin. The FCS Real-World Evidence team also presents a study on frontline treatment patterns in newly diagnosed multiple myeloma.

The FDA approved Revuforj (revumenib), a menin inhibitor, for relapsed or refractory acute leukemia with KMT2A gene translocation. Results from the AUGMENT-101 trial showed significant efficacy, with nearly two-thirds of patients achieving complete or partial remission. Revuforj is generally safe, with manageable side effects, and is now available at a cost of $39,500 per month.

FDA approved obecabtagene autoleucel for relapsed/refractory B-cell acute lymphoblastic leukemia, nilotinib tablets without mealtime restrictions for chronic myeloid leukemia, revumenib for relapsed/refractory acute leukemia with KMT2A translocation, zanidatamab for previously treated HER2+ biliary tract cancer, and an oral solution of imatinib for select leukemias and other cancers in November 2024.