Revumenib

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.

Sonrotoclax, BeiGene’s next-gen inhibitor, targets WT and mutated Bcl-2 by binding within a hydrophobic groove, addressing first-gen limitations.

The FDA approved revumenib, a first-in-class menin inhibitor, for treating acute leukemias with a KMT2A translocation. Based on the AUGMENT-101 trial, 21.2% of patients achieved complete remission or complete remission with partial hematopoietic recovery. The recommended dose is 270 mg orally twice a day for patients weighing 40 kg or more, with adjustments for body surface area and CYP3A4 inhibitors.

November 2024 saw numerous FDA approvals, designations, and clinical trial advancements in oncology, including revumenib for KMT2A-rearranged acute leukemia, obe-cel for relapsed/refractory B-cell ALL, and fast track designations for ALE.P02 in Claudin-1-positive tumors. Other highlights include orphan drug designations for LBL-024 in neuroendocrine cancer and elraglusib in Ewing sarcoma, and a new drug application for sunvozertinib in EGFR-mutant NSCLC.

SELLAS Life Sciences Group identifies ASXL1 mutation as key predictor of SLS009 response in solid cancers, with 67% efficacy observed in ASXL1 mutated cancers vs 0% in non-mutated cancers. ASXL1 mutations were found in colorectal cancer (CRC MSI-H) and non-small cell lung cancer (NSCLC), supporting targeted SLS009 clinical trials.