New Clinical Data Highlight Revumenib’s Potential Beyond KMT2A-Rearranged Leukemia

New results presented at the 2025 European Hematology Association (EHA) Congress have expanded on revumenib’s (Revuforj; Syndax) therapeutic potential in genetically defined acute myeloid leukemia (AML). The first-in-class menin blocker was approved by the FDA in November 2024 for the treatment of relapsed or refractory (R/R) acute leukemia with a KMT2A translocation in adult and pediatric patients.¹ The first approval was a big step in precision oncology, but the new data hint that its help may reach more patients with a different gene issue, those with NPM1-mutant AML.²

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Revumenib stops the link between the menin protein and KMT2A fusion proteins, a process that drives oncogenic transcriptional programs in certain leukemias. This link promotes malignant cell growth and survival. By blocking this link, revumenib targets a key genetic cause of the disease, differentiating it from conventional chemotherapy.³ The drug is very precise at a molecular level, which granted it breakthrough therapy and orphan drug designations before its late-2024 approval.

Findings in NPM1-Mutant AML

At the 2025 EHA Congress, Syndax Pharmaceuticals presented new AUGMENT-101 (NCT04065399) study data for patients with R/R AML harboring NPM1 mutations. In this group of 77 patients, the complete remission (CR) and CR with partial hematologic recovery (CRh) rate was approximately 26% with a duration of response of 4.7 months and an average time to first response of 2.8 months.² Notably, deep molecular fixes were observed, with measurable residual disease (MRD)-negativity achieved in 63% of evaluable responders. These findings suggest that revumenib’s work goes past its first approved use and may work well in fighting targeted leukemias with genetic mutations.

The depth and durability of responses in NPM1-mutant AML, mainly the high rate of MRD negativity, show that revumenib could address a significant unmet need in these patients. The promising data encouraged the FDA to speed up the review of a supplemental new drug application for the use of revumenib in NPM1-mutant AML. The Prescription Drug User Fee Act is set for a goal date of October 25, 2025, and if approved, revumenib would be one of the few key therapies available for R/R NPM1-mutant AML cases, potentially changing the standard of care.²

Role in Precision Oncology and Pharmacy Practice

As the use of revumenib grows, oncology pharmacists will play a key role in clinical integration. Their responsibilities will cover verifying genetic eligibility through molecular testing, setting the most fitting dosage schedules, managing any adverse effects, and educating both patients and health care groups about how the drug works and what to expect from it. With new data showing it helps in many specific gene-based leukemia types, revumenib (Revuforj; Syndax) boosts the move toward aimed, biomarker-driven cancer treatment.

The new EHA 2025 data show strong evidence that revumenib's therapeutic reach may soon reach past KMT2A-rearranged acute leukemia to include NPM1-mutant AML. The mix of positive remission rates, long-lasting outcomes, and high MRD-negativity marks its chance to help more high-risk groups of patients. With decisions from the FDA still to come, revumenib could soon have a wider part in precision oncology, making its mark as a key innovation in the treatment of acute leukemias.

REFERENCES

1. Le K, Ayaa Kobaissi, PharmD Candidate, Kwon E, Farooq M, Ali A. In a Nod to Precision Oncology, the FDA Approves Revumenib. Pharmacy Times. 2025;7. Accessed August 14, 2025. https://www.pharmacytimes.com/view/in-a-nod-to-precision-oncology-the-fda-approves-revumenib

2. Syndax Presents New Revuforj® (revumenib) Data in Relapsed/Refractory mNPM1 and NUP98r Acute Leukemia from AUGMENT-101 Trial at EHA 2025 | Syndax Pharmaceuticals, Inc. Syndax Pharmaceuticals, Inc. Published 2025. https://ir.syndax.com/news-releases/news-release-details/syndax-presents-new-revuforjr-revumenib-data-relapsedrefractory

3. Reuters Staff. US FDA approves Syndax’s blood cancer drug. Reuters. https://www.reuters.com/business/healthcare-pharmaceuticals/us-fda-approves-syndaxs-blood-cancer-drug-2024-11-15/. Published November 15, 2024.