Syndax Pharmaceuticals announced positive Phase 2 trial results for revumenib, a menin inhibitor, in patients with relapsed or refractory (R/R) mutant NPM1 (mNPM1) acute myeloid leukemia (AML). The trial met its primary endpoint, demonstrating a complete remission (CR) plus CR with partial hematological recovery (CRh) rate of 23% and an overall response rate of 47% in a heavily pre-treated patient population. The company submitted a supplemental New Drug Application (sNDA) for revumenib in this indication in April 2025.
This news is impactful because mNPM1 AML is the most common genetic alteration in adult AML, affecting about 30% of cases, and currently lacks approved targeted therapies. The positive results offer a potential new treatment option for patients with R/R mNPM1 AML, who typically face a poor prognosis. The high response rates observed, even in patients with multiple prior lines of therapy, including venetoclax, are particularly encouraging, potentially changing the treatment paradigm for this patient subset.
The pivotal Phase 2 data showed a median overall survival of 4 months for all patients and 23.3 months for those achieving CR+CRh. The median time to first CR+CRh was 2.76 months. The safety profile was consistent with prior reports, with common serious adverse events including febrile neutropenia, differentiation syndrome, sepsis, pneumonia, anemia, and QTc prolongation.
This positive data for revumenib in R/R mNPM1 AML, coupled with its existing approval for R/R acute leukemia with a KMT2A translocation, positions Syndax to potentially expand revumenib’s market reach significantly. The ongoing and planned trials evaluating revumenib in combination with standard-of-care treatments, and in newly diagnosed patients, could further strengthen its role in the treatment landscape of acute leukemias. This development underscores the potential of menin inhibition as a therapeutic strategy and may lead to improved outcomes for patients with these challenging cancers.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
