Lou, 14 ans, atteinte d’une leucémie aiguë agressive, est décédée le 24 septembre. Cet été, sa famille s’était mobilisée pour lui permettre d’accéder au revuménib, un traitement ciblé difficilement disponible en France.

Revumenib added to intensive chemotherapy produced deep, MRD-negative remissions with a manageable safety profile in newly diagnosed AML.

Wen et al. show that menin inhibition suppresses megakaryopoiesis in the myeloproliferative neoplasms, an area of unmet medical need due to disease progression despite current therapies. In preclinical models, the menin inhibitor revumenib exhibits striking anti-tumor activity, supporting its further investigation as a potential treatment for the disease.

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– Total revenue of $73 million in 2Q26, a 92% year-over-year increase – – Revuforj ® (revumenib) net revenue of $54.7 million in 2Q26, a 91% year-over-year increase – – Niktimvo™ (axatilimab-csfr) net revenue of $60.3 million in 2Q26, a 67% year-over-year increase, resulting in Syndax collaboration

Researchers evaluated an all-oral combination of revumenib, decitabine, and venetoclax in patients with heavily pretreated AML.

Für Patienten mit der häufigsten AML-Mutation gibt es erstmals eine zielgerichtete Therapie: Die FDA hat Revumenib für NPM1-mutierte Leukämie zugelassen. Die Kombinationsdaten zeigen Ansprechraten von 100 Prozent.

Wen et al. show that menin inhibition suppresses megakaryopoiesis in the myeloproliferative neoplasms, an area of unmet medical need due to disease progression despite current therapies. In preclinical models, the menin inhibitor revumenib exhibits striking anti-tumor activity, supporting its further investigation as a potential treatment for the disease.

Für Patienten mit der häufigsten AML-Mutation gibt es erstmals eine zielgerichtete Therapie: Die FDA hat Revumenib für NPM1-mutierte Leukämie zugelassen. Die Kombinationsdaten zeigen Ansprechraten von 100 Prozent.

Menin inhibitors have emerged as a targeted therapeutic strategy for patients with acute leukemias driven by KMT2A gene rearrangements (KMT2Ar) and NPM1 mutations, where aberrant menin-KMT2A interactions sustain HOX-MEIS1-dependent transcriptional programs that are critical for leukemogenesis [1,2,3,4]. Positive early-phase studies led to regulatory approval of both revumenib and ziftomenib for these leukemia subtypes [5, 6]. Despite these advances, acquired resistance frequently limits durability of menin inhibitor treatment response [7]. We previously characterized somatic MEN1 mutations as a recurrent genetic mechanism of on-target resistance to menin inhibition [7]. These mutations impair drug binding while preserving the ability of menin to scaffold leukemogenic chromatin complexes, thereby maintaining HOX gene expression. Currently, detection of MEN1 resistance mutations relies on next-generation sequencing (NGS) of genomic DNA (gDNA) derived from bone marrow aspirates. Although informative, marrow-based testing is invasive and logistically challenging for serial monitoring, particularly in patients with low circulating blast counts or in morphologic remission. Plasma-derived cell-free DNA (cfDNA) analysis offers a complementary strategy that enables minimally invasive, repeated sampling and may better capture these mutations across disease compartments. This is especially advantageous in the pediatric setting, where bone marrow aspiration generally necessitates sedation. High-sensitivity cfDNA assays can detect low-frequency variants, enabling the...

Phase 1/2 SAVE data show revumenib plus decitabine/cedazuridine and venetoclax achieved 88% ORR in heavily pretreated AML patients.

– Real-world study of revumenib shows 82% (9/11) ORR and 64% (7/11) CR/CRh rate among R/R NPM1m or KMT2Ar patients treated with revumenib monotherapy or combinations – – Ph 1 trial of revumenib with intensive chemotherapy in newly diagnosed NPM1m or KMT2Ar AML shows 97% (34/35) CRc and 86% (25/29)

Syndax (Nasdaq:SNDX) reported new Revuforj® (revumenib) data from ASCO 2026, including post-transplant and pharmacokinetic results.In 24 adults and children with high-risk acute leukemia resuming revumenib after HSCT, the 2-year overall survival rate was 90% vs a historical 51%, with low relapse rates and manageable thrombocytopenia. PK data from 335 patients showed flexible dosing with gastric acid reducers, CYP3A4 inhibitors, and varying food conditions.

Syndax (Nasdaq:SNDX) announced that four Revuforj (revumenib) abstracts were accepted for presentation at ASCO 2026, including an oral post-transplant maintenance study in 21 adults and children with acute leukemia.Additional posters cover revumenib pharmacokinetics and designs of two phase 3 trials in newly diagnosed NPM1m or KMT2Ar AML.

– Abstracts highlight strong revumenib activity across the acute leukemia treatment continuum and multiple genetic subtypes – – New real-world data show compelling outcomes and favorable tolerability with revumenib monotherapy and combination use – – Two abstracts highlight encouraging results with

– Total revenue of $64.9 million in 1Q26, a 224% year-over-year increase – – Revuforj ® (revumenib) net revenue of $48.9 million in 1Q26, highlighting leadership in menin inhibition and increasing uptake in R/R NPM1m AML – – Niktimvo™ (axatilimab-csfr) net revenue of $55.1 million in 1Q26,

Actimab-A combinations enhanced in vivo AML cell killing across multiple preclinical models, independent of mutation status, when combined with standard-of-care targeted and non-targeted therapies including revumenib (menin-KMT2A inhibitor), gilteritinib (FLT3 inhibitor), and azacitidine...