Drug intelligence / Profile preview

ruxolitinib + decitabine

Development stage
Phase 2
Lead developer
Incyte
Modality
Small Molecules
Administration
Oral, Intravenous
01

Overview

Ruxolitinib + decitabine is an investigational **small‑molecule combination regimen** that pairs the selective Janus kinase 1/2 (JAK1/JAK2) inhibitor ruxolitinib with the hypomethylating agent decitabine for the treatment of advanced myeloid malignancies, particularly accelerated‑phase or blast‑phase myeloproliferative neoplasms (MPN‑AP/BP) and high‑risk acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) in the peri–allogeneic stem cell transplant setting.[1][4][6] Ruxolitinib suppresses aberrant JAK‑STAT signaling to reduce malignant myeloid proliferation and inflammatory cytokine production, while decitabine incorporates into DNA and inhibits DNA methyltransferases, leading to DNA hypomethylation, re‑expression of silenced genes, and antileukemic effects.[1][2][4][6] Preclinical and early‑phase clinical studies have shown synergistic antileukemic activity, acceptable tolerability, and meaningful response rates when ruxolitinib and decitabine are administered together in 28‑day cycles or as part of intensified busulfan–cyclophosphamide conditioning regimens before allogeneic hematopoietic stem cell transplantation.[1][4][6]

Other names
ruxolitinib and decitabinedecitabine/ruxolitinibruxolitinib combined with decitabine
02

Targets

DNADNMT1 (DNA (cytosine-5)-methyltransferase 1)JAK1 (Janus kinase 1)JAK2 (Janus kinase 2)

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