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Drug intelligence / Profile preview
azacitidine + ruxolitinib
- Development stage
- Phase 2
- Lead developer
- Incyte
- Modality
- Small Molecules
- Administration
- Subcutaneous, Intravenous, Oral
01
Overview
Azacitidine + ruxolitinib is an investigational combination therapy consisting of two small molecule drugs, azacitidine and ruxolitinib, used primarily in the treatment of myelofibrosis (MF) and other myeloproliferative neoplasms (MPNs). Azacitidine is a hypomethylating agent that incorporates into DNA and RNA, leading to DNA hypomethylation and cytotoxicity in abnormal hematopoietic cells. Ruxolitinib is a selective inhibitor of Janus kinase 1 (JAK1) and Janus kinase 2 (JAK2), blocking aberrant JAK-STAT signaling implicated in MPN pathogenesis. The combination has demonstrated synergistic effects, improving spleen response rates, bone marrow fibrosis, symptom burden, and overall survival compared to monotherapy. It is being studied in phase 2 clinical trials for MF with promising efficacy and manageable safety profile[1][3][4][8].
02
Targets
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