Drug intelligence / Profile preview

cyclophosphamide + ruxolitinib

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

Overview

Cyclophosphamide + ruxolitinib is a combination therapy consisting of two small molecule drugs with distinct but potentially synergistic mechanisms. Cyclophosphamide is an alkylating agent that cross-links DNA, leading to cell death, and also exerts immunosuppressive and immunomodulatory effects depending on the dose. Ruxolitinib is a selective inhibitor of Janus kinase 1 (JAK1) and Janus kinase 2 (JAK2), blocking the JAK-STAT signaling pathway involved in cytokine-mediated immune responses. This combination has been investigated for its ability to modulate immune cell populations—such as reducing T helper 17 cells while increasing regulatory T cells—and for prophylaxis or treatment of graft-versus-host disease (GvHD) after allogeneic hematopoietic stem cell transplantation, as well as in experimental models of glomerulonephritis. The rationale for combining these agents lies in their complementary actions on both malignant and immune cells.

02

Targets

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