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KTX-1001 + pomalidomide + dexamethasone is an investigational **combination therapy** under study for the treatment of **relapsed and refractory multiple myeloma**, with particular focus on patients with high-risk cytogenetics such as the t(4;14) translocation. - **KTX-1001** is a first-in-class, oral, small molecule inhibitor of MMSET/NSD2, a methyltransferase implicated in oncogenic gene expression in high-risk multiple myeloma. By inhibiting NSD2, KTX-1001 reduces the epigenetic mark H3K36me2, restoring normal gene transcription and re-sensitizing tumor cells to therapy. - **Pomalidomide** is an immunomodulatory drug (IMiD) that enhances immune cell cytotoxicity, inhibits angiogenesis, and has direct anti-myeloma activity. - **Dexamethasone** is a corticosteroid that exerts anti-inflammatory and immunosuppressive effects, widely used in myeloma regimens for its ability to reduce tumor burden and manage immune-related side effects. This combination seeks to exploit synergistic activity for heavily pre-treated or refractory multiple myeloma, particularly in molecularly defined high-risk populations.
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