Drug intelligence / Profile preview

rigosertib + azacitidine

Development stage
Unknown
Lead developer
Onconova Therapeutics
Modality
Small Molecules
Administration
Oral (rigosertib), Intravenous/subcutaneous (azacitidine)
01

Overview

Rigosertib + azacitidine is an investigational combination therapy for myeloid malignancies, primarily myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). Rigosertib is a small molecule Ras-mimetic that inhibits Ras-effector pathways by interfering with the RAS-binding domain of RAF kinases and inhibiting both the RAS-RAF-MEK and PI3K pathways. Azacitidine is a hypomethylating agent that incorporates into DNA and RNA, leading to inhibition of DNA methyltransferase and subsequent epigenetic modulation. The combination aims to overcome resistance to hypomethylating agents in high-risk MDS or AML patients. Clinical studies have shown promising response rates in both HMA-naïve and HMA-refractory populations, with manageable toxicity profiles[1][2][4][5].

Brand names
Vidaza (azacitidine)
02

Targets

DNMT (DNA methyltransferase)PI3K (Phosphoinositide-3-kinase regulatory subunit 6)RAF1 (c-Raf-1 (Y340D/Y341D))TUBB (Tubulin (alpha and beta subunits))

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