obefazimod

Obefazimod is an orally administered, once-daily small molecule drug in late-stage clinical development for the treatment of chronic inflammatory diseases, primarily ulcerative colitis and Crohn’s disease. It is the first and only molecule known to enhance the expression of microRNA-124 (miR-124), a natural regulator of the inflammatory response. By upregulating miR-124, obefazimod stabilizes levels of multiple cytokines and chemokines under dysregulated inflammatory conditions, restoring immune homeostasis without affecting normal immune function. This novel mechanism positions obefazimod as a potential first-in-class therapy—sometimes referred to as a "miR-124 enhancer." The drug was originally conceived for HIV infection but has since shown significant promise in treating ulcerative colitis and other inflammatory bowel diseases. Obefazimod binds to the cap binding complex (CBC) 80/20 and modulates RNA processing[1][2][3][4][8].

Other names

8-chloro-N-[4-(trifluoromethoxy)phenyl]quinolin-2-amine