Obefazimod

**Semantic type:** Pharmacologic Substance

**Definition:** An orally available, small molecule binder of the cap binding complex (CBC) 80/20, with potential antiviral and anti-inflammatory activities. Upon oral administration, obefazimod binds to the CBC, a complex at the 5'-end of the pre-mRNA transcript that promotes the initial interaction with transcription and processing machinery. This leads to a conformational change in the CBC and enhanced splicing of viral RNA variants and upregulation of the anti-inflammatory microRNA, miR-124, via splicing of a long noncoding RNA at the miR-124-1 locus. In human immunodeficiency virus type 1 (HIV-1)-infected cells, obefazimod interacts with the CBC of HIV-1 mRNA and inhibits viral replication by preventing Rev-mediated export of unspliced HIV-1 transcripts to the cytoplasm. Rev facilitates the nuclear export of unspliced or incompletely spliced viral pre-mRNAs, an essential step in HIV-1 replication. In inflammatory conditions, miR-124 reduces the production of various inflammatory mediators, including interleukin 6 (IL-6), tumor necrosis factor alpha (TNF-alpha), and C-C motif chemokine 2 (CCL2; MCP-1.). miR-124 plays a critical role in innate and adaptive immune responses and is a critical mediator of cholinergic anti-inflammatory action.

**Synonyms:** - 8-Chloro-N-(4-(trifluoromethoxy)phenyl)quinolin-2-amine - ABX 464 - ABX-464 - ABX464 - CBC 80/20 Binder ABX-464 - Cap Binding Complex 80/20 Binder ABX-464 - OBEFAZIMOD - Rev Inhibitor ABX-464 - SPL-464