AVA-ADR-001

Conclusions To our knowledge, AVA-ADR-001 is the first disclosure of a selective small molecule inhibitor of ADAR1 p150. AVA-ADR-001 is a potent and selective first-in-class ADAR1 inhibitor which has shown significant IFN induction in vitro and in vivo in the tumor microenvironment resulting in substantial tumor growth inhibition as monotherapy and synergistically in combination with Anti-PD1.

To our knowledge no selective small molecule inhibitors of ADAR1 have been reported so far and AVA-ADR-001 is the first disclosure of such an inhibitor. AVA-ADR-001 is a potent and selective first-in-class ADAR1 inhibitor which has shown significant IFN induction in various cancer cell lines and in vivo in the tumor microenvironment resulting in substantial tumor growth inhibition as monotherapy and synergistically in combination with Anti-PD1. Considering the immune-suppressive and pro-metastatic role of ADAR1, AVA-ADR-001 serves as a promising starting point for novel ADAR1 inhibitors as therapeutic modalities in cancer immunotherapy.