The direct NLRP3 inhibitor and Phase 3 small molecule anticancer agent, RRx‐001, protects aged triple transgenic Alzheimer’s disease model mice from CNS degeneration and cognitive decline

RRx‐001 is an aerospace‐derived, intravenously administered NLRP3‐specific inhibitor with confirmed CNS‐penetrant properties and a favorable safety profile in a Phase 3 clinical trial. It is under investigation as an anticancer agent, a chemoradioprotector for oral mucositis and a prophylactic for acute graft vs. host disease (aGVHD). RRx‐001 has been shown to reduce interleukin‐1β (IL‐1β) production in vivo and to attenuate the severity both of experimental autoimmune encephalomyelitis (EAE), a disease model of multiple sclerosis, and dextran sulfate sodium (DSS)‐induced colitis.