RGX-104

RGX-104 (also known as IPN0007) is a first-in-class, orally bioavailable small molecule agonist of the Liver X Receptor (LXR), a nuclear receptor that plays a critical role in lipid metabolism and immune regulation. Developed by Inspirna (formerly Rgenix) in collaboration with Ipsen, RGX-104 targets the tumor microenvironment by inducing the expression of Apolipoprotein E (ApoE). ApoE subsequently binds to the LRP8 receptor on myeloid-derived suppressor cells (MDSCs), triggering their apoptosis and thereby relieving the immunosuppression that typically hinders T-cell-mediated anti-tumor responses. RGX-104 is currently being evaluated in Phase 1/2 clinical trials for the treatment of advanced solid tumors, including non-small cell lung cancer and melanoma, as well as lymphomas, both as a monotherapy and in combination with immune checkpoint inhibitors like nivolumab.