RGX-202-01

RGX-202-01 is an oral, first-in-class small molecule inhibitor of the creatine transporter SLC6A8. By inhibiting SLC6A8, RGX-202-01 blocks the import of phosphocreatine into cancer cells, leading to depletion of intracellular phosphocreatine and ATP, which induces apoptosis. This mechanism targets energy metabolism in tumor cells and has shown particular activity in KRAS-mutant colorectal cancer. The drug is being developed for use in combination with standard chemotherapy regimens (FOLFIRI and bevacizumab) for advanced or metastatic colorectal cancer. Clinical studies have demonstrated robust target inhibition and antitumor activity, especially in KRAS-mutant tumors[1][4][5].