ERX-41

ERX-41 is an orally bioavailable small molecule therapeutic developed by EtiraRx for the treatment of various solid tumors, including pancreatic cancer, triple-negative breast cancer (TNBC), glioblastoma, and ovarian cancer. It specifically targets lysosomal acid lipase A (LIPA), a protein involved in protein folding within the endoplasmic reticulum (ER). By binding to LIPA, ERX-41 disrupts protein folding, leading to excessive ER stress. Because aggressive cancer cells already operate under high basal levels of ER stress, the additional stress induced by ERX-41 triggers apoptosis while sparing healthy cells. The compound was originally discovered by researchers at UT Southwestern and has shown significant preclinical efficacy in hard-to-treat cancers.