ERX-208

ERX-208 is a small molecule investigational drug being developed by EtiraRx for the treatment of ovarian cancer. It functions by binding to lysosomal acid lipase (LIPA), which induces a robust endoplasmic reticulum (ER) stress response in cancer cells, ultimately leading to cell death via apoptosis. Preclinical studies have demonstrated that ERX-208 is active against all four molecular subtypes of ovarian cancer with high potency (IC50 of approximately 50-100 nM), representing a five-fold increase in potency compared to its related compound, ERX-41. The drug has shown significant anti-tumor efficacy in various preclinical models, including cell line-derived xenografts (CDXs), patient-derived xenografts (PDXs), and patient-derived organoids (PDOs), with no observable toxicity at efficacious doses. ERX-208 is currently in preclinical development with plans to advance toward clinical trials.