Polaryx Therapeutics, Inc.

Polaryx Therapeutics, Inc. (Nasdaq: PLYX) is a clinical-stage biotechnology company developing therapies for rare, pediatric lysosomal storage disorders (LSDs). Its pipeline includes four candidates: PLX-200, an oral gemfibrozil formulation and its lead program; PLX-300, an oral small molecule in IND-enabling studies; PLX-100, a preclinical oral combination therapy; and PLX-400, a preclinical gene therapy. The small-molecule programs are designed to address lysosomal dysfunction, inflammation and neuronal loss. Polaryx is advancing PLX-200 for multiple LSDs, including CLN2 and CLN3 neuronal ceroid lipofuscinosis, Krabbe disease and Sandhoff disease, and plans a Phase 2 open-label basket trial across those indications. It is also pursuing PLX-200 through the FDA’s 505(b)(2) pathway. The company focuses on diseases that include neuronal ceroid lipofuscinoses, Krabbe disease, Tay-Sachs and Sandhoff diseases, and Niemann-Pick types A and B. Its programs are at preclinical or clinical development stages; the company describes approximately 50,000 patients across the United States, Europe and select other regions as affected by LSDs.