Polaryx Therapeutics is a clinical-stage biotech developing therapies for lysosomal storage diseases (LSDs), a group of roughly 50 rare inherited metabolic disorders that impair lysosomal function and typically cause severe neurological decline in infants and young children. Polaryx focuses on a subset of LSDs with high unmet need: neuronal ceroid lipofuscinoses (NCLs), Krabbe disease, Sandhoff disease, and Niemann-Pick disease types A and B. The company has no approved products and no revenue. Polaryx's lead candidate, PLX-200, is a pediatric oral solution formulation of gemfibrozil — an existing FDA-approved lipid drug — being repurposed as a disease-modifying therapy for multiple LSDs simultaneously. PLX-200 works by activating PPARα, which upregulates TFEB, a regulator of lysosomal function, driving clearance of accumulated waste and reducing neuroinflammation. Because gemfibrozil is already FDA-approved for adults, Polaryx is pursuing the 505(b)(2) regulatory pathway, which can leverage existing safety data to reduce development risk. PLX-200 is cleared to proceed into SOTERIA, a Phase 2 basket trial across CLN2, CLN3, Krabbe, and Sandhoff disease, expected to initiate in the second half of 2026. The pipeline also includes PLX-300 (cinnamic acid, IND-enabling stage), PLX-100 (gemfibrozil plus vitamin A, preclinical), and PLX-400 (an intranasal AAV gene therapy, preclinical). Polaryx is a lean organization with 11 employees, outsourced manufacturing, and research licensed from Rush University Medical Center.