Polaryx Therapeutics, Inc.

Polaryx Therapeutics, Inc. is a clinical-stage biotechnology company focused on discovering, developing, and commercializing disease-modifying therapies for rare, pediatric lysosomal storage disorders (LSDs). The company's approach integrates small molecule therapies, including a combination therapy, and a gene therapy. Its small molecule drug candidates target lysosomal dysfunction, neuroinflammation, and neuronal loss. The most advanced product candidate is PLX-200 (gemfibrozil), an oral small molecule for the treatment of LSDs. PLX-200 is a repurposed, reformulated drug pursued through a 505(b)(2) regulatory pathway and designed as a novel oral solution. Polaryx Therapeutics is advancing PLX-200 through a Phase 2 proof-of-concept basket trial, SOTERIA (PLX-200-600), expected to initiate in the second half of 2026. SOTERIA is an open-label, multi-indication master study for certain LSDs, which represent approximately one quarter of the LSD population. These include Classic Late Infantile Neuronal Ceroid Lipofuscinosis (CLN2) and Juvenile Neuronal Ceroid Lipofuscinosis (CLN3) subtypes of neuronal ceroid lipofuscinosis (NCLs), Krabbe disease, and Sandhoff disease. The company held a pre-investigational new drug (IND) submission meeting in April 2025, submitted an IND application to the U.S. Food and Drug Administration (FDA) for the SOTERIA trial in August 2025, and received a safe to proceed letter in October 2025.