Polaryx Therapeutics is a clinical-stage biotechnology company developing novel disease-modifying therapies for rare, pediatric lysosomal storage diseases (LSDs). Its pipeline includes PLX-200 (gemfibrozil, oral small molecule), PLX-300 (cinnamic acid), PLX-100 (combination therapy), and PLX-400 (gene therapy). Lead candidate PLX-200 is advancing through a Phase 2 basket trial (SOTERIA) targeting CLN2, CLN3, Krabbe disease, and Sandhoff disease, with an FDA-cleared IND received in October 2025.
Repurposed, reformulated oral small molecule for treatment of multiple LSDs including CLN2, CLN3, Krabbe, and Sandhoff disease; advancing through Phase 2 SOTERIA basket trial via 505(b)(2) pathway.
PLX-300 (cinnamic acid)
platform
Novel oral small molecule in IND-enabling studies for LSDs including GM2 gangliosidosis, Krabbe disease, and Niemann-Pick Disease types A and B.
PLX-100
platform
Preclinical oral combination therapy comprising PLX-200 (PPAR agonist) and vitamin A (RXR agonist) for treatment of LSDs, including CLN2.
PLX-400
platform
Preclinical gene therapy for LSDs; being explored as monotherapy or in combination with PLX-200.
Partnerships
Partner
Type
Description
Mstone Partners Healthcare Limited
strategic
Controlling stockholder; signed Services Agreement in November 2021 to provide mission-critical operational services.
Origin — Data Provenance
This profile was extracted from SEC EDGAR filings (PEM-signed by sec.gov).
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