Polaryx registers Phase 2 basket trial of PLX-200 across four pediatric lysosomal disorders

Polaryx Therapeutics added one new pediatric trial to the registry this week.

• PLX-200, an oral solution also known as gemfibrozil, is being tested in a Phase 2 study titled "An Open-Label, Multicenter, Phase 2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients With Lysosomal Storage Disorders (SOTERIA)"¹. The trial covers pediatric participants with lysosomal storage disorders including CLN2, CLN3, Sandhoff disease, and Krabbe disease¹, with dosing given twice daily for about 101 weeks, split between a 5-week titration period and a 96-week maintenance period¹. The study is listed as not yet recruiting, with an estimated enrollment of 24 participants¹, and an estimated start date of 2026-12-01¹.