PLX-200 Preclinical Data Show Motor Recovery and Myelin Protection in Krabbe Model
Polaryx’s PLX-200 (reformulated gemfibrozil) restored motor function in GALC-/- mouse models by reducing psychosine accumulation, astrogliosis and neuro-inflammation and protecting myelin. The FDA granted a safe-to-proceed letter in October 2025 for the Phase 2 SOTERIA trial in four lysosomal storage disorders, starting H1 2026.
1. Preclinical Efficacy in Krabbe Model
In GALC-/- mouse models of Krabbe disease, orally administered PLX-200 reduced psychosine accumulation, astrogliosis and neuro-inflammation while preserving myelin. Treated animals displayed restored motor function and increased lifespan compared with untreated controls.
2. FDA Safe-to-Proceed and SOTERIA Trial Launch
Polaryx received a safe-to-proceed letter from the FDA in October 2025, greenlighting the Phase 2 SOTERIA basket trial. The open-label, single-arm study will evaluate PLX-200’s safety, tolerability and clinical activity in CLN2, CLN3, Krabbe and Sandhoff diseases, with initiation planned for the first half of 2026.
3. Mechanism of Action and Broader Development
PLX-200 activates PPARα and recruits PPARβ to myelin gene promoters, boosting lysosomal biogenesis, autophagy and anti-inflammatory pathways. This multi-modal mechanism supports its potential application across multiple rare lysosomal storage disorders beyond Krabbe disease.