Polaryx Picks CRO for PLX-200 Phase 2 Basket Trial in Four LSDs
Polaryx Therapeutics has appointed a leading contract research organization to conduct SOTERIA, its Phase 2, open-label basket trial of PLX-200 evaluating safety, tolerability and clinical activity in four lysosomal storage disorders. The company received an FDA safe to proceed letter in October 2025 and plans to initiate the trial in H1 2026.
1. CRO Partnership
Polaryx has engaged a contract research organization with demonstrated expertise in rare pediatric lysosomal storage disorder trials and established relationships with key opinion leaders and patient advocacy groups to manage site selection, patient recruitment and trial operations for SOTERIA.
2. Trial Design and Scope
Designed as an open‐label, single‐arm Phase 2 basket trial, SOTERIA will evaluate the safety, tolerability and clinical activity of PLX-200 orally across CLN2, CLN3, Krabbe and Sandhoff disease cohorts, utilizing natural history comparisons for CLN2 and CLN3 arms.
3. Regulatory Status and Timeline
Following an FDA safe to proceed letter received in October 2025, Polaryx plans to launch patient enrollment in the first half of 2026 at sites in the United States, Europe and Asia, aiming for a resource‐efficient pathway to pivotal studies and potential conditional marketing authorization.