Polaryx Therapeutics, Inc has selected a leading contract research organization (CRO) to support its SOTERIA phase 2 basket trial, advancing the clinical development of its lead candidate, PLX-200, across multiple lysosomal storage disorders (LSDs).

The SOTERIA trial is an open-label, single-arm study designed to evaluate the safety, tolerability, and clinical activity of PLX-200 in patients with CLN2, CLN3, Krabbe disease, and Sandhoff disease. Polaryx estimates these four LSDs represent around one quarter of the overall patient population for these rare disorders. The trial will run at sites in the US, Europe, and Asia or other jurisdictions, with a flexible design aimed at resource-efficient data collection.

“Our selection of a CRO partner marks an important milestone for us as we continue to advance the clinical development of SOTERIA and move closer to the clinic,” Lisa Bollinger, chief medical officer at Polaryx Therapeutics, said: “Our new partner brings meaningful experience working with and managing LSD and rare pediatric trials, as well as deep-rooted relationships with the dedicated key opinion leaders and patient advocacy groups who work closely with patients and their families.”

The SOTERIA trial received a safe to proceed letter from the FDA in October 2025 and is expected to initiate in the first half of 2026. For the CLN2 and CLN3 cohorts, analyses will compare PLX-200 treatment with natural history data as a control arm, providing insights into clinical activity against disease progression. Polaryx intends to use the results to inform the design of future trials, including potential pivotal studies, and may seek conditional marketing authorisation should the data show compelling activity.

PLX-200 is an orally available formulation containing gemfibrozil, a lipid-regulating agent approved for adults with high triglycerides. Preclinical studies indicate PLX-200 crosses the blood-brain barrier, supporting its potential to address neurological manifestations across multiple LSDs. Polaryx believes this mechanism, along with the drug’s established safety profile, could accelerate clinical development and reduce associated costs.

SOTERIA represents a strategic, flexible approach to validate PLX-200’s preclinical science in rare pediatric LSD populations while generating critical data to guide Polaryx’s next steps in clinical development.