PARAMUS, NJ — February 17, 2026 — Leads & Copy — Polaryx Therapeutics, Inc. (Nasdaq: PLYX), a clinical-stage biotechnology company, has chosen a leading contract research organization (CRO) for its SOTERIA trial. This Phase 2 trial is open-label and single-arm, designed to assess the safety, tolerability, and clinical activity of Polaryx’s lead candidate, PLX-200, across four different lysosomal storage disorders (LSDs).
The SOTERIA trial will evaluate PLX-200 in patients with CLN2, CLN3, Krabbe disease, and Sandhoff disease. Polaryx believes these four LSDs represent approximately one-quarter of the total LSD patient population. The trial is designed to be both flexible and resource-efficient, providing essential data for the future clinical development of PLX-200.
PLX-200 is an orally available compound comprised of gemfibrozil. Gemfibrozil is an FDA-approved lipid regulating agent in the fibrate family which has only been approved in a capsule form for adult patients with very high elevations of serum triglyceride levels to decrease serum triglycerides and very low-density lipoprotein cholesterol and increase high density lipoprotein cholesterol. Preclinical trials have documented gemfibrozil’s ability to cross the blood-brain barrier (BBB). The company believes that the established safety profile of gemfibrozil in adults, combined with PLX-200’s ability to cross the BBB, could position it as a potential treatment for unmet needs in multiple rare LSD indications.
Polaryx received a “safe to proceed” letter from the FDA in October 2025 and is actively collaborating with its CRO to begin the SOTERIA trial in the first half of 2026. The trial sites will be located in the United States, Europe, and Asia, or other foreign jurisdictions. For the CLN2 and CLN3 cohorts, the trial will incorporate analyses comparing natural history data as a control arm to PLX-200’s treated arm. Should the data demonstrate compelling clinical activity, Polaryx may seek conditional marketing authorization.
Polaryx Therapeutics, Inc. focuses on developing treatments for rare orphan lysosomal storage disorders (LSDs). Founded in 2014, the company aims to deliver safe and effective treatments addressing the pathophysiology of these diseases. Polaryx integrates small molecule therapies and gene therapy to potentially address both the genetic and downstream pathological features of LSDs. The company’s small molecule drug candidates share similar modes of action that have been demonstrated to address lysosomal dysfunction, neuroinflammation, and neuronal loss in validated animal models.
“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,” said Lisa Bollinger, M.D., Chief Medical Officer of Polaryx Therapeutics, Inc. “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.”
Polaryx continues to pursue its mission of addressing significant unmet medical needs in rare pediatric disorders by responsibly advancing programs that have the potential to deliver meaningful therapeutic options for patients and their families.
Source: Polaryx Therapeutics
