Polaryx Selects CRO for SOTERIA Phase 2 Basket Trial, Plans Initiation in 1H 2026

Polaryx Therapeutics, Inc. (Nasdaq: PLYX) has selected a leading contract research organization (CRO) to support SOTERIA, its Phase 2, open-label, single-arm basket trial evaluating PLX-200 in four lysosomal storage disorders (LSDs). The company plans to initiate the trial in the first half of 2026.

The SOTERIA trial is designed to assess the safety, tolerability, and clinical activity of Polaryx's lead drug candidate, PLX-200, in CLN2, CLN3, Krabbe disease, and Sandhoff disease, which together represent approximately one quarter of the LSD population. Polaryx received a "safe to proceed" letter from the FDA in October 2025 and plans to initiate SOTERIA in the first half of 2026 across trial sites in the United States, Europe, and Asia.

PLX-200 is an orally available compound comprised of gemfibrozil, an FDA-approved lipid regulating agent in the fibrate family. The ability of gemfibrozil to cross the blood-brain barrier has been documented in third-party preclinical trials, and its safe use in adults has been well-established over several decades, which Polaryx believes accelerates clinical development and reduces associated costs.

The Chief Medical Officer of Polaryx stated that the selection of a CRO partner marks an important milestone for the company as it continues to advance the clinical development of SOTERIA and move closer to the clinic. The CRO partner brings meaningful experience working with and managing LSD and rare pediatric trials, as well as deep-rooted relationships with dedicated key opinion leaders and patient advocacy groups.

For the CLN2 and CLN3 cohorts, although the entire trial is open label, these cohorts will incorporate analyses comparing natural history data as a control arm to PLX-200's treated arm. A natural history study is a preplanned observational study intended to track the course of the disease. Should the data demonstrate compelling clinical activity, Polaryx may seek conditional marketing authorization.

Polaryx Therapeutics is a clinical-stage biotechnology company focused on developing patient-friendly small molecule and gene therapy treatments for rare orphan lysosomal storage disorders. Founded in 2014, the company seeks to deliver safe, effective, and patient-friendly treatments that address the underlying pathophysiology of these catastrophic diseases. The shares of the company's common stock began trading on the Nasdaq Capital Market on February 2, 2026. PLYX has traded between a low of $2.20 and a high of $48.91; the stock is currently trading at $3.64, up 50.80%.