PARAMUS, N.J. – August 25, 2026

Polaryx Therapeutics, Inc. announced continued operational progress toward initiating its SOTERIA Phase 2 basket trial evaluating PLX-200 across four rare pediatric lysosomal storage disorders (LSDs). The company remains on track to begin the study in the fourth quarter of 2026, following key regulatory, clinical and financing milestones that have established the infrastructure needed to move the program toward clinical execution. The update highlights trial readiness for PLX-200, rather than new clinical efficacy findings, as Polaryx prepares to activate study sites and begin patient enrollment.

SOTERIA Moves Toward Q4 2026 Trial Initiation

Polaryx Therapeutics has completed several operational steps supporting the planned launch of SOTERIA. The company received FDA authorization to proceed, engaged an experienced contract research organization (CRO) to support trial execution and secured Fast Track Designation for PLX-200 across all four planned indications. Polaryx also completed a $10 million financing intended to support SOTERIA initiation and key clinical milestones. The trial has been registered on ClinicalTrials.gov as NCT07740512, with information covering eligibility and other study details. Participating clinical sites are expected to be added and updated as activation progresses. Together, these developments indicate that the program has moved from regulatory preparation toward the practical execution required for Phase 2 clinical development.

PLX-200 Basket Trial Targets Four Pediatric LSDs

SOTERIA is designed as a Phase 2, open-label, multicenter basket trial evaluating the safety, tolerability, pharmacokinetics and clinical activity of oral PLX-200 in pediatric patients with CLN2 disease, CLN3 disease, Krabbe disease and Sandhoff disease. The study is planned to include children between 2 and 15 years of age across the four disease cohorts. A sentinel group will provide an initial assessment of safety and tolerability before broader enrollment proceeds. After screening and dose titration, participants are expected to enter a 96-week maintenance period, with an interim analysis planned using 52-week maintenance data. The trial will also incorporate regular clinical and biomarker assessments, allowing Polaryx to generate information across multiple rare LSD populations from a single development program. For the CLN2 and CLN3 cohorts, treatment outcomes will be evaluated alongside established natural history data, providing a framework for assessing potential treatment effects against the expected progression of these disorders.

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Polaryx Builds Clinical Execution Strategy for PLX-200

The operational progress surrounding SOTERIA represents an important step in Polaryx’s clinical development strategy for PLX-200. Rather than relying on a single disease indication, the basket design allows the company to investigate the candidate across four devastating pediatric disorders and identify populations where the drug may show the strongest clinical potential. Fast Track Designation across all four indications is also expected to support continued interaction with the FDA as clinical information becomes available. Polaryx has previously reported preclinical activity associated with lysosomal dysfunction, neuroinflammation and neuronal loss, while the upcoming SOTERIA trial is intended to provide clinical evidence needed to guide subsequent development. If compelling clinical activity emerges, the company could use the resulting data to inform potential pivotal studies and expedited regulatory strategies. With regulatory authorization, CRO support, financing and trial registration now in place, the immediate focus is on site activation, patient enrollment and execution of SOTERIA in Q4 2026. The upcoming trial therefore represents the next major catalyst for PLX-200 and a critical test of Polaryx’s multi-indication development approach.

Source: Polaryx Therapeutics,press relese