Polaryx Therapeutics (PLYX) investor relations material

Strategic overview

• PLX-200 is an oral PPARα agonist being developed for CLN2, CLN3, Sandhoff and Krabbe disease, with an estimated ~50K total LSD patients across targeted indications.

• The program targets rare pediatric disorders with high unmet need; the presentation positions its disease-modifying approach for multiple LSDs.

• Pipeline candidates include PLX-100, a PLX-200/vitamin A combination; PLX-300, cinnamic acid; and PLX-400, an AAV-9 gene therapy.

SOTERIA trial and catalysts

• SOTERIA is a flexible, open-label, single-arm Phase 2 basket trial evaluating PLX-200 in CLN2, CLN3, Sandhoff and Krabbe, with potential expansion to additional LSDs.

• Trial initiation was planned for Q4 2026, initially at U.S. sites followed by other jurisdictions; the study was listed on ClinicalTrials.gov as NCT07740512.

• The design includes a six-patient sentinel group, safety monitoring by an independent data monitoring committee, and a 96-week maintenance period after titration.

• Catalysts include early safety and biomarker readouts, interim analyses, 54- and 102-week efficacy data versus natural history, and potential BTD or expedited approval discussions.

• CLN2 and CLN3 outcomes will be assessed against DEM-CHILD and URBC natural-history controls, respectively; endpoints include safety, indication-specific clinical activity, plasma NfL and GFAP.

Formulation and mechanism

• A proprietary 15 mg/mL oral solution was developed for pediatric usability; an optimized 60 mg/mL solution is 4x more concentrated and is expected to improve compliance and dose control.

• The optimized formulation uses a proprietary buffer and solubilization system; the presentation reports no API degradation and improved bioavailability in a three-month stability report.

• PLX compounds activate the PPARα-TFEB pathway, promoting lysosomal gene expression, lysosomal biogenesis and cellular waste clearance.

• Proposed additional effects include anti-inflammatory IL-1Ra and SOCS3 expression, increased GDNF and reduced apoptosis through BAD phosphorylation.