Polaryx Therapeutics (PLYX) Corporate presentation summary

Strategic focus and market opportunity

• Dedicated to rare pediatric lysosomal storage disorders (LSDs) with disease-modifying drug candidates targeting highly unmet orphan indications.

• Aims to establish new standards of care for LSDs, unlocking multi-billion-dollar market opportunities.

• Multiple regulatory designations and a validated open-label basket trial design support accelerated development.

• Proprietary oral formulations and robust IP protection enhance patient adoption and market defense.

Pipeline and clinical development

• Lead candidate PLX-200 is an oral small molecule with IND-approved Phase 2 basket trial (SOTERIA) for CLN2, CLN3, Krabbe, and Sandhoff diseases.

• PLX-200 activates PPARα, promoting lysosome biogenesis and anti-inflammatory gene expression.

• Additional candidates include PLX-300 (antioxidant/anti-inflammatory), PLX-100 (combination therapy), and PLX-400 (gene therapy).

• SOTERIA trial initiation expected in H1 2026 across US, Europe, and Asia.

Clinical trial design and strategy

• SOTERIA Phase 2 is a flexible, open-label, single-arm basket trial evaluating PLX-200 in multiple LSDs for up to 101 weeks.

• Sentinel groups in CLN2 and CLN3 cohorts assess early safety and tolerability.

• Trial design allows for accelerated approval discussions if compelling data emerge.

• Adaptive design leverages natural history data and enables inclusion of additional indications.