Polaryx Therapeutics (PLYX) investor relations material

Company overview and business model

• Clinical-stage biotechnology company focused on developing novel, disease-modifying therapies for rare, pediatric lysosomal storage disorders (LSDs).

• Pipeline includes small molecule therapies (PLX-200, PLX-300, PLX-100) and a preclinical gene therapy (PLX-400), targeting multiple LSDs with high unmet need.

• Lead candidate PLX-200 (gemfibrozil) is being advanced through a Phase 2 proof-of-concept basket trial (SOTERIA) for CLN2, CLN3, Krabbe disease, and Sandhoff disease, with trial initiation expected in the first half of 2026.

• Multi-modal approach aims to address both genetic and downstream pathological features of LSDs, leveraging mechanisms such as lysosomal biogenesis, reduction of neuroinflammation, and neuronal support.

• Business model includes leveraging regulatory designations (orphan drug, fast track, rare pediatric disease) and pursuing the 505(b)(2) pathway for accelerated development.

Financial performance and metrics

• No products approved for sale and no revenue generated to date; operations funded primarily through $21.7 million in equity financing.

• Net loss of $30.4 million for the year ended December 31, 2024, and $7.5 million for the nine months ended September 30, 2025; accumulated deficit of $98.1 million as of September 30, 2025.

• Cash and cash equivalents of $5.7 million and working capital of $5.3 million as of September 30, 2025.

• Operating expenses for the nine months ended September 30, 2025, were $6.9 million, primarily driven by research and development costs related to PLX-200.

• Management expects existing capital to fund operations through the third quarter of 2026 and anticipates the need for additional financing thereafter.

Use of proceeds and capital allocation

• The direct listing registers 47,343,297 shares for resale by existing stockholders; the company will not receive any proceeds from these sales.

• Proceeds from prior financings have been allocated to research and development, clinical trial preparation, and general corporate purposes.

• Future capital needs will be driven by clinical trial progression, manufacturing scale-up, regulatory activities, and potential commercialization efforts.