Polaryx Therapeutics, Inc. · NASDAQ:PLYX
Polaryx Therapeutics — Investor Presentation (October 2026)
33 slides from Polaryx Therapeutics's investor presentation, October 2026
Slide 1: Polaryx Therapeutics Corporate Presentation
Polaryx Therapeutics Corporate Presentation October 2026 ©2026 Polaryx Therapeutics, Inc. All rights reserved. 1
Slide 2: Disclaimers and Forward-looking Statements
Disclaimers and Forward-looking Statements Other than statements of historical facts, all statements included in this presentation are forward-looking statements, including statements about our plans for our current and future product candidates, the anticipated trial design and timing of initiation
Slide 3: About Polaryx Therapeutics
Section 1 About Polaryx Therapeutics 3
Slide 4: Guided by Leaders with a Strong Track Record in Rare Diseases
Guided by Leaders with a Strong Track Record in Rare Diseases Management Team Alex Yang, JD, LLM G. Michael Landis, CPA Will Charlton, MD, MAS Jay Vijayan, PhD, MBA Andrew O Eddy Zhu, PhD, PMP Minsu Kang, PhD Chief Executive Officer, Chair of Chief Financial Officer, Chief Medical Officer Chief Scie
Slide 5: Polaryx Therapeutics' Key Highlights
Polaryx Therapeutics’ Key Highlights Experienced Team in Orphan Pediatric Diseases Marked by High Unmet Need 1 Deep expertise in orphan pediatric diseases backed by a proven track record in the acquisition of Epygenix Therapeutics by Harmony Biosciences (NASDAQ:HRMY); PLX-200 addresses significant u
Slide 6: Our Clinical Development Strategy
Our Clinical Development Strategy Advancing SOTERIA: A phase 2 open-label basket trial Drug Targeted Timing of Key Catalysts Mechanism Indication Candidate 4Q 2026 2027 2028 2029 2030 PPARα SOTERIA Basket Trial PLX-200 Agonist (CLN2 disease, CLN3 disease, Interim Analysis 1 Interim Analysis 2 Toplin
Slide 7: The SOTERIA Phase 2 Trial
The SOTERIA Phase 2 Trial Highly flexible trial designed with multiple inflection points (NCT07740512) SOTERIA is a flexible, single-arm, Phase 2 basket trial evaluating PLX-200 across 4 target indications, including CLN2, CLN3, Sandhoff and Krabbe disease, with the potential to expand into addition
Slide 8: The SOTERIA Phase 2 Trial
The SOTERIA Phase 2 Trial Open-label basket trial with multiple catalysts designed to drive value and expedite approval CLN2 and CLN3 subjects will be evaluated against natural history control arms (DEMCHILD for CLN2 and URBC for CLN3), with favorable data supporting expedited approval discussions •
Slide 9: SOTERIA Progress Update
SOTERIA Progress Update Europe/UK Now Live On Clinicaltrials.gov United States (NCT07740512) Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Polaryx plans to initiate SOTERIA in the fourth quarter Patients of 2026 with trial sites in the United States followe
Slide 10: Scientific Background and Preclinical Studies
Section 2 Scientific Background and Preclinical Studies 10
Slide 11: Our Novel Drug Formulation
Our Novel Drug Formulation Improving pediatric patient compliance and potentially enhancing dosing accuracy • Meaningful current competitive advantage through delivery of proprietary patient-friendly LSD treatments by proprietary oral formulation – Designed to improve bioavailability versus existing
Slide 12: Our Target Indications: Lysosomal Storage Disorders
Our Target Indications: Lysosomal Storage Disorders What are Lysosomal Storage Disorders (LSDs)? Neuronal Ceroid Lipofuscinosis (NCL) • Subset within LSDs with 13 known variations3 and no known cure, each related to a single gene mutation resulting in loss of lysosomal protein function 70+ 1 in 5,00
Slide 13: Our Small Molecule Drugs
Our Small Molecule Drugs Disease-modifying MoAs to affect key disease etiology and pathology for multiple LSDs ETIOLOGY PATHOLOGY Diminished Lysosomal Gene Expression Waste Build-Up due to Diminished Lysosomal Function Cycle of Inflammation & Neuronal Death Waste Accumulation Pro-Inflammatory Molecu
Slide 14: Disease-Modifying Mechanism of Actions
Disease-Modifying Mechanism of Actions 1 Gene expression and lysosomal biogenesis PLX Therapies Activate PPARα-TFEB Pathway to Promote TFEB PLX-100 Lysosomal Biogenesis and Cellular Waste Gene PLX-200 PPARα PPARα PLX-300 Multiple Gene TFEB Expression • PLX compounds activate Peroxisome Proliferator-
Slide 15: Disease-Modifying Mechanism of Actions
Disease-Modifying Mechanism of Actions 2 Reduction of inflammation PLX Therapies Promote Anti-Inflammatory Gene Expression to TFEB PLX-100 Facilitate Reduction of Inflammation Gene PLX-200 PPARα PPARα PLX-300 Multiple Gene TFEB Expression • PLX compounds activate PPARα, which also promotes the trans
Slide 16: Disease-Modifying Mechanism of Actions
Disease-Modifying Mechanism of Actions 3 Neuronal survival and cellular function improvement PLX Therapies Up-regulate GDNF Expression to Promote TFEB PLX-100 Neuronal Survival and Function Gene PLX-200 PPARα PPARα PLX-300 Multiple Gene TFEB • Glial cell line-derived neurotrophic factor (GDNF) is a
Slide 17: PLX-200 In Vitro Human Data
PLX-200 In Vitro Human Data Up-regulation of PPT1, TPP1 and CLN3 genes by Up-regulation of lysosome biogenesis in CLN2 patient PLX-200 in human cells fibroblasts Fold Change LysoT Time (h) PLX-200 (25µM) Merged PLX-200 PLX-200 Source: Ghosh, A., Corbett, G. T., Gonzalez, F. J., & Pahan, K. (2012). G
Slide 18: Summary of PLX Therapies' Preclinical Data
Summary of PLX Therapies’ Preclinical Data Target Indications Animal Model Supportive Non-Clinical Data Mechanism of Action • Increased lifespan • Showed improved motor activity • Lowered storage material PPARα and TFEB-mediated lysosomal CLN2 Cln2-/- • Increased anti-in lammatory actors ( OC 3 and
Slide 19: Go-To-Market Strategy
Section 3 Go-To-Market Strategy 19
Slide 20: SOTERIA Basket Trial Indications
SOTERIA Basket Trial Indications PLX-200’s oral, disease-modifying approach targets multiple LSDs through a flexible basket trial strategy Disease Addressable Patient Population1 Current Standard of Care PLX-200 Differentiation BRINEURA® is the only approved disease- ✓ Oral solution may offer a much
Slide 21: Contact Us
Contact Us Polaryx Therapeutics 140 E. Ridgewood Avenue, Suite 415 South Tower, Paramus, NJ 07652 US Email: info@polaryx.com STRICTLY PRIVATE 21
Slide 22: Appendix
Section 4 Appendix @ QO
Slide 23: Appendix: Animal Model Studies
Section 4.1 Appendix: Animal Model Studies 23
Slide 24: Preclinical Proof-of-Concept
Preclinical Proof-of-Concept 1 Gene expression and lysosomal biogenesis PLX-200 increases lysosome biogenesis and cellular PLX-200 increases TFEB level and lowers the burden of waste clearance in Cln2/- mice model storage material in Cln3Δex7/8 mice model PLX-200 Relative ATP Synthase Subunit C Leve
Slide 25: Preclinical Proof-of-Concept
Preclinical Proof-of-Concept 2 Reduction of inflammation PLX-200 increase SOCS3 and IL-1Ra anti-inflammatory PLX-200 reduces the level of proinflammatory molecules protein levels in Cln2/- mice model in the SBF¹ cortex of Cln3ΔJNCL mice model Motor Cortex Striatum CLN2(-/-) - + + + CLN2(-/-) - + + +
Slide 26: Preclinical Proof-of-Concept
Preclinical Proof-of-Concept 3 Neuronal survival and cellular function improvement PLX-200 increases the expression o GD F in a PPARα- PLX-200 increases pBAD levels and reduces apoptosis dependent manner in Cln2/- mice brain WT PLX-200 - CLN2(-/-) + + + CLN2(-/-) - + + + - CLN2(-/-) + + + Veh - - +
Slide 27: Appendix: Preclinical Comparisons
Section 4.2 Appendix: Preclinical Comparisons 27
Slide 28: Establishing a New Standard of Disease-Modifying MoAs
Establishing a New Standard of Disease-Modifying MoAs DISEASE ETIOLOGY DOWNSTREAM PATHOLOGY LSD Disease Reduced Lysosomal Protein Function Build-Up of Waste Material Cycle of Inflammation & Neuronal Death Cascade Promotes TFEB expression via PPARα activation, Promote anti-inflammatory gene expressio
Slide 29: CLN2: Comparison of PLX-200 and BRINEURA Preclinical Studies
CLN2: Comparison of PLX-200 and BRINEURA® Preclinical Studies PLX-200 demonstrated more favorable survival outcomes compared to BRINEURA® PLX-200 (gemfibrozil) Status at Time of Publication In Development In Development Mouse Model CLN2-/- CLN2-/- (Experimental Starting Age) (4 Weeks) (4 Weeks) ● ◑
Slide 30: CLN2: Comparison of PLX-200 and BRINEURA Survival Plots
CLN2: Comparison of PLX-200 and BRINEURA® Survival Plots PLX-200 demonstrated more favorable survival outcomes compared to BRINEURA® PLX-200 (gemfibrozil) • N = 40 (untreated), 40 (vehicle), 40 (treated) • N = 14 (untreated), 16 (vehicle), 16 (enzyme) • Increased median survival rate from approx. 11
Slide 31: Sandhoff: Comparison of PLX-200, Nizubaglustat, and Miglustat Preclinical Studies
Sandhoff: Comparison of PLX-200, Nizubaglustat, and Miglustat Preclinical Studies Jeyakumar et al. (1999) PLX-200 AZ-301 Miglustat (gemfibrozil) (nizubaglustat) Status for Sandhoff Disease In Development In Development Discontinued Mouse Model Hexb-/- Hexb-/- Hexb-/- (Experimental Starting Age) (12
Slide 32: Sandhoff: Comparison of PLX-200 and AZ-301 Survival Plots
Sandhoff: Comparison of PLX-200 and AZ-301 Survival Plots PLX-200 demonstrates more favorable survival outcomes in Hexb-/- mouse model compared to nizubaglustat PLX-200 AZ-301 (gemfibrozil) (nizubaglustat) WT Hexb-/- Hexb-/- + PLX-200 • Median survival increases from 110 (vehicle) to 180 • Median su
Slide 33: Comparative Profiling of Fibrate Analogues
Comparative Profiling of Fibrate Analogues Gem ibrozil’s distinct unctional and pharmacological advantages over other alternatives Gemfibrozil Fenofibrate Pemafibrate Bezafibrate Ciprofibrate Clofibrate ARα A Stimulates Lysosomal ✓ ✓ ✓ ✓ ✓ ✓ Biogenesis ARβ A Promote Myelination and ✓ X X ✓ X X Oligo
Polaryx Therapeutics — Investor Presentation (October 2026) on 6ix