XORTX Therapeutics, Inc. · TSXV:XRTX

XORTX Therapeutics — Investor Presentation (March 2020)

21 slides from XORTX Therapeutics's investor presentation, March 2020

  1. Slide 1: Redefining How Kidney Disease Is Treated

    REDEFINING HOW KIDNEY DISEASE IS TREATED INVESTOR PRESENTATION MARCH 2020 Non Confidential

  2. Slide 2: Forward Looking Statements

    FORWARD LOOKING STATEMENTS This document contains forward-looking information pursuant to applicable securities law. All information that addresses activities or developments that XORTX expects to occur in the future are forward-looking statements. Forward-looking statements are based on the estimat

  3. Slide 3: Key Highlights

    KEY HIGHLIGHTS Few treatment options for Progressive Kidney Disease due to Diabetes or Polycystic Kidney Scarce Asset in a Disease. None address this mechanism of disease action. Large Market • ~ 46 MM people in the U.S. have Progressive Kidney Disease; ~10 MM with Diabetic Nephropathy and ~120,000

  4. Slide 4: The Problem & The Opportunity

    THE PROBLEM & THE OPPORTUNITY ~360 Progressive Kidney Disease U.S. population Million • Accelerating decline of filtration (GFR) • Increased creatinine Increased urine protein loss With • • Abdominal pain ~46 Progressive • Declining health / wellness Million Kidney Disease in the U.S. Diabetes Facts

  5. Slide 5: End Stage Renal Disease – A Crisis

    END STAGE RENAL DISEASE – A CRISIS Diabetic Nephropathy is responsible for ~45% of end stage renal disease (ESRD) while, Polycystic Kidney Disease is responsible for ~5% ESRD – A Life Altering Event • 4 hours per day on dialysis • Any therapy that can Loss of ability to work full time • • Dependence

  6. Slide 6: The XORTX Solution

    THE XORTX SOLUTION XORTX is developing therapies to treat Progressive Kidney Disease where uric acid levels are accelerating disease progression, this approach is based on XORTX patent that protect this mechanism of action. Two “First-in-Class” Opportunities 1 Polycystic Kidney Disease (ADPKD) 2 Dia

  7. Slide 7: XORTX Technology Platform

    XORTX TECHNOLOGY PLATFORM Lead Program: Autosomal Dominant Polycystic Kidney Disease (ADPKD) XRx-008: ‘XORLO’ • A Proprietary OXYPURINOL Formulation • With 3x bioavailability filed as composition of matter - worldwide Patent - ‘PCT’ 2014 (until 2034) • Oxypurinol NDA was submitted to the FDA for the

  8. Slide 8: Two Similar Independent Phase 2 Pilot Results in Progressive Kidney Disease

    1 TWO SIMILAR INDEPENDENT PHASE 2 PILOT RESULTS IN PROGRESSIVE KIDNEY DISEASE Recent Phase 2 Clinical Trial Success in progressive kidney disease increases the probability for translational success of this program Lowering and managing blood uric acid appears to stop or slow kidney disease progressi

  9. Slide 9: Controlling Uric Acid Levels

    1 CONTROLLING URIC ACID LEVELS - Promises a ‘durable’ life changing therapy for Chronic Kidney Disease Years Quality of Life Big Picture – Reduce Healthcare Costs • Each year in the US, ~ 650,000 patients start dialysis at a cost to government of ~$90,000 per patient • Recent phase 2 trials suggest

  10. Slide 10: Diabetic Nephropathy - Kidneys Sensitive to Uric Acid Injury

    2 DIABETIC NEPHROPATHY - Kidneys of TYPE 2 DN patients are exquisitely sensitive to uric acid injury Diabetic Nephropathy: Q1< 3.2 mg/dL • Most common cause of chronic kidney disease Q2= 4.4 mg/dL NORMAL Range Q3 =5.0 mg/dL (CKD) and end-stage renal disease (ESRD) Q4 > 6.2 mg/dL HIGH Range worldwide

  11. Slide 11: Competitive Landscape & Advantage

    COMPETITIVE LANDSCAPE & ADVANTAGE 1 • No drugs are currently approved in the US to treat progressive kidney disease caused by Diabetic Nephropathy or Polycystic Kidney Disease and accelerated by hyperuricemia 2 • XORTX granted patent claims: “use of all uric acid lowering agents” as a therapy includ

  12. Slide 12: Commercialization Pathway

    COMMERCIALIZATION PATHWAY 1 Polycystic GMP Manufacturing Estimated IND Kidney Peak Sales Orphan Designation Disease Bioavailability results US$1.8B/Y Program ADPKD Worldwide License $4B/Y XRx-008 I------ Phase 3 Pivotal Study (SPA) - (30 months) ------I 2019 2020 2021 2022 2023 2 Estimated Co-Dev/ G

  13. Slide 13: Capital Structure

    CAPITAL STRUCTURE Market Capitalization ~$11 million Common Shares Outstanding 81,179,118 – 85% tightly held Warrants 18,399,084 Options 2,150,000 Fully-diluted Shares Outstanding 101,728,202 Public Listings Longer term plan for NASDAQ listing 13

  14. Slide 14: Action Item Plan for Value Creation / Recognition

    ACTION ITEM PLAN FOR VALUE CREATION / RECOGNITION In 2018 and 2019, XORTX focused In 2020 and 2021, XORTX will focus on on program value creation: broadcasting “technology value” & gaining recognition: 1. Advanced and accelerated ADPKD program with FDA 1. Investor Relations  Roadshows - North Ameri

  15. Slide 15: Key Takeaways

    KEY TAKEAWAYS Scarce Assets Addressing a Large Market Opportunity Strong Intellectual Property Portfolio Initial Data Strongly Positions the Company For Clinical Success Accelerated Development Pathway Experienced Management Team With Significant Expertise in Drug Development 15

  16. Slide 16: Appendix

    APPENDIX INVESTOR PRESENTATION Non Confidential

  17. Slide 17: Management Team

    MANAGEMENT TEAM Dr. Allen W. Davidoff, James Fairbairn, CPA, Dr. Alan Moore, Brian Mangal Ph.D. ICD.D Ph.D. Co-founder, CEO CFO Co-founder, Senior Director, Business Clinical and Development Regulatory Consultant • 15 years drug development • 20+ years in senior finance • Extensive clinical • 15 yea

  18. Slide 18: Board of Directors

    BOARD OF DIRECTORS W. Bruce Rowlands – Chairman, Audit Committee Member • Biotechnology and investment banking background • Former Senior VP, Lorus Therapeutics, a leading Canadian biotechnology company and VP and Director, Dominick and Dominick Securities Canada • Former Chairman and CEO, Eurocontr

  19. Slide 19: Advisory Board

    ADVISORY BOARD Dr. Petter Bjornstad, MD • Clinical Researcher and Assistant Professor of Pediatrics and Medicine, University of Colorado School of Medicine • NIH and NDRF funded translational research with a track record of research potential and success • Authored over 70 peer reviewed manuscripts

  20. Slide 20: Strong Patent Protection – Covering All Uric Acid Lowering Agents

    STRONG PATENT PROTECTION – COVERING ALL URIC ACID LOWERING AGENTS USE IN PROGRESIVE KIDNEY DISEASE Patent Claims Term US 9,155,740 Method of use claims covering uric acid lowering 2025 * agents (Xanthine Oxidase Inhibitors) in the * estimated GATT treatment of “Diabetic Nephropathy” Acid extension o

  21. Slide 21: Contact Information

    Contact Information: Dr. Allen Davidoff [email protected] 1-403-455-7727 INVESTOR PRESENTATION Non Confidential

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