XORTX Therapeutics, Inc. · TSXV:XRTX
XORTX Therapeutics — Investor Presentation (March 2021)
29 slides from XORTX Therapeutics's investor presentation, March 2021
Slide 1: A Leader Redefining How Kidney Disease is Treated
A Leader Redefining How Kidney Disease is Treated Investor Presentation – March 2021 Non Confidential
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
Slide 3: Key Highlights
Key Highlights Focused on developing XORTX clinical development stage products target xanthine oxidase and uric acid lowering, clinical stage which has been shown to have an important clinical effect. Significant unmet global market assets to treat opportunities. kidney disease Three Proprietary Thr
Slide 4: XRx-008 – Autosomal Dominant Polycystic Kidney Disease (ADPKD)
XRx-008 – Autosomal Dominant Polycystic Kidney Disease (ADPKD) Development Timeline: ** Planning to Initiate Pivotal Study under SPA in second half of 2020 ** 2020 2021 2022 2023 2024 2025 $15 m Mfg GMP(9 months) US Listing Finalize IND Finalize ODD Global BA Study Pharma Protocol/SPA Ph III Ph3 Piv
Slide 5: XRx-101 – A Coronavirus / Acute Kidney Injury Therapy
XRx-101 – A Coronavirus / Acute Kidney Injury Therapy Development Timeline: ** Depending on FDA Feedback Compassionate Use or Initiate Pivotal Study Q4 of 2020 ** 2020 2021 2022 2023 2024 2025 Pharma Mfg GMP(7 months)Co-Dev Finalize IND $XXX m $XXX m $XXX m COVID COVID COVID BA Study Design Ph III P
Slide 6: Xanthine Oxidase and Uric Acid: Injury and Kidney Disease
Xanthine Oxidase and Uric Acid (UA): Injury Kidney Disease High UA Levels Predict Lowering UA Protects Kidney Diabetic Nephropathy Health in Human Clinical Trials Endothelial High Blood NO Depletion Pressure Vascular Damage Normalized Blood Pressure Oxidative Soletsky B et. al., Uric Acid Reductio
Slide 7: Decreasing Uric Acid levels in Diabetes Patients may delay progression to ESRD
Decreasing Uric Acid levels in Diabetes Patients may delay progression to ESRD and the need for dialysis or transplant XORTX Therapies are designed to delay or reverse progression of kidney disease, to prevent ESRD ESRD – A Life Altering Event • 4 hours per day on dialysis • Loss of ability to work
Slide 8: Strong Patent Protection – Covering all Uric Acid Lowering Agents
Strong Patent Protection – Covering all Uric Acid Lowering Agents Use in Progressive 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
Slide 9: XRx-008 in Polycystic Kidney Disease (PKD)
XRx-008 in Polycystic Kidney Disease (PKD) XRx-008: A Novel Formulation of Oxypurinol • XRx-008 - a proprietary formulation of Oxypurinol with 3X increased bioavailability – wholly owned by XORTX and developed “In-house” • Increased oral dosing range • Increased tolerability increases compliance and
Slide 10: XRx-008 – Uric Acid Lowering in ADPKD Provides Clinically Meaningful Benefit
XRx-008 – Uric Acid Lowering in ADPKD Provides Clinically Meaningful Benefit Serum uric acid (SUA) concentrations Decreasing uric acid in ADPKD patients reverses the are higher in APDKD patients than rate of glomerular filtration rate decline. controls. (Mejias et al. Hyperuricemia, gout, and (Han e
Slide 11: XRx-008 – Autosomal Dominant Polycystic Kidney Disease (ADPKD)
XRx-008 – Autosomal Dominant Polycystic Kidney Disease (ADPKD) Development Timeline: ** Planning to Initiate Pivotal Study under SPA in second half of 2020 ** 2020 2021 2022 2023 2024 2025 Mfg GMP(9 months) Finalize IND Finalize ODD BA Study Protocol/SPA Ph III Ph3 Pivotal Study under SPA - (30 mont
Slide 12: XRx-101 – A Novel Formulation of Oxypurinol for COVID-19
XRx-101 – A Novel Formulation of Oxypurinol for the treatment of COVID-19 / Coronavirus • XRx-101 - a proprietary formulation of xanthine oxidase inhibitor, inhibit oxygen radical and uric acid production, ameliorate kidney injury associated with COVID-19 infection. • Current evidence suggests benef
Slide 13: Coronavirus – COVID-19 – Progression & High Uric Acid
Coronavirus – COVID-19 – Progression & High Uric Acid COVID-19 Infection is Vascular Lung Infection/ Hypoxia Lytic Virus Endothelium Injury Cell debris – DAMP’s DNA Kidney Injury Xanthine oxidase Uric Acid >60% with AKI in Cardiac/ Neurological Hospital Injury UA Inflammation Pro Coagulation Hyp
Slide 14: Acute Kidney Injury occurs early during infection and is associated with hyperuricemia
Acute kidney Injury occurs early during infection and is associated with hyperuricemia. AKI is persistant. Proposed Clinical Trial “Unpublished” - Preliminary study results @ Mount Sinai, NY: Hospitalized individuals with AKI – 63% had very high uric acid levels. N>1100 individuals, where greater th
Slide 15: COVID-19 Viral Infection & Acute Kidney Injury (AKI) New York
COVID-19 Viral Infection & Acute Kidney Injury (AKI) New York – 1/2 Preliminary Data: “MAKE” – Major Adverse Kidney Events Hospitalized individuals in New York Uric acid normal range: 3-6 mg/dL Hyperuricemia is associated with MAKE (dialysis or death). Hyperuricemia occurs early in COVID-19 infectio
Slide 16: COVID Infection is producing a substantial, emerging, AKI market
COVID Infection is producing a substantial, emerging, AKI market The World Health Organization investigative team that visited China in February, led by Canadian expert Dr. Bruce Aylward, reported last week that it places the R0 of COVID-19 at between 2 and 2.5 – a number it considers “relatively hi
Slide 17: A Crisis Driven – Significant Market Opportunity
A Crisis Driven – Significant Market Opportunity XRx-101 for Coronavirus Induced Acute Kidney Disease Opportunity #1a: Orphan Disease – Phase 3 XRx-101 in Coronavirus Infection Suppression of Viral and Acute Kidney Injury ~ Hospitalized Individuals U.S. 4,000,000 ~50,000,000 Individuals Worldwide Co
Slide 18: XRx-101 – A Coronavirus / Acute Kidney Injury Therapy
XRx-101 – A Coronavirus / Acute Kidney Injury Therapy Development Timeline: ** Depending on FDA Feedback Compassionate Use or Initiate Pivotal Study Q4 of 2020 ** 2020 2021 2022 2023 2024 2025 Pharma Mfg GMP(7 months)Co-Dev Finalize IND BA Study Design Ph III Ph3 Pivotal Study (8 months) NDA filing
Slide 19: Two Significant Market Opportunities
Two Significant Market Opportunities Progressive Kidney Disease Opportunity #1: Opportunity #2: Orphan Disease – Phase 3 Diabetic Kidney Disease – Phase 2 XRx-008 in Autosomal Dominant XRx-221 in Type 2 Diabetic Polycystic Kidney Disease (ADPDK) Nephropathy ~ 150,000 Individuals U.S. ~ 10,000,000 In
Slide 20: Capital Structure
Capital Structure Market Capitalization ~$26 million Common Shares Outstanding 109,976,717 Warrants 25,170,626 Options 5,600,000 Fully-diluted Shares Outstanding 140,747,343 Public Listings Near term plan for US listing 20
Slide 21: Key Takeaways
Key Takeaways Scarce Assets Addressing a Large Market Opportunity Strong Intellectual Property Portfolio Initial Data Strongly Positions the Company For Clinical Success Important Interim Data Readouts Experienced Management Team With Significant Expertise in Drug Development 21
Slide 22: Contact Information
Contact Information: Dr. Allen Davidoff [email protected] or 1-403-455-7727 Non Confidential
Slide 23: Appendix
Appendix INVESTOR PRESENTATION Non Confidential
Slide 24: Management Team
Management Team Dr. Allen W. James Fairbairn, Dr. Alan Moore, Brian Mangal Dr. David Sans, Davidoff, Ph.D. CPA, ICD.D Ph.D. FAARM, Ph.D., M.B.A. CEO CFO Senior Clinical and Director, Business Director, Corporate Regulatory Consultant Development Development • 15 years drug • 20+ years in senior deve
Slide 25: 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
Slide 26: Advisory Board
Advisory Board Dr. Petter Bjornstad, MD - Neprhologist • 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 review
Slide 27: Effect of Xanthine Oxidase Inhibition in Influenza in Mice
Effect of Xanthine Oxidase Inhibition in Influenza in Mice Purpose: Evaluate biochemical parameters of influenza virus lung infection Model: sBALF – influenza virus infected mice Biochemical After infection, mice showed 170x increase in adensine Characteristics: deaminase (ADA) and 400 fold increase
Slide 28: Effect of Xanthine Oxidase Inhibition in Influenza in Mice
Effect of Xanthine Oxidase Inhibition in Influenza in Mice Uric acid increases substantially as influenza infection progresses. Xanthine oxidase inhibition, greatly increases survival during influenza infection. 2mg (p=0.01) 1mg (p=0.04) Ctrl Figure 4: Catabolic profile of adenosine in s-BALF. Figur
Slide 29: Competitive Landscape Analysis - Well Differentiated Compared to Landscape
Competitive Landscape Analysis - Well Differentiated Compared to Landscape Current Agents are Ineffective at Best CLASS IP ADVANTAGES LIMITATIONS Uricosurics Varied Decrease uric acid absorption/ reuptake Inappropriate: Increase kidney stone formation Uricase Off Patent Acute IV use Cannot be used c
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