Pyc Therapeutics Limited · ASX:PYC
Pyc Therapeutics — Investor Presentation (November 2023)
21 slides from Pyc Therapeutics's investor presentation, Nov 17, 2023
Slide 1: Life-changing science: Polycystic Kidney Disease program
For personal use only Life-changing science Polycystic Kidney Disease program November 2023
Slide 2: Disclaimer
Disclaimer The purpose of this presentation is to provide an update of the factors, many of which are outside PYC’s control. Important factors For personal use only business of PYC Therapeutics Limited (ASX:PYC) [‘PYC’]. These slides that could cause actual results to differ materially from assumpti
Slide 3: PYC Therapeutics
PYC Therapeutics For personal use only • Is a clinical-stage drug discovery & development company • Creates first-in-class RNA drugs for patients with genetic diseases and inadequate treatment options • Develops drugs with the highest likelihood of success in human studies (5x higher than the indust
Slide 4: PYC has added a first-in-class drug candidate to its pipeline for PKD
PYC has added a first-in-class drug candidate to its pipeline for the >5 million people worldwide with Polycystic Kidney Disease Historical progress Progress expected within 12 months For personal use only DISCOVERY PRE-CLINICAL IND ENABLING CLINICAL TRIALS MARKETED MARKET SIZE^ Retinitis Pigmentosa
Slide 5: PYC's drug candidate for PKD has defining features
PYC’s drug candidate for Polycystic Kidney Disease has 6 defining features Addresses a major unmet patient need1 For personal use only 1 2 Overcomes the delivery challenge2 3 Has already demonstrated efficacy2 4 Will progress to human trials in 20245 5 Has a high-velocity path to a >$10 billion p.a.
Slide 6: PYC-003: A first-in-class drug candidate for Autosomal Dominant PKD
For personal use only PYC-003 A first-in-class drug candidate for the treatment of Autosomal Dominant Polycystic Kidney Disease (PKD) PYC THERAPEUTICS 6 PYC THERAPEUTICS 6
Slide 7: PKD patient experience
PKD patient experience For personal use only For patients with PKD, the erratic onset of pain contributes to the unpredictability of daily living, dictates career and family planning, is socially limiting, and renders those affected unable to establish and pursue their long-term life goals¹ PYC THER
Slide 8: Polycystic Kidney Disease is an area of major unmet patient need
1. Polycystic Kidney Disease is an area of major unmet patient need There is an urgent need for treatments with disease-modifying potential in Polycystic Kidney Disease For personal use only Healthy adult kidney Polycystic kidney Polycystic Kidney Disease High prevalence PKD affects 1 in every 1,000
Slide 9: The delivery challenge
The delivery challenge ‘While everybody else was so hyped and giving For personal use only Nobel Prizes for CRISPR and all that, we realized those weren’t really the limitations” ‘The limitations were really delivery’1 - George Yancopoulos President and CSO, Regeneron PYC THERAPEUTICS 9 1. https://e
Slide 10: PYC's proprietary delivery technology overcomes the major challenge in the kidney
2. PYC’s proprietary delivery technology overcomes the major challenge for precision medicine in the kidney PYC’s drug candidate for PKD can reach all of the cells affected by the disease - PYC-003* achieves a For personal use only broad, even and deep distribution throughout the kidney following a
Slide 11: The 'killer experiment'
The ‘killer experiment’ For personal use only Patient organoids/3D kidney models are the ‘most informative model for therapeutic testing in PKD’1 - Peter C. Harris Leading PKD Genetic Researcher, Mayo Clinic PYC THERAPEUTICS 11 1. Sieben CJ, Harris PC. Experimental Models of Polycystic Kidney Diseas
Slide 12: PKD drug candidate has demonstrated efficacy in 3D kidney models
3. PYC’s PKD drug candidate has already demonstrated efficacy in 3D kidney models from patients with the disease PYC’s drug candidate for PKD is effective in ‘the killer experiment’ - Treatment with PYC-003 results in personal use only decreased frequency and area of cysts in a 3D model of PKD deriv
Slide 13: High-velocity path to market
High-velocity path to market ‘[Accelerated approval is] a way of getting there For personal use only that will benefit individuals in medical need, at the earliest possible timepoint while ultimately giving us the opportunity to get the data we need for turning those to traditional approvals’1 - Pet
Slide 14: PYC-003 will progress to human trials in 2024
4. PYC-003 will progress to human trials in 20241 2024 2025 2026 2027 For personal use only IND-Enabling Fast Track & Orphan Drug Designations studies are expected to be available for PKD2,3 GLP-Toxicology Studies Regulatory Approval New Drug Phase 1a/1b Phase 2 Application (NDA)*3 Biomarker efficac
Slide 15: PYC-003 high-velocity path to market with potential approval after Phase 2
5. PYC-003 has a high-velocity path to market with the potential for approval after a Phase 2 clinical trial PYC-003 Clinical Development Plan with Accelerated Approval*1 For personal use only 2025-2026 2026-2027 2027 Ph2 Pivotal for NDA Commercialisation Ph1 SAD / MAD Accelerated Accelerated Approv
Slide 16: Drugs targeting single gene diseases are more likely to succeed in human trials
6. Drugs targeting single gene diseases are 5 to 8 times more likely to succeed in human trials PYC focuses on severe diseases caused by a single gene For personal use only These have the highest likelihood of success in the clinic1-3 How likely is a drug targeting a monogenic disease to succeed in
Slide 17: PYC-003 addresses the root cause of the disease
For personal use only PYC-003 Addresses the root cause of the disease with potential to regenerate damaged kidneys and restore function PYC THERAPEUTICS 17 PYC THERAPEUTICS 17
Slide 18: PKD is caused by a mutation in one copy of a single gene
PKD is caused by a mutation in one copy of a single gene Unaffected PKD Patient For personal use only 80% of PKD patients have a PKD1 DNA mutation in one copy of PKD1 DNA1 PKD1 mRNA Wild type PC1 protein Haploinsufficient PC1 protein PC1 protein expression (100%) (~50% of wild-type levels)2 Balanced
Slide 19: PYC-003 addresses this genetic mutation at its root cause
PYC-003 addresses this genetic mutation at its root cause PKD Patient PKD Patient + PYC-003 For personal use only 80% of PKD patients have a PKD1 DNA PYC-003 mutation in one copy of PKD1 DNA1 effectively PKD1 mRNA ✓ Increases PKD1 mRNA PYC-003 Haploinsufficient PC1 protein (~50% of wild-type levels)
Slide 20: PYC-003 addresses the root cause of PKD in animals and human kidney cells
PYC-003 addresses the root cause of PKD in animals and human kidney cells In Animals1 In Human Kidney Cells2 For personal use only Safe & effective upregulation of PKD1 mRNA Upregulation of PC1 protein in human kidney cell line in mouse kidney 3 days after a single 20 mg/kg dose PC1 is the rate-limi
Slide 21: PYC's new drug candidate for Polycystic Kidney Disease
PYC’s new drug candidate for Polycystic Kidney Disease For personal use only • Has the potential to change the lives of > 5 million patients worldwide with this condition1,3 • Is >5x more likely to succeed in human trials2 • Addresses a target market worth greater than US $10 billion per annum4 • Is
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