Pyc Therapeutics Limited · ASX:PYC
Pyc Therapeutics — Investor Presentation (November 2024)
12 slides from Pyc Therapeutics's investor presentation, Nov 28, 2024
Slide 1: Polycystic Kidney Disease Program Investor Webinar
For personal use only Life-changing science Polycystic Kidney Disease Program Investor webinar November 2024
Slide 2: Disclaimer
Disclaimer The purpose of this presentation is to provide an update of the business which are outside PYC’s control. Important factors that could cause For personal use only of PYC Therapeutics Limited (ASX:PYC) [‘PYC’]. These slides have been actual results to differ materially from assumptions or
Slide 3: Executive Summary
Executive Summary • On 27 November 2024, PYC released its pre-clinical data pack in support of PYC-003 – a For personal use only first-in-class RNA conjugate for the treatment of Autosomal Dominant Polycystic Kidney Disease (PKD) due to mutations in the PKD1 gene1 • In December 2024, PYC will make t
Slide 4: There is an urgent need to create treatment options for the PKD patient community
There is an urgent need to create treatment options for the PKD patient community For personal use only Healthy adult kidney Polycystic kidney Polycystic Kidney Disease High prevalence PKD affects 1 in every 1,000 people meaning >5 million people worldwide have the disease1,2 Life-changing Half of a
Slide 5: Targeting the root cause of PKD (insufficient PC1 protein expression)
Targeting the root cause of PKD (insufficient PC1 protein expression) may be the only therapeutic option in this disease For personal use only “It remains possible that multiple pathways that are directly regulated by the polycystins concur in the prevention of cyst formation and may need to be conc
Slide 6: The PKD1 gene in isolation drives polycystic kidney disease
The PKD1 gene in isolation drives polycystic kidney disease1 For personal use only “These observations collectively point to PKD1 as the primary, if not the sole, factor governing cyst onset and growth”1 1. In the ~75% of PKD patients with a PKD1 mutation PYC THERAPEUTICS 6 2. Lakhia, R., Ramalingam
Slide 7: PYC-003 acts directly on the functional PKD1 transcript to upregulate PC1 protein
PYC-003 acts directly on the functional PKD1 transcript to upregulate PC1 protein expression PKD1 DNA PKD1 RNA PC1 Protein Kidney For personal use only Unaffected PKD patient Reduced functional PC1 dosage PYC-003 treated PYC-003 PKD patient PYC THERAPEUTICS 7 Created using BioRender
Slide 8: How much PC1 protein will be sufficient to make a meaningful impact on disease progression?
How much PC1 protein will be sufficient to make a meaningful impact on disease progression? For personal use only 1. Ong AC, Harris PC. A polycystin-centric view of cyst formation and disease: the polycystins revisited. Kidney Int. 2015 Oct;88(4):699-710. doi: 10.1038/ki.2015.207. Epub 2015 Jul 22.
Slide 9: PYC-003 increases PC1 protein levels by >1.5-fold
PYC-003 increases PC1 protein levels by >1.5-fold1 PYC-003 addresses the root cause of PKD in human kidney cells2 For personal use only PYC-003 increases levels of PC1 protein (the missing protein that causes PKD) in a human kidney cell line ✱ 2.0 ✱ PC1 Protein Fold Change over UT 1.5 1.0 0.5 1.0 1.
Slide 10: Potential of disease-modifying approaches in PKD foreshadowed by animal models
The potential of disease-modifying approaches in PKD are foreshadowed by the results of animal models For personal use only “Even if one could have hypothesized that re-expressing PKD genes would slow disease progression, the rapidity and completeness of the reversal are astonishing and are likely i
Slide 11: PYC-003 will progress to human trials in 2025
PYC-003 will progress to human trials in 20251 Clinical pathway1-3 For personal use only 2024 2025 2026 2027 2028 2029 Phase 2 Pivotal for Marketed GLP tox. Phase 1 SAD/MAD NDA drug Accelerated Approval ✓ Complete Regulatory Established accelerated approval path submission for for PKD in the US allo
Slide 12: Q&A
PYC-003 for Polycystic Kidney Disease (PKD) For personal use only Q&A PYC THERAPEUTICS 12
PYC Therapeutics — Investor Presentation (November 2024) on 6ix