Pyc Therapeutics Limited · ASX:PYC
Pyc Therapeutics — Corporate Update (August 2025)
25 slides from Pyc Therapeutics's corporate update, Aug 1, 2025
Slide 1: Life-changing science: Q3 investor webinar
For personal use only Life-changing science Q3 investor webinar August 2025
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: Q3 investor webinar agenda
Q3 investor webinar agenda • An introduction to PYC For personal use only • Near-term milestones • Focus on Polycystic Kidney Disease and Phelan-McDermid Syndrome • Why is a disease-modifying approach so important in these two indications? • Potential for reversal compared to arrest of disease progr
Slide 4: Introduction to PYC
For personal use only Life-changing science Introduction to PYC
Slide 5: An introduction to PYC Therapeutics
An introduction to PYC Therapeutics • PYC is a drug discovery and development company focused on creating life-changing For personal use only new therapies for patients who have genetic diseases and no treatment options available today • PYC’s strategy is to use RNA therapeutics to increase gene exp
Slide 6: Highlights of PYC's pipeline – clinical-stage assets
Highlights of PYC’s pipeline – 3 clinical-stage assets 1 2 3 4 5 Disease-modifying In areas of major With the highest Validated in patient- Generating human For personal use only drug candidates unmet need probability of success derived models efficacy data in 2025 5x Each of PYC’s In a disease with
Slide 7: PYC has built a pipeline of drug candidates
PYC has built a pipeline of drug candidates with the potential to become the standard of care in areas of major unmet need Historical progress Progress expected in 20251 For personal use only Program Pre-clinical Clinical Prevalence2 PYC-003 Polycystic Kidney Disease 1 in 1,000 PYC-002 Phelan-McDerm
Slide 8: Upcoming milestones
For personal use only Life-changing science Upcoming milestones
Slide 9: All clinical-stage programs will deliver human efficacy data
All 3 of PYC’s clinical-stage programs will deliver human efficacy data over the coming 12 months1 For personal use only 2025 2026 Program Q3 Q4 Q1 Q2 Polycystic Phase 1a/1b study Open-Label Multiple Ascending Dose data data to be presented Kidney Disease at ANZSN conference2 Phelan- Pre-clinical da
Slide 10: Polycystic Kidney Disease deep dive
For personal use only Life-changing science Polycystic Kidney Disease deep dive
Slide 11: Why is a disease-modifying approach in PKD so attractive?
Why is a disease-modifying approach in PKD so attractive? • The potential for substantial patient-impact driven by a combination of: For personal use only • High prevalence; • High morbidity; • The absence of treatment options available for ~95% of patients1; and • Animal models of PKD suggesting th
Slide 12: The total addressable market for PKD exceeds US$15bn p.a.
The total addressable market for PKD exceeds US$15bn p.a.1 Tolvaptan is used by <7% of the addressable patient population2 For personal use only • Despite limited patient uptake, 2023 sales of Tolvaptan exceeded US$1.5bn2 Estimated number of patients with ADPKD USA EUROPE Japan >100,000 >150,000 >20
Slide 13: PYC-003 first drug candidate targeting underlying cause of PKD
PYC-003 is the first drug candidate that directly targets the underlying cause of PKD to enter human trials Unaffected ADPKD patient For personal use only A mutation in one copy of the PKD1 PKD1 DNA gene leads to limited or non- functional mRNA form the affected allele1 PKD1 mRNA Wild type PC1 prote
Slide 14: Re-expressing PC1 protein in animal models rescues PKD phenotype
Re-expressing PC1 protein in animal models completely rescues the PKD phenotype1 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 indicative of a unique
Slide 15: Re-expressing the polycystins quote
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 concomitantly targeted. Thus, re- expressing the polycystins might ultimately remain the best — or possibly the only — way to
Slide 16: PYC-003 restores PC1 protein expression
PYC-003 restores PC1 protein expression towards levels seen in unaffected individuals PYC-003 increases PC1 expression back towards Wild-Type levels For personal use only in patient-derived models 1.7-fold increase in PC1 protein in PKD patient-derived iPSCs1 1.8 1.6 1.4 (fold-change over UT) PC1 pr
Slide 17: Unique delivery profile in vivo
This control of target gene expression is complemented by a unique delivery profile in vivo PYC-003 has a broad, even and deep distribution within the kidney at safe and well-tolerated For personal use only doses in NHPs Distribution in NHP kidney1 Distribution in NHP kidney PYC-003 treated PBS Cont
Slide 18: Phelan-McDermid Syndrome (PMS) deep dive
For personal use only Life-changing science Phelan-McDermid Syndrome (PMS) deep dive
Slide 19: PMS is caused by haploinsufficiency of the SHANK3 protein
PMS is caused by haploinsufficiency of the SHANK3 protein1,2 Unaffected neurons PMS neurons For personal use only Unaffected SHANK3 SHANK3 haploinsufficiency SHANK3 protein protein levels (50-65% of unaffected) Proper synaptic Impaired synaptic protein Synapse protein protein interaction interaction
Slide 20: PMS demonstrates potential reversibility in animal models
PMS also demonstrates potential reversibility in animal models For personal use only “Genetic restoration of Shank3 in rodents has been shown to reverse core deficits even in adult animals”1 1. Delling, J.P., Boeckers, T.M. Comparison of SHANK3 deficiency in animal models: phenotypes, treatment stra
Slide 21: Video interview – potential for reversibility in humans
Video interview – potential for reversibility in humans For personal use only PYC THERAPEUTICS 21 Clinical Trials in Phelan-McDermid syndrome - What we have learned https://www.youtube.com/watch?v=UdHmA-iqcXM&t=3480s
Slide 22: Video – patient journey and regression
Video – patient journey and the phenomenon of regression For personal use only PYC THERAPEUTICS 22 CureSHANK - Darus: A Regression Story (Phelan-McDermid syndrome) https://www.youtube.com/watch?v=Xdb11g2ZnG4
Slide 23: Video – neuronal communication with and without PYC-002
Video – neuronal communication (with and without PYC-002) Untreated PMS-derived neurons PYC-002 treated PMS-derived neurons (day 29) For personal use only PYC THERAPEUTICS 23
Slide 24: Synthesis – 12-month forward view
Synthesis – 12-month forward view For personal use only 2025 2026 Program Q3 Q4 Q1 Q2 Polycystic Phase 1a/1b study Open-Label Multiple Ascending Dose data data to be presented Kidney Disease at ANZSN conference2 Phelan- Pre-clinical data pack released3 McDermid Syndrome Retinitis Updated Phase 1/2 s
Slide 25: Q&A
For personal use only Life-changing science Q&A