Slides
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Q2 Investor Webinar May 2025 Life-changing science For personal use only
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PYC THERAPEUTICS 2 The purpose of this presentation is to provide an update of the business of PYC Therapeutics Limited (ASX:PYC) [‘PYC’]. These slides have been prepared as a presentation aid only and the information they contain may require further explanation and/or clarification. Accordingly, these slides and the information they contain should be read in conjunction with past and future announcements made by PYC Therapeutics and should not be relied upon as an independent source of information. Please contact PYC and/or refer to the Company's website for further information. The views expressed in this presentation contain information derived from publicly available sources that have not been independently verified. No representation or warranty is made as to the accuracy, completeness or reliability of the information. Any forward-looking statements in this presentation have been prepared on the basis of a number of assumptions which may prove incorrect and the current intentions, plans, expectations and beliefs about future events are subject to risks, uncertainties and other factors, many of which are outside PYC’s control. Important factors that could cause actual results to differ materially from assumptions or expectations expressed or implied in this presentation include known and unknown risks. Because actual results could differ materially to assumptions made and PYC’s current intentions, plans, expectations and beliefs about the future, you are urged to view all forward-looking statements contained in this presentation with caution. This presentation should not be relied on as a recommendation or forecast by PYC. Nothing in this presentation should be construed as either an offer to sell or a solicitation of an offer to buy or sell shares in any jurisdiction. Disclaimer PYC THERAPEUTICS 2 For personal use only
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PYC THERAPEUTICS 3 Objectives for today 1) Provide a detailed update on each of PYC’s pipeline program updates a) Progress (year to date) b) Objectives for the remainder of 2025 2) Outline the platform implications of near-term program milestones 3) Contextualise PYC’s progress within the broader commercial landscape For personal use only
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An introduction to PYC Life-changing science For personal use only
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PYC THERAPEUTICS 5 Each of PYC’s pipeline programs address the root cause of the target disease Disease-modifying drug candidates In a disease with no established standard of care and worth between $1 and $10 billion p.a.1 In areas of major unmet need With a 5x higher probability of success than the industry average2 With the highest probability of success Generating critical data this year - high-value human data readouts in areas of major unmet patient need3 Generating human efficacy data in 2025 A ‘quantitative cure’ for the single-gene disease targeted Validated in patient- derived models 1 2 3 4 5 5x 1. Utilising the prevalence for each indication outlined and referenced on page 7 of this presentation and the median orphan drug price from Evaluate Pharma 2. King EA, Davis JW, Degner JF. Are drug targets with genetic support twice as likely to be approved? Revised estimates of the impact of genetic support for drug mechanisms on the probability of drug approval. PLoS Genet. 2019 Dec 12;15(12):e1008489. doi: 10.1371/journal.pgen.1008489. PMID: 31830040; PMCID: PMC6907751. Pre-print version of article 3. Subject to the risks and uncertainties outlined in this document and the Company’s ASX disclosures of 17 February 2025 PYC THERAPEUTICS 5 An introduction to PYC – differentiated drug development For personal use only
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PYC THERAPEUTICS 6 Pre-clinical Prevalence2 Retinitis Pigmentosa Type 11 Clinical Autosomal Dominant Optic Atrophy Phelan-McDermid Syndrome Historical progress Progress expected in 20251 Polycystic Kidney Disease VP-0013 PYC-001 PYC-003 PYC-002 1 in 100,000 1 in 1,000 1 in 35,000 1 in 15,000 Program Platform and discovery programs PYC THERAPEUTICS 6 1. Based on management’s latest estimates accurate as at 27 March 2025 and subject to successful realisation of developmental milestones in each program as well as satisfaction of regulatory requirements and subject to all other risks customary to a biotechnology company developing novel drug candidates including those risks outlined to the ASX in the Company’s disclosures of 17 February 2025 2. See references in Company presentation of 14 March 2024 for source material on prevalence by indication 3. PYC 96.2% ownership of VP-001 (3.8% ownership by Lions Eye Institute, Australia) and 100% ownership of all other pipeline programs PYC has built a pipeline of drug candidates with the potential to become the standard of care in areas of major unmet need For personal use only
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Program-level updates Life-changing science For personal use only
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PYC THERAPEUTICS 8 2026 RP11 VP-001 Monogenic blinding eye disease of childhood Gene: PRPF31 ADPKD PYC-003 Most common lethal single-gene disorder in the world Gene: PKD1 ADOA PYC-001 Monogenic blinding eye disease of childhood Gene: OPA1 PMS PYC-002 Monogenic CNS disease of childhood Gene: SHANK3 2025 Program Ph1/2 study Registrational study Ph 1b MAD studyPh 1a SAD study Human safety Clinical proof-of- concept2 Ph 1b MAD studyPh 1a SAD study (Healthy Volunteers) Human safety (single dose) Finalisation of the pre-clinical data pack Regulatory approval to commence FIH studies Clinical proof-of-concept1 PYC THERAPEUTICS 8 All 4 development programs in PYC’s pipeline are on track to deliver on their 2025 objectives Open-label extension of Ph 1/2 study Program ‘Go/No Go’ decision If ‘Go’ – IND-enabling studies Regulatory approval to commence FIH studies SAD study Ph 1a SAD study (PKD patients) 1. Subject to the risks and uncertainties outlined in the Company’s ASX disclosures of 17 February 2025 We are here For personal use only
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Platform milestones Life-changing science For personal use only
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PYC THERAPEUTICS 10 2026 RP11 VP-001 Monogenic blinding eye disease of childhood Gene: PRPF31 ADPKD PYC-003 Most common lethal single-gene disorder in the world Gene: PKD1 ADOA PYC-001 Monogenic blinding eye disease of childhood Gene: OPA1 PMS PYC-002 Monogenic CNS disease of childhood Gene: SHANK3 2025 Program Ph1/2 study Registrational study Ph 1b MAD studyPh 1a SAD study Human safety Clinical proof-of- concept2 Ph 1b MAD studyPh 1a SAD study (Healthy Volunteers) Human safety (single dose) Finalisation of the pre-clinical data pack Regulatory approval to commence FIH studies Clinical proof-of-concept1 PYC THERAPEUTICS 10 Upcoming clinical milestones in each of PYC’s programs will also help validate PYC’s delivery platform Open-label extension of Ph 1/2 study Program ‘Go/No Go’ decision If ‘Go’ – IND-enabling studies Regulatory approval to commence FIH studies SAD study Ph 1a SAD study (PKD patients) 1. Subject to the risks and uncertainties outlined in the Company’s ASX disclosures of 17 February 2025 We are here 1 3 2 For personal use only
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When to deal Life-changing science For personal use only
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PYC THERAPEUTICS 12 PYC THERAPEUTICS 12 Maximising value from the pipeline 1. Stifel Building Value in Biotech February 2024 For personal use only
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Life-changing science Q&A For personal use only