Slide 1: RP11 Program - Presentation at EURETINA 2026
2 October 2026 For personal use only RP11 PROGRAM - PRESENTATION AT EURETINA 2026 • PYC is a precision medicine company dedicated to changing the lives of patients with genetic diseases who have no treatment options available • The Company is progressing a drug candidate (known as VP-001) that addre
Slide 2: About PYC Therapeutics
About PYC Therapeutics PYC Therapeutics (ASX: PYC) is a clinical-stage biotechnology company creating a new generation of RNA therapies to change the lives of patients with genetic diseases. The Company utilises its proprietary drug delivery platform to enhance the potency of precision medicines wit
Slide 3: A Phase 1/2 Repeat-Dose, Open-Label Study of VP-001
rsonal use only A Phase 1/2 Repeat-Dose, Open-Label Study of VP-001; a peptide conjugate of oligonucleotide designed to treat PRPF31-related Retinitis Pigmentosa Associate Professor Fred Chen Lions Eye Institute, Perth, Western Australia The University of Western Australia University of Melbourne, V
Slide 4: Disclosure
Disclosure rsonal use only • PYC Therapeutics (SAB), Novartis (SF), Roche (SF), Bayer (SF), Allergan (SF), Apellis (AB) • I am a co-inventor of the VP-001 PYC THERAPEUTICS 2
Slide 5: Prevalence of inherited retinal diseases: Australia
Prevalence of inherited retinal diseases: Australia rsonal use only Western Australia 1158 with IRD in WA 3 million population 1:2,600 has IRD PYC THERAPEUTICS
Slide 6: Genes identified in Western Australian Retinal Degeneration Study
rsonal use only 0 50 100 150 250 200 Inconclusive ABCA4 USH2A PRPH2 PRPF31 BEST1 RPGR RPGR carrier RHO EYS ABCC6 CRB1 GUCY2D PDE6B RP1 CHM CHM carrier NR2E3 RS1 BBS1 CACNA1F CNGB3 HK1 MT-TL1 CRX HGSNAT OPN1LW COL2A1 RP1L1 SAG TRPM1 VHL CDHR1 CEP290 IMPG1 MYO7A RP2 carrier ADGRV1 ARL3 BBS10 COL4A5 ca
Slide 7: Number of individuals with top genes
Number of individuals with top 10 genes 160 rsonal use only 140 120 100 80 60 40 20 0 ABCA4 USH2A PRPH2 PRPF31 BEST1 RPGR RPGR carrier RHO EYS ABCC6 CRB1 PYC THERAPEUTICS 40 in WA => 400 in Australia 1:75,000 4,500 people in the USA
Slide 8: PRPF31 (RP11) causes disease through haploinsufficiency
PRPF31 (RP11) causes disease through haploinsufficiency rsonal use only Unaffected individual RP11 patient PRPF31 PRPF31 PRPF31 PRPF31 PRPF31 PRPF31 DNA RNA Protein DNA RNA Protein Functional PRPF31 expression = 100% Functional PRPF31 expression = ~50% PRPF31 is a crucial splicing factor in the reti
Slide 9: A large indigenous family with RP11
A large indigenous family with RP11 rsonal use only PYC THERAPEUTICS
Slide 10: Non penetrance in RP11 led to the discovery of a treatment
Non penetrance in RP11 led to the discovery of a treatment rsonal use only Affected RP11 patient Non penetrant RP11 PRPF31 PRPF31 PRPF31 PRPF31 PRPF31 PRPF31 DNA RNA Protein DNA RNA Protein Functional PRPF31 expression = ~50% Functional PRPF31 expression = ~70% Non penetrance in RP11 is likely to be
Slide 11: VP-001 shows the ability to rescue the haploinsufficiency causing RP11
VP-001 shows the ability to rescue the haploinsufficiency causing RP11 in patient-derived models PRPF31 mRNA Expression in Patient iPSC-RPE with VP-001 6 days post 3) Rescues appearance and rsonal use only Unaffected RP11 RP11 + VP-001 Scrambled control oligo Dose-dependent target gene rescue to wil
Slide 12: VP-001 clinical trials program
VP-001 clinical trials program Natural History Study (Quokka) rsonal use only Progress to date Open Label Phase 1a SAD Phase 1b MAD Study Extension Phase 2/3 Study1 Platypus Wallaby • Safety & Tolerability • Safety & Tolerability • Efficacy • Efficacy • Primary: Low-Luminance Visual • Low-Luminance
Slide 13: Outcome Measures
Outcome Measures rsonal use only • Safety • Low-luminance visual acuity • Mean change from baseline • Proportion of eyes with 10 letter gain/loss PYC THERAPEUTICS
Slide 14: Summary of VP-001 safety data generated in RP11 patients
Summary of VP-001 safety data generated in RP11 patients rsonal use only VP-001 Safety Outcomes: RP11 patients1 • No Treatment Related-Serious Adverse events observed in any subjects dosed with VP-001 to date • Including subjects who have received repeat doses of VP-001 • Treatment-Emergent Adverse
Slide 15: RP11 patients treated with VP-001 have shown improvements in visual acuity
RP11 patients treated with VP-001 have shown improvements in visual acuity compared to natural history (Quokka study)1 rsonal use only Change in Low-Luminance Visual Acuity (LLVA) in RP11 patients2,3 35 30 Change from baseline in LLVA 25 20 VP-001 Treated Eyes2 15 (ET-DRS letters) 10 Mean VP-001 Tre
Slide 16: A greater proportion of RP11 eyes treated with VP-001 show clinically meaningful improvements
A greater proportion of RP11 eyes treated with VP-001 show clinically meaningful1 improvements in LLVA rsonal use only Trial(s) Eye >10 letter % of eyes >10 letter % of eyes Stable % of eyes (analysis gain at loss at (between +9 eligible ‘n’) multiple multiple and -9 at all5 timepoints2 timepoints2
Slide 17: Multiple RP11 patients have reported improved vision and quality of life
Multiple RP11 patients have reported improved vision and quality of life after treatment with VP-001 Reported feedback from patients in PYC’s RP type 11 Phase 1/2 clinical trial1 rsonal use only “I see airplanes in the sky (never have before), stars at night, animals/creatures along the road…” - “My
Slide 18: Long-term data will be used to finalize the design of a potential registrational trial
Long-term data from the ongoing Phase 2 study will be used to finalize the design of a potential registrational trial in RP111 A potential registrational trial1,2: rsonal use only • Primary endpoint – mean change in Low Luminance Visual Acuity (LLVA) • Supported by key secondary endpoint of proporti
Slide 19: Acknowledgements
Acknowledgements rsonal use only Dr. David Birch Dr. Lesley Everett Dr. Thiran Jayasundera Dr. John Chancellor RP11 Patients and Retina Foundation OHSU Casey Eye Kellogg Eye Center Alkek Eye Center families, AIRDR, Sue of Southwest Institute University of Michigan Baylor College of Medicine Fletcher