Pyc Therapeutics Limited · ASX:PYC

Pyc Therapeutics — ARVO 2024 (Association for Research in Vision and Ophthalmology)

20 slides from Pyc Therapeutics's conference presentation, May 6, 2024

  1. Slide 1: Presentation of RP11 Clinical Trial Data at ARVO Conference

    31 October 6 May 2024 2022 PRESENTATION OF RP11 CLINICAL TRIAL DATA AT ARVO CONFERENCE • PYC is conducting clinical trials of the first investigational drug candidate with disease-modifying potential in a blinding eye disease of childhood called Retinitis Pigmentosa type 11 (RP11) • PYC is currently

  2. Slide 2: A/Prof. Chen will also present data from one patient within cohort 3

    A/Prof. Chen will also present data from one patient within cohort 3 (30 micrograms of VP-001) of the Single Ascending Dose (SAD) study. This patient is notable for the fact that they are at an earlier stage of disease progression than the other patients enrolled in the SAD study. Assessment of the

  3. Slide 3: Contacts

    This ASX announcement should not be relied on as a recommendation or forecast by the Company. Nothing in this ASX announcement should be construed as either an offer to sell or a solicitation of an offer to buy or sell shares in any jurisdiction. CONTACTS: INVESTORS and MEDIA info@pyctx.com pyctx.co

  4. Slide 4: Phase 1 First-in-Human Study of VP-001

    Phase 1 First-in-Human Study of VP-001 A Peptide Conjugated Oligonucleotide for the Treatment of Retinitis Pigmentosa Type 11 Dr Fred Chen Lions Eye Institute, Perth, Australia ARVO 2024 1

  5. Slide 5: Executive Summary – Update on VP-001 for RP11

    Executive Summary – Update on VP-001 for RP11 Introduction to VP-001 for RP11 1. RP11 is a progressive and blinding eye disease for which there are no treatments available for patients 2. RP11 is caused by insufficient expression of one gene (PRPF31) in the retina 3. VP-001 addresses the underlying

  6. Slide 6: RP11 is a progressive and blinding eye disease with no available treatments

    1. RP11 is a progressive and blinding eye disease for which there are no treatments available for patients Degenerative sight of an RP11 patient Retinitis Pigmentosa (RP)1,2 6 YEARS OLD • A severe and progressive blinding eye disease that begins in childhood 26 YEARS OLD • Affects 1 in every 3,500 p

  7. Slide 7: RP11 is caused by insufficient expression of one gene in the retina

    2. RP11 is caused by insufficient expression of one gene in the retina Unaffected individual RP11 patient1 PRPF31 PRPF31 PRPF31 PRPF31 PRPF31 PRPF31 DNA RNA Protein DNA RNA Protein Functional PRPF31 expression = 100% Functional PRPF31 expression = ~50% PYC THERAPEUTICS 4 4 1. Hafler BP, et al. Cours

  8. Slide 8: VP-001 addresses the underlying cause of RP11 and leads to functional benefit

    3. VP-001 addresses the underlying cause of RP11 and leads to functional benefit in patient-derived cell and organoid models 1. VP-001 is capable of completely rescuing the haploinsufficiency responsible for causing the disease in patient-derived models a) Upregulation of PRPF31 mRNA in RP11 iPSC-RP

  9. Slide 9: VP-001 is now generating data as the first drug candidate in clinical trials for RP11

    Phase 1 First-in-Human Study of VP-001 VP-001 is now generating data as the first drug candidate in clinical trials for RP11 6

  10. Slide 10: PLATYPUS: Study Design

    PLATYPUS: Study Design PYC THERAPEUTICS 7 7

  11. Slide 11: PLATYPUS: Safety and Exploratory Efficacy Endpoints

    PLATYPUS: Safety and Exploratory Efficacy Endpoints Safety monitored by evaluation of: Exploratory efficacy evaluated using: • Ocular and non-ocular adverse events (AEs) • Perimetry • Clinical chemistry parameters • Microperimetry • Best-corrected visual acuity (BCVA) • BCVA • Perimetry • SD-OCT • M

  12. Slide 12: VP-001 is safe in all dose cohorts in SAD (no serious adverse events)

    4a. VP-001 is safe in all dose cohorts in SAD (no serious adverse events) No Serious Adverse Events (SAEs) were observed (across all dose cohorts) Dose (patient ID) and study eye TEAE Relationship to VP-001 3 mcg (001-1-001) Vitreous floaters (OU) Not Related OS Vitreous opacities (OU) Not Related V

  13. Slide 13: VP-001 is safe in all dose cohorts in SAD (no significant change in ocular measurements)

    4b. VP-001 is safe in all dose cohorts in SAD (no significant change in any ocular measurements of safety) No significant changes in any ocular measurements indicating safety (across all cohorts) Retinal Central Subfield Thickness Measurements (used as representative data) All Cohorts Pooled Cohort

  14. Slide 14: Encouraging signs of efficacy signal in patient treated with 30 µg VP-001

    5. Encouraging signs of efficacy signal (microperimetry data) in patient treated with 30 µg VP-001 (8 week follow up comparison to fellow eye) Baseline (December 2023) Week 8 Post-Dosing Findings Microperimetry measurements (#005-1-002) Mean threshold (baseline) 12.5 Mean threshold (week 4) 13.1 Stu

  15. Slide 15: Microperimetry – Subject #005-1-002, Study Eye, Week 8 Post-Dosing – FAF & OCT

    Microperimetry – Subject #005-1-002, Study Eye, PLATYPUS, Week 8 Post-Dosing – FAF & OCT PYC THERAPEUTICS 12 12

  16. Slide 16: Encouraging signs of efficacy signal in patient treated with 30 µg VP-001

    5. Encouraging signs of efficacy signal (microperimetry data) in patient treated with 30 µg VP-001 (8 week follow up comparison to fellow eye) This patient is at earlier stage of disease progression than other patients in SAD 1 Greater microperimetry sensitivity improvements in treated eye (in 15 lo

  17. Slide 17: Protocol amendments filed with FDA to increase dosing of VP-001

    6. Protocol amendments filed with FDA to increase dosing of VP-001 • Protocol amendments to increase doses, filed with FDA for: • Addition of Cohort 4 (75 µg) to SAD study • Multiple-Ascending Dose (MAD) study to include repeat dosing of 30 µg and 75 µg • Both protocol amendments have revised inclus

  18. Slide 18: Phase 1 First-in-Human Study of VP-001 Conclusion

    Phase 1 First-in-Human Study of VP-001 Conclusion 15

  19. Slide 19: Conclusion – VP-001 is safe and shows encouraging signs of efficacy

    Conclusion – VP-001 is safe and shows encouraging signs of efficacy • VP-001 is safe in all dose cohorts tested to date • No serious adverse events observed, no significant changes in ocular measurements indicating safety • Encouraging signs of efficacy signal observed (microperimetry data) • In a s

  20. Slide 20: Acknowledgements

    Acknowledgements Dr. Mark Pennesi Dr. David Birch Dr. Lesley Everett Dr. Thiran Jayasundera Dr. John Chancellor Retina Foundation Retina Foundation OHSU Casey Eye Kellogg Eye Center Alkek Eye Center of Southwest of Southwest Institute University of Michigan Baylor College of Medicine Dr. Fred Chen D

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