Pyc Therapeutics Limited · ASX:PYC
Pyc Therapeutics — ARVO 2026
70 slides from Pyc Therapeutics's conference presentation, May 4, 2026
Slide 1: Presentation of Ophthalmology Clinical Trial Data at ARVO Conference
4 May 2026 PRESENTATION OF OPHTHALMOLOGY CLINICAL TRIAL DATA AT ARVO CONFERENCE • PYC is a precision medicine company dedicated to changing the lives of patients with genetic diseases who have no treatment options available • PYC is developing two investigational drug candidates that have the potent
Slide 2: About PYC Therapeutics and Forward looking statements
PYC today announces that data from the ongoing Phase 1/2 trials of both VP-001 and PYC- 001 in RP11 and ADOA respectively will be presented at the Association for Research in Vision and Ophthalmology (ARVO) conference in Denver, Colorado between 3 and 7 May 2026. The presentations will highlight the
Slide 3: Approved for release by the Board; Contact us
This ASX announcement was approved and authorised for release by the Board of PYC Therapeutics Limited CONTACT US Investor relations and media contact investor@pyctx.com pyctx.com | ACN 098 391 961 3
Slide 4: SUNDEW: A Phase 1A Single Ascending Dose Study of PYC-001
SUNDEW: A Phase 1A Single Ascending Dose Study of PYC-001; a peptide conjugated oligonucleotide designed to treat OPA1 mutation-associated Autosomal Dominant Optic Atrophy Patrick Yu-Wai-Man1, Clare Fraser2, Doron Hickey3, Andrea Vincent4, George Mitchell5, Timothy Masarei5, Sri Mudumba5, Paula Cunn
Slide 5: Adaptive optics imaging, microperimetry and optoretinography following VP-001
Adaptive optics imaging, microperimetry and optoretinograhy following intravitreal injection of VP-001 in patients with PRPF31-associated retinitis pigmentosa Jessica I. W. Morgan Nivedhitha Govindasamy, Peiluo Xu, Yu You Jiang, Raymond L. Warner, Thiran Jayasundera, Sandeep Grover, George Mitchell,
Slide 6: We thank the following funding sources
We thank the following funding sources: NIH R01EY030227 NIH P30EY001583
Slide 7: The people who make it happen!
The people who make it happen! Jing Zhang Yu You Jiang Peiluo (Angela) Xu Nivedhitha Govindasamy Shaofeng Huang Raymond L. Warner Thanks to Alfredo Dubra for sharing the AOSLO optical design and software and Austin Roorda for sharing AO microperimetry technology. Disclosure: I am an inventor on a pa
Slide 8: Retinitis Pigmentosa
Retinitis Pigmentosa 6 years old 26 years old • Affects 1 in 3,000- 5,000 individuals • Rods degenerate initially with subsequent degeneration of Patients experience night blindness followed by loss of cones and RPE peripheral and then central vision - legal blindness occurs in the 4th or 5th decade
Slide 9: Retinitis Pigmentosa type 11
Retinitis Pigmentosa type 11 • 1-3% of RP RP11 patient • Caused by mutations on the PRPF31 DNA PRPF31 RNA PRPF31 Protein PRPF31 gene • Autosomal dominant inheritance Functional PRPF31 expression = ~50% • VP-001: a RNA therapeutic that leads to upregulation of PRPF31
Slide 10: Experimental treatment of RP11 using VP-001
Experimental treatment of RP11 using VP-001 Progress to date Open Label Phase 1a SAD Phase 1b MAD Study Phase 2/3 Study1 Extension Platypus Wallaby • Safety & Tolerability • Efficacy • Safety & Tolerability • Primary: Low-Luminance Visual Acuity • Efficacy • Secondary: Microperimetry • Low-Luminance
Slide 11: Patient 1 treatment timeline
Patient 1 1st injection 2nd injection T0 T +12 weeks 30 mcg (005-1-002, Platypus/Dingo) Baseline Post treatment 1 Post treatment 2 Pre treatment AO T +6 weeks T +18 weeks T -2 days
Slide 12: Patient 1 and 2 treatment timelines
Patient 1 1st injection 2nd injection T0 T +12 weeks 30 mcg (005-1-002, Platypus/Dingo) Baseline Post treatment 1 Post treatment 2 Pre treatment AO T +6 weeks T +18 weeks T -2 days Patient 2 1st injection 2nd injection 3rd injection T0 T +7 weeks T +15 weeks 75 mcg (003-1-003, Wallaby/Dingo) Baselin
Slide 13: Treatment timelines and imaging methods
Patient 1 1st injection 2nd injection T0 T +12 weeks 30 mcg • Adaptive optics scanning (005-1-002, Platypus/Dingo) light ophthalmoscopy • Optoretinography • Adaptive optics Baseline Post treatment 1 Post treatment 2 microperimetry Pre treatment AO T +6 weeks T +18 weeks T -2 days • OCT Patient 2 1st
Slide 14: Study objective: evaluate retinal structure and function
Evaluate retinal structure and function in PRPF31- associated retinitis pigmentosa before and after unilateral treatment with experimental VP-001
Slide 15: Patient 2 Baseline, Pre-treatment
Patient 2 Baseline, Pre-treatment
Slide 16: Patient 2: Degenerative microcysts over time
Patient 2: Degenerative microcysts over time Injected eye
Slide 17: Patient 2: Degenerative microcysts over time
Patient 2: Degenerative microcysts over time Control eye
Slide 18: Patient 2: Degenerative microcysts over time
Patient 2: Degenerative microcysts over time Injected eye Control eye
Slide 19: Patient 2: Degenerative microcysts over time
Patient 2: Degenerative microcysts over time Baseline Week 29 Injected eye Control eye
Slide 20: Patient 2: Peak cone density over time
Patient 2: Peak cone density over time
Slide 21: Patient 2: Peak cone density over time
Patient 2: Peak cone density over time
Slide 22: Patient 2: Peak cone density over time
Patient 2: Peak cone density over time 106,039 cones/mm2
Slide 23: Patient 2: Peak cone density over time
Patient 2: Peak cone density over time 106,039 cones/mm2
Slide 24: Patient 2: Peak cone density over time
Patient 2: Peak cone density over time *Arrows depict VP-001 injections 106,039 cones/mm2
Slide 25: Optoretinography
Optoretinography • Objective measure of photoreceptor function. • An optical signal that occurs in response to a visible stimulus.
Slide 26: Optoretinography
Optoretinography *I a ta o.*f ’ i a EN i BS. gett hl °. > ao. 15 “a eo * % ” 7 ~-stcwe®
Slide 27: Optoretinography
Optoretinography — a _ oS _ N_@_ Stes
Slide 28: Optoretinography
Optoretinography ef 7 6 5 P| - qi 2 1 0 A aa °o 0s 1 1.5 2 25
Slide 29: Optoretinography
Optoretinography 10' —-—- CIE Function v10 A. 2 | bs. = y, S. Ss Pa o 4 S104” t < ) 3s Q L102 \ 109+ 450 500 550 600 650 700 tt tt ot Wavelength (nm) Non-invasive assessment of human cone photoreceptor function
Slide 30: Optoretinography
Optoretinography 10" —-—- CIE Function Action (relative) 2.3 S a 3. 10750 500 550 600 650 700 tt tt ot Wavelength (nm) EOMESICATOBtIes Express Non-invasive assessment of human cone photoreceptor function wn = Session 2 Amplitude n w 1 2 3 4 5 Session 1 Amplitude Repeatability and reciprocity of the
Slide 31: Optoretinography
Optoretinography 10' wn —---- CIE Function 10 v2 o0 : s” £4 38 5 a = 2 € 26 10" <3 = c a < 5 t Ss = o 4 I 2102, 4 5 4 32 co, g 2 7) 10°4 1 0 450 500 550 600 650 700 1 2 3 4 5 1T 27 #47 «8F 16T tt tt ot Wavelength (nm) Session 1 Amplitude EOMESICATOBtIes Express fsomeseromserness | Non-invasive asses
Slide 32: Optoretinography, Patient 2 Baseline, OS
Optoretinography, Patient 2 Baseline, OS
Slide 33: Optoretinography, Patient 2 Baseline, OS
Optoretinography, Patient 2 Baseline, OS
Slide 34: Optoretinography, Patient 2 Baseline
Optoretinography, Patient 2 Baseline Baseline ORG amplitude is reduced from normal.
Slide 35: Optoretinography, Patient 2 over time
Optoretinography, Patient 2 over time Post-injection 1 Post-injection 2 Post-injection 3 Post-injection Baseline 6 weeks 11 weeks 17 weeks 29 weeks Injected eye Injected eye
Slide 36: Optoretinography, Patient 2 over time
Optoretinography, Patient 2 over time Post-injection 1 Post-injection 2 Post-injection 3 Post-injection Baseline 6 weeks 11 weeks 17 weeks 29 weeks Injected eye Injected eye
Slide 37: Optoretinography, Patient 2 over time
Optoretinography, Patient 2 over time Post-injection 1 Post-injection 2 Post-injection 3 Post-injection Baseline 6 weeks 11 weeks 17 weeks 29 weeks Injected eye Injected eye
Slide 38: Optoretinography, Patient 2 over time
Optoretinography, Patient 2 over time Post-injection 1 Post-injection 2 Post-injection 3 Post-injection Baseline 6 weeks 11 weeks 17 weeks 29 weeks Injected eye Injected eye
Slide 39: Optoretinography, Patient 2 over time
Optoretinography, Patient 2 over time Post-injection 1 Post-injection 2 Post-injection 3 Post-injection Baseline 6 weeks 11 weeks 17 weeks 29 weeks Injected eye Injected eye
Slide 40: Optoretinography, Patient 2 over time
Optoretinography, Patient 2 over time Post-injection 1 Post-injection 2 Post-injection 3 Post-injection Baseline 6 weeks 11 weeks 17 weeks 29 weeks Injected eye Injected eye
Slide 41: Optoretinography, Patient 2 over time
Optoretinography, Patient 2 over time Post-injection 1 Post-injection 2 Post-injection 3 Post-injection Baseline 6 weeks 11 weeks 17 weeks 29 weeks Control eye Control eye
Slide 42: Optoretinography, Patient 2 over time
Optoretinography, Patient 2 over time Parafovea Near transition zone
Slide 43: AO microperimetry
AO microperimetry
Slide 44: AO microperimetry
AO microperimetry • Image the retina with infrared illumination with AOSLO. • Deliver a 550 nm stimulus to targeted locations, repeatedly. • Stimulus size varied by eccentricity but remained the same across time points for all locations. • Yes/No response for each trial. • QUEST adaptive staircase o
Slide 45: AO microperimetry
AO microperimetry 1r me om e 0.85 5 e 8 0.6+ ee 5 © omee B 04+ E - 02+ -° e 0 1 J-.---l.. 1 -30 -20 -10 0 Trial intensity (dB) Baseline
Slide 46: AO microperimetry
AO microperimetry Fraction seen -30 -20 -10 0 Trial intensity (dB) Baseline
Slide 47: AO microperimetry
AO microperimetry 25 1 20 0.8 5 g 8 0.6 2" c 2 = Brot ms 04 5 © a Wwe 0.2 8 ) 0 30 -20 -10 0) Ss & ££ & ¥£ Trial intensity (dB) FF WX ah ps Timepoints Baseline
Slide 48: AO microperimetry – sensitivity over time
AO microperimetry – sensitivity over time Post-injection 1 Baseline 6 weeks
Slide 49: AO microperimetry – sensitivity over time
AO microperimetry – sensitivity over time Post-injection 1 Post-injection 2 Baseline 6 weeks 11 weeks
Slide 50: AO microperimetry – sensitivity over time
AO microperimetry – sensitivity over time Post-injection 1 Post-injection 2 Post-injection 3 Baseline 6 weeks 11 weeks 17 weeks
Slide 51: AO microperimetry – sensitivity over time
AO microperimetry – sensitivity over time Post-injection 1 Post-injection 2 Post-injection 3 Post-injection Baseline 6 weeks 11 weeks 17 weeks 29 weeks
Slide 52: Sensitivity over time, Patient 2
Sensitivity over time, Patient 2 Injected eye
Slide 53: Sensitivity over time, Patient 2
Sensitivity over time, Patient 2 Control eye Injected eye
Slide 54: Patient 1
Patient 1
Slide 55: Patient 1
Patient 1
Slide 56: Sensitivity over time, Patient 1
Sensitivity over time, Patient 1 Control eye Injected eye
Slide 57: Summary
Summary Cone density ORG amplitude AO sensitivity • Cone density, ORG amplitude, and AO sensitivity can be used to assess experimental therapies for retinal disease. • Cone structure and function were maintained or improved following treatment with VP-001. • The data support future studies to assess
Slide 58: A Phase 1B Multiple Ascending Dose Study of VP-001
A Phase 1B Multiple Ascending Dose Study of VP-001; a peptide conjugate of oligonucleotide designed to treat PRPF31-related Retinitis Pigmentosa Dr. Fred Chen Lions Eye Institute, Perth, Australia ARVO 2026
Slide 59: VP-001 for the treatment of Retinitis Pigmentosa type 11 (RP11)
VP-001 for the treatment of Retinitis Pigmentosa type 11 (RP11) Introduction to RP11 and VP-001 1) Patients with RP11 experience progressive and irreversible vision loss beginning in childhood – there are no treatment options available 2) RP11 is caused by a haploinsufficiency of one gene (PRPF31) i
Slide 60: Patients with RP11 experience progressive and irreversible vision loss beginning in childhood
1) Patients with RP11 experience progressive and irreversible vision loss beginning in childhood1-3 Illustration of the degeneration in sight experienced by an RP11 patient1-3 6 years old 26 years old 46 years old Patients experience night blindness followed by loss of peripheral and then central vi
Slide 61: RP11 is caused by a haploinsufficiency of one gene (PRPF31)
2) RP11 is caused by a haploinsufficiency of one gene (PRPF31) in the retina 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
Slide 62: VP-001 rescues the haploinsufficiency causing RP11 in patient-derived models
3) VP-001 shows the ability to rescue the haploinsufficiency causing RP11 in patient-derived models PRPF31 mRNA Expression in Patient iPSC-RPE 6 days post VP-001 treatment 3) Rescues appearance and Unaffected RP11 RP11 + VP-001 Scrambled control oligo Dose-dependent target gene rescue to wild type l
Slide 63: VP-001 is the first clinical stage drug candidate for RP11
4) VP-001 is the first clinical stage drug candidate for RP11 and has the potential to become the first approved treatment option1,2 FDA Type D meeting Q1 20263 Progress to date Open Label Phase 1a SAD Phase 1b MAD Study Phase 2/3 Study1 Extension Platypus Wallaby • Safety & Tolerability • Efficacy
Slide 64: VP-001 is safe and well-tolerated in RP11 patients
5) VP-001 is safe and well-tolerated in RP11 patients • 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 Safety outcomes1 • Treatment-Emergent Adverse Events were mostly mild, and procedure re
Slide 65: Efficacy endpoints that matter most for patients with RP11
6) The efficacy of VP-001 is being evaluated using endpoints that matter most for patients with RP11 1 Assessment of a patient’s ability to see in low-light conditions - providing a direct assessment of rod function Low-Luminance Visual Acuity (LLVA) LLVA is a more sensitive marker of impaired centr
Slide 66: Clinically meaningful improvements in visual acuity
6(a)(i) RP11 patients treated with VP-001 have shown clinically meaningful improvements in visual acuity1 Human efficacy: Change in Low-Luminance Visual Acuity (LLVA) in RP11 patients2,3 25 20 Change from baseline in LLVA 15 VP-001 Treated Eyes2 (ET-DRS letters) 10 Mean VP-001 Treated Eyes Line of f
Slide 67: Greater proportion of treated eyes show meaningful LLVA improvements
6(a)(ii) A greater proportion of RP11 eyes treated with VP-001 show clinically meaningful1 improvements in LLVA 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 timepoints3 timepoints3 timepoi
Slide 68: Clinically meaningful improvements in retinal sensitivity by microperimetry
6(b) RP11 patients treated with VP-001 have shown clinically meaningful1 improvements in retinal sensitivity (as assessed by microperimetry) Bar graphs showing mean number of loci improved by >7dB from baseline for patients enrolled in DINGO2,3 8 ✱✱✱ Mean number of loci improving by >7dB from baseli
Slide 69: Patients report improved vision and quality of life after treatment
6(c) 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 “I see airplanes in the sky (never have before), stars at night, animals/creatures along the road…” - “My central vi
Slide 70: Long-term data will be used to finalise registrational trial design
7) Long-term data from the ongoing Phase 2 study will be used to finalise the design of a potential registrational trial in RP111 • It is expected that the final registrational trial will involve2: • Primary endpoint – mean change in Low Luminance Visual Acuity (LLVA) • Supported by key secondary en