Pyc Therapeutics Limited · ASX:PYC
Pyc Therapeutics — Foundation Fighting Blindness Retinal Therapeutics Innovation Summit 2025
25 slides from Pyc Therapeutics's conference presentation, May 2, 2025
Slide 1: Foundation Fighting Blindness Presentation Materials
31 October 2 May 2025 2022 FOUNDATION FIGHTING BLINDNESS PRESENTATION MATERIALS PERTH, Australia and SAN FRANCISCO, California – 2 May 2025 For personal use only PYC Therapeutics Limited (ASX:PYC) (PYC or the Company) is a precision medicine Company dedicated to changing the lives of patients with g
Slide 2: For personal use only
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. This ASX announcement was approved and authorised for
Slide 3: Advancing PRPF31-Related Retinitis Pigmentosa Treatment: Key Insights from the VP-001 Phase 1B Study
Advancing PRPF31-Related Retinitis For personal use only Pigmentosa Treatment: Key Insights from the VP-001 Phase 1B Study Retinal Therapeutics Innovation Summit May 2, 2025 Lesley Everett, MD, PhD Paul H. Casey Ophthalmic Genetics Division Casey Eye Institute Oregon Health & Science University Port
Slide 4: Co-Investigators and Co-Authors
For personal use only Co-Investigators and Co-Authors Dr. David Birch Dr. Thiran Jayasundera Dr. John Chancellor Retina Foundation Dr. Sandeep Grover Kellogg Eye Center Alkek Eye Center Dr. Byron Lam of the Southwest University of Florida University of Michigan Baylor College of Medicine Bascom Palm
Slide 5: Disclosures
Disclosures Investigator or Co-Investigator for Clinical Trials (no financial disclosures): For personal use only 4D Molecular Therapeutics Ocugen Ascidian Therapeutics SepulBio Atsena Therapeutics Sparing Vision Beacon Therapeutics Spark Therapeutics Biogen SpliceBio Editas Medicine Sanofi Foundati
Slide 6: Retinitis Pigmentosa type 11 is a progressive blinding eye disease with no treatment options
Retinitis Pigmentosa type 11 (RP11) is a progressive blinding eye disease for which there are no treatment options (PRPF31) Degenerative sight of an RP11 patient Retinitis Pigmentosa (RP)1,2 For personal use only 6 YEARS OLD • A severe and progressive blinding eye disease that begins in childhood •
Slide 7: RP11 is caused by insufficient expression of PRPF31 in the retina and RPE
RP11 is caused by insufficient expression of PRPF31 in the retina and RPE PRPF31 expression For personal 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% https
Slide 8: VP-001 is designed to address the underlying cause of RP11
VP-001 is designed to address the underlying cause of RP11 • Cell-penetrating peptide conjugated to an oligonucleotide to modulate CNOT3 expression • Intravitreal injection (50 µl) For personal use only Without VP-001 With VP-001 ASO CNOT3 Exon 15 Intron Exon 16 Intron Exon 17 Intron Exon 18 Exon 15
Slide 9: VP-001's Robust Preclinical Proof of Concept
VP-001’s Robust Preclinical Proof of Concept VP-001 shows the ability to rescue PRPF31 haploinsufficiency in patient-derived models For personal use only PRPF31 mRNA Expression in Patient iPSC-RPE 6 days post VP-001 treatment Unaffected RP11 RP11 + VP-001 Scrambled control oligo 3) Rescues morpholog
Slide 10: Overview of VP-001 Clinical Trials
Overview of VP-001 Clinical Trials Current stage For personal use only Data from the extension study Completed Enrollment Phase 1/2 will inform the final design and Phase 1/2 studies Open-Label Extension Study duration of the registrational trial PLATYPUS: Single Ascending Dose Study and Part B Exte
Slide 11: Overview of the Phase 1/2 studies of VP-001 in RP11
Overview of the Phase 1/2 studies of VP-001 in RP11 Overview of PLATYPUS Overview of WALLABY For personal use only Primary Outcome: Safety & Tolerability Primary Outcome: Safety & Tolerability Single Ascending Dose (SAD) study SAD ‘Part B’ Multiple Ascending Dose (MAD) study Extension study • n = 3
Slide 12: Inclusion criteria progressively refined to seek an accelerated efficacy signal
The inclusion criteria was progressively refined to seek an accelerated efficacy signal in the ongoing studies of VP-001 Study Cohorts Inclusion criteria For personal use only • PRPF31 mutation 3, 10, 30 mcg PLATYPUS • Light perception SAD Study 75 mcg • PRPF31 mutation • At least one of the below:
Slide 13: Key outcomes from ongoing clinical studies of VP-001 in RP11
For personal use only Key outcomes from ongoing clinical studies of VP-001 in RP11
Slide 14: Overview of patient exposure to VP-001 in the Phase 1/2 trials
Overview of patient exposure to VP-001 in the Phase 1/2 trials Study Cohort Patient ID Received repeat doses of > 30 µg VP-001 Number of doses > 30 µg VP-001 patient has received SAD 3 µg Patient 1 Yes – patient was eligible for Part B study 2 (first dose was < 30 µg) SAD 3 µg Patient 2 Yes – patien
Slide 15: No treatment emergent-serious adverse events (TE-SAEs)
No treatment emergent-serious adverse events (TE-SAEs) • No Treatment Emergent-Serious Adverse events observed in For personal use only any subjects dosed with VP-001 to date • Including subjects who received repeat doses of VP-001 • Treatment-Emergent Adverse Events were mostly mild and/or Safety p
Slide 16: Dose-dependent improvement in low-luminance visual acuity
A dose-dependent improvement in low-luminance visual acuity was observed in the first study of VP-001 for Retinitis Pigmentosa type 11 20 For personal use only Change from baseline in Low-Luminance 3µg treated eyes (n=3) 10µg treated eyes (n=2)3 Visual Acuity (ET-DRS letters) 15 30µg treated eyes (n
Slide 17: Transition to therapeutically relevant repeat doses
Transition to therapeutically relevant repeat doses (subsequent data shown will reflect patients treated with multiple 30 or 75 µg doses) Overview of PLATYPUS Overview of WALLABY For personal use only Primary Outcome: Safety & Tolerability Primary Outcome: Safety & Tolerability Single Ascending Dose
Slide 18: Improved vision after VP-001 treatment on two registrational endpoints
Improved vision after VP-001 treatment on two registrational endpoints with high impact for patients with RP11 I For personal use only 1 LLVA is a more sensitive marker of M P Low-Luminance impaired central vision than BCVA R O Baseline Visual Acuity and has been linked to higher V E Week 16 (+4 let
Slide 19: Improvements in low-luminance visual acuity within 12 months
RP11 patients treated with VP-001 have shown improvements in low- luminance visual acuity within 12 months VP-001 treated eyes1 show improvement in LLVA compared to the disease progression observed in the Natural History Study (NHS) of patients with RP112,3 For personal use only 20 1 LLVA 15 Mean VP
Slide 20: Greater proportion of treated eyes show LLVA improvement versus untreated eyes
A greater proportion of RP11 eyes treated with VP-001 show improvement in LLVA compared to untreated eyes RP11 patients treated with VP-001 have shown improvements in LLVA relative to untreated RP11 Natural History Study subjects For personal use only 100 Data from 56-weeks 1 LLVA onwards will be as
Slide 21: Microperimetry improvements observed in VP-001 treated eyes
Microperimetry improvements have been observed in VP-001 treated eyes VP-001 treatment Baseline Week 32 12.5 dB 15.1 dB For personal use only 7 loci improved by >7dB Patient 1 2 MP 0.2 dB 6.3 dB 23 loci improved by >7dB Patient 2 4.6 dB 4.9 dB Patient 3 12 loci improved by >7dB PYC THERAPEUTICS Mean
Slide 22: Bilateral improvements in retinal sensitivity following unilateral injection
Bilateral improvements in retinal sensitivity following unilateral injection VP-001 treatment UNTREATED Baseline Week 32 Baseline Week 32 12.5 dB 15.1 dB 8.1 dB 10.2 dB For personal use only Patient 1 2 MP 0.2 dB 6.3 dB 0.3 dB 4.7 dB Patient 2 4.6 dB 4.9 dB 4.5 dB 4.6 dB Patient 3 The phenomenon of
Slide 23: Clinically meaningful improvements in microperimetry in RP11 patients
Clinically meaningful improvements in microperimetry have been observed in RP11 patients treated with VP-0011 There is a high degree of confidence that it is likely attributable to a genuine treatment effect rather than random chance2 For personal use only 60 10 VP-001 treated eyes 2 MP % of eyes wi
Slide 24: Patients report improved vision and quality of life after VP-001
Multiple RP11 patients have reported improved vision and quality of life after treatment with VP-001 - “For the first time, I’ve seen airplanes in the sky- it was amazing! When we travel to the national parks and stay in our camper van, now I can get around at dusk by myself in unfamiliar For person
Slide 25: Acknowledgements
Acknowledgements OHSU Ophthalmic Genetics Clinical Trials Funding & Support Paul Yang, MD, PhD Hardew Mahto Foundation Fighting Blindness Career Development Mark Pennesi, MD, PhD Alexis Mooney Award (2022-2027) CD-GE-0822-0831-OHSU For personal use only Andreas Lauer, MD Andrew Lamborn Steven Bailey
PYC Therapeutics — Foundation Fighting Blindness Retinal Therapeutics Innovation Summit 2025 on 6ix