Slide 1: Presentation of RP11 Clinical Trial Data at APVRS 2024
2231 November October 2024 2022 PRESENTATION OF RP11 CLINICAL TRIAL DATA AT APVRS 2024 • PYC is developing the first drug candidate (VP-001) for a blinding eye disease of childhood called Retinitis Pigmentosa type 11 (RP11) For personal use only • A/Prof. Fred Chen of the Lions Eye Institute will pr
Slide 2: PYC's RP11 Program Overview and About PYC Therapeutics
registrational endpoints in Retinitis Pigmentosa (RP) and are closely correlated to experienced disability in patients with RP 4. PYC is currently preparing for the transition into a registrational trial for VP-001 in patients with RP11 including engaging with the US FDA in 1H 2025. PYC’s RP11 Progr
Slide 3: Forward looking statements
other factors, many of which are outside the Company’s control. Important factors that could cause actual results to differ materially from assumptions or expectations expressed or implied in this ASX announcement include known and unknown risks. Because actual results could differ materially to ass
Slide 4: VP-001 for the treatment of Retinitis Pigmentosa type 11
For personal use only VP-001 for the treatment of Retinitis Pigmentosa type 11 (RP11) APVRS Presentation - Results of ongoing SAD and MAD studies in RP11 patients PYC Therapeutics 23 November 2024 1
Slide 5: Disclaimer
Disclaimer The purpose of this presentation is to provide an update of the business which are outside PYC’s control. Important factors that could cause For personal use only of PYC Therapeutics Limited (ASX:PYC) [‘PYC’]. These slides have been actual results to differ materially from assumptions or
Slide 6: Executive Summary
Executive Summary • VP-001 is the first drug candidate to have entered human trials in a blinding eye disease of For personal use only childhood called Retinitis Pigmentosa type 11 (RP11) • VP-001 has disease-modifying potential in RP11 as it addresses the underlying cause of the disease • This pres
Slide 7: VP-001 is the first drug candidate to have entered human trials in RP11
VP-001 is the first drug candidate to have entered human trials in RP11 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 26 YEARS OLD • Affects 1 in every 3,500 people (RP11 a
Slide 8: VP-001 has disease-modifying potential in RP11
VP-001 has disease-modifying potential in RP11 as it addresses the underlying cause of the disease Insufficient PRPF31 CNOT3 reduces VP-001 inhibits CNOT3 to For personal use only expression causes RP111 PRPF31 expression2 increase PRPF31 expression Untreated RP11 patient RP11 patient treated with V
Slide 9: VP-001 is currently progressing through Phase 1/2 trials in patients with RP11
VP-001 is currently progressing through Phase 1/2 trials in patients with RP11 For personal use only Clinical pathway2 FDA special designations 2023 2024 2025 2026 2027 2028 Progress Potentially accelerating path to market: Natural History study 1. Fast Track – Granted in 20232 Ph1 Single 2. Orphan
Slide 10: Two concurrent multiple dose studies in patients with RP11
VP-001 is currently progressing through two concurrent multiple dose studies in patients with RP11 Single Ascending Dose (SAD) study SAD ‘Part B’ Multiple Ascending Dose For personal use only Extension study (MAD) study Cohort 4 Cohort 4 Cohort 2 (75 mcg) (75 mcg) (75 mcg) Cohort 3 Cohort 3 Cohort 1
Slide 11: Patients treated with VP-001 have seen meaningful improvements in vision
Patients who have received VP-001 have seen meaningful improvements in vision in their treated eye I Efficacy highlights For personal use only M 1 P R Improvement in LLVA in VP-001 O Baseline Low-Luminance V Month 4 (+6 letters in treated eye, n=8)1 treated eyes compared to E Visual Acuity (LLVA) M
Slide 12: Improvements in functional vision (LLVA) observed following VP-001 treatment
1) Improvements in functional vision (LLVA) have been observed following treatment with VP-001 VP-001 treated eyes1 show greater mean change from baseline in LLVA compared to untreated eyes For personal use only 20 16 12 Mean LLVA Change from Baseline 8 I (Treated eye - untreated eye) (EDTRS letters
Slide 13: Improvements in functional vision (LLVA) observed following VP-001 treatment
1) Improvements in functional vision (LLVA) have been observed following treatment with VP-001 A greater % of VP-001 treated eyes1 demonstrate > 5, > 10, > 15 letter change from baseline in LLVA For personal use only > 5 letter improvement > 10 letter improvement > 15 letter improvement 100 100 100
Slide 14: Improvements in mean retinal sensitivity observed following VP-001 treatment
2) Improvements in mean retinal sensitivity have been observed following treatment with VP-001 Retinal sensitivity as assessed by microperimetry (MP) is enhanced in eyes1 treated with VP-001 For personal use only 4 3 Beacon Therapeutics (AGTC-501) Change from Baseline in Treated Eye5 Mean Sensitivit
Slide 15: Reduction in the number of scotomas observed following VP-001 treatment
3) A reduction in the number of scotomas1 has been observed following treatment with VP-001 Eyes treated2 with VP-001 demonstrate a reduced number of scotomas (nonfunctional area of retina1) only 8 4 Change from Baseline 2 use I M Number of Scotomas 0 Patients receiving P personal (+95% CI) > 30 µg
Slide 16: PYC will now seek to initiate a registrational study in RP11
PYC will now seek to initiate a registrational study in RP11 designed to support a New Drug Application for VP-0011 For personal use only 2025 Q1 Q2 Q3 Q4 Objectives • Finalise • Align with FDA • Initiate registrational study in RP11 MAD/SAD on pivotal open label study design extension • Discuss Sup
Slide 17: Acknowledgements
Acknowledgements For personal 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 Fl