Slide 1: Q3 Investor Update
For personal use only Life-changing science Q3 Investor Update 30 August 2024
Slide 2: 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 3: Today's topics
Today’s topics • Introduction to PYC For personal use only • Industry trends • RNA therapies continue to feature as a major emerging therapeutic modality • PYC’s progress • PYC’s RNA therapeutic pipeline is progressing well with all programs set to deliver a meaningful milestone in 20241: • RP type
Slide 4: Introduction to PYC
For personal use only Life-changing science Introduction to PYC
Slide 5: PYC discovers and develops new drugs for patients with severe diseases
For personal use only PYC discovers and develops new drugs for patients who have severe diseases and no treatment options available. The Company is specifically interested in the creation of precision therapies that address the underlying cause of diseases driven by a single genetic mutation. PYC TH
Slide 6: PYC has carefully selected the domain in which it operates
PYC has carefully selected the domain in which it operates PYC’s strategy For personal use only 1 2 3 4 Pillar Single gene disorders Effective in human 3D Haploinsufficient Drugs that overcome ‘mini-organs’ disease mechanism the delivery challenge3 Clinical insights Highest probability Where RNA dru
Slide 7: PYC has built a pipeline of first-in-class drug candidates
PYC has built a pipeline of first-in-class drug candidates with disease-modifying potential For personal use only Historical progress Progress expected in 20241 Program Pre-clinical Clinical Prevalence2 VP-0013 Retinitis Pigmentosa Type 11 1 in 100,000 PYC-003 Polycystic Kidney Disease 1 in 1,000 PY
Slide 8: Industry trends
For personal use only Life-changing science Industry trends
Slide 9: PYC applies its RNA technology in rare single-gene disorders
PYC applies its RNA technology in the context of ‘rare’ single- gene disorders For personal use only “Across the genetics landscape, the winners will be those who prioritize the right conditions for treatment – and who have the capacity to carry through implementation” “With some 6,000 so-called mon
Slide 10: Rare disease is becoming more attractive for drug developers
Rare disease is becoming more attractive for drug developers For personal use only “Keeney cited regulators' growing willingness to consider surrogate endpoints for rare diseases as bolstering the larger business model for developing these drugs”1 PYC THERAPEUTICS 10 1. FierceBiotech 5 March 2024 Af
Slide 11: RNA is now leading the market with stellar clinical results
For personal use only “RNA is now leading the market with stellar clinical results and outstanding long-term potential1” PYC THERAPEUTICS 11 1. Stifel Biopharma Market Update 12 August 2024
Slide 12: PYC's pipeline progress and RP11 deep dive
For personal use only Life-changing science PYC’s pipeline progress and RP11 deep dive
Slide 13: The focus of today's discussion is the immediate future in RP11
The focus of today’s discussion is the immediate future in RP11 PYC’s path to market is staged with human data read-outs for first-in-class drugs with disease-modifying potential1 For personal use only Program 2024 2025 2026 2027 2028 RP11 Ph1/2 study (underway) Registrational study NDA Product laun
Slide 14: RP11 program progressing through two concurrent multiple dose studies
PYC’s RP11 program is progressing through two concurrent multiple dose studies For personal use only Single Ascending Dose (SAD) study SAD extension Multiple Ascending Dose (MAD) study Cohort 4 Cohort 4 Cohort 2 (75 mcg) (75 mcg) (75 mcg) Cohort 3 Cohort 3 Cohort 1 (30 mcg) (30 mcg) (30 mcg) Expecte
Slide 15: Objectives
Objectives For personal use only • Introduce the relevant efficacy endpoints in the SAD and MAD studies • State the results observed to date • Provide context on why these are relevant and what we are looking for in the ongoing repeat dose studies1 PYC THERAPEUTICS 15 1. Based on management forecast
Slide 16: RP type 11 program has potential to be the first approved therapy
PYC’s Retinitis Pigmentosa Type 11 program has the potential to be the first approved therapy in this indication1 Degenerative sight of an RP11 patient Retinitis Pigmentosa (RP)2,3 For personal use only 6 YEARS OLD • A severe and progressive blinding eye disease that begins in childhood 26 YEARS OLD
Slide 17: What is microperimetry and why is it an endpoint in RP11 trials?
What is microperimetry and why are we interested in it as an endpoint in the RP11 trials? Microperimetry has been used extensively to characterise functional vision in a wide range of retinal For personal use only conditions, often detecting subtle defects in retinal sensitivity that precede visual
Slide 18: How do we monitor progression using microperimetry over time?
How do we monitor progression using microperimetry over time? Post single 30 µg treatment with PYC’s drug candidate For personal use only Baseline Week 4 Week 8 Week 16 Multiple patients in PYC’s ongoing Single Ascending Dose study have improved visual function after a single dose of PYC’s investiga
Slide 19: How can microperimetry be used as a registrational endpoint?
How can microperimetry be used as a registrational endpoint? The FDA has issued guidance on the use of microperimetry as a registrational endpoint1-3 For personal use only 1) Continuous outcomes (slopes) Clinically relevant difference in slope of mean sensitivity of a pre-defined area* (i.e., a slop
Slide 20: What do the results from the SAD show?
What do the results from the SAD show? Improved retinal function following treatment with VP-001 For personal use only Retinal sensitivity as assessed by microperimetry (MP) in SAD cohort 3 and 4 patients over time 4 3 I Change from Baseline in Treated Eye# Mean Sensitivity (whole grid) (dB) Beacon
Slide 21: What have other companies seen in Phase 1/2 trials in other forms of RP?
What have other companies seen in Phase 1/2 trials in other forms of Retinitis Pigmentosa? For personal use only PYC Therapeutics MeiraGTx – J&J Beacon Therapeutics VP-001 MGT009 AGTC-501 Disease (causative gene) Retinitis Pigmentosa (PRPF31) X-linked Retinitis Pigmentosa (RPGR) Efficacy in patient-
Slide 22: What other endpoints might we consider for the registrational study?
What other endpoints might we consider for the registrational study? For personal use only Microperimetry correlates with Low Luminance Visual Acuity (LLVA) and experienced disability in Retinitis Pigmentosa • Patients with Retinitis Pigmentosa show a more pronounced visual acuity deficit at low lum
Slide 23: Q&A
For personal use only Q&A