Slide 1: Successful Efficacy and Toxicity Results
For personal use only Successful Efficacy and Toxicity Results
Slide 2: Context
Context For personal use only PYC recently announced a series of successful results for its lead drug program and RNA therapeutics platform: ▪ Successful Efficacy Result on Path to First in Human Studies (see ASX Announcement 1 April 2020) ▪ Successful Toxicity Results Confirm Safety Advantage (see
Slide 3: PYC's successful efficacy readouts - Three primary messages
PYC’s successful efficacy readouts - Three primary messages For personal use only Patient impact Our target market has thousands of patients burdened by a high unmet need with no other drugs in clinical development, estimated to be worth >USD 1 billion Financial case We have enough capital to fund o
Slide 4: Patient impact: lead program targets a market with significant unmet need
Patient impact: PYC’s lead program targets a market with a significant unmet need For personal use only Prevalence 4,000-8,000 patients in ▪ Retinitis Pigmentosa affects 1 in 2,500 to 4,000 people the Western World with no ▪ 1.5-3% of Retinitis Pigmentosa is RP11 available treatment Impact/ 150-250k
Slide 5: Financial case: probability of reaching a market outcome is relatively high
Financial case: The probability of reaching a market outcome is relatively high For personal use only RP11 represents a $1-2B market Probability of success, Phase 1 to market, % x4 53 PYC’s lead drug 14 All Orphan Ocular Orphan (ex-oncology) Source: Wong et al. ‘Estimation of clinical trial success
Slide 6: Financial case: ability to fund lead program to market
Financial case: PYC has the ability to fund its lead program to market NOTE: DOES NOT REPRESENT A For personal use only COMMITMENT ON BEHALF OF PYC IND Phase 1 Phase 2 Approval Timeline, ~15-18 ~12 ~24 ~6 Months months months months months 30 Overseas spend Australian spend net of the R&D rebate Pot
Slide 7: Scientific validation: drug restored function in RP11-diseased cells
Scientific validation: PYC’s drug has restored function in RP11- diseased cells across the two major assays Impact of RP11 For personal use only Structure of the Retina Restoration Healthy RP11 Impact of RP11 with PYC Drug ▪ Shorter ‘stunted’ connecting cilium in TBD the photoreceptors ▪ Lower ‘phag
Slide 8: What is phagocytosis and how do we measure it?
Scientific validation: What is phagocytosis, why does it matter, and how do we measure it? What is phagocytosis and why does it How do we measure if PYC’s drug improves For personal use only matter? phagocytosis? Treated with Untreated PYC’s drug Treatment group treated with PYC’s drug (CPP-ASO) Flu
Slide 9: Drug increases phagocytosis in RP11-diseased cells
Scientific validation: PYC’s drug has shown it can increase phagocytosis in RP11-diseased cells Fig 1. Phagocytosis assay, 5mM 6hr timepoint For personal use only ** A) B) Untreated CPP-ASO RPE CPP-ASO Untreated NOTE: Green ‘specks’ are Phagocytosed outer- segments, with increased frequency of green
Slide 10: What is cilia length and how do we measure it?
Scientific validation: What is cilia length, why does it matter, and how do we measure it? What is cilia length and why does it How do we measure if PYC’s drug improves cilia length? For personal use only matter? Treated with Untreated PYC’s drug Treatment group Cellular treated with PYC’s level dru
Slide 11: Drug increases cilia length in RP11-diseased cells (RPE)
Scientific validation: PYC’s drug has shown it can increase cilia length in RP11-diseased cells (RPE) Fig 2. Cilia frequency and length assay, 5mM at Fig 3. Cilia frequency and length assay, For personal use only 5 days Penetrant patient 2.5mM at 5 days, Non-penetrant patient **** UNTREATED RPE Stun
Slide 12: Drug reaches and performs its function on its target gene
Scientific Validation: PYC’s drug successfully reaches and performs its intended function on its target gene For personal use only Photo- Fig 4. Exon skipping, retinal organoid Fig 5. Exon skipping, RPE receptors Day 14, 2 treatments Day 5, single treatment RPE 71 70 42 44 0 0 Untreated Patient 1 Pa
Slide 13: Successful efficacy results in context of in vivo results to date
Successful efficacy results in context of in vivo results to date Captured in previous pages Captured in following For personal use only pages Milestone Human cells 3D ‘retina in a dish’ Animal models Humans Patient impact and models Revenue PYC’s lead drug Has reversed the Has proven to be highly I
Slide 14: Drug shows functional and durable effects in animals
PYC’s drug shows functional and durable effects in animals Efficient and functional ASO delivery to Durable Effect reduced dosing frequency For personal use only target retinal cells in vivo Fig 6a. Exon skipping in mice retinas Fig 6b. Exon skipping in mice retinas 1.6mg IVT injection 1.6mg IVT inj
Slide 15: Drug shows minimal local toxicity in vivo
PYC’s drug also shows minimal local toxicity in vivo, highly important in the sensitive retina For personal use only Fig 7. Retinal stress marker expression in mice Day 5 post single 1.6mg IVT injection ▪ The safe delivery of drugs to the retina is critically important for ophthalmic (eye) therapeut
Slide 16: Safety profile of PYC's CPP is a major competitive advantage
Retinal cells are sensitive – the safety profile of PYC’s CPP is a major competitive advantage For personal use only Fig 8. OCT imaging from mice at day 21 post IVT injection (n=1) PYC lead CPP PYC control CPP No evidence of retinal thinning Evidence of severe retinal thinning GFAP 1.4x CPP with ele
Slide 17: For further information, please contact
ENDS For further information, please contact: For personal use only INVESTORS Kaggen Ausma CBO info@pyctx.com This ASX announcement was approved and authorised for release by the Board of PYC Therapeutics Limited. 17
Slide 18: About PYC Therapeutics
About PYC Therapeutics PYC Therapeutics (ASX: PYC) is a drug development company solving a major challenge in the development of a revolutionary For personal use only new class of drugs – delivering large drugs into cells. Cell Penetrating Peptides (CPPs) can overcome ‘the delivery challenge’ and pr