Pyc Therapeutics Limited · ASX:PYC
Pyc Therapeutics — Investor Presentation (June 2020)
26 slides from Pyc Therapeutics's investor presentation, Jun 22, 2020
Slide 1: Investor Presentation June 2020
For personal use only Investor Presentation June 2020
Slide 2: PYC Therapeutics: Mission Statement
PYC Therapeutics: Mission Statement For personal use only PYC is part of the ‘precision medicine revolution’ targeting rare diseases… …with a differentiated technology… $ …a lead program treating a rare disease in the eye (a >US$1Bn p.a. market)… …and a pipeline of further programs 2
Slide 3: Corporate Overview
For personal use only Corporate Overview 3
Slide 4: PYC Corporate Snapshot
PYC Corporate Snapshot (ASX: PYC) Financial Information (18 June 2020, AUD) For personal use only Share Price Performance (12 months) Share price $0.09 0.10 60,000,000 Number of shares 2,930M Daily Volume Traded On-Market Market Capitalisation $264M 0.09 Daily Close Price - PYC 50,000,000 Cash (31-M
Slide 5: PYC has deep expertise in the management and advisory team
PYC has deep expertise in the management and advisory team World-leading RNA, rare disease and clinical expertise + Commercial expertise to drive growth & value For personal use only Professor Sue Fletcher (PhD, BSc) Douglas Huey (MBA (Hons)) Chief of R&D (Perth) Executive Director & CEO (Boston) Le
Slide 6: PYC is dedicated to changing the lives of patients with rare genetic diseases
PYC is dedicated to changing the lives of patients with rare genetic diseases – a unique opportunity in drug development The need: Rare genetic diseases represent an The opportunity: Developing drugs to address For personal use only urgent unmet patient need these needs has better odds of reaching p
Slide 7: Our Approach & Progress to Date
For personal use only Our Approach & Progress to Date 7
Slide 8: PYC is part of the 'precision medicine revolution'
PYC is part of the ‘precision medicine revolution’ For personal use only RNA Therapeutics Examples DNA Therapeutics Examples RNA-related small Antisense Oligos siRNA mRNA Gene Therapy CRISPR molecules 8
Slide 9: Combination of RNA drug design and intracellular drug delivery platforms
What makes PYC different is the combination of our RNA drug design and intracellular drug delivery platforms For personal use only RNA drug design Intracellular delivery Therapeutic PMO1 CPP CPP-PMO We design antisense oligos that …and combine these drugs with …to create safe and effective can preci
Slide 10: PYC's drugs can access cells beyond the reach of competitive RNA technologies
PYC’s drugs can access cells (and diseases) beyond the reach of competitive RNA technologies The challenge for most RNA drugs PYC’s Cell Penetrating Peptides For personal use only The cell membrane has evolved over PYC’s proprietary Cell Penetrating Peptides hundreds of millions of years to keep (CP
Slide 11: Applying CPP-PMO technology towards rare genetic eye diseases
PYC is initially applying its CPP-PMO technology towards rare genetic eye diseases For personal use only Discovery Pre-clinical Clinical Current Programs Retinal advantages VP-001 Retinitis IND Filing Expected 2H21 Broad drug distribution (more cells Pigmentosa 11 treated) than AAV gene therapy Re
Slide 12: Genetic eye diseases have a devastating impact on patients
Genetic eye diseases have a devastating impact on patients For personal use only Losing eyesight can be frightening and overwhelming for patients, with major consequences on physical and mental health and overall quality of life Genetic eye diseases like Retinitis Patients begin to lose their vision
Slide 13: PYC's technology has several advantages over alternatives
PYC’s technology has several advantages over alternatives For personal use only Patient Control of gene Accessibility tolerability expression to providers PYC’s drugs can be Retinal cells are more sensitive RNA therapies administered via administered via intravitreal to gene over-expression, which i
Slide 14: Our lead drug targets a down-regulator of the RP11 gene
Our lead drug targets a down-regulator of the gene underlying Retinitis Pigmentosa Type 11 (RP11) For personal use only Healthy eye Eye with RP11 PYC’s lead drug We all have two copies of each gene in People with RP11 have only one healthy Our drug knocks down a protein that our chromosomes gene (an
Slide 15: Proof of concept in multiple patient derived models
Proof of concept in multiple patient derived models We have proven that our drug modulates target gene expression in multiple patient derived models… For personal use only Exon skipping, retinal organoid Exon skipping, Retinal Pigment Epithelial Day 14, 2 treatments (n=2) Day 5, single treatment 70
Slide 16: RP11 patients experience lost functionality of Retinal Pigment Epithelial cells
RP11 patients experience lost functionality of Retinal Pigment Epithelial cells (RPE) Structure of the Retina – target cells in the back Phagocytosis – the ‘self-repair’ process where RPE For personal use only of the eye (RPE) cells ‘clear away’ debris from the photoreceptors. If outer segments are
Slide 17: Our lead drug can restore RPE functionality in patient derived models
We have proven that our lead drug can restore RPE functionality in patient derived models Phagocytosis assay, 5µM 6hr timepoint For personal use only A) Green ‘specks’ are Phagocytosed outer-segments B) Intensity of phagocytosis per RPE cell (more green = improved functionality) p<0.01 Untreated PYC
Slide 18: Lead drug is competitively differentiated without causing toxicity
PYC’s lead drug is competitively differentiated in achieving this functional correction without causing toxicity in the retina For personal use only Retinal stress marker expression in mice Retinal thinning in mice Day 5 post single 1.6µg IVT injection Day 21 post IVT injection OCT imaging PYC CPP-P
Slide 19: Path to Market & Next Steps
For personal use only Path to Market & Next Steps 19
Slide 20: Path to market advantages over conventional clinical development pathways
The path to market for our lead drug has several major advantages over conventional clinical development pathways For personal use only Stage IND-enabling Phase 1 Phase 2/3 Approval Timeline 18 months 15 months 18 months 6 months Advantage Rationale Higher probability • Precision medicines have a
Slide 21: Upcoming development milestones for VP-001
Upcoming development milestones for VP-001 For personal use only Stage Q3 20 Q4 20 Q1 21 Q2 21 Q3 21 Q4 21 Clinical & IND Submission Orphan drug designation Regulatory development Pre-IND meeting Pre-Clinical Acute Rabbit toxicity Acute Monkey toxicity Large animal PK Long term animal toxicity Final
Slide 22: Addressable Market for our Lead Drug
Addressable Market for our Lead Drug Retinitis Pigmentosa (RP) prevalence For personal use only 1 in 2,500-4,0001 proportion of people in the population with RP Proportion of RP that is autosomal dominant (adRP) 30-40%1 Inherited in a dominant pattern No competitors Proportion of adRP that is RP11 i
Slide 23: Other Applications for PYC's Drug Delivery Technology
Other Applications for PYC’s Drug Delivery Technology For personal use only PYC’s delivery technology Penetration – reaches the Ganglion Traffics deepest layer of cells within the cell layer through the retina – the RPE vitreous Inner Distribution – achieves a broad nuclear distribution througho
Slide 24: Advancing our lead program will validate our drug delivery platform
Advancing our lead program into the clinic will validate our drug delivery platform for retinal disease For personal use only The Retina is a high value target Proven Delivery in the Eye Develop Further Applications 1.6ug IVT injection in mice % truncated SMN1 transcript Diseases primarily affecting
Slide 25: Upcoming milestones
Upcoming milestones For personal use only Progressing Our Lead Drug (VP-001 for Retinitis Pigmentosa 11) Acute animal toxicity data – 2H 2020 Orphan drug designation for VP-001 for RP11 – 2H 2020 Clinical manufacturing studies – 1H 2021 Large animal PK data – 1H 2021 Large animal long term
Slide 26: Disclaimer
Disclaimer For personal use only The purpose of this presentation is to provide an update of the business of PYC Therapeutics Limited (ASX:PYC) [‘PYC’]. These slides have been prepared as a presentation aid only and the information they contain may require further explanation and/or clarification. A