Pyc Therapeutics Limited · ASX:PYC
Pyc Therapeutics — Investor Presentation (February 2023)
37 slides from Pyc Therapeutics's investor presentation, Feb 3, 2023
Slide 1: Life-changing science: Corporate Presentation
For personal use only Life-changing science Corporate Presentation 3 February 2023
Slide 2: 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’]. Theseslides have been prepared as a presentation aid only and the information they contain may require further explanation and/or clarification. Ac
Slide 3: Executive Summary
Executive Summary For personal use only • PYC is a drug developer: PYC discovers and develops RNA therapies for patients with genetic diseases • Platform technology: PYC’s scalable drug delivery technology overcomes the major limitation on RNA therapies – reaching the target • Multi-asset pipeline:
Slide 4: PYC discovers and develops RNA therapies for genetic diseases
For personal use only PYC discovers and develops RNA therapies to change the lives of patients with genetic diseases PYC THERAPEUTICS 4
Slide 5: Urgent need for treatments for rare genetic diseases
There is an urgent need to create treatments for patients with rare genetic diseases For personal use only There are approximately 6,000 known rare diseases1 affecting 400 million people worldwide 1 in every 2 patients diagnosed with a rare disease is a child There are no treatment options available
Slide 6: Strategy anchored on four critical features
PYC is creating therapies for these patients through a strategy anchored on four critical features For personal use only A HIGHER PROBABILITY A FASTER PATH LIKELY RAPID UPTAKE ORPHAN DRUG OF SUCCESS TO MARKET IN MARKET PRICING PYC focuses on The potential for First-in-class drugs in Median list pric
Slide 7: Dominant trend in the pharmaceutical industry: RNA therapeutics
PYC’s drug development efforts are supported by the dominant trend in the pharmaceutical industry today… For personal use only “We are in the midst of a therapeutic revolution, the likes of which have not been seen…” “RNA Therapeutics will change the standard of care for many diseases” PYC THERAPEUT
Slide 8: RNA therapies' Achilles heel: delivery into cells
… However, RNA therapies have an ‘Achilles heel’ – they struggle to get inside the cells where their targets reside For personal use only RNA drug Outside the cell Inside the cell The ‘delivery challenge’ has limited the number of diseases for which RNA therapies have been successfully created PYC T
Slide 9: PYC's RNA delivery platform overcomes the delivery challenge
PYC’s RNA delivery platform overcomes this primary challenge – ensuring enough drug reaches its target For personal use only PYC combines existing RNA drug design PYC’s drug delivery platform is used to assist technology with its proprietary drug delivery the RNA drug reach its target inside the cel
Slide 10: Pipeline of RNA therapies built on the platform
PYC has built a pipeline of RNA therapies on this platform technology For personal use only Current progress Progress expected in 2023 Organ Program Target Discovery Pre-clinical IND enabling Clinical Trials Marketed VP-001* PRPF31 Retinitis Pigmentosa Type 11 PYC-001 OPA1 Autosomal Dominant Optic A
Slide 11: Scaling the platform into clinical read-outs
PYC is now focusing on scaling its platform into clinical read-outs across multiple indications For personal use only PYC IS HERE Scale into multiple clinical 3 safety and efficacy read-outs Evolution to a clinical- 2 stage multi-asset company Objectives • Establish human safety of PYC’s platform te
Slide 12: VP-001 Retinitis Pigmentosa Type 11 (RP11)
For personal use only Life-changing science VP-001 Retinitis Pigmentosa Type 11 (RP11) PYC THERAPEUTICS 12
Slide 13: VP-001 for Retinitis Pigmentosa Type (RP11)
VP-001 for Retinitis Pigmentosa Type 11 (RP11) 1. RP11 is a progressive and blinding eye disease For personal use only 2. There are no treatments available for patients with RP11 3. RP11 is caused by insufficient expression of one gene (PRPF31) in the retina 4. VP-001 restores PRPF31 expression back
Slide 14: RP11 is a progressive and blinding eye disease
1. RP11 is a progressive and blinding eye disease For personal use only Degenerative sight of an RP11 patient Retinitis Pigmentosa (RP)1,2 6 YEARS OLD • A severe and progressive blinding eye disease that begins in childhood • Affects 1 in every 3,500 people 26 YEARS OLD • Patients experience night b
Slide 15: No treatments available for patients with RP11
2. There are no treatments available for patients with RP11 For personal use only 5,000 – 10,000 patients USD $150,000 p.a. Estimated addressable RP11 patient population Median list price of orphan drugs (per patient2) in the western world1 Patients are waiting FDA concessions RP11 patients on retin
Slide 16: RP11 is caused by insufficient expression of PRPF31
3. RP11 is caused by insufficient expression of one gene (PRPF31) in the retina For personal use only Unaffected RP11 patient individual DNA RP11 patients have a mutation in one copy of the PRPF31 gene RNA The mutation leads to an unstable RNA message that is rapidly degraded PRPF31 This leads to ce
Slide 17: VP-001 restores PRPF31 expression in retina in a dish models
4. VP-001 restores PRPF31 expression back to levels seen in unaffected individuals in ‘retina in a dish’ models1 For personal use only A single treatment of VP-001 restores PRPF31 expression to wild-type levels in cellular models derived from patients with RP11 RP11 patient Unaffected RP11 patient w
Slide 18: Increased expression improves appearance of affected cells
5. This increase in gene expression visibly improves the appearance of the affected cells in RP11 'retina in a dish' models For personal use only Retinal pigmented epithelium (RPE) cells derived from: 1. AN ‘UNAFFECTED' INDIVIDUAL 2. A PATIENT WITH RP11 3. A PATIENT WITH RP11 AFTER A SINGLE DOSE OF
Slide 19: Pre-clinical data supports disease modification potential
6. Pre-clinical data supports the potential of VP-001 to drive disease modification in the clinic For personal use only PK/Tox PD Functional PK/Retinal Delivery Target engagement Phenotypic rescue NHP Durable drug presence in retina with NOAEL defined as highest dose assessed in GLP tox studies Rabb
Slide 20: PYC set to generate human safety and efficacy data for VP-001
7. PYC is set to generate human safety and efficacy data for VP-001¹ For personal use only Phase 1/2 Human Trial Pivotal Trial Part 1 Part 2 • Single Ascending Dose Study • Multiple dose expansion study • Dependent on outcome of • 3+3+3 dose escalation in RP11 • 3 groups of 10 patients each Phase 1/
Slide 21: Monogenic disease drugs 2-5x more likely to succeed
8. Drugs targeting monogenic diseases are 2-5x more likely to succeed in human studies¹ For personal use only • RP11 has a single underlying genetic origin - it is caused by mutations in the PRPF31 gene • Disorders of this nature are known as ‘monogenic’ or ‘Mendelian’ disorders • Drug targets assoc
Slide 22: PYC-001 Autosomal Dominant Optic Atrophy (ADOA)
For personal use only Life-changing science PYC-001 Autosomal Dominant Optic Atrophy (ADOA) PYC THERAPEUTICS 22
Slide 23: PYC-001 for Autosomal Dominant Optic Atrophy
PYC-001 for Autosomal Dominant Optic Atrophy (ADOA) For personal use only 1. ADOA is a progressive and blinding eye disease 2. There are no treatments available for patients with ADOA 3. ADOA is caused by insufficient expression of one gene (OPA1) in the cells that form the optic nerve in the eye 4.
Slide 24: ADOA is a progressive and blinding eye disease
1. ADOA is a progressive and blinding eye disease For personal use only ADOA patient sight deterioration Autosomal Dominant Optic Atrophy (ADOA) 10 years old • A progressive and irreversible blinding eye disease • 9,000 – 16,000 addressable patients in the western world1,2 30 years old • Median age
Slide 25: No treatments available for patients with ADOA
2. There are no treatments available for patients with ADOA For personal use only 9,000 – 16,000 patients USD $150,000 p.a. Estimated addressable ADOA patient Median list price of orphan drugs (per patient2) population in the western world1 Patients are waiting FDA concessions ADOA patients on retin
Slide 26: ADOA caused by insufficient expression of OPA1
3. ADOA is caused by insufficient expression of one gene (OPA1) in the cells that form the optic nerve in the eye For personal use only Affected cell type Mechanism of disease causation In ADOA, these are the retinal ganglion cells (RGCs), Patients with ADOA have a mutation in one copy of that make
Slide 27: PYC-001 increases OPA1 expression in patient-derived models
4. PYC-001 increases OPA1 expression in models derived from patients with ADOA1 For personal use only Low dose PYC-001 treatment increases OPA1 protein expression in cells* derived from ADOA patients 1.3-fold Unaffected ADOA ADOA + PYC-001 individual ✱✱✱✱ 1.5 (normalised to beta-tubulin) DNA OPA1 pr
Slide 28: PYC-001 expected to enter clinical trials next year
5. PYC-001 is expected to enter clinical trials next year^ For personal use only 2023 2024 Anticipated • Lead candidate selected • IND-enabling studies completed development timeline and • IND-enabling studies initiated • IND submission milestones • Initiation of natural history • Initiation of a co
Slide 29: PYC-002 Phelan-McDermid Syndrome
For personal use only Life-changing science PYC-002 Phelan-McDermid Syndrome PYC THERAPEUTICS 29
Slide 30: PYC-002 for Phelan-McDermid Syndrome (PMS)
PYC-002 for Phelan-McDermid Syndrome (PMS) For personal use only 1. Phelan-McDermid Syndrome (PMS) is a severe neurodevelopmental disorder caused by insufficient expression of the SHANK3 gene in neurons in the brain 2. Patients with PMS have no treatment options available that address the underlying
Slide 31: PMS is a severe neurodevelopmental disorder
1. Phelan-McDermid Syndrome (PMS) is a severe neurodevelopmental disorder For personal use only Signs and symptoms of PMS Phelan-McDermid Syndrome (PMS)1,2 Poor head control • Rare genetic neurodevelopmental disorder causing life-long disability Intellectual disability Weak cry • ~28,000 addressable
Slide 32: No treatment options address the underlying cause of PMS
2. Patients with PMS have no treatment options available that address the underlying cause of the disorder For personal use only ~28,000 patients USD $150,000 p.a. Estimated addressable PMS patient population Median list price of orphan drugs (per patient3) in the western world1,2 Disease-modifying
Slide 33: PYC-002 addresses the root cause of PMS
3. PYC-002 addresses the root cause of PMS and has the potential to be a first-in-class disease-modifying therapy For personal use only 2.2-fold 2.2-fold, p = 0.0056 ** Fold-change over Untreated PYC-002 hit sequences increase 3 SHANK3 protein levels by >2-fold in a neuronal cell line*1 2 Further st
Slide 34: PYC's RNA drug reaches affected neurons in vivo
4. PYC’s RNA drug modality effectively reaches the cells affected in PMS (neurons) in vivo (in animal models) For personal use only The advantages of PYC’s next generation RNA drug (‘PPMO’) are clearly evident in animal models evaluating the ability of the RNA drug to engage its target in different
Slide 35: Corporate Overview
For personal use only Life-changing science Corporate Overview PYC THERAPEUTICS 35
Slide 36: Executive & Board
Executive & Board PYC’s management has discovered and delivered drugs to market For personal use only Alan Tribe Dr Rohan Hockings Sri Mudumba Andrew Taylor Prof Sue Fletcher Chairman Chief Executive Officer Chief Research Officer Chief Financial Officer Chief Scientific Officer Experience commercia
Slide 37: Life-changing science
For personal use only “These [COVID] vaccines… are only the tip of the iceberg in the coming RNA medical technology revolution”¹ Life-changing science Life-changing science 1 https://newsroom.unsw.edu.au/news/science-tech/rna-technologies-explained-long-and-short-it
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