Pyc Therapeutics Limited · ASX:PYC
Pyc Therapeutics — Investor Presentation (April 2021)
29 slides from Pyc Therapeutics's investor presentation, Apr 22, 2021
Slide 1: Corporate Presentation April 2021
For personal use only Corporate Life-changing science Presentation April 2021
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’]. These slides have been prepared as a presentation aid only and the information they contain may require further explanation and/or clarification. A
Slide 3: Highlights
Highlights For personal use only PYC is an RNA therapeutics company focused on applying distinctive P-PMO technology Leading-edge RNA Novel Technology Platform: Cell Penetrating Peptide-PMOs Therapeutics solve delivery challenge and get more drug safely into cells Development programs in First focus
Slide 4: PYC's growing Executive Leadership team built across the US and Australia
PYC’s growing Executive Leadership team built across the US and Australia For personal use only Leadership team Sahm Nasseri Dr Sue Fletcher Chief Executive Officer US, Exec. Director Chief Scientific Officer Extensive experience in commercial drug development with Leading global expert and pioneer
Slide 5: Platform Technology: Proprietary P-PMOs
For personal use only Platform Technology: Proprietary P-PMOs 5
Slide 6: PYC's core therapeutic technology brings together two distinctive components
PYC’s core therapeutic technology brings together two distinctive components For personal use only PMO (Phosphorodiamidate Cell-penetrating peptide Morpholino Oligomer) Latest generate anti-sense Naturally-derived peptide library oligonucleotide chemistry, neutral (500M sequences) charge Precision a
Slide 7: Platform preclinically validated across ocular, CNS and systemic applications
PYC’s platform has been preclinically validated across a range of applications- ocular, CNS and systemic For personal use only Retinal delivery Brain delivery Systemic delivery PMO delivery in the retina, Day 28 in the mouse eye post PMO delivery in the brain, Day 5 in the mouse brain post PMO deliv
Slide 8: Choosing the right peptide for the right application is a key development step
Choosing the right peptide for the right application (cell, tissue, indication) is a key development step For personal use only PYC’s library leverages millions of years of This is combined with an efficient screening and evolution by micro-organisms to create a validation pipeline structurally dive
Slide 9: Applying our technology to create life changing treatments, initial focus on eye
PYC is applying our technology to create life changing treatments, with an initial focus on diseases of the eye For personal use only PYC is a multi-asset drug development company Estimated patients in Program overview Indication and stage of development western world Organ Program Target Lead Disco
Slide 10: VP-001 for the treatment of Retinitis pigmentosa type 11
For personal use only VP-001 for the treatment of Retinitis pigmentosa type 11 Matthew, living with RP11 10
Slide 11: VP-001 for Retinitis pigmentosa type 11
VP-001 for Retinitis pigmentosa type 11 For personal use only Retinitis pigmentosa (RP) is a genetic, blinding eye disease ▪ Retinitis pigmentosa type 11 (RP11) is a form of RP caused by mutation in the PRPF31 gene ‒ Severe, progressive blinding eye disease ‒ Onset between the ages of 10 and 20 ‒ Le
Slide 12: VP-001 targets a negative regulator of the gene underlying RP11
VP-001 targets a negative regulator of the gene underlying RP11 For personal use only Healthy eye Patients with RP11 VP-001 We all have two copies of each gene in Patients with RP11 have only one healthy VP-001 skips an exon in a gene (CNOT3) our chromosomes gene (and one mutated gene) that acts as
Slide 13: VP-001 has demonstrated a strong preclinical efficacy signal in vivo and in patient models
VP-001 has already demonstrated a strong preclinical efficacy signal in vivo and in patient derived models For personal use only PYC has demonstrated delivery to the target cells, And shown upregulation of the target protein, PRPF31, in Retinal Pigment Epithelium (RPE), for 28 days in the patient iP
Slide 14: Dose dependant response shows no increasing acute toxicity in the mouse
The dose dependant response shows no increasing acute toxicity in the mouse For personal use only Dose dependant increase in effect … … and no increase in toxicity markers Exon 7 skipping, Day 7 in the mouse eye ddPCR Gfap expression, for Day 7 in theGFAP expression mouse eye in Neural Retina at D7
Slide 15: VP-001 corrects important functional deficits associated with RP11
VP-001 has demonstrated the ability to correct important functional deficits associated with RP11 For personal use only Scanning electron microscopy of retinal pigmented epithelium (RPE) derived from control and patient iPSC. Images selected as representative of full data set. These results demonstr
Slide 16: VP-001 Key Takeaways and Next Steps
VP-001 Key Takeaways and Next Steps For personal use only 1. RP11 represents a large, underserved market with no disease-modifying therapies available nor in clinical Next Steps: development • Larger animal (rabbit 2. To date, VP-001 has been shown to: and NHP) PK, Ocular ▪ Reach the target cell fol
Slide 17: VP-002 for the treatment of Autosomal dominant optic atrophy
For personal use only VP-002 for the treatment of Autosomal dominant optic atrophy Elina, living with ADOA 17
Slide 18: VP-002 for Autosomal dominant optic atrophy
VP-002 for Autosomal dominant optic atrophy For personal use only Autosomal dominant optic atrophy (ADOA) is a genetic, blinding eye disease ▪ ADOA-OPA1 is a form of RP caused by mutation in the OPA1 gene1 ‒ Severe, progressive blinding eye disease ‒ Onset between the ages of 5 and 20 ‒ Primarily af
Slide 19: ADOA is largely caused by mutations in the OPA1 gene
ADOA is largely caused by mutations in the OPA1 gene affecting the retinal ganglion cells For personal use only ADOA is caused by the optic nerve cells (retinal ganglion cells, RGCs) losing their ability to transmit Healthy Healthy visual signals to the brain Light retina RGCs ▪ This can cause sever
Slide 20: Ability to increase critical OPA1 protein levels across PMO candidates
We have shown an ability to increase the critical OPA1 protein levels across a range of potential PMO candidates For personal use only Change in OPA1 protein levels, 50mM PMO treatment, patient fibroblasts For upregulation benchmark see poster ‘Antisense oligonucleotide mediated increase of OPA1 exp
Slide 21: VP-002 Key Takeaways and Next Steps
VP-002 Key Takeaways and Next Steps For personal use only Next Steps: ▪ PYC has designed multiple PMOs capable of • Report results from correcting the OPA1 protein haploinsufficiency in patient-derived cells derived from ADOA patients preclinical models in the first half of 2021, ▪ Achieved >100% pr
Slide 22: Beyond Rare Ocular Disease: PYC-001 for Diabetic Retinopathy
For personal use only Beyond Rare Ocular Disease: PYC-001 for Diabetic Retinopathy 22
Slide 23: Diabetic Retinopathy
Diabetic Retinopathy For personal use only ▪ Diabetic retinopathy (DR) is caused by changes in the blood vessels of the retina. In some cases, blood vessels may swell and leak fluid, or grow on the surface of the retina, resulting in loss of vision. ▪ The incidence of DR is expected to grow from ~8
Slide 24: PYC-001 Key Takeaways and Next Steps
PYC-001 Key Takeaways and Next Steps For personal use only ▪ PYC has leveraged the unique advantages of its RNA therapeutics technology to create a modified Next Steps: VEGF therapy that addresses these shortcomings. ▪ PYC’s approach alters the type of VEGFA produced • Report results from by cells i
Slide 25: P-PMOs in the Central Nervous System and Other High Unmet Need Conditions
For personal use only P-PMOs in the Central Nervous System and Other High Unmet Need Conditions 25
Slide 26: Initial results show great promise for PYC's P-PMOs in the CNS
Initial results show great promise for PYC’s P-PMOs in the CNS For personal use only PMO delivery to the brain, Smn exon 7 skipping, PMO distribution in the brain, Smn exon 7 skipping, Day 5 post Day 5 post single I.C.V. injection in mice1 single I.C.V. injection in mice2 1 n=2 for all groups expect
Slide 27: Indicates we can access brain regions that matter in neurodegenerative diseases
… and this indicates we can access regions of the brain that matter in neurodegenerative diseases For personal use only MOUSE Cortex Alzheimer’s disease HUMAN Amyotrophic Lateral Sclerosis % denotes Smn exon 7 skipping, Day 5 post Frontotemporal dementia single I.C.V. injection in mice Corpus Callos
Slide 28: P-PMO technology shows broad potential including preferential delivery to high value organs
PYC’s P-PMO technology has demonstrated broad potential including preferential delivery to high value organs For personal use only CPP-PMOs demonstrate strong uptake across high value … and preferential delivery to certain organs tissues include heart and diaphragm … such as the Kidney Exon 7 skippi
Slide 29: Looking forward to numerous critical value inflection points throughout 2021
We are looking forward to numerous critical value inflection points throughout 2021 For personal use only VP-001 for Retinitis pigmentosa type 11 VP-002 for Autosomal Dominant Optic Atrophy VP-001 Multiple interim data readouts in Full data readout in VP-001 GLP readouts for RP11 large animals (ocul
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