Pyc Therapeutics Limited · ASX:PYC
Pyc Therapeutics — Investor Presentation (April 2021)
24 slides from Pyc Therapeutics's investor presentation, Apr 15, 2021
Slide 1: April Investor Update: Life-changing science
For personal use only April Investor Life-changing Update science 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: Who's on today's call
Who’s on today’s call For personal use only PYC US Development and Corporate Hub PYC Australia Discovery Hub Sahm Nasseri, Chief Executive Officer US Professor Sue Fletcher, Chief Scientific Officer Extensive experience in commercial drug development Leading global expert and pioneer in RNA therapeu
Slide 4: PYC's focus is exploiting our distinctive PPMO technology
PYC’s focus is exploiting our distinctive PPMO technology For personal use only PMO (Phosphorodiamidate Cell Penetrating Peptide Morpholino Oligomer) Latest generation ASO, neutral Naturally-derived (uncharged) Sequence diversity Precision and flexibility Screened upfront for efficacy and Safer prof
Slide 5: 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 Program overview Indication and stage of development Estimated patients1 Organ Program Target Lead Discovery IND-enablin
Slide 6: PYC has pushed our technology further than ever before in 2020
PYC has pushed our technology further than ever before in 2020 For personal use only Demonstrated the effectiveness of PYC’s lead program, VP-001, in gold- standard pre-clinical models for Retinitis pigmentosa type 11 Expanded PYC’s drug pipeline to 3 development candidates, demonstrating the scalab
Slide 7: 2021 is a pivotal year for PYC
2021 is a pivotal year for PYC as we progress multiple candidates towards clinical development For personal use only □ VP-001 through large animal studies in mid-2021 paving the way for IND submission in first half of 2022 Execute □ VP-002 for ADOA proof-of-concept and preclinical efficacy readouts
Slide 8: Good progress towards our critical 2021 deliverables
We are making good progress towards our critical 2021 deliverables For personal use only □ VP-001 through large animal studies in mid-2021 paving the way for IND submission in first half of 2022 Execute □ VP-002 for ADOA proof-of-concept and preclinical efficacy readouts □ PYC-001 for DR to proof-of
Slide 9: 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
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 ‒ Pa
Slide 12: VP-001's core goal is to increase levels of PRPF31 protein
VP-001’s core goal is to increase levels of PRPF31 protein For personal use only PRPF31 protein levels, RPE, 5 µM treatment, (n=1 per patient) 2.5 Day 2 Day 5 Day 12 Untreated Treated Fold change over untreated 2.0 1.5 Anticipated disease correction threshold 1.0 0.5 0.0 Patient 1 Patient 2 Patient
Slide 13: Increasing PRPF31 levels has improved function in pre-clinical models
Increasing PRPF31 levels has improved function in the gold- standard pre-clinical models For personal use only These results demonstrate VP-001’s ability to correct the structural deficiency in patient derived retinal cells that is one of the key causes of vision loss in RP11 patients1 1 Buskin A. D
Slide 14: 2021's key milestones for VP-001 centre on large animal studies
2021’s key milestones for VP-001 centre on large animal studies For personal use only Rabbit pharmaco- Rabbit Dose-range Non-human kinetics [PK] and finding [DRF] primates [NHPs] GLP animal toxicity Formal regulatory tissue distribution toxicity study DRF toxicity study studies (FDA) engagement 3Q21
Slide 15: 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 15
Slide 16: 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 gene ‒ Severe, progressive blinding eye disease ‒ Onset between the ages of 5 and 20 ‒ Primarily aff
Slide 17: VP-002 is targeted at increasing levels of OPA1 protein
VP-002 is targeted at increasing levels of OPA1 protein For personal use only Change in OPA1 protein levels, 50µM PMO treatment, patient fibroblasts For upregulation benchmark see poster ‘Antisense oligonucleotide mediated increase of OPA1 expression using TANGO technology for treatment of autosomal
Slide 18: Expansion into Central Nervous System diseases
For personal use only Expansion into Central Nervous Life-changing System diseases science 18
Slide 19: Beyond the eye: Bringing PPMO to the Central Nervous System
Beyond the eye: Bringing PPMO to the Central Nervous System For personal use only Neurological disease is a substantial There is a clear need for better treatments and increasing global burden Solve delivery to right part of the brain at #1 cause of disability globally safe dose levels Solve deliver
Slide 20: Initial results show great promise for PPMO in the CNS
Initial results show great promise for PPMO 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 PYC PPM
Slide 21: Indicates we can access regions of the brain that matter
… 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 22: Where to from here in the CNS?
Where to from here in the CNS? For personal use only 2H 2021 Refine our CNS delivery platform • Determine cell level delivery • Optimise our CPP selection for our target brain region and cell type Focused discovery effort in neurodegeneration • Select target pathway • Progress a potential candidate
Slide 23: 2021 will be a transformative year for PYC Therapeutics
2021 will be a transformative year for PYC Therapeutics For personal use only • Furthest a PYC Therapeutic has ever advanced in preclinical development—testing VP-001 in larger animals ahead of IND submission • Multiple ocular assets running in parallel with key catalysts throughout 2021 • Expansion
Slide 24: Q&A
For personal use only Q&A 24
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