Pyc Therapeutics Limited · ASX:PYC
Pyc Therapeutics — Corporate Update (October 2023)
17 slides from Pyc Therapeutics's corporate update, Oct 5, 2023
Slide 1: Investor update on ADOA drug program
For personal use only Life-changing science Investor update on ADOA drug program 5 October 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’]. 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: Executive Summary
Executive Summary • An introduction to Autosomal Dominant Optic Atrophy (ADOA) For personal use only • Patient journey • Underlying genetic cause of the disease • An introduction to PYC-001 – a first-in-class investigational drug candidate for ADOA • PYC-001 addresses the root cause of ADOA • PYC-00
Slide 4: An introduction to Autosomal Dominant Optic Atrophy (ADOA)
For personal use only An introduction to Autosomal Dominant Optic Atrophy (ADOA) PYC THERAPEUTICS 4
Slide 5: ADOA is a progressive and blinding eye disease of childhood
ADOA is a progressive and blinding eye disease of childhood for which there are no treatment options available Autosomal Dominant Optic Atrophy (ADOA) For personal use only Degenerative sight of an ADOA patient 10 YEARS OLD • An addressable market size of ~A$2 billion per annum1,2,4 • 9,000 – 16,000
Slide 6: OPA1 protein deficiency triggers bioenergetic deficits leading to loss of vision
The deficiency of OPA1 protein in ADOA patients triggers a cascade of bioenergetic deficits that culminate in cell death and loss of vision Unaffected bioenergetics Bioenergetics in ADOA For personal use only Wild type OPA1 protein Haploinsufficient expression (100%) OPA1 ~50-70% expression Slim and
Slide 7: An introduction to PYC-001 for ADOA
For personal use only An introduction to PYC-001 for ADOA PYC THERAPEUTICS 7
Slide 8: PYC-001 addresses the root cause of ADOA
PYC-001 addresses the root cause of ADOA - insufficient expression of OPA1 protein in the cells that form the optic nerve For personal use only Mechanism of disease causation Treatment with PYC-001 ADOA patients have a mutation in one copy In ADOA, the retinal ganglion cells PYC-001 treatment increa
Slide 9: NHP results supporting clinical development of PYC-001
For personal use only NHP results supporting clinical development of PYC-001 PYC THERAPEUTICS 9
Slide 10: Non-clinical models provide early insight into clinical outcomes
Non-clinical models provide early insight into clinical outcomes – particularly in monogenic diseases “We need to understand as early as possible whether a drug candidate is safe and works in patients, not wait to find out in For personal use only clinical trials which can be expensive and time-cons
Slide 11: Drug delivery is critical to the success of RNA therapeutics
Drug delivery is critical to the success of RNA therapeutics in clinical development For personal use only “While everybody else was so hyped and giving Nobel Prizes for CRISPR and all that, we realized those weren’t really the limitations The limitations were really delivery” George Yancopoulos, Pr
Slide 12: PYC-001 increases OPA1 protein expression in the NHP retina
PYC-001 increases OPA1 protein expression in the NHP retina For personal use only Vehicle • A single 15 µg dose of PYC-001 Untreated control establishes baseline expression of OPA1 protein in the NHP retina RNFL increases OPA1 protein expression in NHPs in the two cellular layers affected by ADOA¹ R
Slide 13: Patient-derived models evaluate impact of PYC-001 on disease cascade
Patient-derived models enable PYC to evaluate the impact of PYC-001 on the disease-causing cascade that leads to blindness in ADOA ADOA Patient Functional outcome For personal use only ADOA patient + PYC-001 Haploinsufficient Restoration towards 100% ~50-70% expression OPA1 OPA1 protein restoration
Slide 14: PYC-001 restores mitochondrial structural defects in patient-derived models
PYC-001 treatment restores mitochondrial structural defects in ADOA patient-derived models Correction of mitochondrial structural defects in ADOA Patient Derived Models following treatment with PYC-001 1 For personal use only ADOA patient ADOA patient ADOA patient ADOA patient + PYC-001 + PYC-001 2.
Slide 15: PYC-001 restores cellular bioenergetics in patient-derived models
PYC-001 treatment restores cellular bioenergetics in ADOA patient-derived models ~1.5-fold increase in cellular bioenergetics in ADOA Patient Derived Models following treatment with PYC-001 1 For personal use only ADOA patient ADOA patient Maximal Respiration Patient 1 Maximal Respiration Patient 2
Slide 16: NHP and patient-derived data linked by ~1.5-fold OPA1 increase
Both the NHP and patient-derived model data is linked by an increase in OPA1 protein expression of ~1.5-fold For personal use only Complexity of a living organism Complexity of the actual human disease 1. Is the drug safe and Non-Human Primates (NHPs)¹ well tolerated in NHPs? RGC OPA1 staining 40 p
Slide 17: PYC is progressing PYC-001 into human trials next year
PYC is progressing PYC-001 into human trials next year For personal use only • FDA Pre-IND meeting late October 2023 • Natural History Study commencing 4Q23 • GLP animal studies commencing 4Q23 to support IND lodgement • Anticipated IND lodgement Q2 2024 PYC THERAPEUTICS 17 Managements best estimate