Pyc Therapeutics Limited · ASX:PYC
Pyc Therapeutics — 44th annual JP Morgan Healthcare Conference 2026
40 slides from Pyc Therapeutics's conference presentation, Jan 13, 2026
Slide 1: JP Morgan Corporate Presentation
13 January 2026 JP MORGAN CORPORATE PRESENTATION PERTH, Australia and SAN FRANCISCO, California – 13 January 2026 PYC Therapeutics Limited (ASX:PYC) (PYC or the Company) is a precision medicine Company dedicated to changing the lives of patients with genetic diseases who have no treatment options av
Slide 2: Contact us
This ASX announcement was approved and authorised for release by the Board of PYC Therapeutics Limited CONTACT US Investor relations and media contact investor@pyctx.com For personal use only pyctx.com | ACN 098 391 961 2
Slide 3: Life-changing science: Corporate Presentation January 2026
For personal use only Life-changing science Corporate Presentation January 2026
Slide 4: Disclaimer
Disclaimer The purpose of this presentation is to provide an update of the business of PYC Therapeutics about the future, you are urged to view all forward looking statements contained in this For personal use only Limited (ASX:PYC) [‘PYC’]. These slides have been prepared as a presentation aid only
Slide 5: PYC's mission: life-changing RNA therapies
For personal use only PYC’s mission is to create life-changing RNA therapies that address the root cause of diseases resulting from insufficient gene expression The Company’s work is dedicated to patients who currently have no treatment options available Sierra – living with Phelan- McDermid Syndrom
Slide 6: An introduction to PYC Therapeutics
An introduction to PYC Therapeutics Precision medicines PYC is a drug discovery and development company focused on creating For personal use only life-changing new therapies for patients who have genetic diseases and no treatment options available today Disease-modifying PYC’s strategy is to use RNA
Slide 7: Pipeline of clinical-stage drug candidates in areas of major unmet need
PYC has created a pipeline of clinical-stage drug candidates in areas of major unmet need Historical progress Progress expected in 20251 For personal use only Program Pre-clinical Clinical Prevalence1 Phase 1/2 Registrational PYC-003 Polycystic Kidney Disease 1 in 1,000 PYC-002 Phelan-McDermid Syndr
Slide 8: Highlights of PYC's pipeline
Highlights of PYC’s pipeline – 4 drug candidates with best-in- indication potential1 1 2 3 4 5 Disease-modifying In areas of major With the highest Validated in patient- Generating human For personal use only drug candidates1 unmet need probability of success derived models data in 2026/2027 Up to 5
Slide 9: Human efficacy data over the coming 24 months
PYC will generate human efficacy data for all 4 of these drug candidates over the coming 24 months1 These data read-outs will highlight the potential of disease-modifying drug candidates in these genetically- For personal use only defined diseases1 PYC-003 in ADPKD2 PYC-002 in PMS3 VP-001 in RP114 P
Slide 10: PYC's platform technologies
For personal use only Life-changing science PYC’s platform technologies
Slide 11: RNA therapies to increase gene expression in haploinsufficiency
PYC designs RNA therapies to increase gene expression in diseases caused by haploinsufficiency For personal use only There are two copies of One copy of a gene is PYC’s drug candidates leverage every gene in humans non-functional in a the ‘good’ copy of the gene to haploinsufficiency restore express
Slide 12: Precision drug design combined with delivery technology
PYC combines this precision drug design expertise with its delivery technology to harness a new class of RNA therapeutic1 1. Design of RNA therapies 2. Drug delivery platform For personal use only Inside the cell Outside the cell1 RNA target RNA Therapeutic Binding Inside the cell Peptide RNA Therap
Slide 13: Mini-human models confirm restoration of gene expression
1. PYC uses ‘mini-human models’1 to confirm restoration of gene expression in the target cell/organ before entering the clinic PYC uses patient tissue samples to create models of The ability of the drug candidate to restore gene For personal use only the specific cells affected in the target indicat
Slide 14: Proprietary drug delivery technology reaches more target cells
2. PYC combines control of gene expression with a proprietary drug delivery technology to reach more target cells PYC validates delivery of each drug candidate in vivo1 Delivery – PYC’s drug candidates reach the target cell For personal use only at safe and well-tolerated doses in vivo External view
Slide 15: Pipeline with disease-modifying potential in substantial markets
The result is a pipeline of drug candidates with disease- modifying potential in substantial markets (>US$1bn p.a.)1 Historical progress Progress expected in 20251 For personal use only Program Pre-clinical Clinical Market Size1 Phase 1/2 Registrational PYC-003 Polycystic Kidney Disease US$10bn p.a.
Slide 16: Autosomal Dominant Polycystic Kidney Disease (PKD) Program
For personal use only Life-changing science Autosomal Dominant Polycystic Kidney Disease (PKD) Program January 2026
Slide 17: Patients with PKD require renal transplantation at median age of 55
Patients with PKD require renal transplantation at a median age of 551 PKD is characterised by progressive growth of fluid-filled cysts that impair kidney function2 For personal use only Age 20 40 60 Endpoint Kidney eGFR Function Blood test Total Kidney Kidney Volume (TKV) Size MRI3 1. See: Cornec-L
Slide 18: Path to market in PKD: single combined P2/3 study
The path to market in PKD incorporates a single combined P2/3 study1 For personal use only Combined Phase 1a/1b Study2 Phase 2/3 Study Part A - SAD in healthy Part B – SAD in ADPKD Part C – MAD in Registrational study Confirmatory extension volunteers Patients PKD Patients Commercialisation Total Ki
Slide 19: Immediate objective in PKD: clinical proof of concept
PYC’s immediate objective in PKD is to demonstrate clinical proof of concept on the registrational endpoint Progress to date For personal use only Combined Phase 1a/1b Study2 Phase 2/3 Study Part A - SAD in healthy Part B – SAD in ADPKD Part C – MAD in Registrational study Confirmatory extension vol
Slide 20: Human safety and pre-clinical efficacy data
Human safety and pre-clinical efficacy data illustrate the potential for impact in this indication1,2 ✱✱✱✱ 2.0 ✱✱✱✱ Untreated Human safety: For personal use only ✱✱✱✱ PYC-003 Healthy volunteers and patients PC1 Fold Change over Untreated ✱✱ 1.5 Normalised to Total Protein Pre-clinical efficacy: Incr
Slide 21: PYC-003 is progressing towards a major unmet patient need
PYC-003 is progressing towards a major unmet patient need Standard of care (Tolvaptan) is used by <7% of the addressable patient population1 For personal use only • Despite limited patient uptake, 2023 sales of Tolvaptan exceeded US$1.5bn1 Estimated number of patients with PKD due USA Europe Japan >
Slide 22: Phelan-McDermid Syndrome (PMS) Program
For personal use only Life-changing science Phelan-McDermid Syndrome (PMS) Program January 2026
Slide 23: Patients with PMS experience life-long disability
Patients with Phelan-McDermid Syndrome (PMS) experience life-long disability PMS is characterised by severe intellectual and physical developmental delays1,2 For personal use only Sierra – living with PMS3 Age 0 5 10 15+ Intellectual disability Speech and developmental delays Skills regression Sympt
Slide 24: PYC-002 follows an established clinical development pathway
PYC-002 follows an established clinical development pathway1,2 Pre-clinical studies GLP. PK and Toxicology Studies Clinical Trials For personal use only For this combination of: • Chemistry: 2ʹMOE PS3 Clinical validation of this modality via the same route of • Administration: intrathecal administra
Slide 25: Immediate objective in PMS: complete GLP toxicology studies
PYC’s immediate objective in PMS is to complete GLP toxicology studies enabling ‘first in human’ trials to commence Progress to date Pre-clinical studies GLP. PK and Toxicology Studies Clinical Trials For personal use only For this combination of: • Chemistry: 2ʹMOE PS3 Clinical validation of this m
Slide 26: Pre-clinical efficacy data illustrate disease-modifying potential
Pre-clinical efficacy data illustrate the disease-modifying potential of this drug candidate1 For personal use only PYC-002 quantitatively restores SHANK3 protein PYC-002 reaches the target cell and modulates gene expression in PMS patient-derived models in vitro1 expression at safe and well-tolerat
Slide 27: PYC-002 is progressing towards a major unmet patient need
PYC-002 is progressing towards a major unmet patient need For personal use only Estimated USA Europe Japan prevalence of 20,000 20,000 5,000 PMS1 PYC THERAPEUTICS 25 1. PMS Foundation
Slide 28: Retinitis Pigmentosa type 11 (RP11) Program
For personal use only Life-changing science Retinitis Pigmentosa type 11 (RP11) Program January 2026
Slide 29: RP11 patients experience progressive and irreversible vision loss
Patients with RP11 experience progressive and irreversible vision loss beginning in childhood1-3 Illustration of the degeneration in sight experienced by an RP11 patient1-3 For personal use only 6 years old 26 years old 46 years old Patients experience night blindness followed by loss of peripheral
Slide 30: First registrational study in RP11 expected in 2026
PYC expects to initiate the first registrational study in RP11 in 20261 VP-001 is the first clinical-stage drug candidate for RP112 FDA Type D meeting Q1 20261 For personal use only Open Phase 1a SAD Label Phase 1b MAD Study Phase 2/3 Study Extension • Safety & Tolerability NDA • Safety & Tolerabili
Slide 31: Immediate objective: align on pathway to an NDA in RP11
PYC’s immediate objective is to align on the pathway to an NDA in RP11 PYC’s immediate objective For personal use only FDA Type D meeting Q1 20261 Progress to date Open Phase 1a SAD Label Phase 1b MAD Study Phase 2/3 Study Extension • Safety & Tolerability NDA • Safety & Tolerability • Efficacy • Ef
Slide 32: Human safety and efficacy data in RP11
Human safety and efficacy data illustrate the potential of this drug candidate in RP111 Human safety: Human efficacy: For personal use only RP11 patients Change in Low-Luminance Visual Acuity (LLVA) in RP11 patients2,3 • No Treatment Related- 20 Serious Adverse events Change from baseline in LLVA 15
Slide 33: VP-001 is progressing towards a major unmet patient need
VP-001 is progressing towards a major unmet patient need For personal use only Estimated USA Europe Japan prevalence of 3,000 3,000 1,200 RP111 PYC THERAPEUTICS 31 1. See: Sullivan L, et al. Genomic rearrangements of the PRPF31 gene account for 2.5% of autosomal dominant retinitis pigmentosa. Invest
Slide 34: Autosomal Dominant Optic Atrophy (ADOA) Program
For personal use only Life-changing science Autosomal Dominant Optic Atrophy (ADOA) Program January 2026
Slide 35: ADOA patients experience progressive and irreversible vision loss
Patients with ADOA experience progressive and irreversible vision loss beginning in childhood1-3 Degenerative sight of an ADOA patient1-3 For personal use only 10 years old 30 years old 50 years old ADOA is the most common inherited optic neuropathy – the median age of onset at 7 years of age2 1. Le
Slide 36: Potential to become the first approved treatment for ADOA
PYC’s drug candidate has the potential to become the first approved treatment for patients with ADOA1 For personal use only Phase 1a SAD Study Phase 1b MAD Study2 Phase 2/3 Study Cohorts 60 mcg 60 mcg (Q12W) Objectives of Phase 1/2/3 trials of PYC-001 • Safety & Tolerability • Efficacy 30 mcg 30 mcg
Slide 37: Immediate objective: demonstrate clinical proof of concept
PYC’s immediate objective in this drug development program is to demonstrate ‘clinical proof of concept’ Progress to date For personal use only Phase 1a SAD Study Phase 1b MAD Study2 Phase 2/3 Study Cohorts 60 mcg 60 mcg (Q12W) Objectives of Phase 1/2/3 trials of PYC-001 • Safety & Tolerability • Ef
Slide 38: Human safety and early efficacy data in ADOA
Human safety and early efficacy data illustrate the potential of this drug candidate in ADOA1 Human safety: Human efficacy: For personal use only ADOA patients2 Change in Visual Acuity (under Low and Normal Contrast) in ADOA patients3 • No Treatment Emergent- Mean improvement in visual acuity at wee
Slide 39: PYC-001 is progressing towards a major unmet patient need
PYC-001 is progressing towards a major unmet patient need For personal use only Estimated USA Europe Japan prevalence ~6,000 patients ~6,000 patients ~2,000 patients of ADOA1,2,3 1. Yu-Wai-Man, P. et al. Ophthalmology. The prevalence and natural history of dominant optic atrophy due to OPA1 mutation
Slide 40: Critical human data generation window with disease-modifying pipeline
PYC is in the critical human data generation window with a pipeline of drug candidates with disease-modifying potential 1 2 3 4 5 Disease-modifying In areas of major With the highest Validated in patient- Generating human For personal use only drug candidates1 unmet need probability of success deriv
PYC Therapeutics — 44th annual JP Morgan Healthcare Conference 2026 on 6ix