Pyc Therapeutics Limited · ASX:PYC
Pyc Therapeutics — Technical Presentation (August 2025)
16 slides from Pyc Therapeutics's technical presentation, Aug 29, 2025
Slide 1: Polycystic Kidney Disease Program – Presentation of Safety Data at ANZSN
29 AUGUST 2025 POLYCYSTIC KIDNEY DISEASE PROGRAM – PRESENTATION OF SAFETY DATA AT ANZSN • PYC is progressing a drug candidate (known as PYC-003) that addresses the underlying cause of Polycystic Kidney Disease (PKD) through clinical trials • The Company today announces that Dr. Aron Chakera will pre
Slide 2: Figure 1. Phase 1a SAD study overview for PYC-003
Figure 1. Phase 1a SAD study overview for PYC-003 Parts A and B of the SAD study will be followed by an Open-Label Multiple Ascending Dose (MAD) study facilitating repeat dosing and evaluation of the optimal dosing regimen of PYC- 003. This study will be conducted alongside a Phase 1b randomised con
Slide 3: Proposed clinical development pathway; About PYC Therapeutics
Figure 3. Proposed clinical development pathway for PYC-003 Next Steps The primary objective of the ongoing Phase 1a/1b study is to evaluate the safety/tolerability profile of PYC-003 with a secondary objective to evaluate the efficacy of the drug candidate in PKD patients. PYC will continue to upda
Slide 4: Contact Us
This ASX announcement should not be relied on as a recommendation or forecast by the Company. Nothing in this ASX announcement should be construed as either an offer to sell or a solicitation of an offer to buy or sell shares in any jurisdiction. This ASX announcement was approved and authorised for
Slide 5: A Phase 1a Clinical Trial of PYC-003 for the Treatment of Patients with ADPKD
A Phase 1a Clinical Trial of PYC-003 for the Treatment of Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD) Dr Aron Chakera September 2025
Slide 6: Disclaimer
Disclaimer The purpose of this presentation is to provide an update of the factors, many of which are outside PYC’s control. Important factors business of PYC Therapeutics Limited (ASX:PYC) [‘PYC’]. These slides that could cause actual results to differ materially from assumptions have been prepared
Slide 7: Objectives for today
Objectives for today • Introduce you to the clinical development pathway for PYC-003 – the first investigational drug candidate to address the root cause of Polycystic Kidney Disease (PKD) • PYC-003 is an RNA therapy that increases Polycystin 1 (PC1) protein expression and rescues the cystic phenoty
Slide 8: ADPKD is an area of major unmet need due to its high prevalence and devastating consequences
ADPKD is an area of major unmet need due to its high prevalence and devastating consequences Healthy adult kidney Polycystic kidney Polycystic Kidney Disease High prevalence ADPKD affects 1 in every 1,000 people meaning >5 million people worldwide have the disease1,2 Life-changing Half of all ADPKD
Slide 9: PYC-003 is the first drug candidate that directly addresses the root cause of ADPKD
PYC-003 is the first drug candidate that directly addresses the root cause of ADPKD to have advanced into human trials PYC-003 • PYC-003 is an antisense oligonucleotide conjugated to a delivery peptide which is administered via intravenous infusion • The oligonucleotide stabilises PKD1 at the mRNA l
Slide 10: PYC-003 is an RNA therapy with disease-modifying potential for ADPKD patients
PYC-003 is an RNA therapy with disease-modifying potential for ADPKD patients PKD1 DNA PKD1 RNA PC1 Protein Kidney Unaffected PKD patient Reduced functional PC1 dosage PYC-003 treated PYC-003 PKD patient 6 Created using BioRender
Slide 11: PYC-003 has a high-velocity path to a New Drug Application
PYC-003 has a high-velocity path to a New Drug Application We are here Planned Planned NDA Phase 1a SAD Study Phase 1b MAD Study Phase 2/3 Study (Parts A, B and C) • Safety & Tolerability • Safety & Tolerability • Safety & Tolerability • Exploratory efficacy • Efficacy • Efficacy • Biomarker (Urinar
Slide 12: PYC-003-CL-001: Phase 1 Study to Evaluate the Safety and Tolerability of PYC-003
PYC-003-CL-001: Phase 1 Study to Evaluate the Safety and Tolerability of Intravenously Administered PYC-003 • Phase 1 Parts A and B are ongoing and an amendment to add Part C (MAD) study is in preparation Phase 1 Study (AU) Part A (Healthy Volunteers) Part B (ADPKD patients) 8
Slide 13: Part B key eligibility criteria are:
Part B key eligibility criteria are: Inclusion criteria Exclusion criteria • Male or female aged 18 to 60 years (inclusive) at the • Presence of potentially confounding genetic time of informed consent. mutations including, but not limited to, the presence of PKD2, HNF1B, GANAB, IFT140, and/or DNAJB
Slide 14: PYC-003 was safe and well-tolerated at doses up to 2.4 mg/kg in Healthy Volunteers
PYC-003 was safe and well-tolerated at doses up to 2.4 mg/kg in Healthy Volunteers • No Treatment Emergent Serious Adverse Events • No changes in serum or urinary electrolytes (creatinine, magnesium, potassium levels) • No evidence of renal injury following administration of PYC-003 Cohort 1 (0.4 mg
Slide 15: No changes in blood chemistry or increases in kidney markers were seen in HVs
No changes in blood chemistry or increases in kidney markers were seen in HVs dosed up to 2.4 mg/kg ALT Serum Magnesium Urea Dosing Dosing Dosing 60 Cohort A1 1.5 Cohort A1 10 Cohort A1 Cohort A2 Cohort A2 8 Cohort A2 Cohort A3 Cohort A3 Urea (mmol/L) Cohort A3 Mg (mmol/L) 40 1.0 ALT (U/L) 6 4 20 0.
Slide 16: The ongoing Phase 1a/1b studies are currently active across 5 sites with 2 more pending activation
The ongoing Phase 1a/1b studies are currently active across 5 sites with 2 more pending activation Activation pending: • Sunshine Hospital in collaboration with Doherty Clinical Trials Mater Hospital Brisbane South Coast Renal • Concord Hospital Linear Clinical Research Westmead Hospital Liverpool H
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