• PYC's drug candidate PYC-003 addresses the underlying cause of Polycystic Kidney Disease (PKD)
• Regulatory approval received to commence human trials of PYC-003
• Initial human safety and efficacy data anticipated within 2025

PYC Therapeutics (ASX:PYC) is a clinical-stage biotechnology company creating precision therapies for patients with genetic diseases. One of the company's assets is an investigational drug candidate (PYC-003) that addresses the underlying cause of Autosomal Dominant Polycystic Kidney Disease (PKD). PYC today announces that it has received all required regulatory approvals to commence human trials of PYC-003. The company will now proceed to initiate a Single Ascending Dose (SAD) study of this drug candidate in healthy volunteers and PKD patients. PKD is a life-changing disease characterized by multiple cysts forming throughout a patient's kidney that increase in size over time, ultimately leading to end-stage renal failure and the need for organ transplantation in the majority of patients by the age of 55. The progression of PYC-003 into human trials is supported by pre-clinical data generated in both animal and patient-derived models, demonstrating high target tissue concentration at safe and well-tolerated doses. The efficacy of this drug candidate has been demonstrated both quantitatively in cells derived from kidneys and animal models, as well as phenotypically in human 3D kidney cyst models derived from patients with end-stage renal failure due to PKD. The SAD study will involve subjects receiving a single intravenous infusion of PYC-003, with the primary endpoint being the assessment of Treatment Emergent Adverse Events and Serious Adverse Events. The study will also collect data on exploratory endpoints directed towards the efficacy of the drug candidate, including urinary biomarkers and Magnetic Resonance Imaging analysis of biometric markers. Dosing of patients in this study is expected to be completed in approximately 12 months, with a budgeted cost of A$10 million.

PYC expects to complete dosing of patients in the SAD study within approximately 12 months, with a budgeted cost of A$10 million.

PYC expects to have initial human safety and efficacy data for its PYC-003 drug candidate within 2025.