PYC Therapeutics Limited (ASX:PYC) has announced the advancement of its investigational drug, PYC-003, into human clinical trials. This marks a significant step forward in addressing the unmet needs of patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD). The company has received all required regulatory approvals to commence the Phase 1 study in Australia.
Clinical Trial Design
The clinical trial will begin with a Single Ascending Dose (SAD) study involving both healthy volunteers (Part A) and Polycystic Kidney Disease (PKD) patients. The primary endpoint of the study will assess treatment-emergent adverse events and treatment-emergent serious adverse events. Exploratory endpoints will also be evaluated, focusing on the efficacy of PYC-003, including urinary biomarkers. Initial safety and efficacy data are anticipated by 2025.
Addressing Polycystic Kidney Disease
PKD is a debilitating genetic disease characterized by the formation of multiple cysts throughout a patient's kidneys. These cysts increase in size over time, destroying the kidney's architecture and function, ultimately leading to end-stage renal failure. The majority of patients require organ transplantation by the age of 55. PKD affects approximately one in every 1,000 people, representing a major unmet need in medicine.
PYC-003: A Precision Therapy
PYC Therapeutics is focused on developing precision therapies for genetic diseases with limited or no treatment options. PYC-003 is designed to address Autosomal Dominant Polycystic Kidney Disease (ADPKD), a severe condition affecting millions worldwide. Recent pre-clinical studies have demonstrated promising results, with effective gene expression control and a favorable safety profile.
