PYC Therapeutics (ASX:PYC) has announced plans to advance its investigational drug candidate, PYC-003, into human clinical trials for Autosomal Dominant Polycystic Kidney Disease (PKD) in early 2025. This decision follows the completion of pre-clinical studies demonstrating safety, efficacy, and targeted delivery in animal models and patient-derived kidney cysts.
PKD is a genetic disorder that causes fluid-filled cysts to grow on the kidneys, leading to their gradual enlargement. The disease is the fourth-leading cause of kidney failure globally and affects approximately 1 in 1,000 people. Symptoms often include high blood pressure, severe back or side pain, frequent urinary tract infections, and blood in the urine. Without intervention, the disease often leads to end-stage renal failure, requiring dialysis or a kidney transplant for survival.
PYC-003 is a first-in-class RNA-based therapeutic designed to restore levels of Polycystin-1 (PC1), a protein deficient in PKD patients. The loss of PC1 contributes to cyst formation and disease progression. Pre-clinical results demonstrated that PYC-003 significantly increased PC1 protein expression in kidney tissue, achieving high concentrations in target cells with no observable adverse effects at therapeutic doses in non-human primates. Additional studies showed its potential to reverse the disease phenotype in patient-derived 3D kidney models. The drug’s delivery profile allows for a dosing interval of approximately two months, offering improved convenience for patients.
Dr Rohan Hockings, PYC’s CEO, commented, “The potential of a disease-modifying treatment for PKD is profound, and these pre-clinical results represent a significant milestone for the PKD community. We look forward to the next phase of development as we bring this therapy closer to patients.”
There are no current therapies addressing the root cause of PKD, and the global PKD treatment market is estimated to exceed US$10bn annually.
PYC Therapeutics plans to submit regulatory filings in Q1 2025, aiming to begin Phase 1 trials shortly thereafter.
Shares are trading 2.67% higher at $1.92.