PYC Therapeutics (ASX:PYC) 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 preclinical studies demonstrating safety, efficacy, and targeted delivery. Animal models and patient-derived renal cysts.

PKD is a genetic disease in which fluid-filled cysts grow in the kidneys and gradually enlarge. The disease is the fourth leading cause of kidney failure worldwide, affecting approximately 1 in 1,000 people. Symptoms often include high blood pressure, severe back and 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 to survive.

PYC-003 is a first-in-class RNA-based therapy designed to restore levels of polycystin-1 (PC1), a protein that is deficient in PKD patients. Loss of PC1 contributes to cyst formation and disease progression. Preclinical results demonstrate that PYC-003 significantly increases PC1 protein expression in kidney tissue and achieves high concentrations within target cells without observable side effects at therapeutic doses in non-human primates. Ta. Additional studies showed the potential to reverse the disease phenotype in patient-derived 3D kidney models. The drug’s delivery profile allows for a dosing interval of approximately 2 months, increasing convenience for patients.

Dr Rohan Hockings, CEO of PYC, commented: We look forward to taking this therapy closer to patients and taking it to the next stage of development. ”

There are no current treatments that address the root causes of PKD, and the global PKD treatment market is estimated to exceed USD 10 billion annually.

PYC Therapeutics plans to file a regulatory filing in the first quarter of 2025 and aims to begin a Phase 1 trial shortly thereafter.

The stock is trading 2.67% higher at $1.92.