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Highlights:
PYC Therapeutics advances its investigational drug, PYC-003, into human clinical trials for Autosomal Dominant Polycystic Kidney Disease (PKD).
Pre-clinical studies have demonstrated safety, efficacy, and targeted delivery of the drug in animal models and patient-derived kidney cysts.
PYC-003 represents a potential disease-modifying treatment for PKD, a condition with no current therapies addressing its root cause.
PYC Therapeutics (ASX:PYC) has announced that it will move its investigational drug candidate, PYC-003, into human clinical trials for Autosomal Dominant Polycystic Kidney Disease (PKD) in early 2025. This decision follows the successful completion of pre-clinical studies, which demonstrated the drug’s safety, efficacy, and its ability to target kidney cysts in animal models and patient-derived cells.
PKD is a genetic disorder that causes fluid-filled cysts to form in the kidneys, eventually leading to their enlargement and impairment. It is one of the leading causes of kidney failure globally, affecting approximately 1 in 1,000 individuals. Symptoms of PKD include high blood pressure, back or side pain, urinary tract infections, and blood in the urine. The disease can progress to end-stage renal failure, which often requires dialysis or a kidney transplant.
PYC-003 is an RNA-based therapeutic designed to restore Polycystin-1 (PC1) protein levels, which are deficient in individuals with PKD. The absence of PC1 contributes to cyst formation and the progression of the disease. In pre-clinical studies, PYC-003 demonstrated its ability to increase PC1 expression in kidney tissue, with no observed adverse effects at therapeutic doses in non-human primates. The drug was also able to reverse the disease phenotype in 3D kidney models derived from patients. Notably, the drug’s delivery system allows for dosing every two months, which could offer increased convenience for patients.
PYC Therapeutics is set to submit regulatory filings in the first quarter of 2025, aiming to commence Phase 1 trials shortly thereafter. This represents a significant step in the development of a potential new treatment for PKD, a disease for which there are currently no therapies addressing its root cause. The global PKD treatment market is estimated to exceed US$10 billion annually, highlighting the potential impact of a successful therapeutic intervention.
