PYC therapies (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 that demonstrate safety, efficacy, and targeted delivery in animal models and patient-derived renal 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 kidney 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 deficient in PKD patients. Loss of PC1 contributes to cyst formation and disease progression. Preclinical results demonstrated that PYC-003 significantly increased PC1 protein expression in renal tissue, reaching high concentrations in target cells with no observable adverse effects at therapeutic doses in non-human primates. Further studies have demonstrated its potential to reverse the disease phenotype in patient-derived 3D kidney models. The drug’s dosing profile allows for a dosing interval of approximately two months, providing greater convenience for patients.
Dr Rohan Hockings, Chief Executive Officer of PYC, commented: “The potential for a disease-modifying treatment for PKD is profound and these preclinical results represent a significant milestone for the PKD community. We look forward to the next phase of development to bring this therapy closer to patients.”
There are currently no therapies that address the root cause of PKD, and the global PKD treatment market is estimated to exceed $10 billion per year.
PYC Therapeutics expects to submit regulatory filings in the first quarter of 2025, with the aim of commencing Phase 1 studies shortly thereafter.
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