POXEL SA announced the publication of preclinical results in autosomal dominant polycystic kidney disease (ADPKD) for PXL770, a novel, first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator. Autosomal dominant polycystic kidney disease, or ADPKD, is a form of chronic kidney disease which is caused by mutations in the PKD1 or PKD2 genes. This causes multiple cysts, or pouches filled with fluid, to form in the kidneys. Autosomal dominant (AD) relates to how the disease is passed down from the parent to child. With ADPKD, cysts develop and grow in the kidneys over time. These cysts continuously grow, causing the kidneys to increase in size and volume. Over time, the growing cysts make it harder for the kidneys to function and eventually lead to kidney failure. Most people with ADPKD have pain, high blood pressure, and kidney failure at some point in their lives.
ADPKD is the fourth leading cause of chronic kidney disease (CKD), affecting 1 in every 400 to 1,000 people (approximately 140,000 patients in the US) and is the most common kidney disorder passed down through family members. More than 50% of ADPKD patients develop renal failure by age 50, followed by dialysis and/or kidney transplantation. Only one drug, tolvaptan, is approved to attenuate progression and is associated with severe liver adverse events and poor tolerability (polyuria).