Lead
Programme
Our lead programme aims to treat Autosomal Dominant Polycystic Kidney Disease (ADPKD), which is characterized by uncontrolled growth of fluid-filled cysts and has limited treatment options. ADPKD is the most common hereditary kidney disorder, affecting over 12 million people worldwide. 50% of patients develop kidney failure by the age of 60 with the only treatment for this being dialysis or a renal transplant. Currently, only one therapy is available to slow down the progression of cyst development and renal insufficiency; however, many patients can’t tolerate this therapy.
Technology
How it works
ADPKD: Kidney Cysts and Complications
ADPKD, or Autosomal Dominant Polycystic Kidney Disease, is a genetic disorder characterized by the formation of multiple fluid-filled cysts in the kidneys.
These fluid filled cysts can lead to kidney enlargement, loss of function, and associated complications.
Genetic Mutations and Cyst Development
Cysts develop in different nephron regions due to abnormal cellular function.
This abnormal function is driven by mutations which disrupt the activity of polycystin proteins increasing cyclic AMP levels in key intracellular sites, leading to increased cell proliferation and fluid transport resulting in cyst formation and expansion.
Mironid's LoAc®: Compounds Target Cystogenesis
Mironid's LoAc® compounds activate long-form PDE4 enzymes and reduce the abnormally elevated cAMP levels that drive disease progression.
LoAc® and Slowing ADPKD Progression
LoAc® directly targets abnormal cyclic AMP, decreasing cyst growth, potentially slowing renal disease progression and delaying or preventing the need for dialysis. Efforts are underway to assess LoAc's® efficacy and safety in slowing disease progression in ADPKD.