It is estimated that approximately 141,000 individuals in the US have autosomal dominant polycystic kidney disease (ADPKD), a monogenic multi-systemic disorder characterized by the development of renal cysts and various extrarenal manifestations.
people in US affected
annual healthcare cost
Median age at death or onset
About 50% of patients with ADPKD will progress to end stage renal disease, with hemodialysis or kidney transplant being the only available treatments. The annual cost of care for these patients is estimated at ~$5.7 billion.
Polycystin 1 (PKD1) genetic mutations underlying ADPKD substantially increase mTORC1 activity, causing progressive enlargement and ultimately failure of the kidneys through formation of cysts and fibrous tissue. Patients with ADPKD experience a range of life-limiting effects of the disease and often progress to dialysis or kidney transplants at some point in their life. Today there are few treatment options for patients with ADPKD, and there is substantial need for new therapies.
Human trials have shown reduced cyst volume following treatment with everolimus, a non-selective mTORC1/mTORC2 inhibitor, but at doses that were either not well-tolerated or were only marginally effective due to dosing limitations associated with mTORC2 inhibition.
We believe that NV-20494, our selective mTORC1 inhibitor from our Navalog class of compounds, will be better tolerated and allow for more effective kidney mTORC1 inhibition than non-selective inhibitors. This could represent a breakthrough in the treatment of ADPKD.