Kadmon begins Phase IIa portion of KD020 trial in autosomal dominant polycystic kidney disease

Start of the Phase IIa portion of the trial follows the unanimous recommendation of the study’s Data Safety Committee after their review of all Phase Ib safety and pharmacokinetics data.

The disease is caused by a mutation in either the polycystin 1 or 2 (PKD1 or PKD2) genes, resulting in the abnormal, uncontrolled growth of renal tubular epithelial cells.

ADPKD is characterized by the formation of cysts in the kidney causing destruction of the kidney parenchyma resulting in loss of renal function.

PKD is the fourth highest cause of kidney failure and clinical symptoms usually develop between the ages of 30 and 40, but they can start earlier and include persistent flank pain, hypertension, kidney and urinary tract infections and hematuria.

In the Phase Ib portion of the trial, KD020 was generally well tolerated, with rash (Grade 2) as the most common > Grade 1 adverse event in the highest dose group (150mg daily).

The Phase IIa trial is designed to assess the activity, safety and tolerability of an alternate dosing schedule of 150mg of KD020 administered thrice times weekly.

Kadmon chairman and CEO Samuel Waksal said autosomal dominant PKD is one of the most common life-threatening genetic diseases, frequently leading to end stage kidney disease requiring dialysis or transplant as early as the fourth decade of life.

"Unlike currently used therapies, which address symptoms but do not delay onset to kidney failure, KD020 is designed to inhibit the molecular pathways central to progression of the disease itself, namely EGFR, Src and VEGFR," Waksal said.

"We are encouraged by the initial tolerability profile of KD020, and look forward to understanding its broader potential in addressing ADPKD through the Phase IIa portion of the study."