Kadmon has started the Phase IIa portion of a Phase Ib/IIa study of KD020 in autosomal dominant polycystic kidney disease (ADPKD), following unanimous recommendation of the study's data safety committee.
KD020 is an orally bioavailable small molecule kinase inhibitor of Src, HER2, EGFR and VEGFR2/KDR, in ADPKD.
The Phase IIa study will assess the activity, safety and tolerability of an alternate dosing schedule of 150mg of KD020 administered three times weekly.
With primary outcome measures of total kidney volume and glomerular filtration rate, the Phase IIa portion of the study is likely to recruit around 25 patients.
In the Phase Ib portion of the study, KD020 was generally well-tolerated, with rash (Grade 2) as the most common > Grade 1 adverse event in the highest dose cohort (150mg daily), according to the company.
Kadmon chairman and CEO Samuel 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."
ADPKD, which is caused by a mutation in either the polycystin 1 or 2 (PKD1 or PKD2) genes, involves the formation of cysts in the kidney, leading to destruction of the kidney parenchyma and loss of renal function.
With no current disease modifying approved therapies, ADPKD is the most prevalent monogenic disease in the US and globally, affecting about 600,000 Americans and about 12.5 million people worldwide.