NEW YORK, NY--(Marketwire - October 24, 2012) - Kadmon Corporation, LLC, today announced that the first patient has been dosed in a Phase 1b/2a study of KD020, a reversible tyrosine kinase inhibitor targeting EGFR, HER2, VEGFR 2/3 and Src, in autosomal dominant polycystic kidney disease (PKD).
PKD is a condition with no known treatments and is associated with a number of co-morbidities as well as early mortality. Affecting more than 600,000 Americans(1) and over 12.5 million people worldwide,(2,3) it is the single largest autosomal disorder with an incidence similar to that of cystic fibrosis, muscular dystrophy, hemophilia, Down syndrome and sickle cell anemia combined. The disease is characterized by the formation of cysts in the kidneys and other organs, causing the kidneys to become enlarged and, in half of affected individuals, leading to kidney failure requiring dialysis or transplant.(4) In fully developed autosomal dominant PKD, a cyst-filled kidney can weigh as much as 20 to 30 pounds. High blood pressure is common with PKD and develops in many patients by age 20 to 30.(4) The average age of patients at time of death is 64 years.
"PKD is an under-recognized disease that has a tremendous impact on affected patients, their families and our healthcare system," said Samuel D. Waksal, Ph.D., Chairman and Chief Executive Officer of Kadmon. "Addressing this unmet medical need with a safe, effective, disease-directed therapy will extend lives, increase productivity and reduce the significant costs associated with treating symptoms and complications, including the cost of dialysis and transplant."
KD020 targets three of the critical molecular pathways that are thought to play a role in the progression of PKD: epidermal growth factor receptor (EGFR), Src and vascular endothelial growth factor receptor (VEGFR). In two PKD animal models, inhibition of these pathways has demonstrated significant improvements in kidney size and function. The Phase 1b/2a study is expected to enroll approximately 200 patients, with primary outcome measures of total Kidney Volume and Glomerular Filtration Rate.
John Ryan, Ph.D., M.D., Executive Vice President, Chief Medical Officer of Kadmon, said: "With advances in molecular biology, we have a greater understanding of what pathways drive disease progression in PKD and how to inhibit them, opening the door for a first-ever approved treatment targeting the disease itself. We look forward to determining the safest dose and demonstrating clinical activity in this study, then moving on to larger outcome studies in the future."
Kadmon Corporation, LLC, is a global company built on a 21st-century paradigm for the translation of innovative science into treatment. The company currently offers products and services for the treatment and management of hepatitis C, and is pioneering novel medicines in areas of serious disease, including oncology, infectious diseases, immunology and neurodegenerative diseases. Emphasizing emerging concepts in molecular biology and genomics, Kadmon is developing treatments and treatment combinations that target the metabolomics and signaling pathways associated with disease, with the goal addressing some of today's most pressing areas of unmet medical need. For more information, visit www.kadmon.com.
(1) Grantham JJ, Nair V, Winklhoffer F. Cystic diseases of the kidney. In: Brenner BM, ed. Brenner & Rector's The Kidney. Vol. 2. 6th ed. Philadelphia: WB Saunders Company; 2000: 1699-1730.
(2) Wilson PD. Polycystic kidney disease. N Engl J Med 2004;350:151-164.
(3) Rossetti S, Harris PC. Genotype-phenotype correlations in autosomal dominant and autosomal recessive polycystic kidney disease. J Am Soc Nephrol 2007;18:1374-1380
(4) U.S. Department of Health and Human Services, National Kidney and Urologic Diseases Information Clearinghouse (NKUDIC).