Kadmon Doses First Patient in Phase 1b/2a Study of KD020 in Polycystic Kidney Disease

NBC News


NEW YORK, NY â€" 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."
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