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Kadmon Corporation Initiates Phase 2a Portion of KD020 Study in Autosomal Dominant Polycystic Kidney Disease

NEW YORK, NY -- (Marketwired) -- 06/19/14 -- Kadmon Corporation, LLC today announced the initiation of the Phase 2a portion of a Phase 1b/2a study of KD020, the Company's orally bioavailable small molecule kinase inhibitor of Src, HER2, EGFR and VEGFR2/KDR, in autosomal dominant polycystic kidney disease (ADPKD). Initiation of the Phase 2a portion of the study follows the unanimous recommendation of the study's Data Safety Committee after their review of all Phase 1b safety and pharmacokinetics data.

ADPKD is the most prevalent monogenic disease in the US and globally, affecting about 600,000 Americans(1) and about 12.5 million people worldwide,(2,3) with no disease modifying approved therapies currently available. ADPKD 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. The disease 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.(4) Clinical symptoms usually develop between the ages of 30 and 40, but they can begin earlier and include persistent flank pain, hypertension, kidney and urinary tract infections and hematuria. Hypertension is particularly common with ADPKD and develops in many patients by age 20 to 30.(4) The average lifespan of ADPKD patients in the US is 64 years, which is more than 10 years less than the average overall average lifespan in the US. The Phase 2a portion of the study is expected to enroll approximately 25 patients, with primary outcome measures of total kidney volume and glomerular filtration rate.

In the Phase 1b 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 (150 mg daily). The Phase 2a study is designed to evaluate the activity, safety and tolerability of an alternate dosing schedule of 150 mg of KD020 administered three times weekly.

"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," said Samuel D. Waksal, Ph.D., Chairman and CEO of Kadmon. "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. We are encouraged by the initial tolerability profile of KD020, and look forward to understanding its broader potential in addressing ADPKD through the Phase 2a portion of the study."

About Kadmon Corporation
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 liver diseases, and is pioneering novel medicines in areas of serious disease, including oncology, immunology and infectious, neurodegenerative and ophthalmic 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 of addressing some of today's most pressing areas of unmet medical need. For more information, visit www.kadmon.com.

This press release contains forward-looking statements. These forward-looking statements are based on management's expectations and are subject to certain factors, risks and uncertainties that may cause actual results, outcome of events, timing and performance to differ materially from those expressed or implied by such statements. The information contained in this press release is believed to be current as of the date of original issue. Kadmon expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations with regard thereto or any change in events, conditions or circumstances on which any such statements are based.

References

(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).

Contact Information
David Pitts
Argot Partners
212.600.1902
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