NOVATO, Calif., Oct. 23, 2013 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. (Nasdaq:RPTP) and DaVita Clinical Research® (DCR®) (NYSE:DVA) today announced a collaboration to screen blood samples from patients with end-stage renal disease (ESRD) in an effort to identify patients with unrecognized late-onset nephropathic cystinosis.
DCR will supply blood samples with clinical data annotation from DCR's biorepository of over four thousand patients with ESRD. The screening effort will employ high-throughput genetic sequencing of the cystinosin, lysosomal cystine transporter (CTNS) gene, mutations of which result in cystinosis. Results of the collaboration may reveal new insights into the prevalence of missed late-onset cystinosis in this at-risk patient population.
"Today, most nephropathic cystinosis patients are identified in infancy by pediatric nephrologists, yet, as with many genetic diseases, later-onset patients have been identified where the clinical manifestations of cystinosis may go unrecognized well into adulthood," noted Christopher M. Starr, Ph.D., Raptor's chief executive officer. "We believe these late-onset patients are likely to present with ESRD as adults and this screening program, initiated with this collaboration, may help adult nephrologists, many of whom are unfamiliar with the late onset form of the disease, identify patients who suffer from cystinosis."
The CTNS gene is responsible for coding for the protein cystinosin, which is responsible for transporting the amino acid cystine out of the lysosome in cells. The accumulation of cystine is toxic to every cell, and can therefore affect all tissues and organs in the body, most notably the kidneys.
"Nephropathic cystinosis is such a rare disease that it is likely that some cases have not been characterized in the adult population," said Amy Young, vice president and general manager of DaVita Clinical Research. "Our biorepository was designed and collected to enable this type of discovery and we are pleased to embark on a project that has the potential to improve clinical outcomes for ESRD patients."
About Nephropathic Cystinosis
Nephropathic cystinosis comprises 95 percent of the diagnosed cases of cystinosis, a rare, life-threatening metabolic lysosomal storage disorder that causes toxic accumulation of cystine in all cells, tissues, and organs in the body. Elevated cystine leads to progressive, irreversible tissue damage and multi-organ failure, including kidney failure, blindness, muscle wasting and premature death. Nephropathic cystinosis is typically diagnosed in infancy and requires lifelong therapy. Left untreated, the disease is usually fatal by the end of the first decade of life. There are an estimated 500 patients living in the United States with cystinosis, and 2,000 worldwide.
Cystine depletion is the primary treatment strategy for nephropathic cystinosis. However, poor adherence to therapy has been a major challenge resulting in poor sustained control of cystine levels, and patients consequently experience poor clinical outcomes, including kidney insufficiency leading to dialysis and kidney transplantation, muscle wasting and in some cases, premature death. Even brief interruptions in daily therapy can permit toxic accumulation of cystine, exposing tissues to renewed, progressive deterioration.
About Raptor Pharmaceuticals
Raptor Pharmaceutical Corp. is a biopharmaceutical company focused on developing and commercializing life-altering therapeutics that treat rare, debilitating and often fatal diseases. The company's first product, PROCYSBI® (cysteamine bitartrate) delayed-release capsules, is FDA approved for the management of nephropathic cystinosis in adults and children 6 years and older. The European equivalent, PROCYSBI® gastro-resistant hard capsules of cysteamine (as mercaptamine bitartrate), received European Commission approval in September 2013 as an orphan medicinal product for the treatment of proven nephropathic cystinosis for marketing in the European Union (EU). Raptor's pipeline also includes RP103 in a Phase 2/3 trial for Huntington's disease and a Phase 2 trial in nonalcoholic fatty liver disease in children. PROCYSBI was granted orphan designation and exclusivity for nephropathic cystinosis in the U.S. and EU and RP103 has received U.S. orphan drug designation for Huntington's disease. For additional information, please visit www.raptorpharma.com.
About DaVita Clinical Research
DaVita Clinical Research (DCR), a wholly owned subsidiary of DaVita HealthCare Partners Inc., uses its extensive, applied database and real-world healthcare experience to assist pharmaceutical and medical device companies in the design, recruitment, and completion of clinical trials including retrospective and prospective pragmatic trials. DCR's scientific and clinical expertise spans the lifecycle of product development with more than 150 client companies. DCR's Biorepository, Early Clinical Research unit (Phase I-IIa) and Clinical Development (Phase IIb through post-marketing) network of physicians and investigative sites, data research, Health Economics & Outcomes Research, and Medical Communications are focused on providing world-class research in both complex/specialty populations and therapeutic areas, and especially in CKD and ESRD populations. To learn more about DCR, visit www.davitaclinicalresearch.com.
FORWARD-LOOKING STATEMENTS
This document contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements are indicated by words or phrases such as "believes," "expects," "anticipates," "estimates," "plans," "continuing," "ongoing", "projected" and similar words or phrases and relate to future events or our future results of operations or future financial performance, including, but not limited to, statements regarding the conduct and results of the collaboration with DaVita Clinical Research and the identification of patients with late onset cystinosis. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, which may cause the Company's actual results to be materially different from these forward-looking statements. Factors which may significantly change or prevent the Company's forward-looking statements from fruition include: that Raptor may be unsuccessful in developing any products or acquiring products; that Raptor's technology may not be validated as it progresses further and its methods may not be accepted by the scientific community; that Raptor is unable to retain or attract key employees whose knowledge is essential to the development of its products; that unforeseen scientific difficulties develop with the Company's process; that Raptor's patents are not sufficient to protect essential aspects of its technology; that competitors may invent better technology; that Raptor's products may not work as well as hoped or worse, that the Company's products may harm recipients; and that Raptor may not be able to raise sufficient funds for development or working capital. As well, Raptor's products may never develop into useful products and even if they do, they may not be approved for sale to the public. Raptor cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Certain of these risks, uncertainties and other factors are described in greater detail in the Company's filings from time to time with the Securities and Exchange Commission (the "SEC"), which Raptor strongly urges you to read and consider, including: Raptor's transition report for the four months ended December 31, 2012 on Form 10-KT filed with the SEC on March 14, 2013, as amended, and Raptor's Quarterly Report on Form 10-Q filed with the SEC on August 9, 2013, as amended, which is available free of charge on the SEC's web site at http://www.sec.gov. Subsequent written and oral forward-looking statements attributable to Raptor or to persons acting on its behalf are expressly qualified in their entirety by the cautionary statements set forth in Raptor's reports filed with the SEC. Raptor expressly disclaims any intent or obligation to update any forward-looking statements.