Raptor Pharmaceutical
Corp. RPTP and DaVita Clinical Research(R) (DCR(R) ) 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(R) (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(R)
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.
CONTACT: Raptor Company Contact:
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