Raptor Pharmaceutical Corp. to Present Cystinosis Research at Lysosomal Disease Network's 2010 WORLD Symposium
By Raptor Pharmaceutical Corp., PRNETuesday, February 9, 2010
Forms European Subsidiary, Raptor Pharmaceuticals Europe BV
NOVATO, California, February 10 - Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq: RPTP),
today announced the presentation of cystinosis data at the Annual Lysosomal
Disease Network WORLD Symposium 2010, being held February 10-12 in Miami,
Florida. Biomarker data from a Phase IIb pilot study of Raptor's proprietary
delayed-release cysteamine bitartrate ("DR Cysteamine") will be the subject
of a poster titled, "Correlation of Plasma Cysteamine and WBC Cystine Levels
at Steady State in Patients Treated with Cysteamine Bitartrate." The results
will also be published in an upcoming issue of the journal Molecular Genetics
and Metabolism.
(Logo: www.newscom.com/cgi-bin/prnh/20071022/NYM074LOGO )
The data supports the effectiveness of pre-dose plasma cysteamine
concentration in determining the correct cysteamine dose for nephropathic
cystinosis ("cystinosis") patients. Pre-dose plasma cysteamine concentration
is an easier biomarker to measure in clinical settings compared to cystine
levels in white blood cells ("WBC"s). Researchers in the pilot study found
that pre-dose plasma cysteamine concentration has the potential to be a
viable alternative to WBC cystine levels as a predictive biomarker for proper
cysteamine dosing.
Patrice P. Rioux, M.D., Ph.D., Chief Medical Officer said, "We are
pleased to share our most recent biomarker findings with the Lysosomal
Disease Network, and have taken advantage of this opportunity to further
contribute to the study and treatment of cystinosis. Our positive Phase IIb
DR Cysteamine clinical trial data demonstrated improved tolerability in
cystinosis patients and potential efficacy with less frequent dosing and a
lower total daily dosage compared to standard of care, immediate-release
cysteamine bitartrate. If we can avoid nocturnal dosing and improve
compliance issues associated with today's standard of care, we believe we
have the potential to improve both quality of life and overall treatment
results for cystinosis patients."
Cystinosis is an inborn metabolic error characterized by the abnormal
transport of cystine, an amino acid, out of the lysosomes. Failure to treat
cystinosis can cause serious health consequences, including renal failure and
resultant kidney transplant, growth failure, rickets, photophobia and
blindness. Symptom onset typically occurs within the first year of life, when
cystine crystals accumulate in various tissues and organs, including the
kidneys, brain, liver, thyroid, pancreas, muscles and eyes.
Separately, Raptor announced the establishment of a Dutch subsidiary,
called Raptor Pharmaceuticals Europe BV, to be used as a base for the
Company's European clinical, regulatory, commercial and business development
activities.
About Raptor Pharmaceutical Corp.
Raptor Pharmaceutical Corp. (Nasdaq: RPTP) ("Raptor") is dedicated to
speeding the delivery of new treatment options to patients by working to
improve existing therapeutics through the application of highly specialized
drug targeting platforms and formulation expertise. Raptor focuses on
underserved patient populations where it can have the greatest potential
impact. Raptor currently has product candidates in clinical development
designed to potentially treat nephropathic cystinosis, non-alcoholic
steatohepatitis ("NASH"), Huntington's Disease ("HD"), aldehyde dehydrogenase
("ALDH2") deficiency, and a non-opioid solution designed to potentially treat
chronic pain.
Raptor's preclinical programs are based upon bioengineered novel drug
candidates and drug-targeting platforms derived from the human
receptor-associated protein ("RAP") and related proteins that are designed to
target cancer, neurodegenerative disorders and infectious diseases.
For additional information, please visit www.raptorpharma.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
relate to future events or our future results of operation or future
financial performance, including, but not limited to the following
statements: that Raptor's results will be published in an upcoming issue of
Molecular Genetics and Metabolism; that pre-dose plasma cysteamine
concentration is effective in determining the correct cysteamine dose for
nephropathic cystinosis ("cystinosis") patients and has the potential to be a
viable alternative to WBC cystine levels as a predictive biomarker for proper
cysteamine dosing; that Raptor's DR Cysteamine has improved tolerability in
cystinosis patients and has potential efficacy with less frequent dosing and
a lower total daily dosage compared to standard of care, immediate-release
cysteamine bitartrate; that Raptor's DR Cysteamine has the potential to
improve the quality of life and overall treatment results for cystinosis
patients; that Raptor's Dutch subsidiary will be used for the Company's
European clinical, regulatory, commercial and business development
activities; and Raptor's ability to successfully develop any of its product
candidates. 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 the joint
proxy statement/prospectus on Form S-4 filed with the SEC on August 19, 2009;
Raptor's annual report on Form 8-K filed with the SEC on February 5, 2010;
Raptor's quarterly report on Form 10-Q/A filed with the SEC on January 15,
2010, all of which are available free of charge on the SEC's web site at
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.
For more information, please contact: Karl Cahill, Investor Relations +1-858-531-6100 kcahill@raptorpharma.com The Ruth Group Sara Ephraim Pellegrino (investors) +1-646-536-7002 spellegrino@theruthgroup.com Janine McCargo (media) +1-646-536-7033 jmccargo@theruthgroup.com
Karl Cahill, Investor Relations of Raptor Pharmaceutical Corp., +1-858-531-6100, kcahill at raptorpharma.com; or Investors: Sara Ephraim Pellegrino, +1-646-536-7002, spellegrino at theruthgroup.com, or Media: Janine McCargo, +1-646-536-7033, jmccargo at theruthgroup.com, both of The Ruth Group for Raptor Pharmaceutical Corp.
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