NOVATO, California, February 10 /PRNewswire/ -- Raptor Pharmaceutical Corp. (Raptor or the Company) , 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.
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 (WBCs). 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. (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 http://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 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.
For more information, please contact: Karl Cahill, Investor Relations +1-858-531-6100 firstname.lastname@example.org The Ruth Group Sara Ephraim Pellegrino (investors) +1-646-536-7002 email@example.com Janine McCargo (media) +1-646-536-7033 firstname.lastname@example.org
SOURCE: Raptor Pharmaceutical Corp.
CONTACT: Karl Cahill, Investor Relations of Raptor Pharmaceutical Corp.,+1-858-531-6100, email@example.com; or Investors: Sara EphraimPellegrino, +1-646-536-7002, firstname.lastname@example.org, or Media: JanineMcCargo, +1-646-536-7033, email@example.com, both of The Ruth Groupfor Raptor Pharmaceutical Corp.