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Raptor Pharmaceutical Completes DR Cysteamine Phase 2b Clinical Trial in Cystinosis

NOVATO, Calif., Nov. 23 /PRNewswire-FirstCall/ -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq: RPTP), today announced results from a Phase 2b clinical trial of its proprietary delayed-release cysteamine bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis ("cystinosis"). The trial, conducted at the University of California, San Diego ("UCSD") General Clinical Research Center, evaluated safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of DR Cysteamine in nine cystinosis patients.

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Phase 2b Clinical Trial Highlights:

  • DR Cysteamine demonstrated improved tolerability and the potential to reduce total daily dosage and administration frequency compared to immediate-release cysteamine bitartrate.
  • Pharmacokinetic evaluation showed that DR Cysteamine had a terminal half-life more than three times longer than the terminal half-life of immediate-release cysteamine bitartrate capsules.
  • Twice-daily DR Cysteamine may achieve the same pharmacodynamic result while using a daily dose 30% lower than immediate-release cysteamine bitartrate capsules administered four times daily.
  • No adverse events recorded during the clinical trial were determined by the principal investigator to be possibly or probably related to DR Cysteamine. Nine adverse events recorded in the clinical trial were determined to be possibly or probably related to immediate-release cysteamine bitartrate.

Raptor plans to meet with the Food and Drug Administration ("FDA") and European Medicines Agency ("EMEA") to discuss the results of its phase 2b study and its plans for a repeat-dose, pivotal, phase 3 clinical trial in cystinosis patients. Upon receiving FDA and EMEA concurrence, Raptor intends to initiate its phase 3 clinical trial at multiple sites in the US and Europe in the first quarter of 2010.

DR Cysteamine is Raptor's proprietary, enteric-coated micro-bead formulation of immediate release cysteamine bitartrate capsules, the current standard of care for treating cystinosis.

Bruce Barshop, M.D., Ph.D., Professor of Clinical Pediatrics at UCSD and principal investigator for the cystinosis clinical trial of DR Cysteamine, stated, "The excellent tolerability of DR Cysteamine, as demonstrated in this study, represents a major step forward in potentially improving the treatment of cystinosis. Raptor's formulation has the potential to improve dosing compliance and long-term outcomes for cystinosis patients."

Raptor's phase 2b clinical trial followed earlier clinical trials with an enteric-coated cysteamine prototype conducted by Ranjan Dohil, M.D., Associate Professor of Pediatrics at UCSD and funded by the Cystinosis Research Foundation ("CRF"). The CRF also supported Raptor's phase 2b clinical trial.

Patrice P. Rioux, M.D., Ph.D., chief medical officer of Raptor, said, "The results from our phase 2b trial are consistent with previous clinical findings from Dr. Dohil and support our plans to provide a twice-daily cysteamine therapy for cystinosis patients. We look forward to continuing our development program of DR Cysteamine in cystinosis with a phase 3 clinical trial."

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.

About DR Cysteamine

DR Cysteamine is an enteric-coated micro-bead formulation of cysteamine bitartrate. Raptor obtained an exclusive, worldwide license to DR Cysteamine, as well as orphan drug designation from the FDA for DR Cysteamine for the potential treatment of nephropathic cystinosis, through its December 2007 acquisition of Encode Pharmaceuticals. In March 2008, Raptor acquired an exclusive worldwide license to intellectual property from the University of California, San Diego ("UCSD") covering use of cysteamine and DR Cysteamine for the potential treatment of non-alcoholic steatohepatitis ("NASH"), a progressive liver disease believed to affect 2-5% of the U.S. population. In October 2009, Raptor and UCSD announced positive interim data from the six-month treatment phase of its Phase 2a clinical study of cysteamine bitartrate in NASH patients. In November 2008 Raptor announced a collaboration with Centre Hospitalier Universitaire d'Angers, in France, to study DR Cysteamine in Huntington's Disease, a rare, genetic neurological disease.

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.

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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 will start a Phase 3 clinical trial in cystinosis patients at multiple US and European sites in the first quarter of 2010, if at all; that DR Cysteamine may allow for a 30% reduction in a patient's total daily cysteamine dose compared to the current standard of care; that DR Cysteamine can be administered in two doses per day instead of four, to achieve the same pharmacodynamic result; that tolerability of DR Cysteamine, as demonstrated in this study, potentially represents a major step forward in the care of cystinosis patients; that Raptor's formulation could result in greatly improved dosing compliance and better long-term outcomes for cystinosis patients; and that any of Raptor's clinical and preclinical drug candidates will result in approved therapeutics. 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 at raising funds to continue its development programs; 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; and that Raptor's products may not work as well as hoped or worse, that the Company's products may harm recipients. 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 current report on Form 8-K as filed with the SEC on November 17, 2009; the joint proxy statement/prospectus on Form S-4 filed with the SEC on August 19, 2009; Raptor's annual report on Form 10-K filed with the SEC on March 27, 2009; and Raptor's quarterly report on Form 10-Q filed with the SEC on August 11, 2009, all of which are available free of charge on the SEC's web site at 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
(858) 531-6100

The Ruth Group
Sara Ephraim Pellegrino (investors) / Janine McCargo (media)
(646) 536-7002 / (646) 536-7033 /

SOURCE Raptor Pharmaceutical Corp.

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