updated 6/6/2011 6:46:36 AM ET 2011-06-06T10:46:36

NOVATO, Calif., June 6, 2011 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq:RPTP), today announced that on June 3 the last clinical trial patient visits were completed and final data analysis will commence this week on the Phase 3 clinical trial of its delayed-release oral formulation of cysteamine bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis ("cystinosis"). The Company announced that 41 patients have completed the nine-week study protocol and the Company expects to report the data from this trial by the end of July 2011. The extension phase of the clinical trial, in which all patients completing the Phase 3 study may elect to continue on DR Cysteamine therapy, is ongoing.

The pivotal Phase 3 clinical trial is designed as an outpatient study of the safety, tolerability, pharmacokinetics ("PK") and pharmacodynamics ("PD") of every 12-hour DR Cysteamine compared to every 6-hour immediate-release cysteamine bitartrate in cystinosis patients. DR Cysteamine is designed for reduced dose frequency and improved tolerability, compared to immediate-release cysteamine, which is the current standard of care in cystinosis. The multi-center, randomized, crossover design of this comparative study is a result of discussions with the U.S. Food and Drug Administration ("FDA") by which FDA provided significant guidance on trial protocol design, clinical endpoints, and statistical analyses.

"We are pleased to see our final patients complete the Phase 3 clinical trial and are gratified that 40 of the 41 patients completing the study have elected to enter our long-term extension study in which they have chosen to remain on the twice-a-day DR Cysteamine formulation," commented Patrice Rioux M.D., Ph.D., CMO of Raptor. "As our earliest enrolled patients began to enter the extension study in August of last year, some patients have now been treated with DR Cysteamine for over 9 months; we currently have over 6 months of extension data on our first 20 patients." 

About Nephropathic Cystinosis

Nephropathic cystinosis is an inborn metabolic error characterized by the abnormal transport of cystine, an amino acid, out of the lysosomes. Poor compliance with current treatments for nephropathic cystinosis can cause serious health consequences, including renal failure and resultant need for a kidney transplant; growth failure; rickets and fractures; 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 Cysteamine and DR Cysteamine

DR Cysteamine is Raptor's proprietary enteric-coated, microbead oral formulation of cysteamine bitartrate designed to potentially reduce dosing frequency and reduce gastrointestinal side effects associated with immediate-release cysteamine bitartrate, which is approved for sale by the FDA and European Medicines Agency ("EMA") to treat nephropathic cystinosis.

In December 2007, Raptor obtained an exclusive, worldwide license from the University of California, San Diego for the development DR Cysteamine for nephropathic cystinosis and cysteamine for other potential indications including Huntington's Disease, NASH and Batten 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 thrombotic disorder.

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 .

The Raptor Pharmaceutical Corp. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=7180

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 DR Cysteamine will reduce dose frequency and improve tolerability compared to immediate-release cysteamine; that final data review will occur and that the Company will report initial findings by the end of July, if at all; that all 40 patients will continue to participate in the Company's long-term extension study in which patients remain on the twice-a-day DR Cysteamine formulation; and that Raptor will be able to successfully develop DR Cysteamine or any of its other 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: Raptor's annual report on Form 10-K filed with the SEC on November 22, 2010; and Raptor's quarterly report on Form 10-Q filed with the SEC on April 14, 2011; 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.

CONTACT: Trout Group (investors)
         Lauren Glaser
         (646) 378-2972
         lglaser@troutgroup.com
         
         The Ruth Group (media)
         Jason Rando
         (646) 536-7025
         jrando@theruthgroup.com

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