updated 1/24/2011 6:45:44 AM ET 2011-01-24T11:45:44

NOVATO, Calif., Jan. 24, 2011 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq:RPTP), today announced it has completed enrollment in its Phase 3 clinical trial of its proprietary delayed-release oral formulation of cysteamine bitartrate ("DR Cysteamine") in patients with nephropathic cystinosis ("cystinosis").

The pivotal Phase 3 clinical trial is designed as an outpatient study of the safety, tolerability, pharmacokinetics ("PK") and pharmacodynamics ("PD") of DR Cysteamine dosed every twelve hours in patients with cystinosis, compared to the current standard of care, immediate-release cysteamine bitartrate, which requires dosing every six hours. Raptor expects over 30 patients to complete the eight-week study protocol. All patients completing the Phase 3 clinical trial have the option of enrolling in a long-term follow-on study where they continue to receive DR Cysteamine twice daily for the extent of the study.

"As defined in our statistical analysis plan, an interim statistical analysis of intra-patient variance after 20 patients had completed the study, led us to determine that our patient enrollment is complete. With enrollment completed, we anticipate that we will be able to meet our goal of reporting top line data from this clinical trial in the first quarter of 2011," remarked Patrice P. Rioux, M.D., Ph.D., Chief Medical Officer of Raptor. "Patient compliance is paramount to improving therapeutic control and achieving optimal long-term treatment outcome, and we believe twice-daily DR Cysteamine has the potential to be as efficacious as immediate-release cysteamine bitartrate for cystinosis patients, but with a more convenient dosing schedule and potentially improved tolerability."

The randomized, crossover design of the pivotal Phase 3 clinical trial is a result of discussions with the U.S. Food and Drug Administration ("FDA") through which the FDA provided significant guidance on trial protocol design, clinical endpoints, and statistical analyses plans. The primary endpoint of the multi-center, global clinical trial is the steady-state white blood cell ("WBC") cystine levels of patients taking DR Cysteamine compared to immediate-release cysteamine bitartrate. Secondary endpoints are the safety and tolerability of DR Cysteamine and the comparability of steady-state PK of DR Cysteamine and immediate-release cysteamine bitartrate.

"DR Cysteamine has the potential to address two critical issues with the current treatment regimen for cystinosis patients, which result in sub-optimal disease control in many of them. I believe this new formulation of cysteamine bitartrate could greatly improve the quality of life and long-term health outcomes of these patients," said Craig Langman, M.D., The Isaac A Abt MD Professor of Kidney Diseases at the Feinberg School of Medicine, Northwestern University, and Lead Investigator in Raptor's Phase 3 clinical trial.

In November 2009, Raptor completed its Phase 2b clinical trial of DR Cysteamine in cystinosis. DR Cysteamine demonstrated improved tolerability and the potential to reduce total daily dosage and administration frequency compared to immediate-release cysteamine bitartrate. Immediate-release cysteamine bitartrate is the only drug therapy approved for marketing by the FDA and European Medicines Agency ("EMA") for this indication. Despite being the standard of care, gastrointestinal side effects and a strict around-the-clock, every 6 hour dosing schedule for immediate-release cysteamine bitartrate create tolerability and compliance issues for cystinosis patients that DR Cysteamine is designed to address.

About Nephropathic Cystinosis

Nephropathic cystinosis is an inborn metabolic error characterized by the abnormal transport of cystine, an amino acid, out of the lysosomes. Failure to treat 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 gastrointestinal side effects associated with immediate-release cysteamine bitartrate, which is approved for sale by the FDA and EMA to treat nephropathic cystinosis, a rare, genetic lysosomal storage disease.

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 Raptor expects over 30 patients to complete the eight-week study protocol; that the patients enrolled in the Phase 3 clinical trial will enroll in a long-term follow-on study where they continue to receive DR Cysteamine twice daily for the extent of the study; that Raptor will meet its goal of reporting top line data from the Phase 3 clinical trial in the first quarter of 2011; that Raptor believes that twice-daily DR Cysteamine has the potential to be as efficacious as immediate-release cysteamine bitartrate for cystinosis patients, but with a more convenient dosing schedule and potentially improved tolerability; that DR Cysteamine has the potential to address two critical issues with the current treatment regimen for cystinosis patients; that DR Cysteamine could greatly improve the quality of life and long-term health outcomes of cystinosis patients; that patient compliance is paramount to improving therapeutic control and achieving optimal long-term treatment outcome; 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 January 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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