Raptor Pharmaceutical Corp. Announces Presentation of PROCYSBI(TM) (RP103) Extension Study Data at the ASN Annual Meeting

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NOVATO, Calif., Oct. 17, 2012 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq:RPTP), announced that Craig B. Langman, M.D., Head of Kidney Diseases, and the Isaac A. Abt, M.D., Professor of Kidney Diseases and Tenured Professor of Pediatrics, Northwestern University Feinberg School of Medicine, will present a Late Breaking Poster session titled "Extended Treatment with RP103 (PROCYSBI™) in Patients with Nephropathic Cystinosis" at Kidney Week 2012, the world's premier nephrology meeting, which is being held October 30-November 4th at the San Diego Convention Center in San Diego, California. Dr. Langman's presentation is on Saturday, November 3rd in a session beginning at 10:00 A.M. PT.

About Nephropathic Cystinosis

Nephropathic Cystinosis is a rare, life-threatening metabolic disorder that causes systemic toxic cystine accumulation. Toxic cystine accumulation causes progressive and irreversible tissue damage and organ failure including renal failure, blindness, CNS toxicities, respiratory deficiencies and muscle wasting. Cystinosis is usually diagnosed in the first years of life and requires lifelong therapy. Left untreated, the disease is fatal by the end of the first decade of life.

About Cysteamine and PROCYSBI™ (RP103)

PROCYSBI™ is Raptor's delayed-release oral medication currently in clinical development for several indications. PROCYSBI™ is an enteric-coated, delayed and extended-release formulation of cysteamine bitartrate. PROCYSBI™ was engineered specifically to allow release of micro-spheronized enteric-coated cysteamine bitartrate in the duodenum for optimal absorption while simultaneously enabling administration every 12-hours.

In December 2007, Raptor obtained an exclusive, worldwide license from the University of California, San Diego to intellectual property related to the development of PROCYSBI™ and other forms of cysteamine for the potential treatment of non-alcoholic steatohepatitis ("NASH"), currently in a Phase 2b clinical trial in the U.S., Huntington's Disease, currently in the Phase 2/3 clinical trial in France, and for the development of PROCYSBI™ for nephropathic cystinosis, which Raptor has recently filed for marketing approval in the U.S. and E.U. The U.S. Food and Drug Administration ("FDA") has accepted for filing Raptor's New Drug Application ("NDA") for PROCYSBI™ for the potential treatment of nephropathic cystinosis and assigned the user fee goal date of January 30, 2013. Raptor's E.U. marketing application of PROCYSBI™ for the potential treatment of nephropathic cystinosis is under review by the EMA, and Raptor expects a decision in the first half of calendar 2013. Raptor has been granted orphan product designation for PROCYSBI™ for the potential treatment for nephropathic cystinosis by the European Medicines Agency and FDA and for the potential treatment of Huntington's Disease by the FDA. Raptor's intellectual property portfolio also includes patents covering the use of transglutaminase inhibitors, a class of molecules chemically similar to cysteamine, in the potential treatment of Huntington's Disease and other neurological disorders from the Weizmann Institute of Science in Israel and Niigata University in Japan; the use of cysteamine and related compounds in the potential treatment of parasitic diseases, including malaria, from McGill University in Montreal, Canada; the use of cysteamine and related compounds in the potential treatment of Parkinson's Disease from Université Laval, Quebec, Canada; and the use of cysteamine and related compounds in the potential treatment of tissue fibrosis from the Seattle Children's Research Institute.

About Raptor Pharmaceutical Corp.

Raptor Pharmaceutical Corp. (Nasdaq:RPTP) ("Raptor") seeks to research, develop, and provide access to medicines that improve life for patients with severe, rare disorders. Raptor currently has product candidates in clinical development designed to potentially treat nephropathic cystinosis, Non-alcoholic Steatohepatitis ("NASH"), Huntington's Disease ("HD"), and aldehyde dehydrogenase deficiency ("ALDH2").

Raptor's preclinical programs are based upon bioengineered novel drug candidates and drug-targeting platforms derived from the human receptor-associated protein and related proteins that are designed to target cancer 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


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 the NIDDK will be able to complete enrollment and complete the Phase 2b clinical trial; that Raptor will be able to supply clinical material and funding for the Phase 2b clinical trial; if the Phase 2b clinical trial is completed, that the resulting clinical trial results will meet its primary objectives and secondary clinical endpoints; that the Phase 2b clinical trial will emulate the results of the Phase 2a open label study; that RP103 will prevent significant GSH depletion and potentially reverse NASH-related liver damage; that the FDA and EMA will deliver a decision regarding marketing approval of RP103 for the potential treatment of nephropathic cystinosis on January 30, 2013 or the first half of calendar 2013, respectively; and that Raptor will be able to successfully develop RP103 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, as amended by Form10-K/A, filed with the SEC on November 11, 2011 and December 19, 2011, respectively; and Raptor's quarterly report on Form 10-Q filed with the SEC on April 9, 2012; 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 EVC Group (media) Janine McCargo (646) 688-0425 jmccargo@evcgroup.com

Source:Raptor Pharmaceutical Corp.