Raptor Pharmaceutical Obtains Tissue Fibrotic Disease Intellectual Property Related to Cysteamine

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NOVATO, Calif., Oct. 10, 2012 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq:RPTP), today announced that the Company has acquired exclusive world-wide rights to intellectual property related to cysteamine and related compounds in the potential treatment of tissue fibrosis from the Seattle Children's Research Institute ("SCRI").

Raptor's agreement with SCRI provides exclusive rights to technology related to compositions and methods for treating patients having or being at risk of developing pathological fibrosis including chronic kidney disease ("CKD"). Raptor holds a portfolio of intellectual property related to composition of matter and methods of use of cysteamine and related compounds, covering therapeutic applications, including genetic diseases, metabolic disorders and neurological diseases. Raptor's RP103, its proprietary, delayed-release oral formulation of cysteamine bitartrate, is in clinical development for the potential treatment of nephropathic cystinosis, a rare metabolic lysosomal storage disease, Huntington's Disease, a genetic neurological disease, and non-alcoholic steatohepatitis ("NASH"), a severe form of non-alcoholic fatty liver disease.

In addition to other fibrotic conditions, the license covers the use of cysteamine or cystamine in the potential amelioration of progressive interstitial renal fibrosis by modulating oxidative stress. Researchers at SCRI have shown in preclinical studies in mice that daily treatment with cysteamine attenuated renal fibrosis, with up to 25% reduction of extracellular fibrotic material observed over a 21-day study period.

"Fibrosis can occur in many organ systems in the body resulting in serious health and in some cases life-threatening consequences," said Patrice P. Rioux, M.D., Ph.D., Raptor's Chief Medical Officer. "This licensing agreement with Seattle Children's Research Institute further expands the applications of our cysteamine formulations in the potential treatment of fibrotic disease, including chronic kidney, liver and severe gastrointestinal diseases, as well as metabolic diseases, and we look forward to continuing to collaborate with the SCRI researchers as we expand our clinical development into these areas."

Daryl Okamura, M.D., Principal Investigator at SCRI and Assistant Professor of Pediatrics at the University of Washington School of Medicine, said, "Kidney dysfunction is a recognized risk factor for poor outcome in a variety of disease states, yet there are few therapies to stop the relentless progression of CKD and alleviate the ongoing oxidative stress within the kidney. We believe that our preclinical results support further development with cysteamine for this indication. We hope to see the study of this potential cysteamine treatment move into a clinical trial."

About Chronic Kidney Disease

CKD affects approximately 26 million Americans and is characterized by the progressive loss of renal function over a protracted period of time. If the progression of CKD is not halted, CKD can develop into end stage renal disease, or chronic renal failure where the kidney no longer functions and the patient requires dialysis or a kidney transplant. There is no cure for CKD. The goals of treatment are to slow disease progression, treat the underlying causes, treat complications of the disease and when necessary, replace lost kidney function. The most common causes of CKD are diabetes mellitus, hypertension and glomerulonephritis. CKD is also caused by genetic disorders, including nephropathic cystinosis.

About Cysteamine and RP103

RP103 is Raptor's proprietary delayed release oral medication currently being investigated in several indications. RP103 is an enteric coated, microbead formulation of cysteamine bitartrate. In December 2007, Raptor obtained an exclusive, worldwide license from the University of California, San Diego for the development of RP103 and other forms of cysteamine for the potential treatment of: Huntington's Disease, currently in a Phase 2/3 clinical trial in France; non-alcoholic steatohepatitis ("NASH"), currently in a Phase 2b clinical trial in the U.S.; and for the development of RP103 for the potential treatment of 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 RP103 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 RP103 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 licensed issued patents related to the potential treatment of Huntington's Disease and other neurodegenerative diseases with cysteamine and related compounds from Niigata University and Weizmann Institute and patent applications for the use of cysteamine and related compounds for the potential treatment of Parkinson's Disease from Laval University, for the use of cysteamine and related compounds for the potential treatment of malaria and other parasitic diseases from McGill University and for the potential treatment of tissue fibrosis from the Seattle Children's Research Institute. Raptor has been granted orphan product designation for RP103 for the potential treatment of nephropathic cystinosis by the EMA and the FDA, and for the potential treatment of Huntington's Disease by the FDA.

About Raptor Pharmaceutical Corp.

Raptor Pharmaceutical Corp. (Nasdaq:RPTP) ("Raptor") seeks to research, produce, and deliver 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"), aldehyde dehydrogenase deficiency ("ALDH2"), 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 and related proteins that are designed to target cancer and infectious diseases.

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

For additional information, please visit www.raptorpharma.com.

About Seattle Children's Research Institute

Located in downtown Seattle's biotech corridor, Seattle Children's Research Institute is pushing the boundaries of medical research to find cures for pediatric diseases and improve outcomes for children all over the world. Internationally recognized investigators and staff at the research institute are advancing new discoveries in cancer, genetics, immunology, pathology, infectious disease, injury prevention and bioethics, among others. As part of Seattle Children's Hospital, the research institute brings together leading minds in pediatric research to provide patients with the best care possible. Seattle Children's serves as the primary teaching, clinical and research site for the Department of Pediatrics at the University of Washington School of Medicine, which consistently ranks as one of the best pediatric departments in the country. For more information, visit http://www.seattleChildrens.org/research.


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 continue to collaborate with the SCRI researchers; that Raptor will expand its clinical development into the areas of fibrotic disease; that preclinical results involving daily treatment with cysteamine in CKD can be duplicated in a clinical trial; 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 July 10, 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.