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Raptor Pharmaceutical Inc. Licenses Intellectual Property Related to Huntington's Disease From the Weizmann Institute of Science and Niigata University

6/2/2010 11:04:31 AM

NOVATO, Calif., June 2, 2010 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq:RPTP), announced that the Company has acquired an exclusive worldwide license to intellectual property related to the potential treatment of Huntington's Disease from the Weizmann Institute of Science in Israel and Niigata University in Japan. In addition, Raptor has added Professor Lawrence Steinman, M.D., an inventor on the Weizmann patent, to its Advisory Board.

The Weizmann and Niigata patents cover 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. These patents add to Raptor's portfolio of intellectual property related to its programs utilizing DR Cysteamine, Raptor's proprietary formulation of delayed-release cysteamine bitartrate, licensed exclusively, with worldwide rights, from the University of California, San Diego ("UCSD").

Ted Daley, President of Raptor's Clinical Division, stated, "This exclusive license covering the Weizmann and Niigata patents significantly strengthens and expands our proprietary position as the compounds claimed in these patents are closely related to our lead clinical compound DR Cysteamine. Additionally, this strategic move to enhance our intellectual property position coincides with our planned clinical trial of DR Cysteamine in collaboration with The Centre Hospitalier Universitaire d'Angers ("CHU d'Angers") for the potential treatment of Huntington's Disease, as well as our future plans to explore potential treatments for multiple indications utilizing cysteamine and DR Cysteamine."

Huntington's Disease is a rare, progressive, and hereditary neurological disease that often leads to death within 15 to 20 years after diagnosis. The disease is thought to affect as many as 20,000 patients in the U.S. There is no currently available drug that targets the defective gene believed to cause Huntington's Disease, which results in the degeneration of certain nerve cells in the brain. The disease is characterized by uncontrollable movements and mood swings or depression, followed by dementia.

Raptor plans to initiate a Phase II clinical trial this summer of DR Cysteamine in patients with Huntington's Disease under a previously announced collaboration agreement with CHU d'Angers of France. The Company is also developing DR Cysteamine as a potential treatment for nephropathic cystinosis ("cystinosis") and non-alcoholic steatohepatitis ("NASH").

Dr. Patrice Rioux, Raptor's Chief Medical Officer stated: "This clinical trial is intended to build on preclinical work published by Drs. Sandrine Humbert and Frederic Saudou from the Curie Institute in France. Their work demonstrated a potential mechanism for cysteamine in an in vivo preclinical Huntington's Disease model that showed increased brain and blood levels of brain-derived neurotrophic factor ("BDNF"), a growth factor known to be deficient in Huntington's Disease patients, through its inhibition of transglutaminase, a key regulating enzyme in BDNF production."

In October 2008, Raptor entered into an agreement with CHU d'Angers to participate in a Phase II clinical study in Huntington Disease patients. The study is sponsored by CHU d'Angers and will be largely funded by a grant from the French government. Raptor will provide the clinical trial materials for the study and has regulatory and commercial rights to the clinical data generated in the study. CHU d'Angers recently received ethics committee approval to begin the clinical trial. The study will be performed in eight clinical sites throughout France in a 96 patient, placebo-controlled, 18-month trial, followed by an open-label trial with all placebo patients rolling onto DR Cysteamine and all non-placebo patients continuing on DR Cysteamine for up to another 18 months. The primary end point of the trial will be based upon the Unified Huntington's Disease Rating Scale ("UHDRS"). Raptor was granted Orphan Drug Designation in the U.S. by the FDA for cysteamine as a potential treatment for Huntington's Disease in May 2008 and is in the process of applying for Orphan Drug Designation with the European Medicines Agency ("EMEA").

Separately, Raptor announced that Dr. Steinman will join the Company's Advisory Board. Dr. Steinman is a leading researcher in neurological disorders including Huntington's Disease and Multiple Sclerosis ("MS"), and currently serves as the George A. Zimmermann Professor of Neurology and Neurological Sciences, Pediatrics and Genetics at Stanford University. He is a member of the Institute of Medicine of the National Academy of Sciences. He was also co-founder of Bay Hill Therapeutics and Neurocrine Biosciences. The addition of Dr. Steinman to the Advisory Board brings world-class expertise in the development of CNS drugs. He has developed two antigen specific therapies, using DNA vaccines, for MS and Type 1 diabetes. He was senior author on the seminal 1992 Nature article that reported the key role of a particular integrin in brain inflammation which led to the development of the drug Tysabri.

Dr. Steinman said, "I'm excited to join the Raptor team and work with them to examine the possible benefits of DR Cysteamine in the treatment of Huntington's Disease. In previous animal studies published in the journal, Nature Medicine, the team that I worked with found that in a Huntington's Disease preclinical model those given cysteamine had fewer tremors and other abnormal movements and reduced weight loss, as compared to the untreated subjects. Additionally, cystamine appeared to work differently than other Huntington's Disease drugs, and it may be capable of adding to the benefits of existing therapies. This could be a powerful option in the treatment of Huntington's Disease."

About Cysteamine and DR Cysteamine

Immediate-release cysteamine bitartrate is approved for sale by the FDA and EMEA to treat cystinosis, a rare, genetic lysosomal storage disease. DR Cysteamine is proprietary enteric-coated, microbead formulation of cysteamine bitartrate in gelatin capsules designed to potentially reduce dosing frequency and gastrointestinal side effects.

Raptor obtained an exclusive, worldwide license from UCSD for the development of cysteamine and DR Cysteamine for a number of potential indications including Huntington's Disease, cystinosis, NASH and Batten Disease. Raptor recently presented positive Phase IIa clinical trial data of cysteamine in NASH patients and recently announced positive Phase IIb clinical trial data in cystinosis patients. Raptor is working with the FDA to finalize a Special Protocol Assessment in the coming weeks and plans to initiate its pivotal Phase III study of DR Cysteamine in cystinosis patients shortly thereafter.

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 this exclusive license covering the Weizmann and Niigata patents significantly strengthens and expands Raptor's proprietary position; that CHU D'Angers will commence its Phase IIa clinical trial of DR Cysteamine in Huntington's Disease, if at all; that Raptor will be able to provide clinical trial material for the CHU D'Angers clinical trial, if at all; that the CHU D'Angers clinical trial will build upon the work of Drs. Humbert and Saudou; that DR Cysteamine in combination with existing therapeutics could be a powerful option in the treatment of Huntington's Disease; that Raptor will be able to obtain Orphan Drug Designation with the EMEA for cysteamine for the potential treatment of Huntington's Disease; that DR Cysteamine could potentially reduce dosing frequency and gastrointestinal side effects; that Raptor will be able to finalize its Phase III protocol with the FDA and commence a Phase III clinical trial in cystinosis, if at all; that cysteamine when given to subjects in a Huntington's Disease preclinical model will reduce tremors or other abnormal movements or produce weight loss as compared to untreated subjects; that cysteamine works differently than other Huntington's Disease drugs; that cysteamine may be capable of adding to the benefits of existing therapies; and Raptor's ability to successfully develop any of its 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 may not be able to raise sufficient funds for development or working capital; 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; and 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 filed with the SEC on February 5, 2010; and Raptor's quarterly report on Form 10-Q filed with the SEC on April 9, 2010, 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.

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