Healthcare Industry News: nephropathic cystinosis
News Release - June 9, 2008
Raptor Pharmaceuticals Corp. Receives FDA Orphan Drug Designation for Cysteamine in Huntington's DiseasePotential Efficacy in Huntington's Disease Already Demonstrated
NOVATO, Calif., June 9 (HSMN NewsFeed) -- Raptor Pharmaceuticals Corp. ("Raptor" or the "Company") (OTC Bulletin Board: RPTP ), today announced that the U.S. Food and Drug Administration ("FDA") has granted orphan drug designation for cysteamine bitartrate ("cysteamine") for the treatment of Huntington's disease ("HD"). Cysteamine is currently approved by the FDA and European Medicines Agency ("EMEA") to treat nephropathic cystinosis ("cystinosis"), a rare lysosomal storage disease. Preclinical results suggest that cysteamine has neuroprotective effects that could potentially help treat HD. Raptor's clinical development subsidiary, Bennu Pharmaceuticals Inc. ("Bennu"), plans to evaluate cysteamine in patients with HD.
HD is a rare and hereditary neurological disease thought to occur in approximately 1 out of every 20,000 people resulting in around 20,000 patients in the U.S. presently. There is currently no drug available that targets the unique molecular defect that is believed to cause HD, a progressive disease that often leads to death within 15 to 20 years after diagnosis. HD is caused by a defective gene resulting 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. Preclinical findings on cysteamine's mechanism of action suggest that it has neuroprotective effects by increasing the levels of BDNF, a neuroprotective neurotrophic factor, in the brain of HD mice. BDNF levels may also be a potential biomarker of efficacy for cysteamine in HD, an important development issue when a therapy is used to prevent or slow clinical manifestation of a disorder.
The 1983 Orphan Drug Act provides important economic incentives to encourage companies to develop potential therapies for the diagnosis, prevention and/or treatment of rare, serious diseases affecting 200,000 persons or less in the U.S. Orphan drug designation by the FDA allows for seven years of market exclusivity following approval of a New Drug Application, as well as reduced regulatory fees and additional regulatory support for research and development initiatives.
Ted Daley, president of Bennu, stated, "Huntington's Disease is a terrible, debilitating disorder with no current cure. Available drugs can only minimize symptoms, such as uncontrollable movements and mood swings. The FDA's decision to grant cysteamine orphan drug designation in Huntington's Disease complements our efforts to develop additional indications for cysteamine. We will be building off of the existing preclinical data that shows cysteamine's safety and potential efficacy to treat HD. We look forward to initiating a Phase II clinical study in HD patients in 2008."
Dr. Christopher M. Starr, Raptor's Chief Executive Officer, stated, "We are delighted that the FDA has granted orphan drug designation for cysteamine in HD. Our team at Raptor is well-versed in developing and gaining regulatory approval of new therapies for orphan drug disorders. We hope to contribute some of this orphan drug expertise and know-how to facilitate Bennu's clinical program for HD."
Through its acquisition by merger of Encode Pharmaceuticals in December 2007, Bennu obtained an exclusive, worldwide license to the intellectual property rights for the development of cysteamine from UC San Diego for the treatment of certain diseases including cystinosis, non-alcoholic steatohepatitis ("NASH") and Huntington's disease.
About Raptor Pharmaceuticals Corp.
Raptor Pharmaceuticals Corp.'s business consists of two segments: its 100% ownership of development stage biotechnology company Raptor Pharmaceutical Inc. ("Raptor Inc."); and its 100% ownership of clinical-stage development company Bennu. Raptor Inc. bioengineers novel drug candidates and drug- targeting platforms derived from the human receptor-associated protein ("RAP") and related proteins, while Bennu advances clinical-stage product candidates towards marketing approval and commercialization.
Raptor Inc.'s preclinical programs target cancer, neurodegenerative disorders and infectious diseases. HepTide(TM) is designed to utilize engineered RAP-based peptides conjugated to drugs to target their delivery to the liver to potentially treat primary liver cancer and hepatitis. In neurodegenerative diseases, engineered RAP peptides called NeuroTrans(TM) are currently undergoing evaluation at Stanford University in cell culture and preclinical models for their ability to enhance the transport of molecules from blood to the brain. In an effort to protect its novel approach, Raptor Inc. currently has five patent applications in review in the U.S., and countries in Europe and Asia, as well as two provisional patent applications licensed from Washington University. In addition, Raptor Inc. has recently submitted two new provisional patent applications in the U.S., the first of which covers a new family of RAP peptides, and the second application to further support and expand its coverage in specific disease indications.
Bennu executes the clinical development of product candidates developed internally at Raptor Inc. and in-licensed candidates that are: 1) new chemical entities in mid- to late-stage clinical development; 2) currently approved drugs with potential efficacy in additional indications; and 3) treatments that may be repurposed or reformulated as potentially more effective or convenient treatments for their currently approved indications. Bennu's initial clinical programs include the treatment of aldehyde dehydrogenase ("ALDH2") deficiency, nephropathic cystinosis and NASH.
For additional information, please visit www.raptorpharma.com.
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: the Company's ability to initiate a Phase II clinical study of cysteamine in Huntington's Disease in 2008, if ever; that the potential benefits reflected in preclinical studies in Huntington's Disease models will be beneficial to patients with Huntington's Disease; the Company will successfully develop cysteamine for any indication, including Huntington's Disease; and the Company will be able to comply with regulations to fulfill the criteria for maintaining Orphan Drug Designation for cysteamine for the treatment of Huntington's Disease. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, which may cause our actual results to be materially different from these forward-looking statements. Factors which may significantly change or prevent our forward looking statements from fruition include that we may be unsuccessful in developing any products or acquiring products; that our technology may not be validated as we progress further and our methods may not be accepted by the scientific community; that we are unable to retain or attract key employees whose knowledge is essential to the development of our products; that unforeseen scientific difficulties develop with our process; that our patents are not sufficient to protect essential aspects of our technology; that competitors may invent better technology; that our products may not work as well as hoped or worse, that our products may harm recipients; and that we may not be able to raise sufficient funds for development or working capital when we require it. As well, our products may never develop into useful products and even if they do, they may not be approved for sale to the public. We caution 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 our filings from time to time with the Securities and Exchange Commission ("SEC"), which we strongly urge you to read and consider, including our Registration Statement on Form SB-2, as amended, that was declared effective on July 10, 2006; our annual report on Form 10-KSB filed with the SEC on November 14, 2007; and our Form 10-QSB filed with the SEC on April 15, 2008, 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 us or to persons acting on our behalf are expressly qualified in their entirety by the cautionary statements set forth in our reports filed with the SEC. We expressly disclaim any intent or obligation to update any forward-looking statements.
Source: Raptor Pharmaceuticals
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