NOVATO, Calif., April 12, 2012 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq:RPTP) today announced that Ted Daley, President, will present an update on its investigational drug candidate, Cysteamine Bitartrate Delayed-Release Capsules (RP103), for the potential treatment of nephropathic cystinosis at the Cystinosis Research Foundation ("CRF") Day of Hope Family Conference to be held from April 20-21. The presentation will include data from the Raptor's Phase 3 clinical trial of RP103 for the potential treatment of cystinosis as well as a regulatory update. Mr. Daley's presentation will take place at Balboa Bay Club in Newport Beach, California on Friday, April 20th at 11:30 a.m. Pacific Time.
Mr. Daley stated, "Raptor always looks forward to these opportunities to share our progress with cystinosis families as part of our ongoing mission to serve this extraordinary community. It will be especially gratifying to be able to report to the Cystinosis Research Foundation Family Conference that we have recently filed our New Drug Application for RP103, since it was the CRF that funded the original clinical studies that first demonstrated the potential benefits of RP103."
"This is a remarkable time for the cystinosis community," said Nancy Stack, President and co-founder of the CRF. "We believe that we are closer than ever to FDA approval of a delayed-release formulation of cysteamine, and researchers continue to make important discoveries about cystinosis gene and stem cell therapies. We feel fortunate that we can again come together as families to support each other and recognize the brilliant researchers who have dedicated their lives to our children and celebrate the significant advances they have made in cystinosis research."
About Nephropathic Cystinosis
Nephropathic cystinosis, an orphan disease, is estimated to effect a population of 2,000 patients worldwide, including 500 patients in the U.S. and 800 patients in Europe. Cystinosis patients have inherited a defective cystine transporter gene which results in body-wide cellular toxicity resulting from the abnormal buildup of the amino acid cystine in the lysosomes. Cystinosis is usually diagnosed in the first year of life and requires lifelong therapy. Cystine crystals accumulate in various tissues and organs, including the kidneys, brain, liver, thyroid, pancreas, muscles and eyes. Left untreated, the disease is fatal by the first decade of life. RP103 reduces cellular toxicity by continuously removing cystine from the lysosome.
About Cysteamine and RP103
RP103 is Raptor's proprietary delayed and extended release oral medication designed to potentially treat the underlying metabolic cause of cystinosis. RP103 is an enteric coated, microbead formulation of cysteamine bitartrate that has been formulated to be sprinkled onto food for administration to patients too young to take oral capsules. Raptor has been granted orphan product designation for RP103 by the EMA and FDA.
In December 2007, Raptor obtained an exclusive, worldwide license from the University of California, San Diego for the development of RP103 for nephropathic cystinosis and for cysteamine for other potential indications including Huntington's Disease currently in a Phase 2/3 clinical trial in France and non-alcoholic steatohepatitis ("NASH") currently in a Phase 2b clinical trial in the US.
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.
For additional information, please visit www.raptorpharma.com.
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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 the FDA will approve a delayed-release formulation of cysteamine, if at all; that researchers continue to make important discoveries about cystinosis gene and stem cell therapies, if at all; 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 and Amendment No. 1 to its Annual Report on Form 10-K/A filed with the SEC on November 14, 2011 and December 19, 2011, respectively; and Raptor's quarterly report on Form 10-Q filed with the SEC on April 9, 2012, 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.