Raptor Pharmaceutical Announces Publication of Data Supporting Potential Efficacy of WntTide(TM) in Treatment of Triple-Negative Breast Cancer

NOVATO, Calif., Mar 4, 2010 (GlobeNewswire via COMTEX) -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq:RPTP) today announced the publication of data on a proprietary, Mesd (Mesoderm development)-based peptide, also known as WntTide(TM), licensed by Raptor from Washington University in St. Louis, in the March 1 Early Addition of Proceedings of the National Academy of Sciences. The paper, titled, "LRP6 Overexpression Defines a Class of Breast Cancer Subtype and Is a Target for Therapy," presents findings from studies conducted by the lab of Dr. Guojun Bu, the inventor of the peptide, and Professor of Pediatrics, Cell Biology and Physiology at Washington University in St. Louis.

Dr. Bu's work evaluated the suppression of LRP6 in inhibiting Wnt signaling and breast tumor growth. Results support the potential efficacy of WntTide(TM) as a targeted treatment for triple-negative breast cancers, a particularly aggressive and difficult-to-treat indication. Abnormal Wnt activation, found in 40% to 60% of breast cancers, is often associated with triple-negative breast cancers. Raptor is currently evaluating WntTide(TM) in a preclinical breast cancer model to inhibit the Wnt-signaling pathway designed to block cancers dependent upon signaling through LRP6.

Breast cancer causes more than 40,000 deaths annually in the U.S. and is the second leading cause of cancer mortality among American women. According to Cancer Research U.K., triple-negative breast cancer account for approximately 15% of all breast cancer cases. While current targeted breast cancer treatments address the overexpression of estrogen and progesterone receptors ("ER/PR") and the HER2 protein, triple-negative breast cancer is clinically negative for expression of these targets and generally cannot be treated with existing targeted treatments.

Dr. Bu stated, "There is an unmet need for targeted treatments that effectively address breast cancer subtypes that have been aggressive and unresponsive to existing chemotherapies and targeted treatments. The data on LRP6 suppression is exciting to me as a researcher, because it suggests a potential new target for developing breast cancer therapies that address aberrant Wnt signaling."

Todd C. Zankel, Ph.D., Raptor's Chief Scientific Officer, stated, "The cancer treatment landscape has long been shifting towards targeted therapies directed at the specific receptors and pathways responsible for the disease. These data suggest that WntTide(TM) could have value as a potential novel approach to a subtype of breast cancers that have not previously benefitted from having a targeted therapy. This preliminary data, considered along with our NeuroTransTM collaboration with Roche, and previously reported preclinical data showing enhancement of liver delivery using LRP1 targeted HepTideTM, point to the scientific interest and potential importance of LRP receptors as drug targets and the potential of our peptide-based LRP targeting technology for a variety of improved drug delivery and therapeutic applications."

Washington University in St. Louis also announced the news in a separate press release which can be found at http://news.wustl.edu/news/Pages/20359.aspx.

About Mesoderm Development Protein ("Mesd") Platform: WntTide(TM)

Human mesoderm development protein ("Mesd") is a chaperone protein necessary for the proper folding of LRP5 and LRP6, two receptor proteins that play a role in the regulation of bone mass, proliferation of tumor cells, and other diseases characterized by overactivation of the Wnt-signaling pathway. In November 2006, Raptor licensed the use of Mesd from Washington University in St. Louis for the potential treatment of cancer and bone density disorders.

WntTide(TM) is a proprietary Mesd-based peptide generated in Dr. Bu's lab at Washington University in St. Louis that is currently under development by Raptor as a potential anti-tumor agent in breast cancers that depend on signaling through LRP5 and LRP6.

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.

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

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

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 potential efficacy of WntTide(TM) as a targeted treatment for triple-negative breast cancers; that WntTide(TM) may be a potential new target for developing breast cancer therapies that address aberrant Wnt signaling; that WntTide(TM) could have value as a potential novel approach to a subtype of breast cancers that have not previously benefitted from having a targeted therapy; the potential importance of LRP receptors as drug targets; the potential of Raptor's peptide-based LRP targeting technology for a variety of improved drug delivery and therapeutic applications; 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'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 current report on Form 8-K filed with the SEC on February 5, 2010; and Raptor's quarterly report on Form 10-Q/A filed with the SEC on January 15, 2010, 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.

This news release was distributed by GlobeNewswire, www.globenewswire.com

SOURCE: Raptor Pharmaceutical Corp.

CONTACT: Raptor Pharmaceutical Corp. Karl Cahill, Investor Relations (858) 531-6100 kcahill@raptorpharma.com The Ruth Group Investors Sara Ephraim Pellegrino (646) 536-7002 spellegrino@theruthgroup.com Media Janine McCargo (646) 536-7033 jmccargo@theruthgroup.com

Posted: March 2010

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