Raptor Expands its Cysteamine Rights for the Treatment of Neurologic Disorders

License Grants Raptor Exclusive Worldwide Rights to the Use of Cysteamine and Related Compounds in the Treatment of MECP2-Associated Conditions

NOVATO, Calif., May 7, 2013 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. (Nasdaq:RPTP) announced that it has licensed pending patents related to the use of cysteamine for the treatment of Rett syndrome and associated disorders of methyl-CpG binding protein 2 (MECP2). Under terms of the agreement, Raptor receives exclusive global rights to develop and commercialize cysteamine and related compounds to treat MECP2-associated disorders. Raptor's license agreement is with the Technology Transfer Accelerator of South Eastern France (SATT Sud Est) that represents the French medical research organizations where the technology was invented, including the Institut Curie, INSERM and Aix-Marseille Université.

"Mutations in the MECP2 gene have been associated with a number of rare disorders affecting cognitive health and motor and autonomic function, most notably Rett syndrome," said Patrice P. Rioux, M.D., Ph.D., chief medical officer of Raptor. "Cysteamine treatment has demonstrated promising results in non-clinical studies, including improved lifespan and reduced motor defects in MECP2-deficient mice, which we believe warrants further investigation. Significant reductions of brain derived neurotropic factor (BDNF) are found in patients with Rett syndrome, and preclinical results have shown that cysteamine increases BDNF in neurons and facilitates its release from the Golgi apparatus."

"This agreement broadens our existing development portfolio in neurologic disorders in which cysteamine may be beneficial for patients with neurodevelopmental conditions as well as neurodegenerative conditions such as Huntington's disease and Parkinson's disease," stated Ted Daley, chief business officer of Raptor. "We are excited to leverage our cysteamine expertise and eagerly anticipate moving forward in the potential development of a novel therapy for patients with Rett syndrome."

Financial terms of the licensing agreement have not been disclosed. When issued, the initial patents covered by this agreement are expected to be valid until 2030.

About Rett Syndrome

Rett syndrome is a neurodevelopmental disorder that affects females almost exclusively. It is characterized by normal early growth and development followed by developmental slowing, slowed brain and head growth, loss of purposeful hand use, problems with walking, seizures and intellectual disability. Perhaps the most severely disabling feature of Rett syndrome is apraxia, the inability to perform motor functions. Nearly all cases of Rett syndrome are caused by a spontaneously occurring mutation in the methyl-CpG binding protein 2, or MECP2 gene on the X chromosome. Because the MECP2 gene does not function properly in individuals with Rett syndrome, insufficient amounts or structurally abnormal forms of the protein are produced and can cause other genes to be abnormally expressed. According to the National Institutes of Health, Rett syndrome affects approximately one in every 10,000 – 15,000 live female births and an estimated 15,000 women and children are living with Rett syndrome.

About Raptor Pharmaceutical

Raptor Pharmaceutical Corp. is a biopharmaceutical company focused on developing and commercializing life-altering therapeutics that treat rare, debilitating and often fatal diseases. The company's first product candidate, PROCYSBI™ (cysteamine bitartrate) delayed-release capsules, has been approved by the FDA and is currently being reviewed by the European Medicines Agency as a potential new treatment for nephropathic cystinosis, a rare metabolic lysosomal storage disease. Raptor's pipeline also includes RP103 in a Phase 2/3 trial for Huntington's disease and a Phase 2 trial in non-alcoholic fatty liver disease (NAFLD) in children. More information is available at 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 statements that cysteamine may be beneficial for patients with neurodevelopmental conditions as well as neurodegenerative conditions and statements regarding the potential development of a novel therapy for patients with Rett syndrome. 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 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 transition report for the four months ended December 31, 2012 on Form 10-KT filed with the SEC on March 14, 2013, which is 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.