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Raptor Pharmaceutical Licenses Intellectual Property For Rett Syndrome From French Research Institutes

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.

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