Raptor Pharmaceutical Corp. 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 commercialise 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, MD, 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.

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.

Raptor Pharmaceutical Corp. is a biopharmaceutical company focused on developing and commercializing life-altering therapeutics that treat rare, debilitating and often fatal diseases.