Date: 2015-01-07
Type of information: R&D agreement
Compound:
Company: NIH (USA) Vtesse (USA - MD)
Therapeutic area: Rare diseases
Type agreement: R&D agreement
Action mechanism:
Disease: Niemann-Pick disease type C and other lysosomal storage disorders
Details: * On January 7, 2015, researchers from the National Institutes of Health have entered into an agreement with biotechnology company Vtesse, Inc., of Gaithersburg, Maryland, to develop treatments for Niemann-Pick disease type C (NPC) and other lysosomal storage disorders. Researchers at the National Center for Advancing Translational Sciences (NCATS) and the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), both parts of NIH, will conduct studies on NPC and other lysosomal storage disorders with funding provided by Vtesse. Vtesse will support the ongoing phase I clinical trial for NPC at the NIH Clinical Center, led by NICHD researchers who have been evaluating the safety of the drug cyclodextrin. Vtesse also plans to collaborate with NICHD to launch a second clinical study of cyclodextrin for the treatment of NPC in the United States and Europe, anticipated to begin in 2015. The use of cyclodextrin for NPC has been granted orphan drug designation in the United States and Europe, providing development incentives for products that demonstrate promise for the diagnosis and treatment of rare diseases or conditions. The NPC project is a collaboration among government, academic and industry researchers, who worked with patient groups and the NPC community to advance knowledge about the disease. Before the TRND project, researchers had found that cyclodextrin showed promise as a treatment for NPC in studies of animal models for the disease. Additional NCATS efforts provided further evidence that cyclodextrin demonstrated potential as a treatment for NPC. Through this agreement, Vtesse will also fund pre-clinical studies led by NCATS researchers to develop various types of the compound delta-tocopherol, a form of vitamin E, to target lysosomal storage disorders. The NCATS team will optimize the delta-tocopherol compounds for further testing as potential single treatments or as a combination therapy with cyclodextrin. Vtesse has exclusively licensed several NCATS patent applications specifically for their use in the treatment of lysosomal storage disorders. In addition to NCATS and NICHD researchers, the NPC project team includes NIH scientists from the Clinical Center, National Human Genome Research Institute, National Institute of Mental Health, National Institute of Neurological Disorders and Stroke, and National Institute on Deafness and Other Communication Disorders. Academic collaborators include Washington University in St. Louis, Albert Einstein College of Medicine in New York City and the University of Pennsylvania in Philadelphia. Janssen Research & Development, LLC, is an industry research collaborator.
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