Novel Therapeutics to Treat Niemann-Pick C Disease and Other Lysosomal Disorders

Niemann-Pick disease, type C (NPC) is a lethal, neurodegenerative disorder that affects children. Presently, no therapies for NPC are approved by the Food and Drug Administration (FDA). Several studies have suggested the potential use of 2-hydroxypropyl-β-cyclodextrin (HPBCD) to treat NPC, but the critical studies and data required for an Investigative New Drug (IND) application to evaluate HPBCD were not available. A multi-institute scientific and clinical team of National Institutes of Health (NIH) intramural researchers and clinicians in a unique collaboration with several academic institutes, industry partners, and patient advocacy groups has generated extensive data to establish safe, effective dosing for the delivery of HPBCD directly into the central nervous system of NPC patients.

The goal of this technology transfer effort is to effectively transfer the valuable preclinical and clinical assets to a private partner who can further develop and commercialize the technology to the benefit of NPC patients. The nominees, each with varied specific expertise but a total commitment to the project goals, worked as a cohesive and effective team to achieve the goals of the project while satisfying the needs and aspirations of other stakeholders.

The goals were realized on December 10, 2014, when NIH signed a unique exclusive license, which for the first time included, along with patent rights, the transfer of an IND, U.S. and European orphan drug designations. In addition, the National Center for Advancing Translational Sciences (NCATS) and the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) signed a joint Cooperative Research and Development Agreement (CRADA) with Vtesse, Inc., a company dedicated to developing drugs for the unmet medical needs of patients suffering from severe and life-threatening lysosomal disorder diseases, including NPC.

The result of this technology transfer effort was a commitment from the industry partner to undertake pivotal clinical trials and commercialization, and also to collaboratively conduct preclinical discovery and development of other novel drugs for NPC and other lysosomal storage diseases. This technology transfer makes it possible for the patients and patient families affected by these devastating disorders to hope for an effective cure in the near future.

Awardees

NIH - National Center for Advancing Translational Sciences:

• Lili Portilla
• Dr. Krishna Balakrishnan
• Dr. Charles Niebylski
• Dr. Elizabeth Ottinger
• Dr. Wei Zheng
• Dr. Juan Marugan

NIH - Eunice Kennedy Shriver National Institute of Child Health and Human Development:

• Dr. Forbes Porter

NIH - National Cancer Institute:

• Dr. Alan Hubbs

NIH - Office of Technology Transfer:

• Dr. Suryanarayana Vepa
• Fatima Sayyid
• Richard Rodriguez

Vtesse, Inc.:

• Dr. Bernardus Machielse
• Dr. Ravi Venkataramani