Vtesse, Inc. www.vtessepharma.com located in Gaithersburg Maryland is developing drugs for Niemann-Pick Disease Type C (NPC) and other severe diseases. Vtesse has signed an agreement with NIH’s National Institute of Child Health and Human Development (NICHD www.nichd.nih.gov to develop treatments for Niemann-Pick disease type C (NPC) along with other lysosomal storage disorders.
Lysosomal storage diseases are part of about 50 rare inherited disorders that usually affect children. NPC affects an estimated one in 100,000 to 150,000 children and is often misdiagnosed and/or underdiagnosed.
Fatty materials accumulate in the cells and tissues of the body and can result in damage to the brain, peripheral nervous system, liver, and other organs and tissues with often fatal results. Affected patients are usually identified in early childhood with ataxia and exhibit progressive impairment of motor and intellectual function and may die before adulthood.
NIH’s National Center for the Advancing Translational Sciences (NCATS) www.ncats.nih.gov program called “Therapeutics for Rare and Neglected Diseases” (TRND) has developed cyclodextrin to help treat NPC.
“Patients and their families are desperate for an effective treatment for NPC,” said NICHD Clinical Director Forbes D. Porter, M.D, PhD who is leading the Phase 1 trial evaluating cyclodextrin.
The goal of TRND projects is to develop therapeutic candidates through a strategy known as de-risking which involves the pre-clinical development of therapeutics to the stage where they attract biotechnology and pharmaceutical companies such as Vtesse to invest in their further clinical development.
Vtesse is supporting the ongoing phase 1 clinical trial for NPC at the NIH Clinical Center http://clinicalcenter.nih.gov led by NICHD researchers to continue to evaluate the safety of the drug cyclodextrin. Vtesse will also fund pre-clinical studies led by NCATS researchers to optimize delta-tocopherol compounds for further testing to see whether the compounds can be used as potential single treatments or used as a combination therapy with cyclodextrin.
In addition, the company also plans to collaborate with NIH to launch a second clinical study of cyclodextrin for the treatment of NPC in the U.S and Europe to begin in 2015. The use of cyclodextrin for NPC has been granted orphan drug designation in the U.S and Europe.