NIH partners with Vtesse Inc. to treat rare disease

The National Institutes of Health in Bethesda has partnered with Vtesse Inc., a Gaithersburg-based biotechnology company, to develop treatments for an often-fatal group of disorders known as lysosomal storage diseases.

Vtesse is providing funding for a phase 1 clinical trial of a treatment for Niemann-Pick Disease Type C and other lysosomal storage diseases, which will be conducted at the NIH Clinical Center.

The study is led by researchers at the National Center for Advancing Translational Sciences and the Eunice Kennedy Shriver National Institute of Child Health and Human Development, which are both part of the NIH.

There are about 50 lysosomal storage diseases, also known as lipid storage diseases, which are inherited, rare disorders that usually affect children. They cause fatty materials to accumulate in the body’s cells and tissues, often resulting in damage to the brain, peripheral nervous system, liver and other organs and tissues.

Researchers have been evaluating the safety of a drug called cyclodextrin. Vtesse is supporting that ongoing study, and thanks to the new agreement, will also help fund a second clinical study of cyclodextrin in this country and Europe. That study is expected to begin sometime this year.

Cyclodextrin has received the orphan drug designation in the U.S. and Europe, which provides incentives for companies and researchers to develop products with the potential to diagnose or treat rare diseases.

“The team at Vtesse offers sincere thanks to NIH for the research done to date preparing a strong pre-clinical package and studying use of cyclodextrin in patients with NPC, which is the basis for optimism as we prepare for the next phase of clinical development,” said Ben Machielse, president and CEO of Vtesse, in a statement.

“Moving forward with this public-private partnership will provide a great opportunity for additional studies to address this rare disease,” Machielse continued. “… We look forward to advancing what we believe will be a successful new treatment option.”