Sucampo Pharmaceuticals said a Phase 1/2a study of its experimental drug VTS-270 (2-hydroxypropyl-beta-cyclodextrin) injected into the spinal theca indicated the drug can slow the symptoms of the progression of Niemann-Pick Disease Type C1 (NPC-1).
NPC1, a rare fatal genetic disorder that primarily affects children and adolescents, affects 2,000 to 3,000 patients worldwide, a Sucampo release said. The disorder causes a progressive decline in neurological and cognitive functions.
The study involved increasing the amount the drug administered through the spinal column in 14 people and observed the patients after 12 weeks and after 18 weeks of getting the medicine.
The researchers found VTS-270 cut down on the evidence of NPC-1 getting worse in significant ways when the researchers used the NPC Clinical Severity Scale, the release said. The NPC Clinical Severity Scale observes a person's skills in walking, their aptitude to learn and how well they can talk, remember, swallow and use their small muscles, among other factors.
The researchers also found that VTS-270 given through the spinal theca didn't have any severe side effects in the study.
There are no approved products for treating NPC-1 in the U.S, the release said.