Investigators have outlined the results of a small, single-arm study of a new drug for Niemann-Pick type C disease which inspired a $200 million biotech buyout earlier this year.
The drug is cyclodextrin, or at least a unique mixture of 2-hydroxypropyl-ß- cyclodextrins (HPßCD) that Vtesse had moved into a pivotal study after gleaning positive data from a study with 14 patients. Sucampo bagged the biotech and its drug for $200 million in April, taking over control of the Phase III.
