The Niemann-Pick disease is poorly understood. Around the globe, however, there are currently several research projects under way. There are encouraging signs, but it seems a suitable form of therapy is still a long way off. In the area of Niemann Pick C there are currently two major known studies:
Cyclodextrin VTS-270
Research with Cyclodextrin (2-hydroxypropyl-β) is originally started by the National Institutes of Health (NIH). For phase 2 of the study, now there is only collaboration with Vtesse. Cyclodextrin has - roughly speaking - the ability to bind substances to itself. The body's cholesterol, which is located in the cells (mainly brain cells) of NPC patients, should therefore be transported with Cyclodextrin. More information about the study and Cyclodextrin is available at www.thenpcstudy.com.
The VTS-270 Clinical Trial: The Patient Experience from Vtesse on Vimeo.
Arimoclomol
The company Orphazyme plans a trial with the drug Arimoclomol. Again a rough explanation: In NPC patients certain proteins are misfolded, respectively have an incorrect structure. This leads to accumulation of endogenous cholesterol. Arimoclomol will now allow for better folding and normalize the structure of the organization responsible for transporting Cholesterol protein of NPC patients. More information about the study and Arimoclomol is available at www.aidnpc.com.
Trappsol/Cyclo
CTD Holdings, a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease, announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Trappsol® Cyclo™ for the treatment of systemic Niemann-Pick Disease Type C (NPC), a rare and fatal genetic disease. Trappsol® Cyclo™ is the Company's proprietary formulation of hydroxypropyl beta cyclodextrin in development for the treatment of NPC; it has previously been designated an Orphan Drug by the FDA and the European Medicines Agency.
Research-News
April 4th , 2017: Takeover of Vtesse by Sucampo (finanzen.ch)
Sucampo Pharmaceuticals and Vtesse declared today that Sucampo acquired Vtesse for a down payment of 200 million USD. Sucampo finances this acquisition by the release of 2.782.678 class A common shares and by 170 million USD in available cash. An external financing was not necessary. press release.