Sucampo Pharmaceuticals, Inc. (Sucampo), a global biopharmaceutical company, today announced that the U.S. Patent and Trademark Office has issued a patent for VTS-270, a novel and well-characterized 2-hydroxypropyl-beta-cyclodextrin (HPβCD) product, under investigation as an intrathecally administered treatment for Niemann-Pick Disease Type C1 (NPC-1). Sucampo Pharmaceuticals, Inc. (Sucampo), a global biopharmaceutical company, today announced that the U.S. Patent and Trademark Office has issued a patent for VTS-270, a novel and well-characterized 2-hydroxypropyl-beta-cyclodextrin (HPβCD) product, under investigation as an intrathecally administered treatment for Niemann-Pick Disease Type C1 (NPC-1).
Hydroxypropyl beta-cyclodextrin compositions and methods
The patent, U.S. Patent No. 9,675,634, relates to proprietary cyclodextrin compositions with a specific fingerprint and purity profile that distinguish VTS-270 from other HPβCD products. The patent expires in 2036.
This disclosure provides mixtures of beta-cyclodextrin molecules substituted at one or more hydroxyl positions by hydroxypropyl groups, the mixture optionally including unsubstituted beta-cyclodextrin molecules, for use as a pharmaceutically active ingredient; methods of making such mixtures; methods of qualifying such mixtures for use in a pharmaceutical composition suitable for intrathecal or intracerebroventricular administration; pharmaceutical compositions suitable for intrathecal or intracerebroventricular administration comprising such mixtures; and methods of using the pharmaceutical compositions for treatment of Niemann-Pick disease Type C.
About Sucampo
Sucampo Pharmaceuticals, Inc. is a biopharmaceutical company focused on the development and commercialization of highly specialized medicines. Sucampo has a late-stage pipeline of product candidates in clinical development for orphan disease areas, including VTS-270, a 2-hydroxypropyl-beta-cyclodextrin product with a specific compositional fingerprint that has been granted orphan designation in the U.S. and Europe and is in a pivotal Phase 2b/3 clinical trial for the treatment of Niemann-Pick Disease Type C-1, a rare progressive genetic disorder. VTS-270 also has been granted breakthrough therapy designation in the U.S. Sucampo has an exclusive option for the North American rights to CPP1-x/sulindac, which is in Phase 3 development for the treatment of familial adenomatous polyposis and has been granted orphan drug designation in the U.S. The company has two marketed products – AMITIZA and RESCULA.
Evidence of the Patent
“The patent is further evidence of the profile of VTS-270, which we believe may provide a much-needed treatment for patients and families living with NPC-1.” VTS-270, which is currently in a pivotal Phase 2b/3 trial, has been granted breakthrough therapy designation in the U.S. and orphan designation in both the U.S. and EU. Results from the pivotal trial are expected in mid-2018. NPC-1 is a rare, progressive and ultimately lethal genetic disorder affecting an estimated 2,000-3,000 patients globally. Effective treatment of NPC remains a high unmet need, with no approved products for patients in the U.S.
