ROCKVILLE, Md., April 18, 2017 (GLOBE NEWSWIRE) -- Sucampo Pharmaceuticals, Inc. (Sucampo) (NASDAQ:SCMP), a global
biopharmaceutical company, today announced the publication of a new study in an independent scientific journal that confirms the
unique composition of VTS-270, a 2-Hydroxypropyl-beta-cyclodextrin (HPβCD) product under investigation as a novel treatment for
Niemann-Pick Disease Type C1 (NPC-1).
NPC-1 is a rare, progressive and ultimately lethal genetic disorder affecting an estimated 2,000-3,000 patients globally. The
results were published today in an article entitled, “Characterization of hydroxypropyl-beta-cyclodextrins used in the treatment of
Niemann-Pick Disease type C1,” in PLOS ONE, the world’s first multidisciplinary, Open Access journal, and are available at
http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0175478.
In this study, researchers demonstrated that VTS-270 has a low degree of consistently specific hydroxypropylation, or degree of
substitution (DS), as well as a low level of doubly charged hetero-dimers as seen through an ion mobility false color
representation, or heat map. The analysis was based on ion distribution and abundance profiles using mass spectrometry methodology
as a means for assessing key molecular activity.
“We are continuing to advance the science around VTS-270 as a potential treatment for NPC,” said Peter Kiener, D. Phil, Chief
Scientific Officer of Sucampo. “These study results further support the specific compositional fingerprint and purity of VTS-270
that distinguishes it from other HPßCD mixtures. The defined and reproducible fingerprint is likely inextricably linked to the
specific potential clinical efficacy and safety of VTS-270.”
HPβCDs are complex mixtures of different species, and variations in production may lead to differences in composition including
differing degrees of hydroxypropylation of the cyclodextrin ring, also known as degrees of substitution. The average degree of
substitution is a critical characteristic of the complexity of HPβCDs mixtures, as it influences the ability to bind to other
molecules, and affects the degree of aqueous solubility, both of which may ultimately influence the biological activity.
About Sucampo Pharmaceuticals, Inc.
Sucampo Pharmaceuticals, Inc. is focused on the development and commercialization of specialized medicines that meet major unmet
medical needs of patients worldwide. Sucampo has two marketed products – AMITIZA, its lead product, and RESCULA – and a late-stage
pipeline of product candidates in clinical development for orphan disease areas. VTS-270 is a mixture of
2-hydroxypropyl-B-cyclodextrins with a specific compositional fingerprint that has been granted orphan designation in the U.S. and
Europe and is in a pivotal Phase 2/3 clinical trial for the treatment of Niemann-Pick Disease Type C-1. Sucampo has an 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. A global company, Sucampo is headquartered in Rockville,
Maryland, and has operations in Japan and Switzerland. For more information, please visit www.sucampo.com.
The Sucampo logo and the tagline, The Science of Innovation, are registered trademarks of Sucampo AG. AMITIZA is a registered
trademark of Sucampo AG.
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Contact: Sucampo Pharmaceuticals, Inc. Silvia Taylor Senior Vice President, Investor Relations and Corporate Affairs 1-240-223-3718 staylor@sucampo.com
