Sucampo Pharmaceuticals has acquired rare disease company Vtesse for $200 million. The deal provides Sucampo with VTS-270, which is in a pivotal study for the treatment of Niemann-Pick Disease Type C1 (NPC-1) with results expected in mid-2018. Sucampo says it provides the capabilities and resources to accelerate the global development and potential commercialization of VTS-270.

Niemann-Pick Disease Type C1 is a rare genetic disorder that begins impacting the lives of those affected from birth to early adulthood. Clinical symptoms do not slow or reverse, with complications from neurological manifestations being the primary cause of eventual fatalities. Incidence of NPC-1 is estimated between 1:100,000 to 1:150,000 live births with an estimated 2,000-3,000 cases globally. NPC-1 results in early death in the vast majority of cases with currently no approved treatments for the disease in the U.S.

VTS-270 is a well-characterized mixture of 2-hydroxypropyl-ß-cyclodextrins (HPßCD) with a specific compositional fingerprint that distinguishes it from other HPßCD mixtures. It is administered by an intrathecal infusion to directly address the neurological manifestations of disease. Preclinical and early clinical studies suggest that the administration of VTS-270 may slow or stop certain indicators of NPC-1, an ultra-orphan, progressive and fatal disease caused by a defect in lipid transport within the cell. 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.

“We are extremely pleased to announce the acquisition of Vtesse,” said Peter Greenleaf, chairman and chief executive officer, Sucampo. “Sucampo brings significant capabilities to Vtesse and its program, and we believe that this acquisition not only has the potential to make an important difference in the lives of patients, their families, and the dedicated physicians who care for them, but also to create value for shareholders. We welcome the employees of Vtesse to our team and look forward to accelerating the global development of VTS-270 in the hopes of bringing this novel treatment to patients afflicted by Niemann-Pick Disease Type C1 in the U.S. and around the globe.”

Ben Machielse, president, founder and chief executive officer, Vtesse, said, “The Vtesse team remains fully committed to the NPC community and will provide continuity to the patients, families, and clinical sites in cooperation with Sucampo. We recognize that Sucampo shares our commitment to the patients and caregivers of NPC and provides us with the best opportunity to bring this important treatment to NPC-1 patients in the U.S. and around the globe. Together, we will accelerate the global development and commercialization of VTS-270, relying on the complementary capabilities at Sucampo. Our commitment to the patients, families and physicians remains steadfast.”

Since its launch in January 2015, the Vtesse team has fully enrolled the registrational study of VTS-270 in NPC-1 at 20 clinical trial sites across the globe, providing broad access for study-eligible patients. The team has also been developing a device to assist healthcare providers with administration of VTS-270 to patients, and has supported compassionate use of the drug candidate.