Drug Research
Drug Discovery & Development

Sucampo Pharmaceuticals buys rare disease firm Vtesse

Published 04 April 2017

Sucampo Pharmaceuticals has acquired rare disease company Vtesse for upfront consideration of $200m.

Sucampo funded the acquisition through the issuance of 2,782,678 shares of Sucampo Class A common stock and $170 million of cash on hand; no external financing was utilized.

Strategic and Financial Benefits of the Transaction

  • Acquisition provides Sucampo with VTS-270, which is in a pivotal study for the treatment of Niemann-Pick Disease Type C1 (NPC-1)
  • Builds on Sucampo’s capabilities, global development platform and focus on specialized areas of high, unmet medical need
  • Fully-enrolled global pivotal clinical trial, with results expected in mid-2018
  • Sucampo provides capabilities and resources to accelerate the global development and potential commercialization of VTS-270
  • Aligns with Sucampo’s patient-focused mission and contributes to goal of building an increasingly diversified, global biopharmaceutical company
  • Product is expected to be launched and accretive to earnings beginning in 2019
  • Vtesse team will continue to support the advancement of VTS-270
  • About Niemann-Pick Disease Type C1
  • 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
  • Estimated 2,000-3,000 cases globally
  • NPC-1 results in early death in the vast majority of cases
  • Currently no approved treatments for the disease in the U.S.

About VTS-270

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.  Effective treatment of NPC remains a high unmet need, with no approved products for patients in the U.S. 

Results from the pivotal trial are expected in mid-2018.

Peter Greenleaf, Chairman and Chief Executive Officer of Sucampo said: “We are extremely pleased to announce the acquisition of Vtesse. 

“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, Drs, President, Founder and Chief Executive Officer of Vtesse Inc. 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.

Source: Company Press Release