Sucampo bolsters pipeline with rare disease buy
- Sucampo Pharmaceuticals has put down $200 million upfront in cash and stock to snag rare disease biotech Vtesse, including its clinical stage Niemann-Pick disease type C1 drug, VTS-270.
- The Vtesse team is staying on board to continue the ongoing Phase 2b/3 pivotal-stage clinical trial for VTS-270. This prospective, randomized, double-blind, controlled is expected to readout in mid-2018 and the drug could reach the market as early as 2019.
- Sucampo will pay $170 million in cash, plus issue 2.8 million shares to fund the acquisition. It also agreed to pay royalties and a portion of any revenues that are generated from selling the Priority Review Voucher the company receives if its lead drug is approved.
Gaithersburg, Maryland-based Vtesse was the first spinout from Cydan Development, a drug accelerator in Cambridge, Mass. that has a focus on rare genetic diseases. The startup launched in 2015 to develop VTS-270, a mixture of cyclodextrins, that has potential to slow or stop certain signs of Niemann-Pick disease type C1.
“With the addition of Sucampo’s commercial expertise, once approved, NPC patients in the U.S. and around the globe will benefit,” said Chris Adams, co-founder and CEO at Cydan.
VTS-270 has breakthrough designation from the Food and Drug Administration, which has potential to speed it to market. Mizuho analysts estimate the drug could have peak sales of $240 million.
Vtesse is also working on earlier stage programs for lysosomal storage diseases, including next-generation therapeutics for Niemann-Pick disease type C1.
This acquisition expands Sucampo's pipeline, which until now has been largely focused around late-stage drugs for gastrointestinal disorders; lubristone for pediatric functional constipation in Phase 2 and Phase 3, and CPP-1X/sulindac in Phase 3 for familial adenomatous polyposis (FAP).
In mid-2016 it culled its lead drug, cobiprostone, after failure of a Phase 2a trial for the prevention of oral mucositis in patients that are undergoing radio chemotherapy for head and neck cancer and another in proton pump inhibitor (PPI)-refractory non-erosive reflux disease (NERD) or symptomatic gastroesophageal reflux disease (sGERD).
The two companies are going to establish a foundation to support research related to Niemann-Pick disease type C1. Vtesse’s equity holders have set aside a portion of the transaction proceeds, and Sucampo intends to match this from its corporate funds.
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