International Niemann–Pick Disease Alliance

An Exciting Announcement from Vtesse

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Exciting News for the NPC Community

An Exciting Announcement from Vtesse

I wanted to let you know that this morning, Vtesse announced an agreement to be acquired by Sucampo Pharmaceuticals—a global biopharmaceutical company that, like us, is focused on pursuing scientific innovation with a passion for improving the lives of patients.
This is an extremely important and positive step for us and we are excited about what is ahead alongside the Sucampo team. This represents an incredible opportunity for the patient community because it connects us with an established, global partner committed to furthering our mission of developing medicines that will benefit patients with extremely rare, life-threatening diseases, and more specifically Niemann-Pick Disease Type C.
With your support, we have advanced the science, achieved crucial regulatory designations, started up sites around the world and fully enrolled the clinical trial for VTS-270. But now we needed to build in certainty and remove the risks of having to continually seek additional financing to accomplish what we initially set out to do – study VTS-270 with the aim of obtaining approval.
Not only will Sucampo provide much needed capital and resources for our shared effort, but they also have the late-stage drug development expertise to help bring VTS-270 to patients. Sucampo’s management team has extensive experience dealing with life-threatening pediatric diseases and the company has a growing footprint in the pediatric and orphan disease space.
In Sucampo, we have found a team that provides us the capabilities and global reach to serve the NPC patient community worldwide and steer the VTS-270 pivotal trial to completion. Sucampo respects the NPC patient community and is committed to continuing and growing the partnership that Vtesse has started.
At the same time, I want to reassure you and your family that our Vtesse team will remain focused on addressing the needs of patients with NPC – and that you should still expect the same level of commitment from us that you have come to rely on. The teams working on the trial remain intact. Sucampo is clear that we are a better team together, and they are committed to supporting patients, their families, and doctors.

Several weeks ago, we reached a significant milestone by completing enrollment in the Phase 2b/3 clinical trial of VTS-270. We do not expect this development to cause our current clinical trial to change nor its progress to slow.
As we move forward with Sucampo, we will continue to maintain an open dialogue with you. You can find information on today’s announcement as well as additional information on Sucampo by visiting our website at Additionally, we will be holding town hall webinars. You will receive an invite for those shortly. If you have immediate questions, please do not hesitate to reach out to me at any time.
Maintaining the trust and transparency we’ve developed is a top priority at this time, and we truly believe this is the best outcome to help the community advance the science. As always, I thank you for your ongoing support, and look forward to your continued partnership as we work to make this much-needed treatment for NPC a reality.

Carrie Burke
Sr. Director, Advocacy


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