New real-world data research partnership between International Niemann-Pick Disease Registry and Mandos Health

The International Niemann-Pick Disease Registry and Mandos Health collaboration will see real-world data from the registry used in the evaluation of Mandos Health’s investigational product VTS-270 as a potential treatment for people with Niemann-Pick type C1.

The International Niemann-Pick Disease Registry (INPDR) and biopharmaceutical company Mandos Health have recently announced they are entering into a new partnership to deploy real-world evidence (RWE) to advance the treatment of Niemann-Pick disease type C1 (NPC1). The collaboration will provide Mandos Health access to INPDR’s database of real-world data (RWD) in order to evaluate the treatment effects of its NPC1 investigational product VTS-270, and fulfills INPDR’s mandate to use the registry data to support clinical research for the benefit of the Niemann-Pick community.

Conan Donnelly, INPDR CEO, said: “The INPDR is delighted to announce that registry data is working hard on behalf of the community to support clinical research. This collaboration with Mandos Health demonstrates that INPDR is serving the Niemann-Pick community in the way it was intended. It also supports our efforts to deepen understanding of Niemann-Pick diseases.”

Niemann-Pick disease type C is a life-limiting neurodegenerative lysosomal storage disorder that affects the body’s ability to metabolize cholesterol and lipids within cells. This ultra-rare genetic disease leads to an accumulation of lipids in the brain, nerves, liver, spleen, bone marrow and lungs, and there are currently no approved treatments.

The INPDR was founded in 2013 to collect RWD, allowing people with Niemann-Pick disease, their families and healthcare providers worldwide to record and share experiences. It is the largest database ever collected about the diagnosis, management, and progression of Niemann-Pick disease, encompassing types A, B and C. It features an anonymized Clinician- and Patient Reported Database, which enables patients to self-enrol online and contribute their data through a series of questionnaires including disease impact, health economics and quality of life. The INPDR database includes information relating to more than 300 patients with NPC1, across 20 clinical sites in 11 countries.

Mandos Health’s VP of Patient & Community Engagement, Cathy Traz, explained: “We are committed to working with the Niemann-Pick community to ensure we have every piece of high-quality evidence incorporated in our analysis of the treatment effects of VTS-270. We chose to partner with INPDR as it provides an unparalleled source of RWD that represents the Niemann-Pick type C1 community and it enables us to address open questions and have further discussions with regulators about the treatment effects of VTS-270 on this ultra-rare condition.”

“This work is only possible as patients, their families and clinicians have committed to participating in the registry, sharing important real-world information about diagnosis, management, and progression of Niemann-Pick diseases,” commented Niemann-Pick UK’s Chief Executive and INPDR Trustee, Toni Mathieson. “We would like to express our sincere gratitude to the community by showing how their valuable data is accelerating research in the field. We enter this partnership guided by our founding principles of integrity and independence, and our mission to advance research and improve health outcomes by accurately documenting the Niemann-Pick patient experience.”

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