A new rare diseases company called Vtesse, backed by Pfizer among others, has been launched with $25 million in funding with the initial goal of developing a treatment for the lysosomal storage disorder Niemann-Pick Disease type C.

Vtesse is the first spin-off for Cydan Development, a US-based “orphan-drug accelerator” and the financing has been led by New Enterprise Associates, with participation from Pfizer Venture Investments, Lundbeckfond Ventures, Bay City Capital and Alexandria Venture Investments. The proceeds will be used to conduct trials of VTS-270, a formulation of cyclodextrin, for NPC, which affects one in 100,000-150,000 children.

Patients with NPC typically develop impaired coordination and movement (ataxia) and other neurological problems as a result of cholesterol buildup in their brain cells.

NIH collaboration

In tandem with the financing, Vtesse will support the ongoing Phase I trial being carried out by researchers from the National Institutes of Health who have been evaluating the safety of cyclodextrin. The latter will transfer its investigational New Drug Application to Vtesse along with all background data on the programme, and there will be a collaboration on a second VTS-270 study for NPC; they will also develop other novel drugs (delta-tocopherol and combinations) for NPC and other lysosomal storage disorders.

Vtesse chief executive Ben Machielse said “we expect to listen to and learn from the physicians, independent researchers, parents and patients who have worked tirelessly for many years to find a treatment for this devastating disorder”. He added that “we are grateful for the work they and the NIH have conducted thus far”, saying that with the spin-off, Vtesse hopes to rapidly advance VTS-270 “which we hope to make widely available to the many young patients suffering from the debilitating effects of NPC”.