Vtesse, Inc. Initiates Phase 2b/3 Clinical Trial of VTS-270 for Treatment of Niemann-Pick Type C1 (NPC) Disease

NPC is a progressive, irreversible, chronically debilitating – and ultimately lethal – genetic disease. It is caused by a defect in lipid transportation within the cell, which leads to excessive accumulation of lipids in the brain, liver and spleen. VTS-270, has shown promise in pre-clinical and clinical studies as a potential treatment for NPC. It is a well-characterized mixture of (2-hydroxypropyl)-beta-cyclodextrin that has been extensively evaluated in pre-clinical and clinical studies at the National Institutes of Health, as well as under individual compassionate-use Investigational New Drug applications (iINDs) and in other academic labs. Most recently, Vtesse announced positive clinical results from the on-going Phase 1 study that demonstrated VTS-270 stabilizes the overall measures of NPC while improving certain disease measures. NPC is a genetic disease affecting an estimated one in 100,000 to 150,000 children and is often misdiagnosed and/or under-diagnosed. Affected patients are usually identified in early childhood with ataxia, exhibit progressive impairment of motor and intellectual function, and often die before adulthood.Vtesse has worked extensively with regulators in the United States and Europe with the goal of conducting its pivotal study under one global protocol that will evaluate safety and efficacy of VTS-270 to support approval of the drug by regulatory agencies in both regions.

Vtesse’s Phase 2b/3 prospective, randomized, double-blind, sham-controlled trial of VTS-270 will be conducted in patients affected by NPC disease. The trial will take place in up to 20 centers in the United States and Europe. It is a three-part, efficacy and safety trial of VTS-270, administered by the lumbar intrathecal route (IT) every two weeks, with a planned enrollment of approximately 51 patients (in Parts A and B). In Part A of the study, researchers will evaluate three different dose levels of VTS-270 versus sham-control to determine the dose level for Parts B and C. All participants in the pivotal trial will be eligible to receive treatment with VTS-270 in Part C, the open-label extension, until the time of regulatory decisions.

Preliminary analyses of the Phase 1 trial, conducted post-hoc, suggest that the rate of disease progression had slowed down (based on a standardized measure) in children treated with VTS-270 as compared to the rate in an age- and disease severity-matched cohort obtained from a separate natural history study of NPC patients.

More information on this Phase 2b/3 study can be found at Vtesse’s clinical trial website: www.theNPCstudy.com. Vtesse launched the website this summer as a resource to patients, parents, caregivers and physicians.  If you would like to contact Vtesse directly, please reach out to Carrie Burke at carrie@vtessepharma.com or 240-801-9268.