Vtesse starts phase 2b/3 clinical trial of VTS-270 to treat NPC disease

The company worked with the US and European regulators to carry out its pivotal study under one global protocol for evaluating safety and efficacy of VTS-270.

Vtesse will undertake the phase 2b/3 randomized, double-blind, sham-controlled trial in about 20 centers in the US and Europe.

About 51 patients will be enrolled to evaluate three different dose levels of VTS-270 versus sham-control in Part A of the study.

The company will use the findings to identify the dose level for Parts B and C.

All patients will receive treatment with VTS-270 in Part C open-label extension, until the time of regulatory decisions, Vtesse said.

Researchers at the National Institutes of Health (NIH) Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) have already carried out a Phase 1 study of VTS-270.

Vtesse said the phase 1 trial’s preliminary analysis indicate that the rate of disease progression had slowed down in children treated with VTS-270, when compared to the rate in an age- and disease severity-matched cohort obtained from a separate natural history study of NPC patients.

Vtesse president and CEO Ben Machielse said: "A key ingredient that enabled us to start the trial so quickly is the complete package of high-quality pre-clinical data that clearly outlines the potential benefits of VTS-270, such as prolonged survival and preservation of neurons, as well as encouraging clinical data from the Phase 1 trial.

"The pivotal trial will allow us to further evaluate safety and efficacy of VTS-270 to build a foundational dataset for the NPC community."

NPC affects an estimated one in 100,000 to 150,000 children and is misdiagnosed and/or under-diagnosed.