International Niemann–Pick Disease Alliance

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

Vtesse, Inc. today announced that the first three patients have been screened for inclusion in its pivotal Phase 2b/3 clinical trial with VTS-270 for treatment of Niemann-Pick Type C1 Disease (NPC). This clinical trial follows a Phase 1 study conducted by researchers at the National Institutes of Health (NIH) Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).

Ben Machielse, Drs., President and Chief Executive Officer of Vtesse, Inc., said, “Starting the pivotal clinical trial for VTS-270 within nine months of the company’s launch underscores our dedication to serve the NPC community. 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.”

He added, “While initiation of the first site at Rush University Medical Center and the screening of three patients is a key milestone, we intend to open several sites in quick succession in the United States and the European Union to ensure availability of sites in close proximity to the patients who are eligible to enroll in the trial.”

For more information, you can view the full press release here.

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