Vtesse, Inc., a company committed to developing drugs that will benefit patients with extremely rare, life-threatening diseases, announced that the first German patient has been dosed in its global, pivotal clinical trial of its investigational drug VTS-270 for Niemann-Pick Type C1 disease (NPC). VTS-270 is a well-characterized mixture of HPbCD with a specific compositional fingerprint that distinguishes it from other HPbCD mixtures.

Prof. Dr. Thorsten Marquardt, Head of Metabolic Disorders, University Hospital Münster, and the study’s lead principal investigator in Germany, noted: “The current clinical program with VTS-270 is aimed at treating the neurological disease that is the primary cause of mortality in children with NPC. Based on preclinical data suggesting that HPbCDs may not cross the blood brain barrier, it is important that we administer the drug through a lumbar puncture to increase the likelihood that the drug can address the neurological symptoms of NPC at doses that will be well tolerated.”

Prof. Dr. Julia B. Hennermann, Head of the Working Group for Lysosomal Storage Diseases at the University Children's Hospital Mainz (known as Villa Metabolica) at the Johannes Gutenberg University Medical Center, dosed the first German patient in the trial. “I’m very happy to be part of this important clinical study, through which we are evaluating the intrathecal use of VTS-270 in children with NPC,” she said. “There is a desperate need for more approved therapies that can arrest the neurological disease associated with NPC. As a clinician and based upon the encouraging Phase 1 clinical data, I am hopeful that VTS-270 can fill this need.”

Outside of the United States and Germany, clinical investigators have enrolled and continue to enroll patients at sites in the United Kingdom, France, Spain, and Turkey in the phase 2b/3 clinical trial of VTS-270. Vtesse expects to have clinical sites available in Australia soon.

“The Vtesse team is committed to conducting this clinical trial at numerous sites around the world, which is critical for the development of VTS-270. We want to make clinical sites easily reachable for the patients who wish to participate in the trial, and are supporting the patients who choose to travel to our clinical sites,” said Ben Machielse, Drs., president and chief executive officer of Vtesse, Inc.  “To address the unmet needs of patients with NPC, which is an ultra-orphan disease, and to further advance science around this drug candidate, we look forward to moving towards the final phase of this clinical trial.”

Vtesse’s ongoing phase 2b/3 prospective, randomized, double-blind, sham-controlled trial of its investigational drug VTS-270 is being conducted in patients affected by NPC disease. It is a three-part efficacy and safety trial of VTS-270, administered by the intrathecal route (IT) every two weeks, with planned enrollment of approximately 51 patients. Intrathecal route of delivery is thought to be important to ameliorating the neurological disease based on studies in pre-clinical models of NPC disease. Phase 1/2 clinical trial data from 14 patients with NPC were presented earlier this year at the 2016 World Symposium on Lysosomal Storage Disease in San Diego, California.

In January 2016, Vtesse announced that the US Food & Drug Administration (FDA) had granted Breakthrough Therapy designation status for VTS-270 for treatment of NPC. Both the FDA and the European Medicines Agency (EMA) had previously granted Orphan Drug status to VTS-270.  

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. The NIH’s National Center for Advancing Translational Sciences (NCATS) and Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) conducted the preclinical research and initiated the drug development phase for VTS-270 in close collaboration with parents and patient support groups. Vtesse is leading the late-stage drug development process.

Vtesse, Inc. is a rare disease company dedicated to developing drugs for patients suffering from diseases that are underserved.