Vtesse, Inc., a company committed to developing medicines to benefit patients with ultra rare, life-threatening diseases, announced that clinicians have dosed the first trial participants in Australia in the company's global, pivotal clinical trial of investigational drug VTS-270 for Niemann-Pick Type C1 disease (NPC).

Neuropsychiatrist Mark Walterfang, MBBS Hons, PhD, FRANZCP, Associate Professor at The Royal Melbourne Hospital, and clinical investigator in the VTS-270 trial, said: "We are honored to lead the way in bringing to Australia this important clinical study, through which we are evaluating the intrathecal use of VTS-270 in children with NPC. There is an urgent unmet global need for approved therapies that can slow progression or even halt the symptoms associated with NPC, and we are hopeful that VTS-270 can help to fill this need."

In addition to sites in Australia, clinical investigators are now enrolling patients with NPC in Vtesse's ongoing study at sites in the United States, Germany, the United Kingdom, France, Spain, and Turkey.  NPC is a progressive, irreversible, chronically debilitating – and ultimately lethal – genetic disease.

Expanding into Australia with our global clinical trial is an important milestone for Vtesse and the NPC community as we seek to determine the overall effectiveness and safety of VTS-270 and to reach our enrollment goals for the study," said Ben Machielse, Drs., president and chief executive officer of Vtesse, Inc. "We had not originally planned to include trial sites in Australia, but the concerted efforts of parent advocacy groups as well as Dr. Mark Walterfang at the Royal Melbourne Hospital and Dr. Michael Fahey at Monash Medical Centre convinced us to initiate multiple sites in this country. As exemplified in this situation, we listen to parents, academicians, researchers and clinicians and work to adapt our plans as needed to meet patient needs and advance the evaluation of VTS-270. We will evaluate options to make the drug available in due time for patients not eligible for the trial."

In November, Vtesse received two important regulatory designations for VTS-270. The United Kingdom's Medicines and Healthcare Products Regulatory Agency (MHRA) granted VTS-270 a Promising Innovative Medicine ("PIM") designation, considered the first step to qualifying for the UK's early access to medicines scheme (EAMS), which aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorisation when there is a clear unmet medical need. In addition, the US Food & Drug Administration (FDA) granted VTS-270 a Rare Pediatric Disease designation.

"We are thrilled to collaborate with Vtesse in bringing this much-needed trial to the NPC community in Australia," said Mandy Whitechurch, Founder and President of the Australian NPC Foundation. "Vtesse has been unwavering in its support, along with Royal Melbourne Hospital, in addressing the unmet needs of the NPC community, and we are excited to see this long-awaited initiative come to fruition. We are very proud of our work and the six years of perseverance it took to find Vtesse, who has been so willing to help us with these patients."

NPC 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) in close collaboration with parents and patient support groups, conducted the preclinical research and initiated the drug development phase for VTS-270. Vtesse is leading the late-stage drug development process for VTS-270.

Vtesse, Inc. is a rare disease company dedicated to developing drugs for patients suffering from diseases that are underserved. Vtesse is working collaboratively with the NIH, other leading academic centers, parents, and patient advocacy groups, to advance a pivotal clinical study of VTS-270 (a well-characterized mixture of HPßCD with a specific compositional fingerprint that distinguishes it from other HPßCD mixtures) to treat NPC, and to conduct pre-clinical discovery and development of other novel drugs for NPC and other lysosomal storage diseases (LSDs).