CTI Clinical Trial and Consulting Services Announces Collaboration With Vtesse, Inc. on Novel Phase 2b/3 Clinical Trial for Treatment of Niemann-Pick Type C1 (NPC) Disease

FOR IMMEDIATE RELEASE [Cincinnati, OH ~ October 20, 2015] CTI Clinical Trial and Consulting Services (CTI), a multi-national, privately held, full-service contract research organization, announces its collaboration with Vtesse, Inc. on a novel Phase 2b/3 clinical trial for the treatment of Niemann-Pick Type C1 (NPC) Disease.

NPC is a genetic disease affecting an estimated one in 100,000 to 150,000 children who typically exhibit progressive impairment of motor and intellectual function, and often die before adulthood. Ground-breaking work in animal models of the disease at multiple academic institutions have highlighted the ability of VTS-270 to preserve neuronal function and significantly improve survival.  Preliminary analyses of the Phase 1 trial 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.

Vtesse's Phase 2b/3 prospective, randomized, double-blind, sham-controlled trial of VTS-270 is being conducted in patients affected by NPC disease. The trial, which CTI is managing with Vtesse, 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.

“We’re thrilled to be partners with Vtesse and the entire NPC community in this effort to develop a drug that may be approved to treat this devastating disease,” according to Timothy J. Schroeder, CTI Chief Executive Officer. “CTI is a leader in rare disease research, with nearly 80% of our work focusing on these unique and often challenging diseases. We work every day to support life-changing therapies that will help patients suffering from illnesses like Niemann-Pick around the world, and are excited to collaborate with such innovative and talented companies that share our mission, such as Vtesse.”

“Because CTI Clinical Trial and Consulting Services is a leader in rare disease research, the organization is an ideal collaborator for the Vtesse team as we conduct our pivotal trial of VTS-270 in Niemann-Pick Disease Type C,” said Ben Machielse, Vtesse Chief Executive Officer. “We anticipate working closely with CTI as we execute flawlessly in this trial with a goal of further evaluating the safety and efficacy of VTS-270 to build a foundational dataset for the NPC community.”

About CTI Clinical Trial and Consulting Services

CTI Clinical Trial and Consulting Services is a global, privately held, full-service contract research organization (CRO), delivering a complete spectrum of clinical trial and consulting services throughout the lifecycle of development, from concept to commercialization. CTI’s focused therapeutic approach provides pharmaceutical, biotechnology, and medical device firms with clinical and disease area expertise in rare diseases, regenerative medicine/gene therapy, immunology, transplantation, nephrology, hematology/oncology, neurology, infectious diseases, hepatology, cardiopulmonary, and pediatric populations. CTI also offers a fully integrated multi-specialty clinical research site that conducts phase I-IV trials. CTI has a passion for helping life-changing therapies succeed in chronically and critically ill patient populations. With clinical trial experience across 6 continents, CTI partners with research sites, patients, and sponsors to fulfill unmet medical needs. CTI is headquartered in Cincinnati, OH, with operations across North America, Europe, Latin America, and Asia-Pacific. For more information visit www.ctifacts.com

About Vtesse

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 and other leading academic centers to advance clinical study of VTS-270 for NPC, and to conduct pre-clinical discovery and development of other novel drugs for NPC and other lysosomal storage diseases (LSDs). Vtesse is led by a highly experienced management team that has been involved in the development of more than 20 approved drugs and vaccines. Its experienced consortium of investors, led by New Enterprise Associates, has committed initial funding that is expected to bring this compound through pivotal clinical trial. Vtesse is based in Gaithersburg, Maryland and is the first spin-out company from Cydan Development, Inc. For more information, visit www.vtessepharma.com. More information on this Phase 2b/3 study can be found at Vtesse's clinical trial website.

Original Press Release