Cytonet LLC Enrolls Patients Into Ongoing U.S. Clinical Trial Evaluating Liver Cell Therapy For Severe Urea Cycle Disorders In Pediatric Population

SALT LAKE CITY & RALEIGH-DURHAM, N.C.--(BUSINESS WIRE)--Cytonet announced today that two new pediatric patients were added in the past four months to the company’s clinical trial of its investigational liver cell therapy (LCT) for the treatment of Urea Cycle Disorders (UCD). The announcement came during the 38th Annual Meeting of the Society for Inherited Metabolic Disorders (SIMD), being held in Salt Lake City, Utah, March 28-31, 2015.

“Longterm Outcome in 3 Children With Urea Cycle Disorder After Hepatocyte Transplantation”

“We continue to actively enroll pediatric patients into this promising study to further evaluate the potential for liver cell therapy to stabilize infants with UCD while they await liver transplantation,” said Sukru H. Emre, M.D., principal investigator and Professor of Surgery and Pediatrics at Yale University School of Medicine where the two new patients were enrolled. “We are greatly encouraged by trial results to date.”

The addition of these two new patients brings the total number of participants enrolled in the multicenter trial being conducted in the U.S. and Canada (CCD05) to 10 since the study began in 2010.

An update on Cytonet’s LCT research is being presented during the SIMD meeting in a poster entitled “Longterm Outcome in 3 Children With Urea Cycle Disorder After Hepatocyte Transplantation,” authored by Aneal Khan, M.D., Assistant Professor of Medical Genetics and Pediatrics at the University of Calgary and Alberta Children’s Hospital in Calgary, Alberta. The poster follows data presented at the July, 2014 World Transplant Congress indicating that liver cell therapy may be an effective tool for treating children with severe UCD until liver transplantation is able to be performed.

Liver cell therapy involves using healthy cells from donated livers that are not used for organ transplantation. These cells are infused into the portal vein in six sessions on six consecutive days. In December 2013, Cytonet submitted a Marketing Authorization Application to the European Medicines Agency seeking approval for its liver cell therapy for the treatment of Urea Cycle Disorders in children.

ABOUT UREA CYCLE DISORDERS

According to the National Urea Cycle Disorders Foundation (NUCDF), Urea Cycle Disorders comprise a group of genetic disorders leading to a deficiency of one of the five enzymes in the urea cycle which is responsible for removing ammonium from the blood stream. These include carbamoyl phosphate synthetase I (CPS I) deficiency, ornithine transcarbamylase (OTC) deficiency, argininosuccinate synthetase (ASS) deficiency (which is also known as citrullinemia), argininosuccinate lyase (ASL) deficiency and arginase 1 deficiency (hyperargininemia), and in addition the co-factor N-acetylglutamate synthetase (NAGS) deficiency. The urea cycle involves a series of biochemical steps in which nitrogen, a waste product of protein metabolism, is removed from the blood and converted to urea. Normally, the urea is transferred into the urine and removed from the body. In patients with Urea Cycle Disorders, the nitrogen accumulates in the form of ammonium, a highly toxic substance, and is not removed from the body, resulting in hyperammonemia (elevated blood ammonium). Ammonium then reaches the brain through the blood, where it may cause irreversible brain damage and, in very severe cases, coma and/or death.

Urea Cycle Disorders are included in the category of inborn errors of metabolism and may cause brain damage and death among newborns and infants.

ABOUT CYTONET

Cytonet is an international biotechnology company which is located in Weinheim and Heidelberg in Germany and in Durham, N.C. in the U.S. The Company develops and produces liver cell products for therapeutic purposes. Cytonet’s goal is to provide alternatives to existing therapies for many diseases, with a particular emphasis on liver diseases. Cytonet is a pioneer and leader in the field of regenerative medicine. For the past several years, Cytonet has worked with internationally-leading metabolic and neonatal centers to study its liver cell therapy for infusion to treat Urea Cycle Disorders in children, using healthy and metabolically functional human liver cells collected from donated livers not used for transplant. For more information, please visit Cytonet’s website at http://www.cytonetllc.com/