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Columbia University Medical Center to Lead New Treatment Trial for Fatty Liver Disease

NEW YORK (Feb 1, 2012)

The Division of Pediatric Gastroenterology at Morgan Stanley Children's Hospital/Columbia University Medical Center is leading a new clinical trial of cysteamine bitartrate as a potential treatment of non-alcoholic steatohepatitis (NASH), an advanced form of non-alcoholic fatty liver disease.

Raptor Pharmaceutical Corp. announced the signing of a cooperative research and development agreement with the National Institute of Diabetes and Digestive and Kidney Diseases to conduct this Phase 2b clinical trial. The clinical trial will evaluate the safety and potential efficacy of RP104, Raptor's proprietary delayed-release tablet formulation of cysteamine bitartrate, as a potential treatment of non-alcoholic steatohepatitis in children. The clinical trial is expected to begin in the first calendar quarter of 2012.

Joel E. Lavine, M.D., Ph.D.
Joel E. Lavine, M.D., Ph.D.

"As obesity rates among children have increased so have obesity-related diseases, including diabetes, high blood pressure, and fatty liver disease," says Joel E. Lavine, M.D., Ph.D., Chief, Gastroenterology, Hepatology and Nutrition at Morgan Stanley Children's Hospital. "In fact, fatty liver disease is now recognized as the most common cause of liver disease among children in the United States. If left unrecognized and untreated, fatty liver disease can lead to significant liver injury. To more effectively manage patients with this condition and to meet demand for referrals, we have established a dedicated clinic at Morgan Stanley Children's Hospital for the study and management of children identified with this disease."

Morgan Stanley Children's Hospital is a member of the NASH Clinical Research Network, which is funded by the National Institutes of Health with the goal of better understanding and treating fatty liver disease. Dr. Lavine is a National Institutes of Health-funded principal investigator for studies related to treatment, natural history, epidemiology, etiopathogenesis and genetics of this condition and is the lead physician and pediatrician for a national 12-year study on fatty liver disease in children and adults. A randomized controlled trial of treatment of fatty liver in children was published this year in the Journal of the American Medical Association, with Dr. Lavine as the lead author. In addition, Dr. Lavine has been invited to deliver the State-of-the-Art Lecture at the 2012 annual meeting of the American Gastroenterological Association and the American Association for the Study of Liver Diseases.

The Cysteamine Bitartrate Delayed-Release for the Treatment of Non-alcoholic Fatty Liver Disease in Children (CyNCh) trial is expected to enroll a total of 160 pediatric participants at 10 U.S. centers in the NIDDK-sponsored NASH Clinical Research Network. Enrolled participants will range in age from 8 to 17 years and have biopsy-confirmed moderate to severe non-alcoholic fatty liver disease (NAFLD). The primary objective of this randomized, multicenter, double-blind, placebo-controlled clinical trial is to evaluate whether 52 weeks of treatment with RP104 in children reverses damage caused by NASH as measured by changes in NAFLD Activity Score (NAS), a histological rating scale of disease activity. Secondary endpoints will include blood markers for liver health, including alanine transaminase (ALT) and aspartate transaminase (AST), as well as safety and tolerability.

For more information about the new clinical trial or the Fatty Liver Disease Clinic at Morgan Stanley Children's Hospital, call (212) 305-NASH, (212) 305-5903, or e-mail us at fattyliver@columbia.edu.

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