Hyperion Therapeutics Announces Initiation of THRIVE Study, a Long-Term Registry of Patients With Urea Cycle Disorders

South San Francisco, Calif. -- 10/31/13
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Hyperion Therapeutics, Inc. (Nasdaq:HPTX) today announced that it has begun enrolling patients in its THRIVE study, a long term registry of patients with urea cycle disorders (UCDs) which will capture clinical outcomes and comparative effectiveness data.

The THRIVE study is a multi-center, prospective, non-interventional study designed to collect data in up to 500 UCD patients as part of their routine management. The study will track long-term outcomes, including hyperammonemic crises, ammonia levels, growth and development, and neurocognitive outcomes. Eligible patients will be enrolled in the study and followed for up to 10 years with annual interim data reports planned. At the time of enrollment, retrospective and baseline data will be collected. Data will also be collected during routine office visits. In addition, the study will capture the duration, dose, frequency, and route of administration of all current standard of care treatments, including ammonia-scavenging agents sodium phenylbutyrate, glycerol phenylbutyrate, or sodium benzoate. THRIVE offers the assistance of a Central Research Coordinating Office which facilitates participation and reduces the burden of study procedures on participating sites for all interested physicians and patients. Interested parties can visit the THRIVE website (www.thriveregistry.com) or contact thriveregistry@unitedbiosource.com to be included in the program.

"We are pleased to initiate this disease registry," said Bruce Scharschmidt, M.D., chief medical officer of Hyperion. "THRIVE meets one of our post marketing commitments to the FDA and will also contribute importantly to our understanding of UCDs and their management." "We have worked closely with the UCD community in designing this registry and are hopeful that it will help define best practices and potential improvements in management that will benefit patients both within and potentially outside the United States."

UCD patients lack enzymes or transporters necessary for the conversion of ammonia to urea and experience heightened levels of ammonia in the bloodstream. Left untreated, UCDs can result in neurological damage, coma, and/or death. Approved by the Food and Drug Administration on February 1, 2013, RAVICTI is an oral medication used for the long-term management of high blood levels of ammonia. In short term clinical studies involving more than 80 UCD patients ages two years and older, RAVICTI was safe and efficacious based on 24-hour ammonia profiles. In addition in long-term follow up studies, patients on RAVICTI maintained average fasting ammonia levels below the upper limit of normal. What makes RAVICTI unique from other ammonia removing medicines is that the active ingredient is released slowly in the body, mostly in the small intestine.

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