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Hyperion Therapeutics Raises $60 Million Series C
Date:6/30/2009

nic acid synthetase (AS) deficiencies. HPN-100, which is dosed orally in liquid form, is under clinical investigation as providing a potential alternative pathway to the urea cycle for the disposal of ammonia through the renal excretion of phenylacetylglutamine, which is formed from phenylacetic acid and glutamine.

About Urea Cycle Disorders

Urea cycle disorders are inherited, inborn errors of metabolism present in an estimated 1 in 10,000 births in the United States. Patients with urea cycle disorders are deficient in one of the key enzymes that comprise the urea cycle, the body's primary vehicle for removing ammonia, a potent neurotoxin, from the bloodstream. Onset may occur at any age depending on the severity of the disorder. Left untreated, urea cycle disorders can cause dangerously heightened levels of ammonia in the bloodstream (hyperammonemia) resulting in brain damage, coma, and/or death.

About Hepatic Encephalopathy

Hepatic encephalopathy (HE) is a serious but potentially reversible neurological disorder that can occur in patients with acute liver failure and, most commonly, in patients with cirrhosis of any etiology. It comprises a spectrum of neurological signs and symptoms ranging from mild (e.g. minimal disorientation) to severe (e.g. coma, death) and is believed to occur when the brain is exposed to gut-derived toxins such as ammonia that are normally removed from the blood by a healthy liver. There are no therapies currently FDA-approved for the treatment of HE.

About BUPHENYL

BUPHENYL is indicated as adjunctive therapy in the chronic management of patients with urea cycle disorders involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). BUPHENYL should not be administered to patients with known hypersensitivity to sodium phenylbutyrate or any component of this preparation. The most common advers
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SOURCE Hyperion Therapeutics, Inc.
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2. Hyperion Therapeutics Completes Phase 2 Data Analysis and Schedules End of Phase 2 Meeting With FDA for HPN-100 in the Chronic Treatment of Urea Cycle Disorders
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