Hyperion Therapeutics Completes Enrollment in Phase 1/2 Clinical Trial in Patients With Urea Cycle...( SOUTH SAN FRANCISCO Calif. July 2...),Health

SOUTH SAN FRANCISCO, Calif., July 22 /PRNewswire/ -- Hyperion Therapeutics, Inc. today announced that it has completed patient enrollment in its Phase 1/2 clinical trial to evaluate the safety, tolerability and ammonia scavenging effects of HPN-100 (formally called GT4P) versus BUPHENYL(R) (sodium phenylbutyrate) in patients with urea cycle disorders (UCD). The company plans to announce top-line results in the fourth quarter of this year.

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"Completing enrollment in this Phase 1/2 study is an important milestone in our development program," said Bruce Scharschmidt, Chief Medical Officer of Hyperion Therapeutics. "This represents the first trial of HPN-100 in patients with UCDs and the results will be important in assessing its potential to improve care for these patients."

About Urea Cycle Disorder

UCDs are inherited, inborn errors of metabolism present in an estimated 1 in 10,000 births in the US. Patients with UCDs lack or 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. UCDs often present in the neonatal period, but onset can occur at any age depending on the severity of the disorder. Left untreated, UCDs can cause dangerously heightened levels of ammonia in the bloodstream (hyperammonemia) resulting in brain damage, coma, and/or death.

About HPN-100

HPN-100 is a pro-drug of phenylbutryrate and a pre-pro-drug of phenylacetic acid (PAA), the active moiety of BUPHENYL(R), the only therapy currently FDA-approved as adjunctive therapy for the chronic management of patients with the most prevalent urea cycle disorders -- carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), and argininosuccinic acid synthetase (AS). HPN-100, which is dosed orally in
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