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"Completing enrollment in this Phase 1/2 study is an important milestonein our development program," said Bruce Scharschmidt, Chief Medical Officer ofHyperion Therapeutics. "This represents the first trial of HPN-100 in patientswith UCDs and the results will be important in assessing its potential toimprove care for these patients."
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About Urea Cycle Disorder
UCDs are inherited, inborn errors of metabolism present in an estimated 1in 10,000 births in the US. Patients with UCDs lack or are deficient in oneof the key enzymes that comprise the urea cycle, the body's primary vehiclefor removing ammonia, a potent neurotoxin, from the bloodstream. UCDs oftenpresent in the neonatal period, but onset can occur at any age depending onthe severity of the disorder. Left untreated, UCDs can cause dangerouslyheightened levels of ammonia in the bloodstream (hyperammonemia) resulting inbrain damage, coma, and/or death.
About HPN-100
HPN-100 is a pro-drug of phenylbutryrate and a pre-pro-drug ofphenylacetic acid (PAA), the active moiety of BUPHENYL(R), the only therapycurrently FDA-approved as adjunctive therapy for the chronic management ofpatients with the most prevalent urea cycle disorders -- carbamylphosphatesynthetase (CPS), ornithine transcarbamylase (OTC), and argininosuccinic acidsynthetase (AS). HPN-100, which is dosed orally in liquid form, provides analternative pathway to the urea cycle for the disposal of waste nitrogenthrough the renal excretion of phenylacetylglutamine, which is formed from PAAand glutamine.
About BUPHENYL(R)
BUPHENYL(R) is indicated as adjunctive therapy in the chronic managementof patients with urea cycle disorders involving deficiencies ofcarbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), orargininosuccinic acid synthetase (AS). BUPHENYL(R) should not be administeredto patients with known hypersensitivity to sodium phenylbutyrate or anycomponent of this preparation. The most common adverse reactions associatedwith BUPHENYL(R) were amenorrhea dysfunction, decreased appetite, body odor(probably caused by its metabolite phenylacetate) and bad taste or tasteaversion. Patients with urea cycle disorders should not take valproic acid,haloperidol, or steroids as these drugs have been reported to increase bloodammonia levels, and probenecid may affect the kidneys' excretion. Use withgreat care, if at all, in patients with congestive heart failure or severerenal insufficiency, and in clinical states where there is sodium retentionwith edema. Use caution when administering to patients with hepatic or renalinsufficiency or inborn errors of beta oxidation. The safety or efficacy ofdoses in excess of 20 grams (40 tablets) per day has not been established.
About Hyperion Therapeutics
Hyperion Therapeutics is a specialty therapeutics company focused on thedevelopment of therapies that address orphan or underserved patientpopulations with unmet medical needs. Hyperion is headquartered in South SanFrancisco, CA. For additional information, visit: http://www.hyperiontx.com.
BUPHENYL is a registered trademark of Ucyclyd Pharma, Inc.
Full Prescribing Information for BUPHENYL(R) is available athttp://www.Buphenyl.com or by contacting Ucyclyd Pharma, Inc.
SOURCE Hyperion Therapeutics, Inc.
