Navigation Links
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

SOUTH SAN FRANCISCO, Calif., Nov. 12 /PRNewswire/ -- Hyperion Therapeutics, Inc. today reported completion of data analysis for its Phase 2 clinical trial designed to evaluate the safety, tolerability and ammonia scavenging effects of HPN-100 versus BUPHENYL(R) (sodium phenylbutyrate), the current standard of care in patients with urea cycle disorders. Data from the trial will be presented at an upcoming medical conference.


"We are encouraged by this trial and the potential to develop a new therapy for patients with urea cycle disorders," said Dr. Bruce Scharschmidt, Senior Vice President and Chief Medical Officer of Hyperion Therapeutics, Inc. "Based on the results, we plan to meet with the Food and Drug Administration in early 2009 to discuss our Phase 3 development program."

"We are pleased with the progress of the HPN-100 clinical program," said Cindy LeMons, Executive Director of the National Urea Cycle Disorders Foundation (NUCDF). "The urea cycle disorder community encourages the development of treatment alternatives."

Hyperion and Ucyclyd Pharma, Inc., a subsidiary of Medicis Pharmaceutical Corporation, entered into a collaboration agreement for HPN-100 in August 2007. Under the terms of the agreement, Hyperion is conducting ongoing research and development of HPN-100 for urea cycle disorders, hepatic encephalopathy, and other forms of hyperammonemia.

About Urea Cycle Disorder

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 HPN-100

HPN-100 is a pro-drug of phenylbutyrate and a pre-pro-drug of phenylacetic acid, 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 deficiencies including those related to carbamylphosphate synthetase, ornithine transcarbamylase and argininosuccinic acid synthetase. HPN-100, which is dosed orally in liquid form, provides an alternative pathway to the urea cycle for the disposal of waste nitrogen through the renal excretion of phenylacetylglutamine, which is formed from phenylacetic acid and glutamine.


BUPHENYL(R) 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(R) should not be administered to patients with known hypersensitivity to sodium phenylbutyrate or any component of this preparation. The most common adverse reactions associated with BUPHENYL(R) were amenorrhea dysfunction, decreased appetite, body odor (probably caused by its metabolite phenylacetate) and bad taste or taste aversion. Patients with urea cycle disorders should not take valproic acid, haloperidol, or steroids as these drugs have been reported to increase blood ammonia levels, and probenecid may affect the kidneys' excretion. Use with great care, if at all, in patients with congestive heart failure or severe renal insufficiency, and in clinical states where there is sodium retention with edema. Use caution when administering to patients with hepatic or renal insufficiency or inborn errors of beta oxidation. The safety or efficacy of doses in excess of 20 grams (40 tablets) per day has not been established.


The National Urea Cycle Disorders Foundation is a nationally-recognized nonprofit organization dedicated to saving children and adults from the catastrophic effects of urea cycle disorders. The Foundation is a lifeline to families and medical professionals all over the world desperately seeking crisis intervention and treatment information. The Foundation's mission is to provide information, guidance and support to patients and their families, to educate healthcare professionals on the identification, diagnosis and treatment of UCDs, to raise awareness so that no child or adult ever goes undiagnosed, and to stimulate and support critical research activities and a CURE. The Foundation is headquartered in Pasadena, California. For more information, visit, or call (626) 578-0833.

About Hyperion Therapeutics

Hyperion Therapeutics is a privately held specialty therapeutics company focused on the development of therapies that address critical unmet needs and underserved patient populations in the areas of gastroenterology and hepatology. Hyperion is headquartered in South San Francisco, CA. For additional information, visit:

SOURCE Hyperion Therapeutics, Inc.
Copyright©2008 PR Newswire.
All rights reserved

Related medicine news :

1. Hyperion Therapeutics Elects Lota Zoth to Board of Directors
2. Hyperion Therapeutics Announces Enrollment of First Patient in Phase 2 Clinical Trial in Patients With Hepatic Encephalopathy
3. Transition Therapeutics Announces First Quarter Fiscal 2009 Financial Results
4. Duska Therapeutics and DSM Pharmaceuticals in Manufacturing Collaboration for PSVT Drug Product
5. Transition Therapeutics to Hold Conference Call on First Quarter Fiscal 2009 Financial Results on Monday, November 10th, at 4:30 P.M. EST
6. Nektar Therapeutics Repurchases $100 Million of Convertible Debt and Reports Third Quarter 2008 Financial Results
7. Cell Therapeutics, Inc. (CTI) Rodman & Renshaw Healthcare Conference Presentation to be Webcast
8. Cornerstone Therapeutics Announces New Board of Directors
9. Orexigen(R) Therapeutics Announces Webcast of Third Quarter Financial Results on November 6, 2008
10. Cornerstone BioPharma Announces Completion of Merger with Critical Therapeutics
11. Cell Therapeutics, Inc. (CTI) Oppenheimer & Co. Healthcare Conference Presentation to be Webcast
Post Your Comments:
(Date:6/26/2016)... Aliso Viejo, California (PRWEB) , ... June 26, 2016 , ... ... for Final Cut Pro X. , "Film editors can give their videos a whole ... artistically," said Christina Austin - CEO of Pixel Film Studios. , ProSlice Levels ...
(Date:6/26/2016)... ... June 26, 2016 , ... Many women ... diagnosed with endometriosis. These women need a treatment plan to not only alleviate ... that can help for preservation of fertility and ultimately achieving a pregnancy. The ...
(Date:6/25/2016)... Miami, FL (PRWEB) , ... June 25, 2016 , ... The temporary closing of Bruton ... Plant City Observer , brings up a new, often overlooked aspect of head lice: ... The closing for fumigation is not a common occurrence, but a necessary one in the ...
(Date:6/25/2016)... ... June 25, 2016 , ... As a lifelong Southern Californian, Dr. Omkar ... M.D from the David Geffen School of Medicine at UCLA. He trained in Internal ... complete his fellowship in hematology/oncology at the UCLA-Olive View-Cedars Sinai program where he had ...
(Date:6/25/2016)... ... June 25, 2016 , ... Conventional wisdom preaches the benefits of moderation, ... the latter, setting the bar too high can result in disappointment, perhaps even self-loathing. ... toward their goal. , Research from reveals that behind the ...
Breaking Medicine News(10 mins):
(Date:6/23/2016)... Research and Markets has announced the addition of ... Forecast to 2022" report to their offering. ... date financial data derived from varied research sources to present ... impact on the market during the next five years, including ... sub markets, regional and country level analysis. The report provides ...
(Date:6/23/2016)... , June 23, 2016 Research ... Pharma News Issue 52" report to their offering. ... in influenza treatment creates a favourable commercial environment for MedImmune ... growing patient base that will serve to drive considerable growth ... vaccine would serve to cap sales considerably, but development is ...
(Date:6/23/2016)... , June 23, 2016 , ... Thursday, July 7, 2016 , , , , LOCATION: , ... , , , , EXPERT PANELISTS:  , , , Frost ... Industry Analyst, Christi Bird; Senior Industry Analyst, Divyaa Ravishankar and Unmesh ... The global pharmaceutical industry is witnessing an exceptional era. Several ...
Breaking Medicine Technology: