Press Releases
Hyperion Therapeutics Enrolls First Patient in Phase II Hepatic Encephalopathy Trial
South San Francisco, California - December 3, 2009Hyperion Therapeutics today announced that the first patient has been enrolled in its phase II clinical trial of investigational compound HPN-100 (glycerol phenylbutyrate). The multi-center study consists of an open label safety component followed by a randomized, double-blind, placebo-controlled study that will enroll approximately 140 patients with episodic hepatic encephalopathy in the US and Eastern Europe. The primary efficacy measure will be the proportion of patients who exhibit a hepatic encephalopathy episode during the four-month treatment period.
About Hepatic Encephalopathy
Hepatic encephalopathy (HE) is a serious but potentially reversible neurological disorder that can occur in patients with cirrhosis of any etiology or acute liver failure. HE 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. Based on the current epidemiological literature, Hyperion estimates that there are approximately one million1,2 patients in the US with cirrhosis, of whom approximately 140,000 have overt HE. There are no therapies currently FDA-approved for the treatment of HE.
About HPN-100 (glycerol phenylbutyrate)
HPN-100 (glycerol phenylbutyrate), an investigational product, is a pre-pro-drug of phenylacetic acid, the active moiety of BUPHENYL® (sodium phenylbutyrate) Tablets and Powder, 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) deficiencies. Glycerol phenylbutyrate is administered orally in liquid form. 17.4 mL of glycerol phenylbutyrate (~ 3.5 teaspoons) delivers the same amount of active ingredient as the maximum daily dose of BUPHENYL (forty tablets or 6.67 teaspoons of powder mixed with food or dissolved in liquid). Glycerol phenylbutyrate holds orphan product designations from the FDA for the maintenance treatment of patients with enzymes of the urea cycle and for the intermittent or chronic treatment of patients with cirrhosis and any grade of hepatic encephalopathy.
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 adverse reactions associated with BUPHENYL 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.
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 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. Hyperion is headquartered in South San Francisco, CA. For additional information, visit www.hyperiontx.com .
BUPHENYL is a registered trademark of Ucyclyd Pharma, Inc.
Full Prescribing Information for BUPHENYL is available at www.Buphenyl.com or by contacting Ucyclyd Pharma, Inc.
1Bell BP, Manos MM, Zaman A, et al. The epidemiology of newly diagnosed chronic liver disease in gastroenterology practices in the United States : results from population-based surveillance. Am J Gastroenterol 2008; 103:2727-2735.
2 Dufour MC. Chronic liver disease and cirrhosis. In digestive diseases in the United States : epidemiology and impact. JE Everhart, Editor, 1994; NIH publication No. 94-1447:615-646.
Press contact:
Christine Nash
Hyperion Therapeutics, Inc.
650-745-7844