Ocera has received Fast Track Designation and Orphan Drug Status and plans to initiate Phase 2 studies with OCR-002 in 2011.
SAN DIEGO, Dec. 15, 2010 /PRNewswire/ -- Ocera Therapeutics, Inc. announced today that it has completed two studies evaluating the safety and pharmacokinetics of OCR-002 (ornithine phenylacetate) which includes healthy volunteers and patients with liver cirrhosis. OCR-002 recently received Orphan Drug status and Fast Track designation by the United States Food and Drug Administration for the treatment of hyperammonemia (excessive ammonia levels) and resultant Hepatic encephalopathy. Patients with liver failure and decompensated cirrhosis may present with confusion and coma, a frequent complication known as acute hepatic encephalopathy (AHE) and an indicator of poor long-term survival.
Orphan Drug designation applies to a compound being developed to treat a rare medical condition. It offers a number of potential incentives, which may include a seven-year period of U.S. marketing exclusivity from the date of marketing authorization, funding for clinical studies, study design assistance, waiver of FDA user fees, and tax credits for clinical research. The Fast Track program is designed to facilitate the development and expedite the review of new drugs that are intended to treat serious or life-threatening conditions, and that demonstrate the potential to address unmet medical needs. Fast Track designated drugs often qualify for priority review, thereby expediting the FDA review process.
"Unlike current treatments that target the production of ammonia in the intestinal tract, OCR-002 directly reduces toxic levels of ammonia in the blood that lead to severe neurologic complications including coma. AHE is associated with substantial morbidity and mortality, and OCR-002 has the potential to be a novel therapeutic option for these patients," stated Dr. Laurent Fischer, CEO of Ocera Therapeutics. "Our receipt of Orphan Drug status and Fast Track designation for OCR-002 reinforce the clinical unmet need in AHE, and demonstrate the importance being placed on accelerating the development of drugs like this in order to reach the patients in need as quickly as possible." Ocera plans to initiate Phase 2 studies in 2011.
OCR-002 data presented at EASL, AASLD, and ISHEN in 2010 have confirmed that OCR-002 can consistently lower ammonia in multiple preclinical models of cirrhosis and acute liver failure and has been correlated with normalization of intracranial pressure, brain edema and neurologic function.
"OCR-002 is a promising new therapy for the acute care of patients with hyperammonemia and hepatic encephalopathy, a patient population for whom treatment options are currently very limited," stated Dr. Tarek Hassanein, Professor of Medicine and Director of Southern California Liver Centers, Coronado, CA.
OCR-002 (ornithine phenylacetate) is a novel injectable treatment with a unique mechanism of action that directly reduces toxic levels of ammonia in the blood. When the liver is unable to detoxify ammonia, patients are at risk of developing hepatic encephalopathy, a condition that can lead to coma and death, requiring hospitalization that cost the U.S. healthcare system $1.2 billion every year. The compound has demonstrated in several published studies that it has a rapid and sustained effect in lowering toxic ammonia levels and can prevent severe neurologic complications in animal models of acute liver failure and liver cirrhosis. Ocera Therapeutics licensed OCR-002 from University College of London in December 2008.
About Hepatic Encephalopathy
Hepatic Encephalopathy (HE) is caused by the accumulation of toxic substances in the bloodstream, including ammonia, that are normally removed by the liver. HE ranges in severity from disorientation to confusion, coma and even death. With severe liver impairment, toxic substances such as ammonia accumulate in the blood and impair the function of brain cells. In acute liver failure, hyperammonemia can lead to cerebral hypertension, which can cause brain edema and death and may require a liver transplant.
About Ocera Therapeutics, Inc.
Ocera Therapeutics, based in San Diego, California, USA, is a privately held biopharmaceutical company focused on the development and commercialization of proprietary compounds to treat liver diseases and gastrointestinal disorders. Ocera has raised $62.5 million dollars in venture financing from Domain Associates, Sofinnova Ventures, Thomas, McNerney & Partners, Greenspring Associates and InterWest Partners. Additional information on the company can be found at www.oceratherapeutics.com.
SOURCE Ocera Therapeutics, Inc.