Ammonia Scavenger Ornithine Phenylacetate (OCR-002) Granted Orphan Disease and Fast Track Status
PALO ALTO, Calif., Jan. 8, 2014 (GLOBE NEWSWIRE) -- Ocera Therapeutics (Nasdaq:OCRX) today announced enrollment of the first patient in its multi-center Phase 2b study, the "STOP-HE" or OCR002-HE209 trial. This study is evaluating the efficacy, safety and pharmacokinetics of OCR-002, or ornithine phenylacetate, in hospitalized patients with liver cirrhosis and an acute episode of hepatic encephalopathy (HE). HE is a serious complication of liver failure and decompensated cirrhosis in the setting of chronic liver disease, where elevation of ammonia concentration in the blood and brain can lead to deterioration of neurocognitive function, ranging from subtle abnormalities to frank (overt) disorientation, stupor, coma and death. OCR-002 is a novel molecule, an ammonia scavenger which is intended to rapidly reduce the concentration of ammonia in the blood when administered as a continuous intravenous infusion. OCR-002 has been granted orphan drug designation by the US Food and Drug Administration (FDA) for the treatment of hyperammonemia and resultant HE in patients with acute liver failure or acute on chronic liver disease.
"HE is a serious disorder that leads to approximately 150,000 hospitalizations per year in the United States and for which the best available treatments are still suboptimal. We need better and more effective treatments," said Robert Brown M.D., Frank Cardile professor of medicine and pediatrics and director, Center for Liver Disease and Transplantation, Columbia University and a principal investigator in the Ocera Phase 2b study.
The study is a randomized double-blind, controlled study of OCR-002 plus current standard of care versus standard of care alone. The study will enroll 200 patients at approximately 40 centers in the United States and Canada. The trial will assess time to achievement of meaningful improvement in HE symptoms. Ocera anticipates completing the study in early 2015.
"We are excited to begin enrolling patients in the Phase 2b study," said Linda Grais, M.D., J.D., CEO of Ocera. "The number of hospitalizations for HE has been increasing steadily over the past decade and we are eager to help these seriously ill patients."
About Hepatic Encephalopathy
Hepatic encephalopathy (HE) is a complication of patients with liver cirrhosis, acute liver failure and acute liver injury. When the liver is no longer able to function normally, there is an accumulation of ammonia in the blood because usual metabolic pathways are impaired. Ammonia elevation impairs brain cell function, and can lead to overt neurocognitive impairment that frequently occurs as acute episodes in the setting of cirrhosis following the occurrence of an inciting factor, such as gastrointestinal bleeding or infection. An acute overt episode of HE is marked by impaired cognition, lethargy, disorientation and a decreased level of consciousness. HE can lead to coma and death due to brain swelling. There are no current drug treatments on the market in the United States for HE that can be given intravenously to hospitalized patients with an acute HE episode.
Ocera Therapeutics, Inc. is a clinical stage biopharmaceutical company focused on the development and commercialization of OCR-002 (ornithine phenylacetate). OCR-002 is an ammonia scavenger which has been granted Orphan Disease and Fast Track status from the FDA to treat hyperammonemia and associated hepatic encephalopathy in patients with liver cirrhosis, acute liver failure and acute liver injury. For additional information, please see www.ocerainc.com.
Forward Looking Statements
This press release contains "forward-looking" statements, including, without limitation, all statements related to the OCR-002 clinical development program, including patient enrollment estimates, expected timing for the receipt of clinical data, and the potential success of OCR-002 in clinical trials. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believe," "expected," "hope," "plan," "potential," "will" and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Ocera's current expectations. Forward-looking statements involve risks and uncertainties and Ocera's actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, including those risks and uncertainties discussed under the heading "Risk Factors" in Ocera's Quarterly Report on Form 10-Q for the quarter ended September 30, 2013 filed on November 14, 2013, as well as other risks detailed in Ocera's subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Ocera undertakes no duty to update this information unless required by law.
Ocera Therapeutics, Inc.