The Acute Liver Failure Study Group Initiates Phase 2a Study of OCR-002 for Treatment of Acute Liver Failure after Acetaminophen Overdose

SAN DIEGO, Nov. 8, 2012 /PRNewswire/ -- Ocera Therapeutics, Inc. announced that the first patient has enrolled in a Phase 2a study to evaluate the safety and tolerability of OCR-002 (ornithine phenylacetate) in the treatment of patients with acute liver injury (ALI) as well as those that have progressed to acute liver failure (ALF) following acetaminophen overdose.

OCR-002 is an investigational drug designed to directly reduce circulating blood levels of ammonia which, at high levels, is neurotoxic, associated with brain injury, and may lead to hepatic encephalopathy and mortality. The investigator-sponsored study, referred to as STOP-ALF, is being conducted by The Acute Liver Failure Study Group, a National Institutes of Health-funded consortium led by UT Southwestern Medical Center.

"When a patient presents with acute liver failure, physicians have no medicines available that can directly lower circulating levels of ammonia, which drive hepatic encephalopathy and can lead to coma and death," stated William M. Lee, M.D., Lead Investigator, and Professor, Internal Medicine, UT Southwestern.  "OCR-002 acts specifically by scavenging for and removing ammonia from the blood, which may prevent the development of hepatic encephalopathy in patients at risk. Our collaboration to study OCR-002 is a key step towards understanding more about a potentially lifesaving drug."

The STOP-ALF study is a Phase 2a multi-center, open-label study that will enroll 24 participants with severe acute liver injury or acute liver failure. The primary endpoint will be safety and tolerability. Secondary endpoints include measurement of OCR-002 plasma concentration, change in venous ammonia and neurological function.

Hepatic encephalopathy is an often-reversible neuropsychiatric abnormality observed in patients with acute liver failure; it is marked by a worsening of brain function when the liver is no longer able to remove toxic substances such as ammonia in the blood. Ammonia accumulation in the blood impairs brain cell function. Signs of hepatic encephalopathy include impaired cognition, uncontrolled movements and decreased levels of consciousness leading to coma and death due to brain swelling.

"Acute liver failure is an orphan disease that carries significant economic costs, can occur from accidental acetaminophen overdose, often impacts young patients, and may require substantial interventions such as liver transplants," said Linda S. Grais, M.D., CEO of Ocera Therapeutics. "The development of novel medicines such as OCR-002 that address some of the most devastating consequences of acute liver failure is urgently needed."

Acute liver failure affects 2,000-3,000 patients in the United States every year and is often caused by acetaminophen overdose. Although only a fraction of the 55,000 acetaminophen overdoses each year leads to acute liver failure, it represents a large socioeconomic burden due to the young population involved and the high health care costs associated with the long term impacts of liver transplant.

About OCR-002

OCR-002 (ornithine phenylacetate) is an ammonia scavenger designed to rapidly lower systemic hyperammonemia and treat hepatic encephalopathy in patients with liver cirrhosis and acute liver failure. OCR-002, through its novel mechanism of action, directly lowers circulating blood levels of ammonia by enabling alternate metabolic pathways in the muscle and kidney in patients with liver failure. OCR-002 is being developed as an injectable for hospitalized patients and as an oral formulation to treat and prevent hepatic encephalopathy in patients with liver cirrhosis.

OCR-002, developed by Ocera Therapeutics, has received Orphan Drug designation in the United States and Europe and has been granted fast track status by the U.S. Food and Drug Administration. OCR-002 is currently being evaluated in a separate Phase 2 study as a treatment for acute hepatic encephalopathy in patients with liver cirrhosis. It is estimated that there are up to one million patients with cirrhosis in the United States, of which 150,000 – 200,000 patients are hospitalized due to complications of encephalopathy costing the healthcare system more than $7.2 billion every year.

For more information about clinical trials related to OCR-002, please visit: www.Clinicaltrials.gov

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 acute and chronic liver diseases and a broad range of gastrointestinal disorders. In addition to OCR-002, Ocera is developing Zysa™ (AST-120, spherical carbon adsorbent) for the treatment of irritable bowel syndrome. Ocera has raised over $60 million dollars in venture financing from Domain Associates, Sofinnova Ventures, Thomas, McNerney & Partners, Greensprings Global Partners and InterWest Partners. Additional information on the company can be found at www.oceratherapeutics.com.  

About the Acute Liver Failure Study Group

The Acute Liver Failure Study Group (AFLSG), led by Dr. William M. Lee, is a consortium of investigators in the United States focused on studying acute liver failure. The group is funded by the National Institutes of Health and focuses on better understanding the mechanisms of liver failure, using this knowledge to develop better disease biomarkers and novel therapeutic strategies.

SOURCE Ocera Therapeutics, Inc.