Ocera in-licensed OCR-002 (ornithine phenylacetate) from University College London. OCR-002 is an ammonia scavenger designed to rapidly lower abnormally elevated systemic ammonia levels (hyperammonemia) and treat hepatic encephalopathy (HE) in patients with liver cirrhosis and acute liver failure.
With severe liver impairment, toxic substances that are normally removed by the liver, such as ammonia, accumulate in the blood. Elevated ammonia levels can cause swelling of the brain. Signs and symptoms of HE include impaired cognition, confusion, and a decreased level of consciousness which can progress to coma and ultimately death. Patients are currently treated with laxatives that help eliminate ammonia in the gut and antibiotics that target ammonia production.
Unlike currently approved therapies for HE, OCR-002 is unique in that it directly lowers circulating blood levels of ammonia through pathways that do not require liver function.
OCR-002 is in development as both an intravenous formulation which is well suited for the hospitalized population with acute HE, and as an oral formulation for potential step-down therapy and to provide a chronic use option to maintain remission of HE in patients with liver cirrhosis.
|Product||Formulation||Indication||Preclinical||Phase 1||Phase 2||Phase 3|
|OCR-002||IV||Acute Hepatic Encephalopathy (HE)*||
*FDA Orphan drug and Fast Track Status
STOP-HE: Phase 2b Trial in Acute HE
Ocera recently completed its Phase 2b placebo-controlled, randomized, double-blind trial, STOP-HE, evaluating the Safety, Tolerability, pharmacokinetics and efficacy of intravenously-administered OCR-002 (Ornithine Phenylacetate) in resolving neurocognitive symptoms of acute Hepatic Encephalopathy in hospitalized patients with elevated ammonia.
The Company expects to meet with the Food and Drug Administration in the third quarter of 2017 to discuss paths forward for IV OCR-002. For more trial-related information, please see our Resources page.
Phase 2a Trial in Patients with Cirrhosis for Chronic HE
Ocera recently completed a single-dose Phase 1 study of orally available OCR-002 in patients with cirrhosis for the prevention of hepatic encephalopathy to understand the pharmacokinetics, PAGN formation and absolute bioavailability in this intended treatment population.
The Company expects to initiate a Phase 2a multi-dose study, also in cirrhotic patients, in the second quarter of 2017 to evaluate a new tablet formulation and steady-state pharmacokinetics. Data from the Phase 2a study is expected by the end of 2017. For more trial-related information, please see our Resources page.