The Phase 1 part showed the treatment to be safe and well tolerated.
HE is a progressive complication of cirrhosis of the liver or liver failure. Its hallmarks include impaired movement and mental changes, including confusion, disorientation, and in more severe cases, loss of important mental functions, coma, and death. Cirrhosis is liver malfunction stemming from long-term scarring.
“Advancing oral OCR-002 into Phase 2a marks another significant milestone in the progression of our pipeline and programs for orphan and other serious liver diseases,” Linda Grais, chief executive officer of Ocera, said in a press release.
“Patients with cirrhosis continue to have significant breakthrough episodes of HE, even with current standard of care for HE prevention,” she said. “We are developing an oral formulation to address this need. Our initial focus will be as a step-down therapy for patients following hospitalization for an acute episode of overt HE, in order to prevent re-hospitalization and relapse of HE.”
OCR-002 is an ammonia scavenger, or therapy that’s designed to rapidly remove ammonia from the blood. Ammonia accumulates when the liver can no longer remove toxic substances from blood. The accumulation impairs brain cell function and can lead to HE.
The treatment has received orphan drug designation in the United States and Europe, and the U.S. Food and Drug Administration has granted it fast track status. The designations will move it through the approval process quicker.
Ocera described the Phase 1 trial as an open-label, crossover study designed to determine how OCR-002 would act in patients with cirrhosis of the liver and decompensated liver disease. It proved safe and well-tolerated, the trial indicated.
The Phase 2a trial is an open-label, multiple-dose, randomized, three-period crossover study designed to evaluate the pharmacokinetics and pharmacodynamics of OCR-002. Pharmacokinetics involves the body’s effect on a drug, whereas pharmacodynamics involves the drug’s effect on the body.
Eighteen patients with significant cirrhosis-related liver problems will receive OCR-002 tablets three times a day in the trial. Some will receive six grams, some 12 and some 21.
The trial results are expected to be announced by the end of 2017.