Ocera Therapeutics has started dosing patients in the Phase IIa part of its Phase I/IIa clinical trial of oral OCR-002 (ornithine phenylacetate) for the treatment of cirrhosis.
OCR-002 is a validated ammonia scavenger currently being developed as a step-down therapy to retain remission of a neurocognitive disorder called hepatic encephalopathy (HE), which is caused due to serious liver disease.
The open-label, multiple-dose, randomised, three-period crossover Phase IIa part of the trial will assess the steady-state pharmacokinetics and pharmacodynamics of three times daily 6g, 12g and 21g of oral OCR-002 in 18 patients with Child-Pugh B cirrhosis.
The top-line results from this part of the trial are expected to be reported by the end of this year.
Ocera Therapeutics CEO Linda Grais said: "Patients with cirrhosis continue to have significant breakthrough episodes of HE, even with the current standard-of-care for HE prevention. We are developing an oral formulation to address this need.
“Our initial focus will be as a step-down therapy for patients following hospitalisation for an acute episode of overt HE, in order to prevent re-hospitalisation and relapse of HE."
The firm previously reported positive findings from the open-label, cross-over Phase I part of the trial.
The Phase I part was designed to determine the pharmacokinetics and the absolute oral bioavailability of single 5g doses of IV OCR-002 and single 5g doses of a liquid oral solution of OCR-002 in patients with Child-Pugh A and C cirrhosis.
The results showed that OCR-002 was safe and well-tolerated, with an absolute oral bioavailability of more than 95%.
Image: Illustration comparing a normal liver with a liver affected by cirrhosis. Photo: courtesy of BruceBlaus.