Novel drug shows efficacy in hepatic insufficiency

A novel drug comprising eight different amino acids showed efficacy in improvement of neurocognitive function, metabolism and other positive outcomes in patients with mild and moderate hepatic insufficiency, according to a press release issued by the manufacturer, Axcella.

The company said the drug, AXA1665, was safe and well-tolerated by patients.

“We believe the data from the AXA1665-002 study show AXA1665’s potential to address multiple fundamental dysregulations associated with cirrhosis and hepatic encephalopathy,” Manu Chakravarthy, MD, PhD, chief medical officer of Axcella, said in the release. “This study replicated findings on amino acid metabolism from our previous short-term study, AXA1665-001, and we were pleased to see those effects sustained through 12 weeks.”

Researchers assessed the safety, tolerability and physiological impact of the drug in a placebo-controlled trial that included 60 patients with mild or moderate hepatic insufficiency. They randomly assigned patients to receive either 29.4 g or 53.9g of AXA1665 or matched placebo in three divided doses per day for 12 weeks. Then, they followed patients for 4 weeks.

In addition to the safety and tolerability of the drug, investigators observed dose dependent improvement in amino acid metabolism and improvements in other outcomes.

“We also noted dose dependent, directionally consistent changes across all three psychometric tests that were utilized, which help to bolster our confidence in AXA1665’s potential to reduce overt hepatic encephalopathy events,” Chakravarthy said in the release. “We look forward to initiating a phase 2 clinical trial to investigate this hypothesis and further evaluate this candidate’s impact on measures of physical function and related patient reported outcomes in patients with advanced liver disease.”