新药对肝功能不全显示功效

StephenW

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.”

StephenW

新药对肝功能不全显示功效

根据制造商Axcella发布的新闻稿，包含八种不同氨基酸的新型药物在轻度和中度肝功能不全患者中显示出改善神经认知功能，代谢和其他阳性结果的功效。

该公司表示，这种药物AXA1665是安全的，患者可以耐受。

Axcella首席医学官Manu Chakravarthy医学博士说：“我们相信AXA1665-002研究的数据表明AXA1665有潜力解决与肝硬化和肝性脑病相关的多种基本失调。” “这项研究重复了我们先前的短期研究AXA1665-001中氨基酸代谢的发现，我们很高兴看到这些作用持续了12周。”

研究人员在安慰剂对照试验中评估了该药物的安全性，耐受性和生理影响，该试验包括60例轻度或中度肝功能不全的患者。他们将患者随机分配为接受29.4克或53.9克AXA1665或匹配的安慰剂，分三天每天服用12周。然后，他们随访患者4周。

除了药物的安全性和耐受性外，研究人员还观察到了氨基酸代谢中剂量依赖性的改善以及其他结果的改善。

Chakravarthy在新闻稿中说：“我们还注意到在所使用的所有三个心理测试中剂量依赖性，方向一致的变化，这有助于增强我们对AXA1665减少明显肝性脑病事件潜力的信心。” “我们期待着开始一项2期临床试验，以研究这一假设，并进一步评估该候选人对晚期肝病患者的身体机能和相关患者报告结局的影响。”