2015年3月27日讯 /生物谷BIOON/--肝脏是人体最为重要的器官之一，也是发病风险最高的器官之一。许多因素都会导致肝脏疾病的产生。例如喝酒过多会导致肝硬化，服用药物过多会导致肝功能损伤，甚至肥胖也会导致脂肪肝。因此，如何拯救多灾多难的肝脏也成为了众多生物医药公司眼中的热点问题。
在众多肝部疾病中，非酒精性脂肪肝炎是发病比较普遍的一种疾病。据统计，在美国有5%的人患有这种疾病，而目前市场上尚没有有效治疗这种疾病的疗法。
Genfit公司开发的GFT505正是希望填补这片空白。而如今，GFT505临床二期研究的失败也为公司的开发计划蒙上了一层阴影。在这次有274名患者参与的为其一年的临床研究中，数据显示GFT505未能与对照组在肝脏脂肪减少和纤维化稳定等关键指标上与对照组有显着性差异。
不过，和其他生物医药公司一样，Genfit公司似乎并不会轻易放弃GFT505。公司表示尽管此次临床二期研究未能收获理想数据，但是研究人员发现此次的失败极有可能是志愿者招募策略失误和研究地点过多造成的。研究人员发现如果仅仅分析非酒精性脂肪肝的早期患者数据，结果将会天差地别。因此，Genfit公司的管理人员计划将进一步开展临床三期研究以确定GFT505的疗效。
不过，投资者显然对此并不买账。数据公布当天，公司股价应声下跌。而令Genfit公司始料不及的是，公司在非酒精性脂肪肝领域最强大的竞争对手Intercept Pharmaceuticals股价大涨，显示投资者内心中的天平已经有所倾斜。Intercept Pharmaceuticals公司开发的奥贝胆酸药物正处于临床三期研究，并已经获得了一部分理想数据。这或许也为Genfit公司平添了几分压力。
更有甚者，此次临床研究的失败，或许将使Genfit失去许多潜在的"金主"。此前一直有传闻称，赛诺菲、诺华和Shire公司等生物医药巨头都有兴趣将Genfit收入囊中。
详细英文报道：
Genfit's in-development drug for a pervasive liver disease failed to beat placebo in a midstage trial, but the company, undaunted, is setting sights on late-stage studies with eyes on what could be a blockbuster market.
The drug, GFT505, is an oral treatment for nonalcoholic steatohepatitis (NASH), which is a liver-scarring disease that affects as many as 5% of Americans but has no approved therapies. In a one-year study on 274 patients, GFT505 failed to chart a statistically significant improvement in NASH symptoms--measured by liver fat reduction and fibrosis stabilization--compared with placebo.
However, take out data from patients with early forms of the disease and the trial is a success, Genfit said, chalking up the overall failure to an enrollment error and the use of too many study sites. And management believes it has found a blueprint for further study, planning to design Phase III trials that focus on more severe NASH patients. Genfit says it plans to submit the Phase II data to global regulatory authorities in hopes of getting a late-stage program started as soon as possible.
Investors seemed less optimistic, sending the biotech's shares down about 5% on the Paris exchange.
Genfit's most advanced competitor in NASH is Intercept Pharmaceuticals ($ICPT), whose obeticholic acid is on its way into Phase III after posting some promising midstage efficacy data, picking up the FDA's coveted breakthrough therapy designation. The news of GFT505's failure sent Intercept's shares up about 9%.
The Phase II setback might throw some cold water on market chatter that Genfit was in line for a buyout, rumors that surfaced earlier this month when Bloombergreported that Sanofi ($SNY), Novartis ($NVS) and Shire ($SHPG) were taking a look at the company.
Elsewhere in NASH, Gilead Sciences ($GILD) splashed into the field this year with a $470 million deal to get its hands on a Phase II treatment for the disease, and the hepatitis C experts at Enanta Pharmaceuticals ($ENTA) are entering the space as well. Shire's $260 million acquisition of Lumena Pharmaceuticals gave it a Phase I NASH treatment, and Conatus Pharmaceuticals is plotting a Phase III effort for its emricasan.
