关键词：Agios,白血病,AG-221,IDH2,Celgene

2014年12月9日讯 /生物谷BIOON/ --对于Agios生物医药公司来说，2014年可以说是幸运的一年。公司在研发领域取得了许多出色的成绩。就在公司宣布其开发的治疗白血病药物AG-221先行试验研究取得成功的几个月后，好医师网，公司另外一项关于AG-221的探索试验又传来佳音。

在此次小规模临床研究中，，研究人员招募了45名患有IDH2（异柠檬酸脱氢酶2）突变阳性的恶性肿瘤患者，以考察这些患者对AG-221的反应情况。这些患者患有包括难以治愈的急性白血病、骨髓增生异常综合征、慢性粒单核细胞白血病等疾病，有些患者甚至已经表现出对化疗的抗性。

结果显示其中有6名患者出现完全响应，9名患者出现伴随血液功能恢复的完全响应，另有10名患者出现部分响应。而在后者中，有9名患者的响应时间长达3-8个月。这一结果是在美国血液学会的年会上公布的。而AG-221的这些数据无疑为公司申请更进一步的临床研究打下了良好的基础。

公司预计将于2015年开展相应的研究。而作为Agios公司合作伙伴的Celgene公司也对这一结果表示满意。Celgene公司此前向Agios公司投入了1亿3千万美元的资金以支持AG-221的研究。负责这一研究的Eytan Stein介绍说，AG-221能够帮助抑制IDH2突变体的产生，从而促使白血病患者体内的肿瘤细胞能够正常分化，变为正常的血液细胞。而这也是AG-221与传统化疗手段最大的不同。

除了AG-221，Agios公司还有一种用于治疗IDH1突变型白血病的药物AG-120正处于临床研发过程中。让我们拭目以待，Agios公司未来将为血液疾病市场带来怎样的改变！（生物谷Bioon.com）

详细英文报道：

Just a few months after Agios Pharmaceuticals highlighted clear indications of success for its early-stage drug AG-221 for various blood cancers, the biotech has followed up with a bonus round of promising results at the annual scientific meeting of the American Society of Hematology. Investigators noted that a growing number of patients with IDH2-mutant positive malignancies have achieved a complete response, with the durability of the response extending out now to 8 months in some cases. And the clinical success is opening the door to a registration study now planned to launch in 2015.

The Phase I dose escalation, single agent trial has now counted an evaluable response from 25 of 45 patients suffering from hard-to-treat acute myeloid leukemia, myelodysplastic syndrome, chronic myelomonocytic leukemia and untreated AML who had declined intensive chemotherapy. Investigators counted 6 complete remissions, nine complete remissions "with various degrees of hematologic recovery" and 10 partial remissions. Nine out of 10 patients had responses lasting from three months to as long as 8 months and ongoing.

The news also marks a boost for Celgene, which followed up on its early partnership by grabbing an option on the drug with a $130 million upfront payment.

Cambridge, MA-based Agios ($AGIO) is operating with a game plan established by David Schenkein, a noted cancer drug researcher and Genentech vet who has become one of the leaders in adaptive trial design, in which drugs can be sped through a compact, blended series of studies capable of delivering solid evidence of efficacy and safety. With the outspoken support of FDA cancer czar Richard Pazdur, studies like this are cutting months and sometimes years out of the development process. And the biotech, a 2009 Fierce 15 biotech, said today that it is mapping out a pivotal program for this drug that can launch next year, with its other clinical-stage drug--the IDH1-mutant inhibitor AG-120--advancing into a registration program in 2016.

"These findings build upon those presented throughout the year," says Eytan Stein, the lead investigator and a physician at Memorial Sloan Kettering Cancer Center. "In addition, they continue to provide evidence that AG-221 can inhibit the IDH2 mutant protein, stop production of the oncometabolite, 2-HG, and allow for cancer cells to become mature, functioning blood cells. This approach is different from traditional chemotherapy that attempts to non-selectively kill cancer cells. I believe AG-221's unique mechanism targeting a specific mutation is the way of the future and represents a highly specific oral therapy that may transform the treatment of a devastating group of blood cancers."

(责任编辑：yixin.zhang)

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