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首页 » 投资 » $26亿的豪赌:Celgene公布克罗恩病反义核酸药物惊人II期疗效数据

$26亿的豪赌:Celgene公布克罗恩病反义核酸药物惊人II期疗效数据

来源:生物谷 2014-10-21 16:20

2014年10月21日讯 /生物谷BIOON/ --在研发阶段就将潜力品种收入囊中,是许多大中型药企惯用的招数,这考验的不仅是财力,更考验眼力。今年4月,生物技术巨头新基(Celgene)就与名不见经传的爱尔兰生物技术公司Nogra制药公司签署了一笔高达26亿美元的协议(相关阅读:Celgene签署$26亿协议获克罗恩病反义药物GED-0301),在支付了7.1亿美金预付款后,将一种处于II期临床的克罗恩病(Crohn's disease,CD)口服反义核酸药物Mongersen(GED-0301)收入囊中。业界认为,Celgene的这一豪赌要么使其成为这一行业最大的赢家,要么成为最大的输家。

近日,Celgene公布了Mongersen为什么值得豪赌的理由。Celgene表示,该药在II期临床中所表现出的惊人疗效,将为克罗恩病的临床治疗带来一场革命。

Mongersen(GED-0301)是一种首创的口服反义寡核苷酸疗法,开发用于中度至重度克罗恩病的治疗。目前,Mongersen已顺利通过一项涉及166例克罗恩病患者的II期研究,而Celgene已迫不及待地计划在今年年底启动Mongersen的III期临床项目。

该项II期临床研究在166例活动性克罗恩病患者中开展,调查了口服反义药物Mongersen的疗效和安全性。数据表明,与安慰剂相比,2种剂量Mongersen治疗组均有显著更高比例的患者实现临床缓解,40mg/天剂量组、160mg/天剂量组、安慰剂组实现临床缓解的患者比例分别为55.0%、65.1%、9.5%,2个治疗组与安慰剂组相比数据均具有统计学显著差异(p<0.0001);10mg/天剂量组与安慰剂组在临床缓解上无显著差异(12.2% vs 9.5%)。与安慰剂组(临床反应率=16.7%)相比,3种剂量Mongersen治疗组临床反应率显著更高:10mg/天剂量组(36.6%,p=0.039)、40mg/天剂量组(57.5%,p<0.0001)、160mg/天剂量组(72.1%,p<0.0001)。不良事件(AEs)和严重不良事件(SAEs)发生率在各个治疗组相似。

今年4月,Celgene与Nogra签订协议,根据协议条款,Celgene将支付7.1亿美元的预付款。若GED-0301研发成功,Nogra公司将有资格获得超过8.15亿美元的开发里程碑款,若GED-0301上市后的年销售额超过40亿美元,Nogra还将获得11亿美元的销售里程碑款,使得该笔交易的最大价值达到了26亿美元。

Mongersen是一种口服反义药物,是一种合成的寡核苷酸,开发用于中度至重度克罗恩病的治疗,Mongersen靶向Smad7信使RNA(mRNA),通过结合Smad7 mRNA,关闭基因的表达,从而降低Smad7的蛋白水平。在克罗恩病患者中,异常高水平的Smad7蛋白干扰了肠道中TGF-β1抗炎信号通路,导致炎症的增加。Mongersen药片的独特配方,被设计为在小肠的远端(末端回肠)和邻近结肠末端(右半结肠),在该部位局部起效,降低Smad7蛋白水平。(生物谷Bioon.com)

英文原文:Celgene's $2.6B deal for Crohn's drug pays off with promising PhII

Celgene ($CELG) bet big on the little-known Irish biotech Nogra Pharma when it partnered on a mid-stage drug for Crohn's disease. And today Celgene spelled out the reasons why it gambled $710 million upfront on a Phase II drug, highlighting data that support a clear case that the therapy can help spur clinical remission in a broad group of patients.

"Clinical remission was achieved by significantly greater proportions of patients receiving Mongersen (GED-0301) 40 (55.0%) and 160 mg/day (65.1%) compared with placebo (9.5%; p<0.0001 for both)," Celgene noted in an abstract. "No significant difference in clinical remission was seen for 10 mg/day (12.2%) vs. placebo. The rate of clinical response was significantly greater among patients receiving 10 (36.6%), 40 (57.5%) or 160 mg/day (72.1%) of Mongersen vs. placebo (16.7%; p=0.039, p=0.0001 and p<0.0001, respectively). The rates of adverse events (AEs) and serious AEs (SAEs) were similar across groups."

Mongersen is an oral antisense oligonucleotide that is designed to zero in on Smad7 near the end of the colon, where it can incite an inflammatory response. Celgene had already seen these results when it bought in for the upfront plus $1.9 billion in development and sales milestones. For analysts, who had to adjust their expectations for Celgene after the deal, the numbers helped underscore the potential for this drug.

"Overall, data from the ~160 patient trial (40 per arm) is at the high end of our expectations and perhaps roughly in-line with consensus Street expectations," notes ISI's Mark Schoenebaum. "Thus, my best guess at this point is that the stock will rise modestly today."

"GED-0301, a first-in-class oral antisense therapy, has the potential to change the treatment landscape for Crohn's disease," said Scott Smith, who runs Celgene's inflammation and immunology group. "Celgene is excited to pursue the clinical development program for this novel therapy in phase III trials in the near future."

Celgene is no stranger to the world of biotech deals. The company has one of the most active partnering groups in the industry, and there's a big interest in diversifying the company's product portfolio outside of cancer.

For now, Celgene appears ready to deliver on that promise. Mongersen is slated to start Phase III before the end of this year.

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