遗憾！C肽替代疗法临床试验宣告失败

Cebix是一家位于美国圣地亚哥的生物技术公司，曾募集大笔资金用于推进C肽替代疗法的研究（治疗I型糖尿病引发的微血管病变），引发业界瞩目，但最近却悄悄偃旗息鼓.

Jacob Fuchs，一名药学专业的学生，也一直很看好C肽对糖尿病病人的疗效。最近，他向Cebix寻问该药物的临床试验进展，却发现公司网站无法打开，电话无法接通，Cebix就像人间蒸发了一样。当记者联系到Cebix的首席执行官Joel Martin时，对方遗憾地说道：“就快关门大吉了！”

Cebix成立于2008年，立足于John Wahren教授提出的假说“C肽对于维持微血管的正常功能非常重要”。John Wahren是瑞典卡罗林斯卡学院临床生理学的名誉教授。

C肽存在于每个人的体内，前胰岛素经酶切变为胰岛素和C肽两部分。患有I型糖尿病的病人，即产生微量或完全不产生胰岛素，特别容易发生微血管损伤，继而引发感觉丧失（神经病变）、肾功能削弱（肾病）、视觉衰退（视网膜病变）等并发症。

基于此，Cebix诞生了，并创立了专属新药，商品名Ersatta，用于治疗上述各种并发症。

“在12月，我们全力以赴进行了Ersatta的IIb临床试验（注：国外II期临床试验分为IIa和IIb两个阶段），在最后关头，竟然发现Ersatta与安慰剂效果没什么区别，哎！” Joel Martin说道，“我们好好研究了实验数据，得出最终结论：没有必要再进行下一步研究，即刻停止Ersatta的所有工作。”

记得在2012年10月份，Cebix获得了多家著名风投的青睐，比如InterWest， Sofinnova Ventures， 以及Thomas， McNerney & Partners等等，一共融资4800万美元，Cebix拿出了3000.9万美元来支持已经进行了一半的临床试验。去年11月，Cebix在一份向监管部门递交的文件中提到，新一轮融资又获得了3200万美元的支持，希望能继续推进Ersatta临床试验直至结束。12月得到如此不堪的试验结果后，Cebix压力巨大，不得不选择关门大吉。

公司CEO Joel Martin并未气馁，谈道“请大家理解，我们只能控制进程，却不能控制结果。我们的团队正在寻求新的创意点，有一天我们会重整旗鼓，荣耀回归！”

英文原文：

Cebix Shuts Down Following Mid-Stage Trial of C-Peptide Drug

San Diego-based Cebix， which raised about $50 million to advance a C-peptide replacement therapy for treating diabetes-related microvascular problems， has quietly folded its tent.

Jacob Fuchs， an Xconomy reader and pharmacy student interested in the use of C-peptide as a potential treatment for diabetic patients， recently inquired about the company’s status. In an e-mail， he noted that the company’s website had shut down and their phone numbers are disconnected. “It’s like they fell off the face of the Earth or something，” he wrote.

When I asked for an update from Cebix CEO Joel Martin， he replied， “Falling off the face of the Earth is pretty close！”

Cebix， founded in 2008， was based on a promising hypothesis. John Wahren， an emeritus professor of clinical physiology at Sweden’s Karolinska Institute， had helped determine that C-peptide plays a key role in keeping the smallest blood vessels healthy.

C-peptide is formed naturally in the body， when insulin is cleaved from pro-insulin. Patients with type 1 diabetes， whose pancreases produce little or no insulin， are also susceptible to microvascular deterioration， including loss of sensation (neuropathy)， loss of kidney function (nephropathy)， and loss of vision (retinopathy).

Cebix was focused on a proprietary replacement peptide， called Ersatta， for treating such complications.

“We concluded a perfectly executed phase 2b in December， just to get definitive results that Ersatta and placebo were， alas， indistinguishable，” Martin explained. “We determined that there was no point in further development and moved to wind down operations. As efficient as ever， we did that in 30 days.”

In October， 2012， Cebix raised $30.9 million to fund the mid-stage trial. At that time， Martin said the company had raised a total of nearly $48 million， primarily from InterWest， Sofinnova Ventures， and Thomas， McNerney & Partners.

In a regulatory filing last November， Cebix disclosed that it had secured nearly $32 million in additional commitments from its investors for a planned $34.9 million round， apparently in anticipation of capital the company would need to move Ersatta into late-stage clinical trials. After getting the lackluster results in December， the company instead shut down.

“The team and I are looking at many NewCo ideas with the support of our investors，” Martin wrote. “We can only control the operations， not the outcomes. We’ll be back in another guise before you know it！”