随着制药巨头们数十亿美元交易如火如荼地进行，生物技术公司新基（Celgene）也已悄然斥资7.1亿美元，从爱尔兰制药商Nogra制药手中获得了一种处于后期临床开发的克罗恩病（Crohn's disease）药物GED-0301。

GED-0301是一种口服反义药物，靶向于Smad7信使RNA（mRNA），该药开发用于中度至重度克罗恩病的治疗。反义药物是一种合成的核酸拷贝，旨在结合导致疾病的基因的mRNA，关闭基因的表达。

根据协议，若GED-0301研发成功，Nagra公司将有资格获得超过8.15亿美元的里程碑款项，若GED-0301上市后的年销售额突破40亿美元，Nagra还将获得11亿美元的销售支付款项，使得这笔交易的最大价值达到了26亿美元。

目前，GED-0301已顺利通过一项涉及166例克罗恩病患者的II期研究，该项研究的数据将在即将到来的科学会议公布。Celgene计划在今年年底将该药推进至III期研究。

GED-0301在II期临床中所表现出的惊人疗效，预示着该药有望成为克罗恩病的一种革命性疗法。

英文原文：Celgene shells out $710M for a PhIII Crohn's drug

As Big Pharma trades billions for whole business units, the deal-inclined Celgene ($CELG) has quietly paid $710 million to get its hands on a late-stage Crohn's disease contender in a deal that spiral out to 10 figures.

Celgene is handing over $710 million upfront to Ireland's Nogra Pharma to get its hands on GED-0301, an oral antisense drug that targets the messenger RNA Smad7 to treat moderate to severe Crohn's symptoms. Antisense therapies are synthetic copies of nucleic acids designed to treat diseases by shutting the down the genes that cause them, usually by binding to mRNA.

Under the agreement, Nogra is in line for up to $815 million more in development milestones and $1.1 billion in sales payouts if the drug ever hits $4 billion in a year, bringing the maximum deal value to about $2.6 billion.

The drug has already made its way through a placebo-controlled Phase II study on 166 Crohn's patients, Celgene said, with data to be unveiled at an upcoming medical meeting. Now the biotech plans to get its new antisense candidate into Phase III by year's end.

"GED-0301 is a potentially transformative therapy that demonstrated striking clinical activity in a phase II trial for Crohn's disease," Celgene Senior Vice President Scott Smith said in a statement. "It strengthens our expanding pipeline of novel therapies intended to address significant unmet medical need in immune-mediated diseases."

Celgene has a well-told penchant for striking up innovative and often byzantine deals with its partners, including lots of back-loaded agreements with risk-sharing clauses. Considering that reputation, the sizable upfront payment involved in the Nogra deal means the Summit, NJ, Big Biotech has considerable faith in '0301's potential.

The drug joins Celgene's small but promising anti-inflammatory pipeline, led by apremilast, a blockbuster hopeful that picked up its first FDA nod to treat psoriatic arthritis last month with a make-or-break verdict on psoriasis expected by September. The company is also developing the Phase I pomalidomide for systemic sclerosis and the early-stage CC-220 for a variety of inflammatory diseases.