3年前，制药巨头罗氏公司决定投入5亿美元用于发展RNA类药物，不过很快公司就发现在这一领域公司面临的困难可能远远超出想象。
不过现在罗氏公司决定卷土重来，近日，罗氏与Santaris公司达成合作共研RNAi药物协议。根据协议，公司首先将投入1000万美元支持此次研究计划，同时还设立了总额高达1亿3千8百万美元的里程碑基金。与此同时，Santaris公司最近也刚刚宣布公司也和葛兰素史克公司以及施贵宝公司建立了研发合作关系。
在这次合作中，罗氏公司将借助Santaris公司的LNA平台来进行相关的RNAi药物研发。Santaris公司称LNA平台能够克服抗体、siRNA难以输送的缺点。罗氏公司与Santaris公司都对这一平台寄予了极大希望，希望能够帮助其取得在RNA药物研究方面的突破。
Three years ago, Roche sent shockwaves ricocheting through the R&D field when it decided to write off a half-billion-dollar initiative in RNA drug research and sell off its dedicated facility in Wisconsin. Tied with a similar RNA retrenchment at other major outfits, the pharma giant's ($RHHBY) retreat sent a clear message that the technology was too imperfect, and the clinical path too long, to warrant a big commitment.
But a year after John Reed took over at Roche's Basel-based research group pRED, Roche is once again doing RNA deals. Joining hands with Santaris--a company with headquarters in Denmark, a facility in San Diego and an American biotech exec newly installed at the helm--Roche is gambling $10 million upfront and up to $138 million in milestones per potential RNA program they commit to in the discovery pact.
This morning Santaris followed up with an announcement that it had struck a new deal with GlaxoSmithKline ($GSK), which follows an earlier deal last year with Bristol-Myers Squibb ($BMY).
Roche is jumping on Santaris' LNA (locked nucleic acid) platform, which the biotech claims can overcome the limitations of antisense and siRNA technologies and deliver new therapies that can do what antibodies and small molecules are incapable of doing: hitting some currently undruggable targets with mRNA and microRNA-targeted therapies that are much better targeted than some of the current RNA therapies in the pipeline.
The big idea behind RNAi was to mimick the way the body silences genes. But delivering the drug became a major challenge, early programs floundered, and Roche joined the drugmakers on the way out the door. Last spring, though, Roche was back with a $392 million RNA deal with Isis. And a company spokesperson says Roche is increasingly confident that these new partnerships can blaze a path to significant new therapies.
"Thanks to past siRNA research we have gained substantial expertise in terms of the technology application, delivery and safety profiling of RNA therapies," the spokesman tells FierceBiotech. "Our recent deals such as Isis and Santaris have been focused on specific applications of antisense. For instance in case of the Locked Nucleic Acid technology we believe there is a potential for antisense molecules to achieve longer duration of effect in target tissue. This particular application would be relevant for our R&D efforts in the area of ophthalmology, as it would mean that longer duration would decrease frequency of treatments."
Roche's pRED, though, has been on a deal spree in recent months, racking up a string of new deals aimed at reviving an R&D arm that had fallen on hard times. This deal with Santaris comes shortly after it announced that Donald deBethizy, the former chief of the troubled biotech Targacept, was being put in charge. Santaris also recently wrapped up a squabble over a tech licensing deal it had had with Enzon.
"We believe the LNA drug platform provides the means to discover novel drugs against a range of targets of high clinical relevance that are difficult--or impossible--to target with other drug platforms," Reed said in a statement. "As such, the partnership will increase our ability to discover and develop important novel medicines that can address significant unmet needs in critical therapeutic areas."
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