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Big Pharma's Turn On RNAi Shows That New Technologies Don't Guarantee R&D Success

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This article is more than 9 years old.

It is not often that a new technology bursts on the scene with this type of hoopla:

“RNAi (RNA interference) is a revolution in biology – a breakthrough in understanding how genes are turned on and off in cells – and represents a completely new approach to drug discovery and development. The importance of its discovery was recognized by the award of the 2006 Nobel Peace Prize for Physiology or Medicine, and RNAi has been heralded as ‘a major scientific breakthrough that happens once every decade or so.’ It is widely considered one of the most promising and rapidly advancing frontiers in biology and drug development today.”

This is taken from the website of Alnylam, a biotech company that is one of the leaders in the RNAi field (along with Tekmira and others), so it is not surprising that it is bullish on this technology. When earlier in the year Sanofi took a $700 million stake in Alnylam, John Carroll reported that “Alnylam execs have been one of the chief proponents of RNAi as the next big product arena, with the potential to rival monoclonal antibodies in terms of revenue potential.” If true, that would be enormous. Antibodies like Humira and Remicade, are two of the largest selling drugs today with annual sales in the billions of dollars.

Thus, it was pretty surprising to see that Novartis, which had an ambitious RNAi research effort, suddenly halted this work. Novartis isn’t the first Big Pharma company to become disenchanted with this field. Merck , enamored with the promise of RNAi, bought the RNAi company, Sirna Therapeutics, for $1.1 billion in 2006. Last January Merck sold Sirna to Alnylam for $175 million and took a huge write-off in the process.

What has gone wrong? A decade ago, there were a variety of issues facing RNAi as therapeutics, but this is true for any new technology. However, the biggest hurdle for RNAi is delivery of such a therapy to the target organ as these large molecules are easily broken down in the body. In 2004, there were a lot of ideas to try to overcome this problem. Unfortunately, progress has been slow and people are beginning to question whether RNAi therapy could ever be broadly applicable to a wide variety of diseases. Novartis confirmed this when they provided its rationale for de-emphasizing this area: “This decision was driven by ongoing challenges with formulation and delivery and the reality that the current range of medically relevant targets where siRNA may be used is quite narrow.” In fact, the delivery challenges were such that ten years ago companies like Pfizer (which had a member of its Board of Directors also serving on Alnylam’s Board) decided to stay on the sidelines waiting for the science to play out a bit more before investing in this technology.

Clearly, there are still believers in this field as evident from Sanofi ’s investment. Indeed, for certain diseases where an RNAi therapeutant can be more readily introduced, such as the eye, or “privileged compartments” such as the liver, RNAi still has potential. But given that these therapies would be expensive due to the high cost-of-goods involved in synthesizing these agents, they would have to be targeted to diseases where the cost of therapy would be justified by the beneficial medical effects. Thus, the potential for RNAi therapy is narrower still. It is possible that new therapies will evolve from this technology. But to say that RNAi therapy will rival monoclonal antibodies in terms of revenue potential – well, that’s a bit of a stretch.