Gene Logic today announced that Pfizer has filed a patent based on a Gene Logic drug repositioning effort. This would appear to be one of the most significant votes of confidence in such efforts by an outside partner.
Drug repositioning is the idea of finding new therapeutic uses for advanced compounds, particularly compounds which are very advanced but failed due to poor efficacy in the originally targeted disease. A number of companies have sprung up in this field -- the two I am most familiar with are Gene Logic and Genstruct -- and at least some large pharmas have in-house programs.
The reality is that many existing drugs have origins in therapeutic areas which are quite different than those they started in. Perhaps the most notorious case is Viagra, which was muddling along as an anti-hypertensive until an unusual side effect was spotted. Minoxidil similarly began in the anti-hypertensive until its side effect was noted. The route to some psychiatric medications began with anti-tuberculosis agents and antihistamines. I doubt that's a complete list.
Gene Logic is one of the original cohort of genomics companies and has been through many iterations of business plan. If memory serves, they were one of several companies originally built around a differential display technology, a way of obtaining mRNA signatures for diseases which predated microarrays. Gene Logic later became one of the major players in the toxicogenomics space, and as part of that effort built a large in-house Affy-based microarray effort. They built microarray databases for a number of disease areas (I've used their oncology database), built a sizable bioinformatics effort, and even acquired their own CRO.
However, none of that could quite be converted into a stream of gold, so over the last year or so the whole mess has been deconstructed, leaving behind the drug repositioning business which had begun as a unit of Millennium (which is one reason I'm familiar with it). They'll even be changing their name soon, to Ore Pharmaceuticals (presumably Overburden and Slag, while appropriate for the mining theme, did not last long in the naming queue).
While there is certainly historical precedent for repositioning, the question remains whether companies can make money doing it, and whether those companies will simply be the big pharmas or the gaggle of biotechs chasing after the concept. Depending on the company, some mixture of in vivo models, in vitro models and computational methods are used. One way to think of it is doing drug discovery, but with a compound which already has safety data on it. There is also extensive interest in the concept in the academic sector, which is a very good thing -- many drugs which may be repositionable have little or no patent life yet, meaning companies will find it difficult to invest in them with any hope for a return.
Gene Logic / Ore has one repositioned drug which has gone through clinical trials, GL1001 (nee MLN4760). This is a drug originally developed by Millennium as an ACE2 inhibitor. Since I'm among the discoverers of ACE2, I tend to key an eye on this one. Millennium gave it a whirl in obesity, but now Gene Logic has found a signal in inflammatory bowel disease in animal models.
That Pfizer bothered to file a patent is significant, as it triggered a milestone payment -- amount unspecified, but these are usually something interesting. But that is still a long way from starting a new trial -- that will be the real milestone, and whichever drug repositioning firm can claim that will really be crowing -- that is, until somebody actually gets a drug approved this way.