BioWorld Insight: CNS Discovery, Glass Half Full or Half Empty?
Anette Breindl, Science Editor for BioWorld Today wrote an interesting article that in BioWorld Insight on the current state of state of CNS discovery. Despite some Big Pharma backing away from CNS, the field is percolating with fresh drug candidates and opportunities led by companies such as Targacept and Rexahn.
A few excerpts that illuminate the CNS opportunity include:
“There’s still a recognition that there’s a large unmet need there,” Targacept chief financial officer Alan Musso, told BioWorld Insight. Targacept chief financial officer. In that sense, pharma’s squeamishness “also creates opportunities,” Musso said.
There is no mystery as to why big pharma might be wary of drug development for CNS disorders. Even in the context of the low success rates of developing drugs for anything at all, CNS diseases are a tough assignment. “All the big pharmas are trying to reduce cost, and in CNS development, there is a higher possibility of failure,” Rick Soni told BioWorld Insight. Soni is president and chief operating officer of Rexahn Pharmaceuticals Inc., of Rockville, Md. which just started enrollment for a Phase IIb trial with its antidepressant Serdaxin.
Despite all those obstacles, CNS drug development has one advantage going for it: “The market’s not disappearing, that’s for sure,” [Michael Jackson, vice president for drug discovery and development at the Sanford-Burnham Medical Research Institute] said, so it’s unlikely big pharma will abandon CNS drug development. Rather it might simply change which parts of the drug discovery marathon it runs.
On the one hand, Rexahn’s Soni said, pharma companies are “cutting back on the basic research . . . and waiting until a small company gets to Phase IIb or Phase III” to in-license promising compounds, rather than looking for them after the proof-of-concept stage. “From a small company perspective, that’s a challenge,” he acknowledged, because the money for those trials has to come from somewhere – read: investors, who are not necessarily thrilled to bankroll later-stage, more expensive trials. Yet “if you are successful, big pharma is willing to value that drug very highly,” Soni said..
Targacept, for example, in addition to its deal for TC 5214, also has partnered with AstraZeneca on other CNS programs. One has been going on since 2005, while two anothers were initiated in 2009. Plus, the low success rate of clinical trials means that “the area is
ripe for innovation,” Musso said.

