Excellent point regarding the importance of considering cash on hand when interpreting cohort size. Companies that are strapped will sometimes run a tiny 10-20 pt Phase IIa in order to attract a private investor--or to get a larger partner to take on the program--via a 'hint of principle', since the size precludes 'proof of principle.' It's not a smokescreen, more like a flaregun, looking for attention and financial rescue. Examples would include NEOT's first 10 pt Alzheimer's 'trial', and Cortex's 15 pt schizophrenia IIa. They were scaled due to lack of cash and (for the Cortex study) some uncertainty as to the suitability of the target, which fits the second category. Another exception would be the complexity/risk of the procedure--e.g. neural cell implants. Titan/Schering and Diacrin both chose to start with very small cohorts, a handful of patients, but not as a smokescreen for pessimism.
The need for attention/financing also accounts for the hyperbole in the NEOT self-description. They need to raise money, but unfortunately, this casts a cloud of diminished credibility over the whole program. They have never discussed more than five or six compounds with me (BTW: AIT 034 and 082 are not just different indications, they are different compounds), and have put virtually all resources behind 082, everything else on the backburner.
If one were to use NEOT's criteria for 'drugs under development', one can only imagine how big the pipeline at NBIX would look.
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