Biotech Values

[masterlongevity]

NO basis is a bit strong. Im sure there are examples, but in both directions. When I was working on Rituxan in 1998, we couldnt enroll a phase 3 in aggressive NHL because a cooperative group study in France was sp positive that everyone was using off-label. We tried to do it but after a year gave up with less than 20% of needed patients enrolled.

The problem of not being able to enroll with an approved therapy is a real problem. The way most people deal with it is to enroll the confirmatory PBO controlled trials in EU or elsewhere if drug is approved in US. Otherwise it will take forever to enroll confirmatory and defeats the purpose. Another company with good mgmt and planning would have started the phase 3 over a year ago and the FDA point would be moot.

I see their mgmt as A)incompetent for the poor planning and execution and also B)unethical because they 1) overpromsied the possibility of AA and 2) are not offering comp use to the sickest DMD patients (the ones who probably couldnt qualify for phase 3)