>> NO basis is a bit strong. Im sure there are examples, but in both directions.
I used strong word because people repeated that assertion as if it were fact. I gave two examples to refute that assertion. I haven't seen any counter example given in genetic disorder space. Slower enrollment than otherwise isn't the same as impossible!
>> Another company with good mgmt and planning would have started the phase 3 over a year ago and the FDA point would be moot.
How? That's another reason I used strong word because people made these types of assertions without any regard to fact. How could they start ph3 over one year ago when they didn't have any drug available for ph3? Poor planning? Oh right, how terrible for a company almost broken forgot to have scale up manufacture! Maybe it costed money it didn't have at the time!
>> 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)
I guess it is a surprise to many the patient groups haven't turned on the company, demanding compassionate use from the company like so many others would. Well, many genetic disorder patient groups are different from others. They have special relationship with companies who are developing drugs specifically for them. Just look at Genzyme during manufacture problems when patients had to give up drugs for extended period of time. Most outsiders crucified the company. However, very few patients abandoned the company and its drugs like others would. Big pharmas don't understand this type of special relationship, which is why they don't do well in this setting. Look at the difference how GSK communicated with patients in their trials last year and how RNA did this year. Night and day!
