Calling seasoned biotech investors here

[alcc]

Would greatly appreciate your insight and perspective, based on your experience with other NDAs you have followed:

1) 25% dropout in controlled PIII trial -- how unusual is this? Any meaningful comparable NDAs come to mind? 

2) Briefing doc -- how much weight do the concerns and criticisms expressed by the writers and reviewers have on the final agency decision process? I assume the internal (post adcom) agency review meetings would include those who drafted the briefing doc, since they essentially are the agency's experts re the subject.



Thanks.

[nadathing]

I can tell you that participating in a P3 trial is challenging. My Bydureon trial lasted 3 years and required 36 visits to the clinic 40 miles round trip). Each visit was 60-90 minutes not including travel. A also had an MRI and a series of tests to even qualify for the study. I was required to keep meticulous records including 5 BS measurements a day and sometimes 8, which included waking at intervals in the late evening and waking in mid morning. I recorded all medications I took including Advil for an occasional headache. I was required to call in if I had a low under 40 and return all of my used medication packaging and syringes. It was tedious but I also had the satisfaction of knowing I was doing something that could conceivably help millions with this dreadful disease. For this, I was compensated $1800 which covered my gas and time. Compensation is not a reason why people participate in studies, contrary to popular belief among many people.

I suspect the Afrezz study was more involved and detailed than mine. It should come at no surprise that some dropped out. In fact, didn't MNKD recruit many more participants than were required knowing this would happen?

[alcc]

I can imagine being a trial subject is quite a burden and I can believe most subjects do not sign up for the little bit of compensation. That is why I am trying to gain some perspective as to whether the 25% dropout in the 171 Gen 2 arm is within or outside 1 SD for dropouts for all PIII trials.

For the 175 trial, in which both arms had to use the inhaler, the dropout rates were 15% and 21%. The equivalent Exubera PIII T1 arm saw 12% dropout; for T2 it was 11%. I would imagine the burdens (i.e. non-adverse event/non-efficacy related) were similar. So, Afrezza dropouts were higher in every case, the average being ~20%, or almost 2x higher. How much of that is related to the overseas cohorts?

My concern is how negatively the FDA will weigh the dropout data in their decision, especially given their conclusion that Afrezza is numerically and statistically inferior to SC RAA (re A1c).

[biotec]

Yes MNKD a think recruited 10% more for this reason, But they had a 25% drop out. To me thats a huge number.This info should have been told last year when the release of the phase 3 results. IMO I remember a lot of neg articles about MNKD holding back info.IDK anymore. If results for the trials was great we should of held 7+ pps since.JMO. OPC might have been right, I thing this Management is very poor. Sorry my last neg post.