Tuck,
<<A couple of Axokine related news items today, both good if not earth shattering:>>
Last night and few past weeks I was occupied with few other things, so my last respond was bit short on *thinking forward*.
<<Nice to have fast track status for severe obese population. This was primary target population in '00. Why they did not started pivotal trial for this population early when PIIIs program started? Or, will they be able to select sub-population from 2000 pts that belong to this category without compromising trial statistic and protocol?>>
While FT does not have direct effect on time to NDA file, it does have significant psychological benefit. FDA acknowledged and send clear message that severe obesity is serious medical problems in US and will only get worse not better in near future. I do believe that REGN clinicians did think about this seriously before they started PIII trials.
Contrary to many analyst prediction (IF all went well with Axokine) that time frame for Axokine NDA is mid-’05 (completion of the two large pivotal trials), I think that REGN have reasonable shot with one pivotal trials and two supportive ones (NDA file in 1Q/2Q-’04). IF Axokine COME OUT CLEAN on any SAEs at 24 months and confirm that weight loss is medically important and SUSTAINABLE, than FDA (by sending message with fast track status) will accept NDA. Why?
Here I will speculate bit with numbers. With 65% retention rate, and with assumption that ~50% of the pts were with 40<BMI<55, and with assumption that ~50% of this severe obese population did have co-morbidity status, than REGN should be able to separate ~250-260 pts in Axokine and ~80 pts on placebo as *target population*. This is in range of the size for pivotal trial, or IF Axokine produce significant weight loss in ITT analysis, this target population should be sufficiently powered to produce conclusive data on weight loss and other clinical parameters important to fast track status. I know that this is STRACHED hypothesis, but it is not out of reach.
I mentioned in my early posts that insiders selling at year-end are concern and that *smell* like pre-BDNF pattern. I spent considerable time on studding and comparing insiders selling at REGN. While at this time it is yellow flag and should be followed closely, it is far from red flag from BDNF-time. I am bit relieved from what I did find because REGN is my biggest bet and can not afford misjudgment. While my view can change at any time, for now am comfortable with REGN investment.
Miljenko |
