Biotech Values

[urche]

Re: Update on GTCB’s U.S. ATryn program:

The proposed design of the Atryn study that is nearing approval by the FDA is very encouraging, I think. This design gives GTCB recognition for the difficulty in performing a rigorous, controlled, double-blind study with this drug. The requirement for only 17 study patients also seems to be a concession to GTCB which had expressed to FDA concern about the expense and difficulty of running a larger trial. The willingness to accept patients from Europe and US is also a burden lessened.

Overall, it appears that patients with hereditary AT deficiency are a relatively predictable lot and GTCB is not going to be burdened with the usual rigors of study design. If FDA and GTCB work out the remaining "refinements" to the protocol it seems to be a fairly modest and feasible study likely to succeed, IMO.

DD, was that you getting nowhere with questions about the precedent for using historical control in study design?