Replies to post #8361 on Biotech Values

[Aiming4]

For those of us that can't listen live...

in-progress updates like this are very valuable and greatly appreciated, thanks... Aiming4.

[DewDiligence]

Re: More detail on GTCB’s U.S. ATryn program:

The 17 patients in the U.S. trial's active arm will be pooled with the 14 patients from the European study whose review is now pending, giving an effective active arm of 31 patients.

What’s remarkable about this is that we already know that the 14-patient data from the European study were basically solid, and hence the U.S. ATryn trial will begin with almost half of the active-arm data already in good shape. Obviously, this improves the probability of a successful trial outcome.

Comments?

[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?

[io_io]

"GTCB has proposed that the pivotal U.S. ATryn trial have an active arm of 17 patients with hereditary antithrombin deficiency and an historical control arm of 35 patients."

Was this approved by the FDA ?

(I mean the Control Arm, not just the Pool Data you referred to)