Replies to post #8382 on Biotech Values

[DewDiligence]

>>
As I understand it, we can expect study arms as follows:
1)ATryn active study arm with 17 new patients from Europe and USA;
2)Historical control arm with 35 preselected patients from Europe and USA.
3) A total of 31 patients from USA and Europe will be compared to 35 historical controls from USA and Europe.
<<

Correct.

>>It is indeed a small coup, Dew, to be allowed to pool data and stack the odds for a favorable outcome with existing data from a similar, positive study. Maybe we should attempt to verify that Cox meant what he said.<<

Dr. Cox paused for a moment as though considering the ramifications, so I assume that he did fully understand the question. I plan to follow up with Tom Newberry to be sure.

[DewDiligence]

Re: GTCB
Pooled 31-patient active arm and other matters:

I just talked to Tom Newberry at length. Dr. Cox indeed spoke correctly in answering the last question on the CC when he agreed that the active arm in the U.S. trial effectively has 31 patients.

Newberry also stressed the importance of the FDA’s agreeing to make clinical symptoms of thrombosis –rather than a thrombosis “hit” on a diagnostic scan— the primary endpoint of the U.S. trial. In the 14-patient data set submitted to the EMEA, the two patients with possible DVT indications from an ultrasound scan did not in fact have clinical symptoms of DVT.

In other words, for the 17 new patients in the active arm of the U.S. trial, patients with outcomes similar to the two European patients with suspected DVT would presumably not be counted as patients with adverse events!

My one misgiving about the U.S. ATryn trial is that GTCB has not sought to obtain an SPA for it. Newberry suggested that, due to the highly unusual nature of this trial [it’s not often that the FDA agrees to a pivotal clinical trial with only 17 new active-arm patients], the amended IND that GTCB and the FDA have negotiated contains as much detail as a typical SPA would contain. I’m not fully convinced on this point, at least not yet.