Biotech Values

[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.