Replies to post #2111 on GTC Biotherapeutics (fka GTCB)

[DewDiligence]

Re: DIC program

>what leo/gtcb should do differently [from the Kybersept study] seems clear:
1. limit enrollment to just the DIC subgroup of sepsis pts
2. limit enrollment to patients with high risk (but not highest risk) of mortality
3. allow no concomitant heparin therapy<

#1 and #3 are a certainty. #2 is less clear because there’s the usual tradeoff between inclusion criteria and speed of enrollment.

The biggest problem I foresee with the DIC program in Europe is that Leo will not be able to mandate the treatment given in the control arm. According to GTC, this looseness is necessary to allow for the different SoC treatments for sepsis in different EU countries.

The lack of a standardized control arm introduces the kind of wildcard that could lower the statistical power of the trial. More of a problem in phase-3 than in phase-2, but a problem nonetheless.