GTC Biotherapeutics (fka GTCB)

[DewDiligence]

Re: Inhaled AAT in CF

For comparison, these were the data from the phase-2 trial
completed by Bayer and the now-defunct PPL in 2003. At
all but the highest dose, the efficacy was essentially no better
than placebo.


Number of Exacerbations as a
Percentage of the Placebo Rate

              All Exacerbations     Severe Exacerbations 
 Dose Group  % of placebo  p-value   % of placebo  p-value 
 250 mg/day       75%      0.21           48%      0.08 
 125 mg/day       81%      0.35           94%      0.85 
 62.5 mg/day     106%      0.88          104%      0.91

Lung Function: % Difference
from Placebo at Six Months

Dose Group   VC    FVC    FEV-1 
 250 mg/day   4.3   1.2     2.1 
 125 mg/day  -0.1  -1.2     0.1 
 62.5 mg/day -1.8  -2.3    -0.6

VC = vital capacity (lung capacity in normal breathing)
FVC = forced vital capacity (lung capacity breathing out hard)
FEV-1 = forced expiry volume in one second (maximum amount breathed out in one second)