Anavex Life Sciences Corp (AVXL)

[Doc328]

I agree with your post and look forward to the placebo controlled blinded results.

The RSBQ is a questionnaire for parents/caregivers. It has 45 questions about the patient and the parent responds 0 for not true, 1 for sometimes true and 2 for often true. Sometimes and often are of course subjective and in an open label trial requiring significant effort to participate in and being told good things about a medicine, placebo effect (by proxy) is likely.

The CGI-I is a simple one question response by the doctor scored 1 to 7: 1 is very much improved 2 is much improved 3 is minimally improved 4 is no change 5 is minimally worse 6 is much worse and 7 is very much worse. Many studies with other disease also include this question. For a disease like Rett, where the caregiver talks to the doctor, if a parent says patient seems better, then the doctor will likely pick 2 if they observe patient being better than last time or 3 if they seem the same though parent says they are better. Parents and doctors would know that the baseline period was off drugs and the treatment period was on.

The glutamate and GABA data is more interesting to me. I am hoping they present the actual blood concentrations with dots to see what the relative changes were and if 1 outlier could explain the variance. Better yet if they connect the baseline and 7 week values with a line for each patient. Although plasma glutamate and GABA are less likely than the questionnaires to have a placebo effect, one might still occur. Stress increases glutamate and the young woman might be less stressed at the follow up visit, especially if the parent is less stressed. Also initial visits are always longer than subsequent visits which could affect stress. They will also measure these in the randomized study so these results will be interesting.