The ignorance on this board is astounding. No one here seems to think critically.
Think for a moment. This was an interim result.
Ignore the placebo for a minute; it does not mean anything in the long run. What matters is 41 % got an average reduction of over 50% in the marker. We do not know the actual numbers but if the 41% got a reduction of over 65-70%; which is possible as the mean is over 50% the trial is a success for that 41%. To me what is dissapointing is that only 41 % got a reduction of over 50%. As these were genetically prescreened the no. of patients that would benefit seems to be less than the 40-50% of fabry disease patients that the company had been touting maybe closer to 20%. This is still enough to support a stock price in early teens by itself ignoring the other trials.
Wait for the actual data in feb. before calling this a failure.
The trial used a ratio of 2 female - 1 male & we might find out thats why they got burned with the screening. Prior data indicates females don't respond as well, but in the end its the placebo group which burned them. Looks like tissue biopsies @ 6 months are a poor indicator of efficacy so they can still file for approval depending on what we get out of the 1 yr data.
They also had some secondary indicators which they will publish in feb. It might well be these show significant improvement. I had not thought of the female factor but it makes sense; it might be most of the significant gains are in the males which would explain the 41% success rate.