Outcomes of four independent trials to validate the efficacy of simvastatin and BMS-204352 following the initial compound screen
| Compound (concentration) | Trial no. (post-initial screen) | % Vehicle-treated responders (n/20) | % Compound-treated responders (n/20) | p value |
|---|---|---|---|---|
| Simvastatin (50 μm) | 1 | 90 (18/20) | 55 (11/20) | 0.0310 |
| 2 | 95 (19/20) | 70 (14/20) | 0.0915 (ns) | |
| 3 | 100 (20/20) | 70 (14/20) | 0.0202 | |
| 4 | 95 (19/20) | 60 (12/20) | 0.0197 | |
| BMS-204352 (50 μm) | 1 | 95 (19/20) | 60 (12/20) | 0.0197 |
| 2 | 90 (18/20) | 60 (12/20) | 0.0648 (ns) | |
| 3 | 100 (20/20) | 75 (15/20) | 0.0471 | |
| 4 | 100 (20/20) | 70 (14/20) | 0.0202 |
The data in the table refer to that depicted in Figure 4A,B. For each trial, the number of vehicle-treated and compound-treated Nf1P1 larvae responding to the stimulus is given, both as a fraction of total larvae tested (n = 20 per trial) and as a percentage. The outcome (p value) of the statistical comparison between these two groups in each trial is also stated. Both compounds reduced the number of responding larvae in all four validation trials, with this reduction reaching significance (p < 0.05) in three out of four trials for each. For trials in which the reduction was not significant, p values were nevertheless approaching 0.05. Statistical analysis (via Fisher’s exact text) was conducted on raw data rather than percentage values. ns = not significant.