PRAN - The meta-analysis of the PBT2 IIa trial demonstrated the percentage of patients who improved was greater in the PBT2 group than the placebo group in all thresholds of improvement in the Composite and Executive Factor z-scores. Using ROC analysis, the improvement of any dimension on the NTB Composite z-score was significantly more likely on PBT2 than on placebo.
I think simply clearing out or preventing the accumulation of amyloid-beta is unlikely to be a successful MOA in achieving efficacy. Regardless, PBT2 has demonstrated other positive activity in mice and human trials. I think the odds of success are small but this is the only drug I know of that has somewhat convincingly demonstrated safety and possible efficacy in a phase 2 trial in patients with mild AZ. All the patients in the trial had been taking an acetylcholinesterase inhibitor for at least 4 months before entry.