Here's the A2-73 treatment score for FragileX Syndrome:
This is the 30000 foot, non-technical, view of the results of the Murine Fragile X study. I like to evaluate efficacy by how far does a drug return the afflicted back to normal. This will be given as a percentage, with 100% being the afflicted was returned to complete normalcy(cannot do better than that!). Oh yes, the afflicted in this case were, of course, mice.
The three most significant parts of the study evaluated, EFFICACY and BIOMARKERS, IMAGING. The most significant and most revealing were the efficacy and biomarkers, so they will be discussed here.
The efficacy was based on these characteristics/measures:
1) Hyperactivity - Open Field Test.................100%
2) Freezing Response - Fear Conditioning........100%
3) Perseverance and Anxiety - Marble Burying.....50%
Also shown above is the percentage that each was improved, compared to normalcy. Hyperactivity and Freezing were totally returned to normal, while "Marble Burying" returned half way to normal.
Weighting each of the three equally (each 33.3% max), efficacy score would be:
The mice on A-73 returned 83.3% back to normal!
The biomarkers were:
1) pAkt....68.7%
2) pERK....39.3%
3) BDNF....86.9%
Also shown above is the percentage that each biomarker was moved towards normalcy. Weighting each of the three equally (each 33.3% max), efficacy score would be 64.3%. This means the biomarkers moved almost 2/3 back to normal. Biomarkers are indicators, the real proof is in the efficacy.
CONCLUSIONS:
It is clear why Fragile X expert, Dr.Hagerman, is very enthusiastic about getting A2-73 trials going for humans, with a hopeful improvements of 83.3%! As quoted in the article "...this neurodevelopment disorder continues to have an unmet therapeutic need.".
The efficacy numbers are amazingly good. The biomarker improvements support and validate the efficacy.
This study adds to the growing body of evidence, validating A2-73 as a powerful new drug.
The usual caveats exist, this is a murine study(not humans), there were not a lot of mice used, there were limited efficacy tests, all IMHO, etc.
Thanks to TTT for posting the link to the Murine Fragile X study.
