Anavex Life Sciences Corp (AVXL)

[falconer66a]

Understanding of Mechanism of Action Not Required

...will validate the Sigma-1 treatment path - assuming the scientists can determine "how it works".

Actually, FDA does not require exact or precise understandings of the biochemistry of the drugs it approves. Correct me if wrong, but these are the hurdles to leap:

1. Safety. Adverse events (side effects) are balanced against therapeutic benefits. Lots of FDA-approved drugs have "black box" warnings, telling of potential cancers and deaths from taking these drugs.

2. Standard of Care-exceeding Efficacy. The new drug has to work better than existing drugs.

3. Minimal teratogenic activities. The drug should not cause birth defects, or create aberrant genetic expressions in utero.

For Anavex 2-73, safety should not be a problem, as revealed in lab rat studies and the people in the Australian study.

Efficacy for all three of the target diseases, Rett, Parkinson's, and Alzheimer's, will be easy to attain. None of those diseases have drugs of any real usefulness. Benefits are minimal and/or non-persisting.

I've seen no data or papers on any sort of teratogenic activities by any of the Anavex molecules. Teratogenicity is determined by the Ames Test, where the drug is included in bacterial growth media. If the drug is teratogenic, able to disrupt normal DNA nucleotide sequences, this can be detected in the bacteria. That is a good indicator of mutagenicity.

Actual teratogenicity is checked by feeding pregnant rats or mice the drug, and monitoring fetal and neo-natal development.

But, FDA doesn't have to have an elaborated diagram of the precise molecular reactions that cause therapeutic success, if they can even be determined. Many drugs work, but actual step-by-step mechanisms of action are still unknown.

Is it safe, and does it beat anything in presently in the pharmacy? Those are the two big questions FDA needs answers to. Well-constructed clinical trials provide the answers.