- Problems with the trial design make it difficult to determine the effectiveness of Amylyx's AMX0035 against ALS.
- AMX0035 combines two compounds - sodium phenylbutyrate and tauroursodeoxycholic acid - that inhibit and scavenge peroxynitrite. This may be the key to the treatment of several neurological diseases.
- Compounds that are better inhibitors and scavengers may produce better results than AMX0035.
- The FDA will likely follow their advisory board's recommendation not to approve AMX0035 based on one trial.
- Amylyx's stock would then drop further. Phase 3 clinical trial results for ALS (and perhaps eventually for Alzheimer's disease) will likely determine the fate of the company.
For further details see:
Amylyx And ALS: Muddied Results