Amylyx Pharmaceuticals

[3][4] The two proposed that taurursodiol and sodium phenylbutyrate together might prevent dysfunction of mitochondria and the endoplasmic reticulum, safeguarding neurons.

[4] Klee and Cohen were advised by Rudolph E. Tanzi, who ultimately served as the founding chair of Amylyx's scientific research board.

After Klee and Cohen were able to achieve strong results in pre-clinical research, Tanzi connected the two to his colleagues at Massachusetts General Hospital, which culminated in clinical trials and FDA approval.

After a disappointing Phase 3 trial of AMX0035 failed to produce significant differences versus a placebo,[8] on April 4, 2024 Amylyx announced they would begin the process of withdrawing Albrioza and Relyviro from the North American market.

[9] Amylyx received praise for keeping its pre-approval promise to stop selling the drug if it turned out to be ineffective, an unusually ethical move which the FDA rarely requires.