FDA gives Amylyx green light for phase 1 ALS trial after clinical hold

After raising questions surrounding dosing levels, the FDA has freed Amylyx’s early-stage amyotrophic lateral sclerosis (ALS) candidate from a clinical hold.

When Amylyx had asked the FDA to approve the launch of in-human studies for AMX0114—an investigational antisense oligonucleotide—the agency restricted dosing to an amount that was lower than the biopharma’s proposed starting dose of 12.5 mg, according to a November 2024 company release.