• A comprehensive review of ALS drug development reveals 53 new drug candidates in clinical trials between 2020-2022, with 13 compounds advancing to Phase 3 trials and showing promising therapeutic potential.
• Five drugs demonstrated particularly strong efficacy: high-dose methylcobalamin, masitinib, AMX0035, CNM-Au8, and tofersen, with AMX0035 receiving FDA approval as the third treatment for ALS.
• The success of clinical trials is influenced by patient population homogeneity, observation duration, and analysis strategies, highlighting the importance of refined trial design in ALS drug development.