On/off dosing regimens of Radicava® (edaravone) IV (intravenous; MTPA) and Radicava ORS® (edaravone) oral suspension (MTPA, hereafter “MTPA oral edaravone”) were US FDA-approved for ALS treatment in 2017 and 2022, respectively. Placebo-controlled trials showed MTPA IV edaravone slows functional decline in patients with ALS vs placebo, though no studies have investigated function and survival of MTPA oral edaravone vs placebo.

Study MT-1186-A01, a phase 3, open-label, 48-week study, evaluated long-term safety and tolerability of MTPA oral edaravone approved on/off dosing, Study MT-1186-A03 was its 96-week extension. Study MT-1186-A02, a phase 3b, double-blind, parallel group study that randomized patients to once daily or approved on/off MTPA oral edaravone regimen, Study MT-1186-A04 was its 48-week extension. Patients from Studies MT-1186-A02/A04 and Studies MT-1186-A01/A02/A03/A04 were propensity score–matched 1:1 on 10 baseline variables with historical, external PRO-ACT placebo patients (not receiving active investigational treatment in their respective clinical trials).

Analysis of MTPA oral edaravone–treated patients with ALS from MT-1186 studies suggests MTPA oral edaravone significantly increases survival and decreases functional decline vs PRO-ACT placebo.