Background: Radicava® (edaravone injection) is a US FDA-approved treatment for amyotrophic lateral sclerosis (ALS) that has been shown to slow the rate of physical functional decline. There is interest in a non-intravenous formulation of edaravone; an ongoing, phase 3 study is currently assessing the safety and tolerability of an investigational formulation of oral edaravone.
Objectives: To assess long-term safety and tolerability of investigational oral edaravone (MT-1186) in patients with ALS over 24 weeks.
Results: The global, multicenter, open-label, phase 3 study evaluated the long-term safety and tolerability of investigational oral edaravone in patients with ALS. The study included an open-label treatment period of 48 weeks, with primary assessments at Weeks 24 and 48. Entry criteria included adults with a diagnosis of definite ALS, probable ALS, probable laboratory-supported ALS, or possible ALS, according to El Escorial criteria; baseline forced vital capacity ?70% predicted; disease duration ?3 years; and who were functioning independently. Patients received a 105-mg dose of oral edaravone administered in treatment cycles. In addition to the primary safety analysis, the study also included exploratory endpoints, such as change from baseline in the revised ALS Functional Rating Scale (ALSFRS-R) score and time to death, tracheostomy, or permanent assisted mechanical ventilation.
A total of 185 patients were enrolled. Overall, oral edaravone was well tolerated in the study. The most common treatment-emergent adverse events were considered by investigators to be consistent with ALS progression and with the edaravone safety profile from previous clinical trials. No other safety concerns were identified.
Conclusions: The first phase 3 trial of oral edaravone provided important information on the long-term safety and tolerability of this new investigational formulation of oral edaravone in patients with ALS.