24-Week Results From Phase 3 Study MT-1186-A01 an Open-Label, Multicenter Safety Study of Oral Edaravone in Subjects With Amyotrophic Lateral Sclerosis
Angela Genge1, Gary L. Pattee2, Gen Sobue3, Philippe Couratier4, Daniel Selness5, Manabu Hirai6, Takeshi Sakata6, Alejandro Salah7, Stephen Apple7
1Mcgill University, 2University of Nebraska Medical Center, 3Nagoya University Graduate School of Medicine and Aichi Medical University, 4CRMR SLA Service de Neurologie, CHU de Limoges, 5Mitsubishi Tanabe Pharma Development America, Inc., 6Mitsubishi Tanabe Pharma Corporation, 7Mitsubishi Tanabe Pharma America, Inc.
Objective:
To assess long-term safety and tolerability of investigational oral edaravone (MT-1186) in patients with amyotrophic lateral sclerosis (ALS) over 24 weeks.
Background:
Radicava® (edaravone) is a US Food and Drug Administration–approved treatment for 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 oral formulation of edaravone.
Design/Methods:

An ongoing, global, multicenter, open-label, phase 3 study is evaluating the long-term safety and tolerability of investigational oral edaravone in patients with ALS. The study includes an open-label treatment period of 48 weeks, with primary assessments at Weeks 24 and 48. Entry criteria include 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 are functioning independently.

Patients receive a 105-mg dose of oral edaravone administered in treatment cycles. In addition to the primary safety analysis, the study also includes exploratory end points, 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.
Results:

A total of 185 patients were enrolled. Overall, oral edaravone was well tolerated in the study. The most common treatment-emergent adverse events were 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 formulation of edaravone in patients with ALS.