Extension Study MT-1186-A04 Evaluating Oral Edaravone (Radicava ORS®) Continued Efficacy and Safety up to an Additional 48 Weeks in Patients With ALS
Angela Genge1, Jeffrey Rothstein2, Shari De Silva3, Lorne Zinman4, Marvin Chum5, Adriano Chio6, Gen Sobue7, Manabu Doyu8, Daniel Selness9, Vesna Todorovic10, Nissim Sasson11, Fumihiro Takahashi9, Michelle Cecic9, Art Wamil9, Stephen Apple9
1Montreal Neurological Institute and Hospital, 2Department of Neurology, School of Medicine, Johns Hopkins University, 3Woodland Research Northwest, 4Sunnybrook Health Sciences Centre, University of Toronto, 5McMaster University Health Sciences Centre, 6Università degli Studi di Torino, Centro Regionale Esperto per la Sclerosi Laterale Amiotrofica (CRESLA), 7Nagoya University Graduate School of Medicine, Aichi Medical University, 8Department of Neurology, Aichi Medical University, 9Mitsubishi Tanabe Pharma America, Inc., 10Mitsubishi Tanabe Pharma Europe, Ltd., 11NStat Solutions, Biostatistical Services
Objective:
Continue to examine the efficacy and safety of investigational once daily and FDA-approved on/off Radicava ORS® (edaravone) oral suspension (Mitsubishi Tanabe Pharma America [MTPA], hereafter “MTPA oral edaravone”) dosing regimens in patients with amyotrophic lateral sclerosis (ALS) for up to an additional 48 weeks in extension Study MT-1186-A04.
Background:
On/off dosing of Radicava® (edaravone) IV (intravenous; MTPA, hereafter “MTPA IV edaravone”) and MTPA oral edaravone were US Food and Drug Administration (FDA)-approved for ALS treatment in 2017 and 2022, respectively. Clinical trials showed MTPA edaravone slows physical functional decline in patients with ALS.
Design/Methods:
Study MT-1186-A04 (NCT05151471) was a phase 3b, multi-center, randomized, double-blind, parallel group extension study for up to an additional 48 weeks following the initial 48 weeks of Study MT-1186-A02, where patients had been randomized to investigational once daily or FDA-approved on/off dose of MTPA oral edaravone (105-mg dose). Patients who met Study MT-1186-A04 eligibility criteria, including Study MT-1186-A02 visit completion, continued in the same treatment regimen they were on during Study MT-1186-A02. The primary efficacy endpoint for MT-1186-A04 was time from randomization in Study MT-1186-A02 to ≥12-point decrease in ALS Functional Rating Scale-Revised (ALSFRS-R) or death, whichever happened first.
Results:
Over 96 weeks including the Study MT-1186-A02 treatment period, results for the primary endpoint indicated daily dosing did not show a statistically significant difference vs FDA-approved on/off dosing. MTPA oral edaravone was well-tolerated and no new safety concerns were identified in either group in Study MT-1186-A04.
Conclusions:
Similar to the results obtained in MT-1186-A02, in MT-1186-A04, daily MTPA oral edaravone did not show superiority to the FDA-approved on/off regimen (same safety, efficacy and tolerability profile) from the time of the randomization date in Study MT-1186-A02 to ≥12-point decrease in ALSFRS-R or death, whichever happened first, and reinforces the appropriateness of the FDA-approved regimen.
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