Abstract Details

Title Extension Study MT-1186-A04 Evaluating Oral Edaravone (Radicava ORS®) Continued Efficacy and Safety up to an Additional 48 Weeks in Patients With ALS

Topic General Neurology

Presentation(s) P9 - Poster Session 9 (11:45 AM-12:45 PM)

Poster/Presentation
Number 2-007

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.

Authors/Disclosures
Stephen Apple PRESENTER	Stephen Apple has received personal compensation for serving as an employee of Mitsubishi Tanabe Pharma America, Inc.
Angela L. Genge, MD (Mcgill University)	Dr. Genge has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for AL-S Pharma. Dr. Genge has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Amylyx. Dr. Genge has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Quralis. Dr. Genge has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MTPA. Dr. Genge has received personal compensation in the range of $0-$499 for serving as a Consultant for WAVE. Dr. Genge has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for eikonizo. Dr. Genge has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for rapa.
Jeffrey D. Rothstein, MD, PhD (Johns Hopkins University)	Dr. Rothstein has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Corcept Therapeutics. Dr. Rothstein has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Lilly Pharmaceuticasl. Dr. Rothstein has received personal compensation in the range of $0-$499 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for ALS Journal. The institution of Dr. Rothstein has received research support from ALSA. The institution of Dr. Rothstein has received research support from MDA. The institution of Dr. Rothstein has received research support from Target ALS. The institution of Dr. Rothstein has received research support from Maryland TEDCO. The institution of Dr. Rothstein has received research support from Dept of Defense. The institution of Dr. Rothstein has received research support from National Insitutes of Health. The institution of Dr. Rothstein has received research support from Corsalex. Dr. Rothstein has received intellectual property interests from a discovery or technology relating to health care.
Shari DeSilva, MD (VAMC)	Dr. DeSilva has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Woodland Research . Dr. DeSilva has received personal compensation in the range of $500,000-$999,999 for serving as a Consultant for Woodland Research.
Lorne H. Zinman, MD	Dr. Zinman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen, MTP, AB Science, Cytokinetics, Amylyx. Dr. Zinman has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amylyx.
Marvin C. Chum, MD (St. Joseph's Healthcare Hamilton - Neuro-Diagnostics)	Dr. Chum has nothing to disclose.
Adriano Chio, MD, FAAN (Dept. of Neuroscience, University of Turin)	Dr. Chio has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cytokinetics. Dr. Chio has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Mitsubishi. Dr. Chio has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Chio has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Corcept.
Gen Sobue, MD (Nagoya University School Of Medicine/Dept. of Neurology)	Dr. Sobue has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Mistubishi Tanabe Pharma Corporation. Dr. Sobue has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Nippon Chemiphar Co., Ltd.. Dr. Sobue has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Mistubishi Tanabe Pharma Corporation. Dr. Sobue has received personal compensation in the range of $0-$499 for serving as an Expert Witness for Takeda Pharmaceutical Company Limited..
Manabu Doyu, MD, PhD	Prof. Doyu has nothing to disclose.
Daniel Selness (Mitsubishi Tanabe Pharma America, Inc)	Mr. Selness has received personal compensation for serving as an employee of Mitsubishi Tanabe.
Vesna Todorovic, MD	Dr. Todorovic has nothing to disclose.
Nissim Sasson, Sr.	Mr. Sasson has nothing to disclose.
Fumihiro Takahashi, PhD	Dr. Takahashi has nothing to disclose.
Michelle Cecic	Mrs. Cecic has nothing to disclose.
Arthur Wamil, MD, PhD	Dr. Wamil has received personal compensation for serving as an employee of Mitsubishi Tanabe.

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