Phase 3, Open-Label, Safety Extension Study of Radicava ORS® (Oral Edaravone) Administered Over 96 Weeks in Patients with ALS (MT-1186-A03)

Topic:

Clinical Trials

Poster Number: P270

Author(s):

Angela Genge, MD, Clinical Research Unit, The Montreal Neurological Institute, Montreal, QC, Canada, Gary Pattee, MD, University of Nebraska, Gen Sobue, MD, PhD, Nagoya University Graduate School of Medicine, Nagoya; Aichi Medical University, Aichi, Japan, Masashi Aoki, MD, Tohoku University School of Medicine, Sendai, Miyagi, Japan, Hiide Yoshino, MD, Neurology Clinic, Chiba, Japan, Philippe Couratier, MD, PhD, CRMR SLA Service de Neurologie, CHU de Limoges, Limoges, France, Christian Lunetta, MD, Istituti Clinici Scientifici Maugeri IRCCS, Neurorehabilitation Unit of Milano, Milan, Italy, Susanne Petri, MD, Hannover Medical School, Hannover, Germany, Daniel Selness, RN, BA, MBA, Mitsubishi Tanabe Pharma America, Inc., Jersey City, New Jersey, USA, Vesna Todorovic, MD, MPhil, Mitsubishi Tanabe Pharma Europe, Ltd, London, United Kingdom, Manabu Hirai, MS, Mitsubishi Tanabe Pharma Corporation, Tokyo, Japan, Alejandro Salah, MD, PhD, MBA, MHA, Mitsubishi Tanabe Pharma America, Inc., Jersey City, New Jersey, USA, Stephen Apple, MD, Mitsubishi Tanabe Pharma America, Inc., Jersey City, New Jersey, USA, Art Wamil, MD, PhD, Mitsubishi Tanabe Pharma America, Inc., Jersey City, New Jersey, USA, Alexander Kalin, MS, Mitsubishi Tanabe Pharma America, Inc., Jersey City, New Jersey, USA, Carlayne E. Jackson, MD, FAAN, University of Texas Health Science Center at San Antonio, San Antonio, Texas, USA

BACKGROUND: Radicava® (edaravone) IV (intravenous; Mitsubishi Tanabe Pharma America [MTPA], hereafter “MTPA IV edaravone”) and Radicava ORS® (edaravone) oral suspension (MTPA, hereafter “MTPA oral edaravone”) were approved by the US Food and Drug Administration (FDA) for the treatment of amyotrophic lateral sclerosis (ALS) in 2017 and 2022, respectively, and studies have demonstrated these approved formulations have similar pharmacokinetics. Study MT-1186-A01 indicated that MTPA oral edaravone was well-tolerated over 48 weeks, with no new safety concerns identified.

OBJECTIVE: To evaluate the safety of MTPA oral edaravone in patients with ALS over 96 weeks.

RESULTS: Study MT-1186-A03 (NCT04577404) was a phase 3, open-label, multi-center, extension study that evaluated the long-term safety of MTPA oral edaravone over an additional 96 weeks in patients who have completed the initial 48 weeks of Study MT-1186-A01. Participants received MTPA oral edaravone (105-mg dose) according to the FDA-approved dosing for MTPA IV edaravone. Upon entering Study MT-1186-A01, patients had definite, probable, probable-laboratory-supported, or possible ALS; baseline forced vital capacity ≥70%; and baseline disease duration ≤3 years. In Study MT-1186-A03, MTPA oral edaravone was well tolerated with no new safety concerns. The most common treatment-emergent adverse events (TEAEs) were fall, muscular weakness, dyspnea, constipation, and dysphagia. These TEAEs were consistent with the safety profile for MTPA edaravone from previous clinical trials.

CONCLUSIONS: MTPA oral edaravone showed no new safety concerns and was well-tolerated during the 96-week study period of MT-1186-A03, for a total of 144 weeks when including Study MT-1186-A01.

Sponsorship: This study was sponsored by Mitsubishi Tanabe Pharma America, Inc.

Acknowledgments: The authors thank Irene Brody, VMD, PhD, of p-value communications, Cedar Knolls, NJ, USA, for providing medical writing support. Editorial support was also provided by p-value communications. This support was funded by Mitsubishi Tanabe Pharma America, Inc., Jersey City, NJ, USA, in accordance with Good Publication Practice Guidelines 2022.