Abstract Details

Title 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

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P1 - Poster Session 1 (8:00 AM-9:00 AM)

Poster/Presentation
Number 11-001

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.

Authors/Disclosures
Angela L. Genge, MD (Mcgill University) PRESENTER	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 pepgen. 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.
Gary L. Pattee, MD (Neurology Associates PC)	The institution of Dr. Pattee has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MTPA pharmaceuticals. The institution of Dr. Pattee has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for MTPA pharmaceiticals. The institution of Dr. Pattee has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Catalyst. The institution of Dr. Pattee has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for General Dynamics US military. The institution of Dr. Pattee has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Otsuka pharmaceuticals.
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..
Philippe Couratier, PhD	Dr. Couratier has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Couratier has received personal compensation in the range of $500-$4,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Elsevier. Dr. Couratier has received personal compensation in the range of $5,000-$9,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Elsevier .
Daniel Selness (Mitsubishi Tanabe Pharma America, Inc)	Mr. Selness has received personal compensation for serving as an employee of Mitsubishi Tanabe.
	No disclosure on file
Takeshi Sakata	Takeshi Sakata has received personal compensation for serving as an employee of Mitsubishi Tanabe.
Alejandro Salah, MD, PhD, MBA, MHA	Dr. Salah has received personal compensation for serving as an employee of Mitsubishi Tanabe Pharma.
Stephen Apple	Stephen Apple has received personal compensation for serving as an employee of Mitsubishi Tanabe Pharma America, Inc.

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