Interim Analysis of the Radicava/Edaravone Findings in Biomarkers from ALS (REFINE-ALS) Study


Clinical Trials

Poster Number: M198


James Berry, MD, Massachusetts General Hospital, Eric A. Macklin, PHD, MS, Massachusetts General Hospital, Alexander V. Sherman, MS, Massachusetts General Hospital, Hong Yu, MS, Massachusetts General Hospital, Vinieth N. Bijanki, Massachusetts General Hospital, Ashlyn D. Butowski, Massachusetts General Hospital, Reena Maharaj, Massachusetts General Hospital, Stephen Apple, MD, Mitsubishi Tanabe Pharma America, Inc., Takuya Kudo, MS, Mitsubishi Tanabe Pharma Corporation, Kinjal Patel, MHA, MBA, Mitsubishi Tanabe Pharma America, Inc.

Background: Radicava® (edaravone) injection gained FDA approval for treatment of ALS based on clinical trial results demonstrating slowing of physical functional decline. Radicava ORS® (edaravone) oral suspension was FDA-approved in May 2022. An assessment of the effects of edaravone on biomarkers in ALS could provide insights into its mechanism of action. Although there are few validated biomarkers of ALS, an array of biomarkers are proposed.

Objectives: Assessment of biomarkers in the REFINE-ALS study may identify quantifiable, biological, nonclinical measures of the pharmacodynamic effect of edaravone in ALS.

Results: This prospective, observational, longitudinal, multicenter US study is following adults with ALS who initiate edaravone (intravenous or oral formulation) treatment as part of their clinical care for ~24 weeks (6 cycles of edaravone) to assess changes in putative biomarkers, both from ALS progression and associated with edaravone treatment.

Assessments at baseline and during cycles 1, 3, and 6, include biomarkers for oxidative stress, inflammation, neuronal injury and death, muscle injury, epigenetics (EpiSwitch™), and the SOMAscan® panel. DNA samples are being collected for genomic sequencing. Historical controls for patients not taking edaravone from the Answer ALS study were matched on baseline characteristics with REFINE-ALS participants for comparisons of results.

Clinical efficacy assessments are performed, and adverse events related to study procedures are being tracked. As of June 2023, 73 participants were enrolled and will be matched with participants from Answer ALS. Biomarker and clinical data from this interim analysis will be presented.

Conclusions: Results from this ongoing study may help to identify prognostic and disease monitoring markers of ALS and predictive and pharmacodynamic biomarkers of edaravone effect. We look forward to gathering more biomarker data evaluating the efficacy of edaravone in treating people with ALS in a real-world setting.

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