Mitsubishi Tanabe Pharma Canada, Inc. Announces Publication of Long-Term Function and Survival Analysis of RADICAVA® Oral Suspension (edaravone) -Treated Patients with ALS

Results from this analysis were published in Muscle and Nerve

TORONTO, Sept. 4, 2025 /CNW/ - Mitsubishi Tanabe Pharma Canada, Inc. (MTP-CA), a subsidiary of Mitsubishi Tanabe Pharma America, Inc. (MTPA), today announced the publication of a retrospective analysis of patients living with amyotrophic lateral sclerosis (ALS) in Muscle and Nerve.¹ The analysis evaluated patients taking RADICAVA^® Oral Suspension (edaravone) compared with Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) ² historical placebo controls (patients who did not receive active investigational treatment, but may have received riluzole in their respective clinical trials), with evidence suggesting treatment with RADICAVA^® Oral Suspension decreased physical functional decline and offering insights into survival.

"ALS remains a fatal neurodegenerative disease with no cure, highlighting the critical need for treatments that can help slow functional decline," said Dr. Angela Genge, Neurologist and Director, ALS Centre of Excellence for Research and Patient Care at The Neuro (Montreal Neurological Institute-Hospital). "This analysis adds to our growing understanding of the potential impact of RADICAVA^® Oral Suspension on disease progression in ALS, and provides valuable insights for healthcare professionals and Canadians impacted by this disease."

The primary analysis, based on data from clinical studies MT-1186-A02/A04, evaluated long-term survival and functional outcomes of 78 propensity score-matched patients treated with RADICAVA^® Oral Suspension compared to 78 matched historical placebo controls from the PRO-ACT database.

The main statistical comparison was made between the Combined (FDA approved On/Off + investigational Once Daily) RADICAVA^® Oral Suspension versus PRO-ACT placebo group. The post-hoc analysis, based on data from clinical studies MT-1186-A01/A02/A03/A04, expanded the evaluation to a broader ALS population (n=210 RADICAVA^® Oral Suspension patients vs. 210 PRO-ACT placebo patients) to confirm robustness of the initial results.

Propensity score matching on 10 baseline variables was used to assess the impact of RADICAVA^® Oral Suspension on function and survival. The analysis showed evidence for a slowing of functional decline and improved survival outcomes with long-term RADICAVA^® Oral Suspension treatment versus PRO-ACT placebo group patients with ALS. The analysis also showed a smaller ALS Functional Rating Scale-Revised (ALSFRS-R) change from baseline, demonstrating a slower rate of functional decline observed over 48 weeks.

Results from this analysis are not generalizable and cannot be used to determine definitive conclusions about the effects of treatment. Results should be interpreted with caution.

This analysis was funded and conducted by MTPA. The full publication is titled, "Analysis of Long-term Function and Survival of Edaravone Oral Suspension-Treated Patients With Amyotrophic Lateral Sclerosis Using PRO-ACT Data as Historical Placebo Controls."

Healthcare professionals may contact MTP-CA's Medical Affairs department for more information.

About the PRO-ACT Database
PRO-ACT Dataset is the world's largest ALS clinical trial data repository, compiling placebo and treatment-arm data from 36 phase II/III clinical trials and over 12,500 fully anonymized longitudinal Subject records funded by The ALS Therapy Alliance, Prize4Life, Inc., Northeast ALS Consortium (NEALS), Neurological Clinical Research Institute of Mass. General Hospital, ALS Finding A Cure, and The ALS Association. Neurological Clinical Research Institute of Mass. General Hospital created and maintained the PRO-ACT Dataset and serves as the coordinating center and data distributor of the PRO-ACT Dataset. Find out more at www.alsdata.org.

About Studies MT-1186-A01/A02/A03/A04
Study MT-1186-A01 (NCT04165824) was a phase 3, global, multicenter, open-label study that evaluated the long-term safety and tolerability of the approved edaravone oral suspension 105 mg On/Off dosing regimen over 48 weeks, and Study MT-1186-A03 (NCT04577404) was its extension study, which provided up to 96 weeks of additional treatment. Study MT-1186-A02 (NCT04569084), a post-marketing commitment following the FDA approval of RADICAVA^® (edaravone), was a phase 3b, multicenter, double-blind, parallel-group, randomized study that evaluated whether investigational Once Daily edaravone oral suspension dosing was superior to the approved On/Off dosing based on Combined Assessment of Function and Survival, and assessed safety and tolerability over 48 weeks in patients with ALS; and Study MT-1186-A04 (NCT05151471) was its extension study, which provided up to 48 weeks of additional treatment.

In Study MT-1186-A02, a pre-planned futility analysis was conducted after 50% of the planned study population (N=190) reached 48 weeks, which assessed the study's primary endpoint and the probability of the study results changing if all participants completed the 48-week study period. Through that interim analysis, the Independent Data Monitoring Committee (IDMC) concluded that there was a low statistical probability for the investigational once-daily dosing regimen to show superiority to the current on/off dosing regimen as measured by the ALS Functional Rating Scale Revised (ALSFRS-R) score at study completion; therefore, study discontinuation was recommended by the IDMC.

About RADICAVA^® Oral Suspension (edaravone)
RADICAVA^® Oral Suspension is indicated for the treatment of patients with amyotrophic lateral sclerosis (ALS). Edaravone was discovered and developed for ALS by Mitsubishi Tanabe Pharma Corporation (MTPC) through an iterative clinical development platform over a 13-year period.

In 2015, edaravone was approved for use as a treatment for ALS in Japan and South Korea. RADICAVA^® was approved by the U.S. Food and Drug Administration (FDA) in May 2017. Marketing authorization for RADICAVA^® IV Infusion was granted in Canada (October 2018), Switzerland (January 2019), Indonesia (July 2020), Thailand (April 2021), and Malaysia (December 2021). In October 2024, Mitsubishi Tanabe Pharma Canada, Inc. (MTP-CA), announced the strategic business decision to discontinue RADICAVA^® IV infusion as fewer people living with ALS were still using RADICAVA^® IV. MTP-CA continues to market the oral formulation, RADICAVA^® Oral Suspension.

RADICAVA^® ORS (edaravone) was approved by the U.S. FDA in May 2022. RADICAVA^® Oral Suspension (edaravone) was authorized by Health Canada in November 2022.

About Mitsubishi Tanabe Pharma Canada, Inc.
Based in Toronto, Mitsubishi Tanabe Pharma Canada, Inc. (MTP-CA) is a wholly owned subsidiary of Mitsubishi Tanabe Pharma America, Inc. (MTPA), with a goal to provide therapies for some of the most difficult-to-treat diseases, including ALS. For more information, please visit www.mt-pharma-ca.com.

About Mitsubishi Tanabe Pharma America, Inc.
Based in Jersey City, N.J., Mitsubishi Tanabe Pharma America, Inc. (MTPA) is a wholly owned subsidiary of Mitsubishi Tanabe Pharma Corporation (MTPC). It was established by MTPC to develop and advance its pipeline as well as commercialize approved pharmaceutical products in North America. For more information, please visit www.mt-pharma-america.com or follow us on X (formerly Twitter), Facebook and LinkedIn.

References:

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¹ Takahashi F, Genge A, Hirai M, et al. Analysis of Long-Term Function and Survival of Edaravone Oral Suspension-Treated Patients With Amyotrophic Lateral Sclerosis Using PRO-ACT Data as Historical Placebo Controls. Muscle Nerve. Published online July 1, 2025. Doi:10.1002/mus.28462

² Pooled Resource Open-Access ALS Clinical Trials Database (PRO-ACT). Neurological Clinical Research Institute, Massachusetts General Hospital. Https://ncri1.partners.org/PROACT.

SOURCE Mitsubishi Tanabe Pharma Canada, Inc.

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