Post-Hoc Study Analysis Examines Early Intervention with Edaravone for ALS Survival

Mitsubishi Tanabe Pharma America announced a post-hoc analysis of its Phase 3 edaravone study reviewing the results of intravenous (IV) edaravone treatment on disease progression milestones and events among people with amyotrophic lateral sclerosis (ALS). In the analysis, a risk reduction was observed for the exploratory composite estimate of time to death, tracheostomy, permanent assisted ventilation (PAV), and hospitalization. The data was highlighted as an oral presentation at the 2021 Muscular Dystrophy Association Clinical & Scientific Virtual Conference.

"While the Phase 3 edaravone study was not designed with survival as an endpoint, this post-hoc analysis allows us to explore insights on the results of early treatment intervention on survival-related events due to ALS progression, including death, hospitalization, ventilation and tracheostomy," said Atsushi Fujimoto, President, MTPA. "We are committed to putting patients first in everything we do, which includes working to explore our Phase 3 data in order to help expand understanding."

This post-hoc analysis assessed the double-blind, placebo-controlled 24-week Phase 3 study of edaravone, including an additional 24-week open-label period with all patients receiving IV edaravone (i.e., edaravone-edaravone [EE] vs. placebo-edaravone [PE] groups). There were no deaths during the double-blind period. During the open-label period, there were two deaths in the EE group and four in the PE group. Survival analysis of the cumulative occurrence of milestone events (death, tracheostomy, PAV and hospitalization due to ALS progression) showed a 53 percent relative risk reduction in the EE group, with a hazard ratio of 0.47. The hazard ratio for death alone was 0.48 for the EE group vs. the PE group. As these results are from a post-hoc analysis, the results should be interpreted with caution and cannot be considered conclusive evidence of efficacy.

"I'm encouraged by the findings of this post-hoc analysis," said Benjamin Rix Brooks, M.D., an ALS specialist and lead author of the study. "We were able to uncover data that reinforces the need to further our research that can show an important association between edaravone use and the potential decrease in functional decline."

This analysis was funded and conducted by MTPA.

The U.S. Food and Drug Administration (FDA) approved RADICAVA® (edaravone) IV on May 5, 2017 as a treatment for amyotrophic lateral sclerosis (ALS). Edaravone was discovered and developed for ALS by Mitsubishi Tanabe Pharma Corporation (MTPC) and commercialized in the U.S. by Mitsubishi Tanabe Pharma America, Inc. MTPC group companies began researching ALS in 2001 through an iterative clinical platform over a 13-year period. In 2015, edaravone IV was approved for use as a treatment for ALS in Japan and South Korea. Marketing authorization was granted in Canada in October 2018 and Switzerland in January 2019.

RADICAVA is indicated for the treatment of amyotrophic lateral sclerosis (ALS).