Extension Study Will Continue Evaluating Oral Edaravone

Mitsubishi Tanabe Pharma America (MTPA) has launched an extension study to assess the long-term effects of an investigational oral formulation of edaravone (MT-1186) for the treatment of amyotrophic lateral sclerosis (ALS).

“As a company, we strive to always put patients first in our efforts to understand this debilitating disease. We hope this extension study will help provide important information to the ALS community,” Atsushi Fujimoto, president of MTPA, said in a press release.

This open-label long-term extension study (NCT04577404) will continue to evaluate oral edaravone in ALS patients who previously participated and completed an earlier Phase 3 clinical trial (NCT04165824). Enrollment into the extension study is underway at three sites in the U.S. More information is available here.

The original open-label Phase 3 study, which became fully enrolled in November, was designed to evaluate the safety and tolerability of oral edaravone, given in the same dosing regimen of Radicava, an approved intravenous (into-the-vein) formulation of edaravone, for 48 weeks (nearly one year).

Patients who complete the original trial now may enroll in the study’s long-term extension study to continue treatment with oral edaravone for up to 96 weeks. As in the original trial, the investigational therapy will be given at a dose equivalent to that of Radicava in 28-day cycles — once daily for 10 days in the first 14 days, followed by 14 days without treatment.

The main goal of the extension study will be to evaluate the therapy’s long-term safety and tolerability, based on the incidence and severity of side effects observed throughout the 96-week treatment course.

Changes in participants’ ALS functional rating scale-revised (ALSFRS-R) scores — a measure of disability — and time until invasive breathing procedures or death also will be assessed.

Findings from the original Phase 3 trial and its long-term extension, both sponsored by Mitsubishi Tanabe Pharma Development America (MTDA), are expected to support future approval requests for this experimental oral formulation of edaravone.

Edaravone is a free-radical scavenger that helps eliminate potentially harmful oxidant molecules resulting from cells’ normal metabolic processes that can cause oxidative stress — cellular damage resulting from the excessive accumulation of free radicals. By removing free radicals and lowering oxidative stress, which also is thought to contribute to nerve cell death in ALS, edaravone is expected to slow ALS progression and reduce patients’ disability.

Approved in 2017, Radicava was the first ALS treatment to be approved in the U.S. in more than two decades. This intravenous formulation of edaravone also is approved to treat ALS in Japan, South Korea, Canada, and Switzerland.