Radicava found to prolong survival in real-world analysis

Presentations note no new concerns with safety, dosing regimen

Lindsey Shapiro, PhD avatar

by Lindsey Shapiro, PhD |

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Adding Radicava (edaravone) to riluzole prolongs survival of amyotrophic lateral sclerosis (ALS) patients by more than three months relative to riluzole alone, according to analyses of real-world data from the ALS/MND Natural History Study Consortium database.

These and other Radicava-related findings were presented by the therapy’s developer Mitsubishi Tanabe Pharma America (MTPA) at the European Network to Cure ALS (ENCALS) Meeting 2024, being held in Stockholm June 17-20.

“Access to real-world data is critical for complex rare diseases like ALS, and we are proud to collaborate with the ALS/MND Consortium to bring important real-world insights to the clinical community,” Gustavo A. Suarez Zambrano, MD, vice president of medical affairs at MTPA, said in a company press release.

Edaravone, the active ingredient in Radicava and Radicava ORS, works to reduce oxidative stress, a type of cellular damage that’s implicated in ALS-related neurodegeneration.

Radicava is given via into-the-vein infusions. Radicava ORS is taken as an oral liquid suspension via mouth or feeding tube. Both formulations are administered in 28-day cycles in which patients are treated on most or all days during the first 14 days, and remain off treatment for the second 14 days.

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Initial results from ongoing collaboration

In the poster, “Treatment Patterns and Survival Benefit of Edaravone–Treated People with Amyotrophic Lateral Sclerosis in the ALS/MND Natural History Consortium,” researchers described survival outcomes in Radicava-treated patients in the real-world setting using data from the ALS/MND Natural History Consortium database.

The study is part of an ongoing collaboration between the consortium and MTPA.

The analysis involved 176 patients receiving Radicava and another standard ALS therapy called riluzole (e.g. Rilutek, Tiglutik, Exservan) and 176 patients receiving riluzole alone. Both groups were generally matched in terms of factors, including disease severity.

Survival analyses over a period of 50 months, or just over four years, showed that median survival time with the Radicava/riluzole combination was 31.1 months (about 2.5 years), compared with 28.8 months, or a little under 2.5 years, with riluzole alone.

When adjusted for potentially influencing factors at treatment’s start, this group difference was statistically significant and amounted to a 3.2-month survival benefit with Radicava.

“Through our collaboration with MTPA, we are thrilled to highlight initial results from a real-world analysis of people living with ALS in our database,” said Alex Sherman, director of Massachusetts General Hospital’s Center for Innovation and Biomedical Informatics and the study’s principal investigator. “We hope to build this momentum as our analysis of treatment patterns in the database continues.”

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Radicava ORS studies find no new concerns

Through the poster “Phase 3, Open-Label, Safety Extension Study of Oral Edaravone Administered Over 96 Weeks in Patients with ALS (MT-1186-A03),” the researchers presented long-term safety findings of Radicava ORS at its approved dose.

The Phase 3 MT-1186-A03 extension study (NCT04577404) evaluated Radicava ORS’ two-year safety in patients who participated in an earlier Phase 3 safety study (NCT04165824).

MT-1186-A03 results showed that no new safety concerns emerged with longer-term Radicava ORS use. The most commonly reported adverse events were falls, muscle weakness, shortness of breath, constipation, and swallowing difficulties, consistent with the therapy’s known safety profile.

In a third poster, “Phase 3b Study MT-1186-A02 to Investigate the Superiority of Daily Dosing vs the FDA-approved On/Off Regimen of Oral Edaravone in Patients with ALS,” researchers shared final results from the Phase 3 MT-1186-A02 study (NCT04569084), a post-marketing study designed to compare the currently approved on/off dosing regimen of Radicava ORS with a once-daily dosing regimen among 384 ALS patients.

Similar to results of a pre-planned analysis conducted when half of the participants reached 48 weeks of treatment, final data showed that the two regimens were similarly effective for slowing disease progression and prolonging survival.

There were no new safety concerns with either regimen.

The findings show that the currently approved regimen is the most appropriate for ALS patients, the company said. This was also the conclusion of an external monitoring committee at the time of the pre-planned analysis, which said that there was a low probability of the daily-dosing regimen showing any benefits over the approved regimen by the trial’s end. The trial was stopped early at that time.

The study findings come at the two year-anniversary of Radicava ORS’s U.S. launch. Since June 2022, more than 9,600 ALS patients have been treated with the oral medication, and 82% have continued treatment for three months or more, according to MPTA.

“Helping people who are living with serious and debilitating diseases is our greatest motivator, and we are honored that for two years, Radicava ORS has provided people with ALS an oral treatment option,”  Yasutoshi Kawakami, MTPA’s president, said in a separate company press release. “We are encouraged by the ongoing research and work being done in the field as we remain dedicated to supporting the ALS community,” Kawakami said.