Mitsubishi Tanabe Pharma America Presents Final Results from the Global, Phase 3b MT-1186-A02 Post-marketing Study of RADICAVA® (edaravone) in ALS

JERSEY CITY, N.J., June 17, 2024 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced the presentation of final results from the global, multi-center, double-blind, Phase 3b MT-1186-A02 study of oral edaravone in amyotrophic lateral sclerosis (ALS). Final results showed the daily dosing regimen of RADICAVA ORS^® (edaravone) did not show superiority to the U.S. Food and Drug Administration (FDA) approved on/off dosing regimen based on the primary endpoint of Combined Assessment of Function and Survival (CAFS) at 48 weeks. Both dosing regimens provided comparable change in ALSFRS-R from baseline to week 48. These findings suggest that the current on/off regimen of RADICAVA ORS is the most appropriate regimen for people living with ALS based on efficacy and safety findings from studies MT-1186-A02 and MCI186-19. ALSFRS-R changes observed at week 48 in Study MT-1186-A02 of RADICAVA ORS were similar to the post hoc assessment of the previous Japanese, Phase 3 Study MCI186-19 of intravenous (IV) RADICAVA^® (edaravone).^1,2 The MT-1186-A02 study found no new safety concerns and reinforces edaravone as a safe product. The full results are included in a poster presentation at the European Network for a Cure for ALS (ENCALS) 2024 meeting taking place in Stockholm, Sweden.

"The safety and efficacy of the on/off dosing regimen of oral edaravone in this Phase 3b post-marketing study was similar to the safety and efficacy of oral edaravone," said Gustavo A. Suarez Zambrano, M.D., Vice President of Medical Affairs at MTPA. "We extend our gratitude to the study participants, investigators, caregivers and clinical trial staff for completing this journey with us."

The MT-1186-A02 study, which was a post-marketing commitment following the FDA approval of intravenous (IV) RADICAVA^® (edaravone), was designed to evaluate the superiority of an investigational once-daily dosing regimen of oral edaravone (105 mg) vs. the FDA-approved on/off dosing regimen administered in 28-day cycles, in people with ALS over 48 weeks. A pre-planned futility analysis, conducted after 50% of the planned study population (N=190) reached 48 weeks, 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 is 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^® (edaravone) and RADICAVA ORS^® (edaravone)
The U.S. Food and Drug Administration (FDA) approved RADICAVA^® (edaravone) on May 5, 2017, and the oral formulation RADICAVA ORS^® (edaravone) on May 12, 2022, for the treatment of amyotrophic lateral sclerosis (ALS). In 2024, the FDA recognized RADICAVA ORS with Orphan Drug Exclusivity based on the major contribution to patient care of the innovative oral formulation. RADICAVA is administered in 28-day cycles by intravenous (IV) infusion. It takes 60 minutes to receive each 60 mg dose. For the initial cycle, the treatment is infused daily for 14 consecutive days, followed by a two-week drug-free period. All cycles thereafter are infused daily for 10 days within a 14-day period, followed by a two-week drug-free period. RADICAVA ORS is taken daily for 14 consecutive days followed by a 14-day drug-free period for the initial treatment cycle. For subsequent treatment cycles, RADICAVA ORS is taken for 10 days within a 14-day period followed by a 14-day drug-free period. RADICAVA ORS should be taken in the morning after overnight fasting. Patients should not eat or drink (except water) within one hour after taking RADICAVA ORS.³

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. (MTPA). The MTPC group companies began researching ALS in 2001 through an iterative clinical platform over a 13-year period. In 2015, edaravone was approved as RADICUT^® for the treatment of ALS in Japan and South Korea. Marketing authorizations were subsequently granted in Canada (October 2018), Switzerland (January 2019), Indonesia (July 2020), Thailand (April 2021), Malaysia (December 2021), and Brazil (February 2024). Marketing authorization for RADICAVA^® Oral Suspension was granted in Canada (November 2022) and Switzerland (May 2023), and RADICUT^® Oral Suspension 2.1% was granted regulatory approval in Japan in December 2022. To date, in the U.S., RADICAVA and RADICAVA ORS have been used to treat over 16,000 people with ALS, with over 1.9-million days of therapy, and have been prescribed by over 2,400 HCPs.^4-6

Hypersensitivity Reactions
RADICAVA (edaravone) and RADICAVA ORS (edaravone) are contraindicated in patients with a history of hypersensitivity to edaravone or any of the inactive ingredients of this product. Hypersensitivity reactions (redness, wheals, and erythema multiforme) and cases of anaphylaxis (urticaria, decreased blood pressure, and dyspnea) have occurred with RADICAVA.

Patients should be monitored carefully for hypersensitivity reactions. If hypersensitivity reactions occur, discontinue RADICAVA or RADICAVA ORS, treat per standard of care, and monitor until the condition resolves.

Sulfite Allergic Reactions
RADICAVA and RADICAVA ORS contain sodium bisulfite, a sulfite that may cause allergic-type reactions, including anaphylactic symptoms and life-threatening or less severe asthmatic episodes in susceptible people. The overall prevalence of sulfite sensitivity in the general population is unknown but occurs more frequently in asthmatic people.

Adverse Reactions
The most common adverse reactions (≥10%) reported in RADICAVA-treated patients were contusion (15%), gait disturbance (13%), and headache (10%). In an open label study, fatigue was also observed in 7.6% of patients receiving RADICAVA ORS.

Pregnancy
Based on animal data, RADICAVA and RADICAVA ORS may cause fetal harm.

To report suspected adverse reactions or product complaints, contact Mitsubishi Tanabe Pharma America, Inc., at 1-888-292-0058. You may also report suspected adverse reactions to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

INDICATION
RADICAVA and RADICAVA ORS are indicated for the treatment of amyotrophic lateral sclerosis (ALS).

For more information, including full Prescribing Information, please visit www.RADICAVA.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 our 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.

About Mitsubishi Tanabe Pharma Corporation
Mitsubishi Tanabe Pharma Corporation (MTPC), the pharma arm of Mitsubishi Chemical Group (MCG), is one of the oldest pharmaceutical companies in the world, founded in 1678. MTPC is headquartered in Doshomachi, Osaka, the birthplace of Japan's pharmaceutical industry. MCG has positioned health care as its strategic focus in its management policy, "Forging the future". MTPC sets the MISSION of "Creating hope for all facing illness". To that end, MTPC is working on the disease areas of central nervous system, immuno-inflammation, diabetes and kidney, and cancer. MTPC is focusing on "precision medicine" to provide drugs with high treatment satisfaction and additionally working to develop "around the pill solutions" to address specific patient concerns based on therapeutic medicine, including prevention of diseases, pre-symptomatic disease care, prevention of aggravation and prognosis. For more information, go to https://www.mt-pharma.co.jp/e/.

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¹ RADICAVA and RADICAVA ORS Prescribing Information. Jersey City, NJ: Mitsubishi Tanabe Pharma America, Inc.; 2022.
² Edaravone (MCI-186) ALS 19 Study Group. Safety and efficacy of edaravone in well defined patients with amyotrophic lateral sclerosis: a randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2017;16(7):505-512.
³ RADICAVA and RADICAVA ORS Prescribing Information. Jersey City, NJ: Mitsubishi Tanabe Pharma America, Inc.; 2022.
⁴ Data on file. Mitsubishi Tanabe Pharma America, Inc.
⁵ Data on file. Mitsubishi Tanabe Pharma America, Inc.
⁶ Data on file. Mitsubishi Tanabe Pharma America, Inc.

SOURCE Mitsubishi Tanabe Pharma America