Mitsubishi Tanabe Pharma America Presents New Results of RADICAVA ORS® (edaravone) from a Phase 3b Efficacy and Safety Extension Study and a Clinical Analysis of Multiple Studies at NEALS 2024

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Oct 21, 2024, 09:00 ET

Phase 3b extension study reinforces current FDA-approved on/off regimen of RADICAVA ORS

Analyses suggest improved survival in people with ALS on RADICAVA ORS compared to PRO-ACT placebo group

JERSEY CITY, N.J., Oct. 21, 2024 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced new findings from two studies of RADICAVA ORS^® (edaravone) presented at the Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) 2024 Annual Meeting. The company presented results from the MT-1186-A04 Study, a Phase 3b extension of the previously reported MT-1186-A02 study which explored the superiority of investigational once daily dosing versus the FDA-approved on/off regimen of RADICAVA ORS in people living with ALS. MTPA also shared results from primary and post-hoc analyses of RADICAVA ORS compared with Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) historical placebo controls, with evidence suggesting treatment with RADICAVA ORS increased survival outcomes and decreased physical function decline versus controls.

"We're pleased to share the findings of the Phase 3b extension study and an analysis of long-term function, which highlight additional data about the impact that RADICAVA ORS can have on function and survival for people living with ALS," said Gustavo A. Suarez Zambrano, M.D., Vice President of Medical Affairs at MTPA. "These results reinforce our commitment to delivering valuable insights to the ALS and medical communities."

Poster Presentation: Phase 3b Extension Study MT-1186-A04 to Evaluate the Continued Efficacy and Safety of Radicava ORS^® (Oral Edaravone) for up to an Additional 48 Weeks in Patients with Amyotrophic Lateral Sclerosis

The randomized, double-blind, multi-center extension study MT-1186-A04 followed patients for 48 weeks after completion of study MT-1186-A02, in which patients had been treated with either investigational once daily or on/off FDA-approved dosing of RADICAVA ORS.
Daily dosing did not show superiority to the approved on/off dosing. No significant difference in ALS Functional Rating Scale-Revised (ALSFRS-R) score reductions between the two groups reinforces that the on/off regimen of RADICAVA ORS is the most appropriate regimen for patients with ALS.

Poster Presentation: Analysis of Long-term Function and Survival of Radicava ORS^®(Oral Edaravone)-Treated Patients with Amyotrophic Lateral Sclerosis vs Propensity Score–Matched PRO-ACT Historical Controls

Patients enrolled in the RADICAVA ORS Studies MT-1186-A02/A04 (prespecified analysis) and Studies MT-1186-A01/A02/A03/A04 (post hoc analysis) were propensity score-matched 1:1 on 10 baseline variables with historical, external, PRO-ACT controls (placebo-arm participants in their respective trials).
These propensity score–matched cohort analyses provide evidence for a slowing of functional decline and improved survival outcomes with long-term RADICAVA ORS treatment versus PRO-ACT placebo group patients with ALS.

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.¹

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 2.0-million days of therapy, and have been prescribed by over 2,400 HCPs.^2-4

IMPORTANT SAFETY INFORMATION

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 Data on file. Mitsubishi Tanabe Pharma America, Inc.
³ Data on file. Mitsubishi Tanabe Pharma America, Inc.
⁴ Data on file. Mitsubishi Tanabe Pharma America, Inc.

SOURCE Mitsubishi Tanabe Pharma America