Mitsubishi Tanabe Pharma America Presents 48-Week Results from Global Phase 3 Safety Clinical Study of RADICAVA ORS® (edaravone), an Oral Treatment for ALS

Data Presented at the European Network to Cure ALS (ENCALS) Meeting 2022

JERSEY CITY, N.J., June 1, 2022 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced 48-week results from the global Phase 3 open-label, multi-center clinical trial (MT-1186-A01) assessing the safety and tolerability of RADICAVA ORS^® (edaravone) in patients with amyotrophic lateral sclerosis (ALS). Details about the findings will be presented at the European Network to Cure ALS (ENCALS) Meeting 2022, being held in Edinburgh, Scotland, from June 1-3.

"We are pleased to present the 48-week safety results, which build upon the 24-week results from the global Phase 3 trial that showed a favorable safety profile to support the approval of RADICAVA ORS," said Gustavo A. Suarez Zambrano, M.D., Vice President of Medical Affairs at MTPA. "Patients are the primary focus of our work as we try to make a difference, and we strongly believe in the potential of the oral formulation of edaravone to help people with ALS."

RADICAVA ORS was approved by the U.S. Food and Drug Administration (FDA) on May 12, 2022, as an oral treatment for ALS.¹ The FDA approval of RADICAVA ORS was supported by several studies, including 24-week results from the global Phase 3 trial (MT-1186-A01) demonstrating the safety and tolerability profile of the treatment in 185 ALS patients (aged ≥18 years to 75 years) across 50 sites in the U.S., Canada, Europe and Japan.²

According to the 48-week results presented at ENCALS, treatment-emergent adverse events (TEAEs) reported by ≥10% of patients were fall (22.2%), muscular weakness (21.1%), constipation (17.8%), dyspnea (10.8%), dysphagia (10.3%) and back pain (10.3%). No serious TEAEs considered to be treatment-related by investigators were reported.

Sixteen subjects (8.6%) discontinued the study due to TEAEs. The most common TEAEs leading to discontinuation were respiratory failure, muscular weakness and pneumonia, consistent with the disease state. There were 12 deaths during the 48-week study period, and none of the deaths were related to the study drug (respiratory failure, worsening of ALS, pneumonia, acute respiratory failure, lung disorder, diabetic ketoacidosis, feeding disorder and suicide).

"I'm encouraged by the findings from the Phase 3 trial, which provide important information on the safety and tolerability of this new formulation of edaravone in patients with ALS," said Gary L. Pattee, M.D., a neurologist and ALS specialist based in Lincoln, Neb. "These data reinforce the potential significance of RADICAVA ORS and its important role in the treatment of this devastating disease."

In addition, the loss of physical function was evaluated as an Exploratory Endpoint and measured by the ALS Functional Rating Scale–Revised (ALSFRS-R), a validated rating instrument for monitoring the progression of disease in patients with ALS.³ The changes from baseline to all post-baseline visits until Week 48 in ALSFRS-R score were estimated using a mixed model for repeated measures (MMRM) analysis from 139 subjects who completed the study. At the beginning of the study, patients had an average ALSFRS-R score of 40 (SD 4.5). At Week 48, the average change from baseline in ALSFRS-R score was −11.3 (95% CI −12.6, −10.1).

Additionally, a separate presentation at the ENCALS meeting will highlight the design of an ongoing Phase 3 open-label extension study (MT-1186-A03) that is evaluating the long-term safety and tolerability profile of RADICAVA ORS for up to 96 weeks. Both the A01 and A03 studies are conducted by Mitsubishi Tanabe Pharma Development America, Inc. (MTDA).

Poster Presentation Details:

• Phase 3, Open-Label, Multicenter Safety Study of Oral Edaravone in Patients With Amyotrophic Lateral Sclerosis (MT-1186-A01): 48-Week Results (Angela Genge, M.D., FRCP; Montreal Neurological Institute and Hospital)
  Poster Session 2 (#i183): June 2, 5:00 p.m. – 6:30 p.m. BST
  
  
• Phase 3, Open-Label, Safety Extension Study of Investigational Oral Edaravone Administered Over 96 Weeks in Patients with ALS (MT-1186-A03) (Angela Genge, M.D., FRCP; Montreal Neurological Institute and Hospital)
  Poster Session 2 (#i184): June 2, 5:00 p.m. – 6:30 p.m. BST

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). RADICAVA is administered in 28-day cycles by 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 Mitsubishi Tanabe Pharma Development America, Inc (MTDA), 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, RADICAVA was approved for the treatment of ALS in Japan and South Korea. Marketing authorizations were subsequently granted in Canada (October 2018), Switzerland (January 2019), China (July 2019), Indonesia (July 2020), Thailand (April 2021) and Malaysia (December 2021). To date, in the U.S., RADICAVA has been used to treat over 6,500 patients, with nearly one-million days of therapy, and has been prescribed by more than 1,600 HCPs.⁴

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's (MTPC) 100 percent owned U.S. holding company, Mitsubishi Tanabe Pharma Holdings America, Inc. It was established by MTPC to commercialize approved pharmaceutical products in North America. For more information, please visit www.mt-pharma-america.com or follow us on Twitter, Facebook and LinkedIn.

About Mitsubishi Tanabe Pharma Development America, Inc.
The U.S. headquarters of Mitsubishi Tanabe Pharma Development America, Inc. (MTDA) is located in Jersey City, New Jersey. MTDA is a wholly-owned subsidiary of Mitsubishi Tanabe Pharma Corporation's 100 percent-owned U.S. holding company, Mitsubishi Tanabe Pharma Holdings America, Inc. For more information, please visit https://mt-pharma-development-america.com/.

About Mitsubishi Tanabe Pharma Corporation
Mitsubishi Tanabe Pharma Corporation (MTPC), the pharma arm of Mitsubishi Chemical Holdings Group (MCHC Group), is one of the oldest pharmaceutical companies in the world, founded in 1678, and focusing on ethical pharmaceuticals. MTPC is headquartered in Doshomachi, Osaka, the birthplace of Japan's pharmaceutical industry. The MCHC Group 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 prioritizing work on "precision medicine" to provide drugs with high treatment satisfaction by identifying patient populations with high potential for efficacy and safety, focusing on the disease areas of central nervous system and immuno-inflammation. In addition, MTPC is 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 ClinicalTrials.gov. 2019. Safety Study of Oral Edaravone Administered in Subjects With ALS. https://clinicaltrials.gov/ct2/show/NCT04165824.
³ Rutkove SB. Clinical measures of disease progression in amyotrophic lateral sclerosis. Neurotherapeutics. 2015;12(2):384-393.
⁴ Data on file. Mitsubishi Tanabe Pharma America, Inc.

SOURCE Mitsubishi Tanabe Pharma America, Inc.