Mitsubishi Tanabe Pharma America Announces Results from the Global Phase 3 Safety Study of Investigational Oral Edaravone in the Treatment of ALS

JERSEY CITY, N.J., Dec. 9, 2021 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced 24-week results from the global Phase 3 open-label study evaluating the safety and tolerability of investigational oral edaravone in the treatment of amyotrophic lateral sclerosis (ALS). Findings from the Phase 3 study, which is conducted by Mitsubishi Tanabe Pharma Development America, Inc. (MTDA), were presented during the Motor Neurone Disease Association (MNDA) virtual 32^nd International Symposium on ALS/MND (December 7-10).

"We are committed to putting patients first in everything we do and are working tirelessly in pursuit of an additional treatment option to help those living with ALS," said Gustavo A. Suarez Zambrano, M.D., Vice President of Medical Affairs at MTPA. "The 24-week treatment results showed a favorable safety profile for investigational oral edaravone. We are grateful to the study participants, their caregivers, investigators and clinical staff at the trial sites and the MTDA staff working diligently on this study."

The global multi-center, open-label study (MT-1186-A01), enrolled 185 ALS patients (aged ≥18 years to 75 years) across 50 sites in the U.S., Canada, Europe and Japan. The primary objective is to evaluate the 24 and 48 weeks safety and tolerability of oral edaravone in patients with ALS. Additionally, a long-term safety extension study (MT-1186-A03) to the ongoing Phase 3 trial will continue to evaluate oral edaravone in patients with ALS for up to 96-weeks.

The 24-week results presented at MNDA include findings from a total of 185 subjects included in the Safety Analysis Set who received at least one dose of study drug. Treatment emergent adverse events (TEAEs) reported by ≥5% of subjects were muscular weakness (16.2%), fall (15.7%), fatigue (7.6%), back pain (7.0%), constipation (7.0%), headache (5.9%) and dyspnea (5.4%). No serious TEAEs considered to be treatment-related by investigators were reported.

Eleven subjects (5.9%) discontinued the study due to TEAEs, two of them related to the study drug. The most common TEAEs were respiratory failure and muscular weakness, consistent with the disease state. There were six deaths during the 24-week study period, and none of the deaths were related to the study drug (respiratory failure (3), ALS (1), pneumonia (1) and suicide (1)).

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 disability in patients with ALS.¹ The changes from baseline to all post-baseline visits until Week 24 in ALSFRS-R score were estimated using a mixed model for repeated measures (MMRM) analysis from 169 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 24, the average change from baseline in ALSFRS-R score was −5.6 (95% CI −6.5, −4.8).

About Oral Edaravone (MT-1186)
MT-1186 is an investigational oral formulation of edaravone being studied in patients with amyotrophic lateral sclerosis (ALS). Edaravone was discovered and developed by researchers together with Mitsubishi Tanabe Pharma Corporation (MTPC), headquartered in Osaka, Japan. More information on the oral edaravone development program is available at ClinicalTrials.gov (NCT04165824 and NCT04577404).

About RADICAVA^® (edaravone) IV
The U.S. Food and Drug Administration (FDA) approved RADICAVA^® (edaravone) IV on May 5, 2017 as a treatment for amyotrophic lateral sclerosis (ALS).² 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. The MTPC group companies began researching ALS in 2001 through an iterative clinical platform over a 13-year period. In 2015, edaravone IV 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), and Thailand (April 2021).

INDICATION
RADICAVA is indicated for the treatment of amyotrophic lateral sclerosis (ALS).

IMPORTANT SAFETY INFORMATION
Before you receive RADICAVA, tell your healthcare provider about all of your medical conditions, including if you:

• have asthma.
• are allergic to other medicines.
• are pregnant or plan to become pregnant. It is not known if RADICAVA will harm your unborn baby.
• are breastfeeding or plan to breastfeed. It is not known if RADICAVA passes into your breast milk. You and your healthcare provider should decide if you will receive RADICAVA or breastfeed.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

What are the possible side effects of RADICAVA?

• RADICAVA may cause serious side effects including hypersensitivity (allergic) reactions and sulfite allergic reactions.
• Hypersensitivity reactions have happened in people receiving RADICAVA and can happen after your infusion is finished.
• RADICAVA contains sodium bisulfite, a sulfite that may cause a type of allergic reaction that can be serious and life-threatening. Sodium bisulfite can also cause less severe asthma episodes in certain people. Sulfite sensitivity can happen more often in people who have asthma than in people who do not have asthma.
• Tell your healthcare provider right away or go to the nearest emergency room if you have any of the following symptoms: hives; swelling of the lips, tongue, or face; fainting; breathing problems; wheezing; trouble swallowing; dizziness; itching; or an asthma attack (in people with asthma).
• Your healthcare provider will monitor you during treatment to watch for signs and symptoms of all the serious side effects.

The most common side effects of RADICAVA include bruising (contusion), problems walking (gait disturbance), and headache.

These are not all the possible side effects of RADICAVA. Call your healthcare provider for medical advice about side effects. You may report side effects to Mitsubishi Tanabe Pharma America, Inc. at 1-888-292-0058 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

For more information, including full Prescribing Information and Patient 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 Corporation
Mitsubishi Tanabe Pharma Corporation (MTPC), founded in 1678, is one of the oldest pharmaceutical companies in the world. Focused on ethical pharmaceuticals, MTPC is headquartered in Doshomachi, Osaka, the birthplace of Japan's pharmaceutical industry. In light of the anticipated changes in healthcare needs in the future, MTPC is advancing business activities under the mission of "Creating hope for all facing illness," and has set the corporate vision for 2030 (VISION 30) to "Be a healthcare company that delivers optimal therapy to each individual." As part of the vision for 2030, MTPC is prioritizing work on "precision medicine" to create effective therapies and preventive methods 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. MTPC is a member of Mitsubishi Chemical Holdings Group. For more information, go to https://www.mt-pharma.co.jp/e/.

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. MTDA is dedicated to research and develop innovative pharmaceutical products that address the unmet medical needs of patients. For more information, please visit https://mt-pharma-development-america.com/.

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¹ Simon, N. G., Turner, M. R., Vucic, S., et al. (2014). Quantifying Disease Progression in Amyotrophic Lateral Sclerosis. Annals of Neurology, 76(5), 643–657.
² RADICAVA^® (edaravone) U.S. Prescribing Information. March 2021.

SOURCE Mitsubishi Tanabe Pharma America, Inc.