12-month Edaravone Clinical Study Data in ALS
Presented at the 27th International Symposium on ALS/MND
Osaka, Japan, December 9, 2016 -- Mitsubishi Tanabe Pharma Corporation (Head Office:Osaka; President & Representative Director, CEO: Dr. Masayuki Mitsuka) has presented datathat show patients with amyotrophic lateral sclerosis (ALS) given edaravone intravenously in
10-14 day cycles for 48 weeks experienced significantly less functional loss as measured by
the ALS Functional Rating Scale-Revised (ALSFRS-R). The data were presented at the 27thInternational Symposium on ALS/MND in Dublin, Ireland.
In the 48-week study, a 24-week double blind placebo controlled phase was followed by a
24-week open label active treatment phase. Data previously presented at the 2016 Annual
Meeting of the American Academy of Neurology, showed edaravone met the primary efficacy
endpoint of mean change in the ALSFRS-R at 24 weeks. The frequency of serious adverse
events was similar to placebo.
“These findings suggest that intervention with edaravone may provide a treatment option topeople living with ALS when therapy is promptly initiated,” said Joseph M. Palumbo, MD, VicePresident, Medical Sciences and Translational Research, Mitsubishi Tanabe Pharma
Development America, Inc.
About ALS
ALS, sometimes called Lou Gehrig’s disease, attacks the nerve cells in the brain and the spinal
cord responsible for controlling voluntary muscles, such as those needed to move, speak, eat
and breathe.1,2 It is one of the most well-known neuromuscular diseases, affecting
approximately two in 100,000 people worldwide.3,4 While it is inherited in 5%–10% of cases,
the cause for the majority of cases is not well understood but may involve genetic andenvironmental factors.5,6 There is currently no cure.6
About Edaravone
Discovered by Mitsubishi Tanabe Pharma Corporation (MTPC), edaravone is described as afree radical scavenger that is believed to relieve the effects of oxidative stress, a likely factor in
the onset and progression of ALS.3,7 Oxidative stress is thought to be an imbalance between
the production of free radicals and the ability of the body to counteract or detoxify their harmful
effects.8 In patients with ALS, there are consistent increases in oxidative stress biomarkers.7
In 2015, edaravone was approved for use as a treatment for ALS in Japan and South Korea. In thesame year, the FDA and the European Commission granted Orphan Drug Designation foredaravone. It is not currently approved by the FDA for any use in the U.S.
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About MT Pharma America, Inc.
Based in Jersey City, NJ, MT Pharma America is a wholly-owned subsidiary of MTPC’s 100%
owned U.S. holding company, Mitsubishi Tanabe Pharma Holdings America, Inc. MT Pharma
America is dedicated to delivering innovative solutions that address the unmet medical needsof patients in the United States. It was established by MTPC to commercialize approved
pharmaceutical products in the U.S. with plans to expand its product line through collaborations
with partners. For more information, go to www.mt-pharma-america.com.
Overview of Mitsubishi Tanabe Pharma
Mitsubishi Tanabe Pharma, which was founded in 1678, has its headquarters in Doshomachi,
Osaka, which is the birthplace of Japan’s pharmaceutical industry. With business centered onethical pharmaceuticals, Mitsubishi Tanabe Pharma is a well-established company and has thelongest history of any listed company in Japan.9 In accordance with the corporate philosophyof “contributing to the healthier lives of people around the world through the creation ofpharmaceuticals,” the Company formulated the key concept of Open Up the Future under theMedium-Term Management Plan 16-20. Through the discovery of drugs that address unmet
medical needs, centered on its priority disease areas — autoimmune diseases, diabetes andkidney diseases, central nervous system diseases, and vaccines — Mitsubishi Tanabe
Pharma will strive to contribute to the health of patients around the world. For more information,
go to http://www.mt-pharma.co.jp/.

1

The Mayo Clinic. Diseases and Conditions: Amyotrophic Lateral Sclerosis.
http://www.mayoclinic.org/diseases-conditions/amyotrophic-lateral-sclerosis/basics/causes/con-20024397. Accessed May 17, 2016.

2

National Institute of Neurological Disorders and Stroke. Amyotrophic Lateral Sclerosis (ALS) Fact Sheet.
http://www.ninds.nih.gov/disorders/amyotrophiclateralsclerosis/detail_ALS.htm. Updated March 14, 2016. Accessed May 17, 2016.

3

Nagase M, Yamamoto Y, Miyazaki Y, et al. Increased oxidative stress in patients with amyotrophic lateral sclerosis and the effect of edaravoneadministration. Redox Rep. 20154Chiò A, Logroscino G, Traynor B, et al. Global Epidemiology of Amyotrophic Lateral Sclerosis: a Systematic Review of the Published Literature.
Neuroepidemiology. 2013;41(2):118-130.

5

ALS Association. Familial Amyotrophic Lateral Sclerosis (FALS) and Genetic Testing. http://www.alsa.org/about-als/genetic-testing-for-als.html
Accessed June 8, 2016.

6

Centers for Disease Control and Prevention. Prevalence of Amyotrophic Lateral Sclerosis — United States, 2010–2011.
http://www.cdc.gov/mmwr/preview/mmwrhtml/ss6307a1.htm. Accessed April 14, 2016.

7Manning, M.M. and Kelly-Worden, M. (2015) Potential Regulators of Sporadic ALS Development and Alternative Therapeutic Options.
Neuroscience & Medicine. 2015; 6, 5-12.

8

9Betteridge, D.J., What is oxidative stress? Metabolism. 2000;49: 3-8.
Research by TOKYO SHOKO RESEARCH, LTD.

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