Mitsubishi Tanabe Pharma America to Present New ALS Research at MNDA's 35th International Symposium on ALS/MND

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Dec 03, 2024, 09:00 ET

JERSEY CITY, N.J., Dec. 3, 2024 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced 10 presentations covering advances in amyotrophic lateral sclerosis (ALS) research at the Motor Neurone Disease Association (MNDA) 35^th International Symposium on ALS/MND, being held in Montreal, Canada, from December 6-8.

"We are honored to participate in this year's MNDA symposium, where we will share insights from our preclinical, clinical and real-world studies on RADICAVA^® (edaravone) and RADICAVA ORS^® (edaravone) and discuss the evolving ALS treatment landscape," said Gustavo A. Suarez Zambrano, M.D., Vice President of Medical Affairs at MTPA. "Our commitment to advancing ALS research is underscored by over eight years of continuous contributions at this symposium. This dedication guides us in leveraging both clinical and real-world evidence to help support the ALS community."

RADICAVA ORS Results:
Final results from two multi-center, Phase 3b, double-blind, parallel group studies will be presented, including a comparison of daily dosing of RADICAVA ORS (edaravone) with the FDA-approved on/off regimen, assessed over 48 weeks (MT-1186-A02) and up to 96 weeks (MT-1186-A04). Additionally, safety data from a Phase 3 open-label safety extension study (MT-1196-A03) will be shared.

Phase 3b Study MT-1186-A02 to Investigate the Superiority of Daily Dosing vs the FDA-approved On/Off Regimen of Oral Edaravone in Patients with ALS (Alejandro Salah, M.D., Ph.D., MBA, MHA; MTPA)
Poster Session B: 5:30 p.m. – 7:00 p.m. ET, Saturday, December 7
Phase 3, Open-Label, Safety Extension Study of Oral Edaravone Administered Over 96 Weeks in Patients with ALS (MT-1186-A03) (Alejandro Salah, M.D., Ph.D., MBA, MHA; MTPA)
Poster Session B: 5:30 p.m. – 7:00 p.m. ET, Saturday, December 7
Phase 3b Study MT-1186-A04 Extension Study 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 (Stephen Apple, M.D.; MTPA)
Poster Session B: 5:30 p.m. – 7:00 p.m. ET, Saturday, December 7

Real World Data:
Presentations will highlight the evolving ALS treatment landscape with insights from the ALS/Motor Neuron Disease (MND) Natural History Consortium (NHC) registry covering treatment patterns, clinical outcomes and survival rates in RADICAVA-treated individuals. Additional presentations include a preliminary analysis of the OPTUM claims database, evaluating treatment combinations in people with ALS. Further highlights include studies on point-of-care quality assessment and at-home telespirometry, evaluating erect and supine slow vital capacity measurements in people with ALS.

Evolution of the Treatment Landscape for Patients Diagnosed With ALS in the United States (Malgorzata Ciepielewska, M.S.; MTPA)
Poster Session B: 5:30 p.m. – 7:00 p.m. ET, Saturday, December 7
Treatment Patterns and Survival Benefit of Edaravone–Treated People With Amyotrophic Lateral Sclerosis in the ALS/MND Natural History Consortium (Malgorzata Ciepielewska, M.S.; MTPA)
Oral Session 2B – Improving Clinical Management: 10:30 a.m. – 12:30 p.m. ET, Friday, December 6
Preliminary Analysis of Treatment Combinations in Patients With Amyotrophic Lateral Sclerosis Enrolled in an US-Based Administrative Claims Database (Malgorzata Ciepielewska, M.S.; MTPA)
Poster Session B: 5:30 p.m. – 7:00 p.m. ET, Saturday, December 7
Smartphone Application–Mediated Supervised At-Home Telespirometry Erect and Supine Slow Vital Capacity (eSVC/sSVC) Measurements in Subjects With Amyotrophic Lateral Sclerosis Identify Statistically Significant Differences in eSVC/sSVC Decline as Function of Non-Invasive Ventilation Treatment Status [NCT05106569] (Eufrosina I. Young, M.D.; SUNY Upstate Medical University)
Poster Session B: 5:30 p.m. – 7:00 p.m. ET, Saturday, December 7
Smartphone Application–Mediated, Supervised, At-Home Telespirometry Vital Capacity Measurements in Amyotrophic Lateral Sclerosis: Comparison of American Thoracic Society/European Respiratory Society Point-of-Care Quality Assessment Algorithms Implemented for Slow Vital Capacity and Forced Vital Capacity [NCT05106569] (Eufrosina I. Young, M.D.; SUNY Upstate Medical University)
Poster Session B: 5:30 p.m. – 7:00 p.m. ET, Saturday, December 7

Preclinical Data:
Two preclinical studies will be presented, showcasing key mechanisms in ALS pathology. The first study demonstrates edaravone's role in modulating glutamate-mediated hyperexcitability in spinal motor neurons, using an ALS animal model. The second study explores how LRSAM1 contributes to TDP-43 clearance, a critical process in ALS, using induced pluripotent stem cells (iPSCs) derived from ALS patients.

Edaravone Reduces the Enhanced Glutamatergic Transmission Onto Motor Neurons in the Spinal Cord of a Mouse Model of Amyotrophic Lateral Sclerosis (Yun Kyung Park, Ph.D.; MTPC)
Poster Session B: 5:30 p.m. – 7:00 p.m. ET, Saturday, December 7
LRSAM1 Protects Neurons in Amyotrophic Lateral Sclerosis by Regulating the TDP-43 Proteostasis (Takayuki Shirakawa, Ph.D.; MTPA)
Oral Session 6A – Proteostasis and Proteotoxicity: 10:30 a.m. – 12:30 p.m. ET, Saturday, December 7

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, and focusing on ethical pharmaceuticals. MTPC is headquartered in Doshomachi, Osaka, the birthplace of Japan's pharmaceutical industry. 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 by identifying patient populations with high potential for efficacy and safety. 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 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