Mitsubishi Tanabe Pharma America Unveils New ALS Research Findings at NEALS 2024 Annual Meeting

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Oct 17, 2024, 09:00 ET

A total of 12 presentations including 6 original posters to showcase MTPA's latest ALS research

JERSEY CITY, N.J., Oct. 17, 2024 /PRNewswire/ -- Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced that twelve presentations following expansive research in amyotrophic lateral sclerosis (ALS) will be shared as part of the Northeast Amyotrophic Lateral Sclerosis Consortium (NEALS) 2024 Annual Meeting, being held virtually October 21-24.

"We are excited to present our latest data in ALS research, from preclinical studies to extensive real-world data on RADICAVA^® (edaravone) at this year's meeting," said Gustavo A. Suarez Zambrano, M.D., Vice President of Medical Affairs, MTPA. "Our unwavering commitment to ALS research drives us to provide valuable insights to patients, healthcare practitioners and researchers alike, with the ultimate goal of providing treatment options to help improve the lives of those affected by this disease."

RADICAVA ORS Results:
A presentation will examine the impact of RADICAVA ORS on survival and function in ALS patients, using data from the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database. For this analysis, patients were propensity score-matched with participants from studies MT-1186 A01-A04. Additionally, multiple presentations will explore RADICAVA ORS through multi-center, phase 3b, double-blind, parallel group studies, including a comparison of daily dosing to the U.S. Food and Drug Administration (FDA)-approved on/off regimen, evaluated over 48 weeks (MT-1186-A02) and up to 96 weeks (MT-1186-A04). Safety data from the phase 3 open-label safety extension study (MT-1196-A03) and a post-marketing safety analysis will also be presented.

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 (Stephen Apple, M.D.; MTPA)
Poster Session: 4:00 p.m. - 6:00 p.m. ET, Wednesday, October 23
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: 4:00 p.m. - 6:00 p.m. ET, Wednesday, October 23
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: 4:00 p.m. - 6:00 p.m. ET, Wednesday, October 23
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: 4:00 p.m. - 6:00 p.m. ET, Wednesday, October 23
Summary of the US Safety Data for Radicava ORS^®: Findings From the Postmarketing Pharmacovigilance (Stephen Apple, M.D.; MTPA)
Poster Session: 4:00 p.m. - 6:00 p.m. ET, Wednesday, October 23

Real World Data:
Data will be presented from an evaluation of treatment patterns, clinical outcomes and survival rates of RADICAVA-treated individuals living with ALS from the ALS/Motor Neuron Disease (MND) Natural History Consortium (NHC) registry. Additionally, a preliminary analysis will examine the treatment combinations in people with ALS enrolled in the OPTUM claims database. Other presentations include two studies focused on point-of-care quality classifications and at-home telespirometry, evaluating erect and supine slow vital capacity in individuals with ALS, as well as an observational study, Track-ALS, assessing participant compliance and the usability of digital health technologies (DHTs) to remotely monitor ALS progression.

Treatment Patterns and Survival Benefit of Edaravone–Treated People With Amyotrophic Lateral Sclerosis in the ALS/MND Natural History Consortium (Stephen Apple, M.D.; MTPA)
Poster Session: 4:00 p.m. - 6:00 p.m. ET, Wednesday, October 23
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: 4:00 p.m. - 6:00 p.m. ET, Wednesday, October 23
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: 4:30 p.m. - 6:30 p.m. ET, Tuesday, October 22
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: 4:30 p.m. - 6:30 p.m. ET, Tuesday, October 22
Compliance of Home-based Remote Digital Monitoring to Assess ALS progression (Track-ALS) (Katie Terrebonne; Emory University)
Poster Session: 4:30 p.m. - 6:30 p.m. ET, Tuesday, October 22

Preclinical Data:
Two preclinical studies will be presented, showcasing the effects of edaravone. The first study examines its impact on spinal motor neurons derived from induced pluripotent stem cells (iPSC) taken from a person with ALS carrying the A382T mutation in TDP-43. The second study investigates the role of edaravone on the modulation of glutamate-mediated hyperexcitability in spinal motor neurons using an ALS animal model.

Transcriptomic Signature Induced by Edaravone in Motor Neurons From an ALS Patient With a TDP-43 Mutation (Satsuki Mikuriya, M.S.; MTPA)
Poster Session: 4:00 p.m. - 6:00 p.m. ET, Wednesday, October 23
Edaravone Reduces the Enhanced Glutamatergic Transmission Onto Motor Neurons in the Spinal Cord of a Mouse Model of Amyotrophic Lateral Sclerosis (Alejandro Salah, M.D., Ph.D., MBA, MHA; MTPA)
Poster Session: 4:00 p.m. - 6:00 p.m. ET, Wednesday, October 23

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