Mitsubishi Tanabe Pharma to Showcase ALS Research at ENCALS 2024

Mitsubishi Tanabe Pharma America, Inc. (MTPA) will showcase eight presentations at the European Network to Cure Amyotrophic Lateral Sclerosis (ENCALS) 2024 Annual Meeting in Stockholm, Sweden, from June 17-20, 2024. The presentations will encompass various aspects of translational research in amyotrophic lateral sclerosis (ALS) and include findings from the crucial Phase 3 trial (MT-1186-A01) that supported the U.S. approval of RADICAVA® (edaravone).

Dr. Gustavo A. Suarez Zambrano, Vice President of Medical Affairs at MTPA, expressed enthusiasm for the broad range of research being presented. The presentations will cover two preclinical studies on RADICAVA's mechanism of action and real-world evidence gathered during post-marketing analysis, offering valuable insights to the ALS research and healthcare community.

The key areas of focus at the ENCALS presentations are:

RADICAVA ORS Results:
Final data from the Phase 3b study MT-1186-A02, involving an investigational once-daily dosing regimen of RADICAVA ORS®, will be presented. This study compared the efficacy of this regimen with the FDA-approved on/off regimen administered in 28-day cycles over 48 weeks in ALS patients. Additionally, the complete results from the Phase 3 open-label safety extension (MT-1186-A03) over 96 weeks will be shared.

Real-World Data:
Presentations will include interim analysis results from the Phase 4 REFINE-ALS study that aims to identify biomarkers to measure edaravone's effect in ALS and evaluate clinical outcomes in real-world settings. Another focus will be on treatment patterns and survival benefits for RADICAVA-treated individuals in the ALS/Motor Neuron Disease Natural History Consortium registry.

Data from Optum Clinformatics® Data Mart:
Preliminary analysis will outline demographics, clinical characteristics, and treatment patterns of ALS patients, utilizing U.S.-based administrative claims data. Additionally, healthcare resource utilization data for RADICAVA ORS-treated patients will be presented.

Preclinical Data:
Research findings will be shared on the effect of edaravone on spinal motor neurons derived from an ALS patient with the A382T mutation in TDP-43, using induced pluripotent stem cells (iPSC). Another study will examine edaravone’s role in modulating glutamate-mediated hyperexcitability in spinal motor neurons in an ALS mouse model.

About RADICAVA® (edaravone) and RADICAVA ORS® (edaravone):
RADICAVA® (edaravone) received FDA approval on May 5, 2017, for the treatment of ALS, followed by the oral formulation RADICAVA ORS® approved on May 12, 2022. The FDA recognized RADICAVA ORS with Orphan Drug Exclusivity in 2024 for its significant contribution to patient care. RADICAVA is administered in 28-day cycles via intravenous infusion, while RADICAVA ORS is taken orally with an initial cycle of daily dosing for 14 days followed by a 14-day drug-free period.

Developed by Mitsubishi Tanabe Pharma Corporation (MTPC), RADICAVA has been administered to over 14,600 ALS patients in the U.S., with more than 1.8 million days of therapy. The drug has also been approved in multiple countries, including Japan, Canada, and Switzerland.

Safety Information:
Patients with hypersensitivity to edaravone or its inactive ingredients should avoid RADICAVA. Hypersensitivity and sulfite allergic reactions, including anaphylaxis, have been reported. Common adverse reactions include contusion, gait disturbance, and headache, with fatigue also noted in patients using RADICAVA ORS. Pregnant women should be cautious as the drug may cause fetal harm.

Mitsubishi Tanabe Pharma America, Inc., headquartered in Jersey City, NJ, is committed to advancing its pharmaceutical pipeline and commercializing products in North America. MTPC, part of the Mitsubishi Chemical Group, focuses on central nervous system diseases, immuno-inflammation, diabetes, and cancer, aiming to provide high satisfaction treatments and "around the pill solutions" for specific patient needs.

The upcoming ENCALS presentations reflect MTPA's dedication to enhancing ALS research and patient care through extensive clinical and real-world data analysis.

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