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To better understand <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) and improve patient care, <a href="https://www.mt-pharma-america.com/" rel="nofollow">Mitsubishi Tanabe Pharma America</a> (MTPA) is partnering with the <a href="https://www.data4cures.org/natural-history-consortium">ALS/MND Natural History Consortium</a> in collecting real-world data on people with the progressive neurodegenerative disorder.
The collaboration will give MTPA access to the consortium&#8217;s data repository, containing demographic, baseline, and longitudinal clinical and scientific data on people with ALS and other motor neuron disorders (MNDs).
The company also will gain access to clinical data covering patients using its ALS treatments, <a href="https://alsnewstoday.com/radicava-edaravone-for-als/">Radicava</a> (edaravone) and the oral formulation <a href="https://www.radicava.com/patient/taking-radicava-ors/">Radicava ORS</a>, dating back when the consortium was founded in 2015.
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<h3>Radicava&#8217;s 2017 approval marked first new ALS treatment for US in 22 years</h3>
Both formulations were developed by <a href="https://www.mt-pharma.co.jp/e/" rel="nofollow">Mitsubishi Tanabe Pharma</a> and are marketed in the U.S. by its subsidiary MTPA. They are used to slow functional decline and extend survival in those with ALS.
When Radicava was <a href="https://alsnewstoday.com/news/fda-approves-radicava-first-new-als-therapy-in-20-years-and-cause-for-hope/">approved by the U.S. Food and Drug Administration in 2017</a>, preceded by approvals in Japan and South Korea, it became the first new ALS treatment available in the U.S. in more than 20 years. To date, the formulations have been used to treat some 10,000 ALS patients in that country, according to MTPA.
&#8220;Access to real-world data is critical with complex and difficult-to-treat diseases like ALS,&#8221; Gustavo A. Suarez Zambrano, MD, MTPA vice president of medical affairs, said in a <a href="https://www.prnewswire.com/news-releases/mitsubishi-tanabe-pharma-america-to-partner-with-alsmnd-natural-history-study-consortium-to-collect-real-world-data-in-als-with-the-goal-of-improving-patient-care-301843078.html">company press release</a>. &#8220;This collaboration will enable us to leverage a comprehensive database of real-world and retrospective clinical trial findings, continuously advancing our scientific understanding of ALS and how to improve care for people living with this disease.&#8221;
More than 2,100 ALS patients from across 10 participating clinics in the U.S. and Italy are currently enrolled in the consortium. Its database uses NeuroBANK, a patient-centric clinical research platform managed by the Neurological Clinical Research Institute (NCRI) at Massachusetts General Hospital (MGH).
The consortium&#8217;s mission is to collect real-world ALS and MND data that could aid scientists in disease research and help to inform clinical trial design. The overarching aim is improved ALS knowledge and care.
&#8220;Through this collaboration, MTPA joins the ranks of visionary companies that understand the importance of capturing high-quality real-world data that help us to better understand, better diagnose and ultimately find new treatment options for ALS,&#8221; said Alex Sherman, director of the Center for Innovation and Biomedical Informatics at the NCRI and the study&#8217;s principal investigator. &#8220;We hope to see this momentum continue and are confident our collaboration with MTPA will have a meaningful impact on the ALS community.&#8221;

MTPA joins with ALS/MND patient registry with goal of better care

A Phase 3b study evaluating the long-term safety and effectiveness of two dosing regimens of <a href="https://alsnewstoday.com/radicava-edaravone-for-als/">Radicava ORS</a> (edaravone) — an approved oral formulation of the medicine — in people with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) is now fully enrolled.
The study was designed to fulfill a post-marketing commitment that <a href="https://www.mt-pharma-america.com/">Mitsubishi Tanabe Pharma America</a> (MTPA) made to the U.S. Food and and Drug Administration (FDA) after the regulatory agency <a href="https://alsnewstoday.com/news/fda-approves-radicava-first-new-als-therapy-in-20-years-and-cause-for-hope/">approved</a> the into-the-vein (intravenous) formulation of Radicava in 2017.
The medication&#8217;s oral formulation, Radicava ORS, <a href="https://alsnewstoday.com/news/fda-approves-radicava-ors-oral-suspension-edaravone-als/">earned</a> its U.S. approval in 2022.
&#8220;We&#8217;re very pleased to announce the completion of trial enrollment in our Phase 3b study of Radicava ORS, representing an important achievement in our ongoing commitment to the fight against ALS,&#8221; Atsushi Fujimoto, president of MTPA, said in a <a href="https://www.prnewswire.com/news-releases/mitsubishi-tanabe-pharma-america-announces-enrollment-completion-for-global-phase-3b-study-of-radicava-ors-edaravone-in-als-301779833.html">company press release</a>.
&#8220;We are deeply grateful to the study participants and their families, as well as study investigators and clinical staff for working so diligently to help us meet this milestone,&#8221; Fujimoto added.
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<h3>Phase 3b study testing 2 different doses of oral therapy</h3>
Radicava ORS is a liquid suspension taken either orally or via a feeding tube. As with the intravenous formulation, it is given in 28-day cycles.
The first cycle consists of daily treatment for 14 days followed by a two-week period (a second 14 days) without the medication. In subsequent cycles, Radicava is taken daily for 10 of the next 14 days, followed again by two weeks without treatment.
Approval of Radicava ORS was based on <a href="https://alsnewstoday.com/news/als-treatment-radicava-ors-fares-well-phase-3-clinical-trial/">clinical trial data</a> indicating that a 105 mg dose of the oral treatment delivers similar amounts of edaravone — Radicava&#8217;s active ingredient — to the body and is as good at slowing disease progression as the intravenous version (60 mg).
Now, the global <a href="https://clinicaltrials.gov/ct2/show/NCT04569084">MT-1186-A02 study (NCT04569084)</a> is designed to compare the approved Radicava ORS on/off dosing regimen against once-daily dosing.
A total of 384 ALS patients, ages 18-75, whose symptoms emerged in the two years prior to the trial, were enrolled across 80 sites in the U.S., Canada, Europe, and Asia.
After an eight-week screening period, participants will either receive Radicava ORS every day, or be given the medication and a placebo. In the medication/placebo group, patients will receive Radicava ORS for 14 days and a placebo for 14 days in the first 28-day cycle, followed by 10 days of treatment and 18 days on a placebo for the next cycles.
The trial&#8217;s main goal is to evaluate changes in disease progression, as assessed by the ALS Functional Rating Scale-Revised (ALSFRS-R), over 48 weeks, or nearly one year. Secondary goals include changes in lung function and in measures of patient-reported quality of life, as well as the time patients live without requiring ventilatory support.
Top-line results from the trial, which already is underway, are expected in 2024.
&#8220;We look forward to seeing how the results of this study evaluating an alternative dosing regimen for Radicava ORS build upon our existing data to support its long-term efficacy and safety profile for people living with ALS, and how it may increase our understanding of the full potential of the medication as a treatment option,&#8221; said Michael D. Weiss, MD, of the University of Washington Medical Center, who is the study&#8217;s lead investigator.
Radicava works by combating oxidative stress, a type of cellular damage that&#8217;s been implicated in ALS. In doing so, it&#8217;s expected to protect nerve cells from damage and slow disease progression.
Intravenous Radicava also is approved in Japan, South Korea, Canada, and Switzerland, among other countries. The oral version has approvals in the U.S., Canada, and Japan.

Tag: Mitsubishi Tanabe Pharma America

MTPA joins with ALS/MND patient registry with goal of better care

Post-marketing trial of 2 Radicava ORS doses now fully enrolled