Study results may help identify ALS surveillance biomarkers as well as predictive biomarkers to measure the effect of RADICAVA® (edaravone)
JERSEY CITY, NJ, November 2, 2022 /PRNewswire/ — Mitsubishi Tanabe Pharma America, Inc. (MTPA) today announced the presentation of an interim analysis from the Phase 4 REFINE-ALS study, designed to identify predictive and pharmacodynamic biomarkers to measure the RADICAVA effect® (edaravone) in people with amyotrophic lateral sclerosis (ALS). The results will be presented as part of the 21st Northeast Amyotrophic Lateral Sclerosis (NEALS) Annual Meeting, held at Clearwater, Florida.of November 1-3.
“The REFINE-ALS study allowed us to examine meaningful biomarkers to improve our understanding of the complexities of ALS and the role that edaravone might play in treating the disease,” said Gustavo A. Suarez Zambrano, MD, vice president of medical affairs at the MTPA. “We are encouraged by these early results and look forward to collecting more biomarker data on these important assessments and sharing our key learnings with the ALS community.”
REFINE-ALS, conducted in collaboration with the Neurological Clinical Research Institute (NCRI) at Massachusetts General Hospital (MGH), is a prospective, observational, multicenter study in people with ALS in the United States and Canada who started treatment with RADICAVA. The study includes biomarker assessments of oxidative stress, inflammation, and neuronal injury and death, as well as biomarker discovery panels, EpiSwitch™ and SOMAscan®, designed to identify and assess epigenetic and protein biomarkers, respectively. Baseline biomarker measurements are taken before starting RADICAVA treatment, at the start of treatment, and at predefined longitudinal time points throughout the 24-week study period.
Of the April 2022, a total of 50 participants were enrolled in the study and matched with 36 participants from the Answer ALS Study, a research project conducted by a collaborative team including the MGH NCRI. Biomarkers and clinical data from 40 participants were included in this interim analysis, with the following results:
- At baseline, the proportion of rapid progressors, as predicted by EpiSwitch™were numerically higher in REFINE-ALS participants compared to Answer ALS participants.
- At baseline, neurofilament light chain (NfL) levels were numerically higher in REFINE-ALS participants (n=38) compared to Answer ALS participants (n=35). Additionally, as hypothesized, NfL levels were found to correlate with the rate of disease progression as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R).
- Despite a higher proportion of EpiSwitch™ “fast” predictions and higher baseline NfL for REFINE-ALS participants compared to Answer ALS, both Answer ALS and REFINE-ALS cohorts progressed at the same rate on the ALSFRS-R score.
“We have seen increased interest in leveraging biomarker research in ALS to assess potential relationships between biomarker changes and disease progression and treatment outcomes,” said the lead researcher. James BerryMD, MPH, MGH NCRI, Boston. “The results of REFINE-ALS may help to better understand the biological effect of treatment of ALS with edaravone using biomarkers. We hope that continued research on ALS biomarkers will serve as an important tool for understand the effects of treatment in ALS.”
REFINE-ALS is a clinical trial affiliated with NEALS and sponsored by the MTPA.
About RADICAVA® (edaravone) and RADICAVA ORS® (edaravone)
The United States Food and Drug Administration (FDA) has approved RADICAVA® (edaravone) on May 5, 2017and the oral formulation RADICAVA ORS® (edaravone) on May 12, 2022, for the treatment of amyotrophic lateral sclerosis (ALS). RADICAVA is administered in 28-day cycles by IV infusion. It takes 60 minutes to receive each 60 mg dose. For the initial cycle, treatment is infused daily for 14 consecutive days, followed by a two-week drug-free period. All subsequent cycles are infused daily for 10 days over 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 over a 14-day period followed by a 14-day drug-free period. RADICAVA ORS should be taken in the morning after an overnight fast. Patients should not eat or drink (except water) for one hour after taking RADICAVA ORS.1
Edaravone was discovered and developed for ALS by Mitsubishi Tanabe Pharma Corporation (MTPC) and Mitsubishi Tanabe Pharma Development America, Inc. (MTDA), marketed in the United States by Mitsubishi Tanabe Pharma America, Inc. (MTPA). The MTPC group companies began their ALS research in 2001 through an iterative clinical platform over a 13-year period. In 2015, RADICAVA was approved for the treatment of ALS in Japan and South Korea. Marketing authorizations were then granted in Canada (October 2018), Swiss (January 2019), China (July 2019), Indonesia (July 2020), Thailand (April 2021) and Malaysia (December 2021). To date, in the United States, RADICAVA and RADICAVA ORS have been used to treat more than 8,000 patients, with more than 1.1 million treatment days, and have been prescribed by nearly 2,000 healthcare professionals.2,3
IMPORTANT SAFETY INFORMATION
Hypersensitivity reactions
RADICAVA (edaravone) and RADICAVA ORS (edaravone) are contraindicated in patients with a history of hypersensitivity to edaravone or to any of the inactive ingredients of this product. Hypersensitivity reactions (redness, wheals and erythema multiforme) and cases of anaphylaxis (hives, decreased blood pressure and dyspnoea) have occurred with RADICAVA.
Patients should be carefully monitored for hypersensitivity reactions. If hypersensitivity reactions occur, discontinue RADICAVA or RADICAVA ORS, treat according to standard of care, and monitor until the condition resolves.
Allergic reactions to sulphites
RADICAVA and RADICAVA ORS contain sodium bisulfite, a sulfite which may cause allergic-type reactions, including anaphylactic symptoms and life-threatening or less severe asthmatic episodes in susceptible individuals. The overall prevalence of sulphite sensitivity in the general population is unknown, but occurs more frequently in people with asthma.
Side effects
The most commonly reported adverse reactions (≥10%) in patients treated with RADICAVA were bruising (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 can cause fetal harm.
To report suspected adverse reactions or product complaints, contact Mitsubishi Tanabe Pharma America, Inc., at 1-888-292-0058. You can also report suspected side effects 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 complete prescribing information, please visit www.RADICAVA.com.
About Mitsubishi Tanabe Pharma America, Inc.
Situated at Jersey City, New JerseyMitsubishi Tanabe Pharma America, Inc. (MTPA) is a wholly owned subsidiary of Mitsubishi Tanabe Pharma Corporation (MTPC), a wholly owned US holding company, Mitsubishi Tanabe Pharma Holdings America, Inc. It was established by MTPC to market pharmaceutical products approved in North America. For more information, please visit www.mt-pharma-america.com or follow us on TwitterFacebook and LinkedIn.
About Mitsubishi Tanabe Pharma Development America, Inc.
The U.S. headquarters of Mitsubishi Tanabe Pharma Development America, Inc. (MTDA) is located at Jersey City, New Jersey. MTDA is a wholly owned subsidiary of Mitsubishi Tanabe Pharma Corporation’s wholly-owned US holding company, Mitsubishi Tanabe Pharma Holdings America, Inc. For more information, please visit https://mt-pharma-development-america. com/.
About Mitsubishi Tanabe Pharma Corporation
Mitsubishi Tanabe Pharma Corporation (MTPC), the pharmaceutical arm of Mitsubishi Chemical Group, is one of the oldest pharmaceutical companies in the world, founded in 1678 and focusing on ethical pharmaceuticals. MTPC is headquartered in Doshomachi, Osakabirthplace of from Japan pharmaceutical industry. The Mitsubishi Chemical Group has positioned healthcare as its strategic axis in its management policy, “Forging the future”. MTPC’s MISSION is to “Create hope for all those who face the disease”. To this end, the MTPC is prioritizing work on “precision medicine” to deliver medicines with high treatment satisfaction by identifying patient populations with high potential for efficacy and safety, focusing on areas pathologies of the central nervous system and of immuno-inflammation. In addition, MTPC works to develop “pill-based solutions” to address specific patient concerns based on therapeutic medicine, including disease prevention, pre-symptomatic disease care, prevention of aggravation and the prognosis. For more information, please visit https://www.mt-pharma.co.jp/e/.
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1 RADICAVA and RADICAVA ORS prescribing information. Jersey City, New Jersey: Mitsubishi Tanabe Pharma America, Inc.; 2022.
2 Data on file. Mitsubishi Tanabe Pharma America, Inc.
3 Data on file. Mitsubishi Tanabe Pharma America, Inc.
SOURCEMitsubishi Tanabe Pharma America, Inc.
