Shionogi to Present RADICAVA® (edaravone) ORS and Digital Health Analyses in ALS at AAN 2026

FLORHAM PARK, N.J.--(BUSINESS WIRE)--Shionogi Inc. (Head Office: Osaka, Japan; Chief Executive Officer: Isao Teshirogi, Ph.D.; hereafter "Shionogi") today announced two presentations highlighting research in amyotrophic lateral sclerosis (ALS) at the American Academy of Neurology (AAN) 2026 Annual Meeting, taking place April 18-22 in Chicago, Illinois, and online. Both presentations will feature encore analyses sharing previously presented findings.

“We look forward to sharing these analyses at AAN and contributing to the ongoing scientific dialogue around ALS,” said Gustavo A. Suarez Zambrano, M.D., Vice President, Medical Affairs, Rare Disease, Shionogi. “These analyses explore a potential ALS prognostic biomarker and examine emerging digital health tools aimed at advancing monitoring and understanding of disease progression.”

RADICAVA ORS^® (edaravone) Data:
An analysis will be presented assessing neutrophil-to-lymphocyte ratio (NLR) changes in response to edaravone oral suspension-treated patients with ALS and examining the predictive value of baseline NLR compared with propensity score-matched PRO-ACT placebo controls. These results further examine the potential of NLR as a prognostic biomarker in ALS.

• Exploratory Post Hoc Analysis of Neutrophil-to-Lymphocyte Ratio as a Novel Response Biomarker for Edaravone Oral Suspension-Treated Patients With Amyotrophic Lateral Sclerosis vs Untreated Propensity Score-Matched PRO-ACT Historical Placebo Controls (Benjamin Rix Brooks, M.D.; Clinical Trials Planning LLC, Charlotte, NC)
  Session: S32: General Neurology 1 (#008): 4:54 pm CT, April 21

Digital Health Insights:
A study will be presented evaluating smartphone-based telespirometry as an at-home tool for ALS monitoring. In a six-month prospective study of patients receiving edaravone, ALSFRS-R decline differed based on non-invasive ventilation (NIV) status. Further analyses highlight the importance of incorporating NIV use into clinical study design and interpretation of respiratory and functional endpoints.

• Smartphone Application–Mediated, Supervised, At-Home Telespirometry Identifies Statistically Significant Differences in Erect and Supine Slow Vital Capacity and ALSFRS-R Decline as a Function of Non-invasive Ventilation Treatment Status in Multicenter, Prospective, Longitudinal, Observational Clinical Study [NCT05106569] (Eufrosina I. Young, M.D.; SUNY Upstate Medical University)
  Session: P7: General Neurology: Community and Population Studies 2 (#002): 8:00 am-9:00 am CT, April 21

About RADICAVA ORS® (edaravone)
The U.S. Food and Drug Administration (FDA) approved RADICAVA ORS® (edaravone) on May 12, 2022, for the treatment of amyotrophic lateral sclerosis (ALS). In 2024, the FDA granted RADICAVA ORS Orphan Drug Exclusivity based on its major contribution to patient care by providing an oral suspension route of administration that avoids the burdens of IV administration. 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. Each 105 mg (5mL) dose of 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 Tanabe Pharma Corporation and commercialized in the U.S. by Tanabe Pharma America, Inc. The Tanabe Pharma 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), Australia (February 2023) 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 ORS, along with the previously available IV RADICAVA® (edaravone), have been used to treat over 22,000 people with ALS, with over 2.8-million days of therapy, and have been prescribed by over 2,800 HCPs.^2-4

INDICATION
RADICAVA ORS® (edaravone) is indicated for the treatment of amyotrophic lateral sclerosis (ALS).

IMPORTANT SAFETY INFORMATION

Hypersensitivity Reactions
RADICAVA ORS® (edaravone) is 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.

Patients should be monitored carefully for hypersensitivity reactions. If hypersensitivity reactions occur, discontinue RADICAVA ORS, treat per standard of care, and monitor until the condition resolves.

Sulfite Allergic Reactions
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® (edaravone)-treated patients and at least 2% more frequently than placebo were contusion (15% vs 9%), gait disturbance (13% vs 9%), and headache (10% vs 6%), respectively. In an open label study, fatigue was also observed in 7.6% of patients receiving RADICAVA ORS.

Pregnancy
Based on animal data, RADICAVA ORS may cause fetal harm.

To report suspected adverse reactions or product complaints, contact 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.

Please see the full Prescribing Information, also available at http://www.RADICAVAORS.com.

About Shionogi in Rare Disease
Shionogi is committed to the research and development of innovative medicines that address unmet medical needs for people worldwide. Rare diseases often have limited treatment options and affect the lives of individuals and families around the world. In the U.S., Shionogi is advancing a rare disease franchise that includes an approved therapy for amyotrophic lateral sclerosis (ALS) as well as clinical programs in Fragile X syndrome, Jordan’s Syndrome and Pompe disease. For more information, view our pipeline here: https://www.shionogi.com/us/en/innovation/pipeline.html.

About Shionogi & Co., Ltd.
Shionogi & Co., Ltd. is a 148-year-old global, research-driven pharmaceutical company headquartered in Osaka, Japan, that is dedicated to bringing benefits to patients based on its corporate philosophy of “supplying the best possible medicine to protect the health and wellbeing of the patients we serve.” The company currently markets products in several therapeutic areas including anti-infectives, pain, CNS disorders and cardiovascular diseases. Shionogi’s research and development currently targets two therapeutic areas: infectious diseases and diseases with unmet medical needs in pain/CNS, including Alzheimer’s disease, oncology, rare diseases, and sleep apnea. For more information on Shionogi & Co., Ltd., please visit https://www.shionogi.com/global/en .

Forward-Looking Statements
This announcement contains forward-looking statements. These statements are based on expectations in light of the information currently available, assumptions that are subject to risks and uncertainties which could cause actual results to differ materially from these statements. Risks and uncertainties include general domestic and international economic conditions such as general industry and market conditions, and changes of interest rate and currency exchange rate. These risks and uncertainties particularly apply with respect to product-related forward-looking statements. Product risks and uncertainties include, but are not limited to, completion and discontinuation of clinical trials; obtaining regulatory approvals; claims and concerns about product safety and efficacy; technological advances; adverse outcome of important litigation; domestic and foreign healthcare reforms and changes of laws and regulations. Also for existing products, there are manufacturing and marketing risks, which include, but are not limited to, inability to build production capacity to meet demand, lack of availability of raw materials and entry of competitive products. The company disclaims any intention or obligation to update or revise any forward-looking statements whether as a result of new information, future events or otherwise.

1. RADICAVA and RADICAVA ORS Prescribing Information. Jersey City, NJ: Tanabe Pharma America, Inc.; 2025.
2. Data on file. Shionogi Inc.
3. Data on file. Shionogi Inc.
4. Data on file. Shionogi Inc.

For Further Information, Contact:
SHIONOGI Website Inquiry Form: https://www.shionogi.com/global/en/contact.html
Shionogi Inc.: ShionogiCommunications@shionogi.com
Shionogi Europe Press Office: pressoffice@shionogi.eu