Shionogi to Partner with ALS/MND Natural History Consortium to Collect Real-World Data in ALS, Reinforcing Investments in ALS Research

FLORHAM PARK, NJ, June 26, 2026 – Shionogi & Co., Ltd. (Head Office: Osaka, Japan; Chief Executive Officer: Isao Teshirogi, PhD; hereinafter, “Shionogi”) today announced its continued partnership with the ALS/MND Natural History Consortium (Consortium) to collect real-world data in amyotrophic lateral sclerosis (ALS).

The goal of this collaboration is to deepen scientific knowledge of ALS through access to real-world data, including data of approved therapies for ALS, such as RADICAVA ORS^® (edaravone). By collaborating with the Consortium, Shionogi is contributing to a broader understanding of ALS as well as the therapeutic effects of currently approved treatments.

“A major scientific challenge in ALS is determining how the disease presents and evolves across diverse patient populations in real-world settings. Access to real-world data is critical to addressing this complexity,” said Gustavo A. Suarez Zambrano, M.D., Vice President, Medical Affairs, Rare Disease, Shionogi Inc. “This partnership will advance our knowledge of ALS and help improve care for people living with the disease, reinforcing Shionogi’s deep commitment to research in the ALS community.”

The partnership is built around access to NeuroBANK^®, a patient-centered observational platform supported by the Neurological Clinical Research Institute (NCRI) at Massachusetts General Hospital (MGH), which enables the collection of high-quality, standardized clinical data during routine clinical visits with minimal additional burden on people living with ALS. To date, over 4,500 people living with ALS from across 18 multidisciplinary clinics in the U.S., Europe, and Israel have enrolled in the study.¹

“ALS remains a devastating disease with profound unmet need, underscoring the urgency to further our understanding of disease progression,” said Alexander Sherman, Director of Center for Innovation and Bioinformatics at NCRI and the Consortium’s Principal Investigator. “By collaborating with Shionogi, we aim to deepen our perspective of ALS in real-world settings to generate insights that can support more informed care for people living with the disease.”

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.²

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 22,800 people with ALS, with over 3.0-million days of therapy, and have been prescribed by over 2,850 HCPs.³

Following the completion of Shionogi’s acquisition of the global RADICAVA business, Shionogi now holds global rights to RADICAVA ORS and RADICAVA IV.

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

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.

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.

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.

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

To report suspected adverse reactions or product complaints, contact Shionogi 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

ALS (Amyotrophic Lateral Sclerosis) is a progressive neurodegenerative disease characterized by the selective degeneration and loss of motor neurons, leading to muscle weakness and respiratory impairment. After onset, muscle atrophy gradually progresses, and ultimately respiratory muscle paralysis severely impacts life expectancy. Globally, the annual incidence is estimated at approximately 1–2 cases per 100,000 population, making it a rare disease for which no fundamental cure currently exists.⁴ Multiple factors, including oxidative stress and glutamate-induced excitotoxicity, are involved in its pathophysiology, and treatment options to slow disease progression remain extremely limited. For this reason, ALS is recognized as a disease with a high level of unmet medical need, and the development and delivery of innovative therapies are strongly required.

The ALS/MND Natural History Consortium, comprising academic medical centers across the U.S., Europe, and Israel, is driving efforts to transform our understanding of ALS and related motor neuron diseases. Through its network of multidisciplinary clinics, the Consortium captures rich, longitudinal, real-world data from people living with ALS. By sharing these insights with researchers worldwide, it is accelerating discovery, informing smarter clinical trial design, and advancing better treatment and care for those living with the 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.

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.

For Further Information, Contact:

SHIONOGI Website Inquiry Form: https://www.shionogi.com/global/en/contact.html

Shionogi Inc.: ShionogiCommunications@shionogi.com

1. The Center for Innovation & Bioinformatics. ALS/MND Natural History Consortium. (2023). 22 May 2026. Available at: https://www.data4cures.org/natural-history-consortium.

2. RADICAVA and RADICAVA ORS Prescribing Information. Florham Park, NJ: Shionogi Inc.; 2026.

3. Data on file. Shionogi Inc.

4. Xu, et al. Global variation in prevalence and incidence of amyotrophic lateral sclerosis: a systematic review and meta-analysis. J Neurol. 2020 Apr;267(4):944-953. DOI: 10.1007/s00415-019-09652-y