September 8, 2021

Phase 2 Trial of MT-3921 Initiated for Treatment ofSpinal Cord Injury
Mitsubishi Tanabe Pharma Corporation (MTPC, Head Office: Chuo-ku, Osaka;President & Representative Director, CEO: Hiroaki Ueno) and Professor Toshihide
Yamashita at the Department of Molecular Neuroscience and Department of NeuroMedical Science, Osaka University Graduate School of Medicine, announced todaythat MTPCʼs research and development subsidiary in the US, Mitsubishi Tanabe
Pharma Development America, Inc. (MTDA) has initiated enrollment of a Phase 2clinical trial of jointly developed MT-3921 for the treatment of spinal cord injury.
With no effective therapy available, spinal cord injury is a primary disease target forregenerative medicines. MT-3921 is a novel humanized immunoglobulin G1 (IgG1)
monoclonal antibody that binds to repulsive guidance molecule A(RGMa) for thetreatment of spinal cord injury. It is a potentially significant addition to thetreatment of neurological disorders, one of the focus areas of MTPCʼs development
pipeline. MTDA was granted Fast Track Designation1 for MT-3921 for the treatmentof spinal cord injury in July 2021 by the U.S. Food and Drug Administration (FDA).
MTDA has initiated a Phase 2 Proof of Concept (POC) study (NCT04683848) to enroll
72 patients with spinal cord injury in the US, Canada, and Japan. It is a multicenter,randomized, double-blind, placebo-controlled, parallel-group study to evaluate thesafety, efficacy, and tolerability of MT-3921. The primary endpoint of the study is achange in Upper Extremity Motor Score (UEMS) after 6 months of treatment.
Professor Yamashitaʼs research team supports the Phase 2 study with preclinicalresearch and scientific communication with the study sites.
Mitsubishi Tanabe Pharma Group and Osaka University Graduate School of Medicine
are committed to advance research and development activities to satisfy unmet
medical needs and to deliver innovative treatments to patients.
1US Food and Drug Administration. Fast Track Information Page:
https://www.fda.gov/ForPatients/Approvals/Fast/ucm405399.htm

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Mitsubishi Tanabe Pharma CorporationCommunication Crossroads Department
Media contacts: TEL +81 6 6205 5119
Osaka University
Graduate School of Medicine
Department of Molecular Neuroscience
Professor Toshihide Yamashita
Media contact: TEL +81 6 6879 3661

About MT-3921
Based on the results of fundamental research by Professor Yamashitaʼs researchteam, MT-3921 is a humanized anti-RGMa antibody jointly developed by MTPC andOsaka University since 2005. Current research concluded that RGMa is associatedwith inhibition of neuronal survival and neuroregeneration, involved in theprogression of inflammation, and may play a role in neurological diseases including
spinal cord injury, stroke, and multiple sclerosis. The data from the preclinical animalstudies conducted by Professor Yamashitaʼs research team and MTPC indicate thattreatment with MT-3921 improves locomotor function and promotes
neuroregeneration.
Pre-clinical studies conducted by Professor Yamashitaʼs team were supported by agrant from the Japan Agency for Medical Research and Development (AMED).
About Spinal Cord Injury
Spinal cord injury (SCI) typically occurs due to a traumatic, sudden onset of external
force to the spinal cord, resulting in neurological impairment or loss of motor,
sensory, autonomic, and/or reflex functions below the neurological level of injury.
SCI is a serious medical condition due to automobile accidents, falls, sports-related
accidents, violence, and other causes. The injuries vary in severity and can cause
motor paralysis, loss of sensation, and disturbance of bladder and rectal function.
Spinal cord injuries affect the health of the patients and place a major burden on
the patientsʼ families and caregivers. Epidemiological studies estimate there areapproximately 18,000 acute SCI incidents in the United States annually. There is noFDA-approved treatment for acute spinal cord injury. The current treatment of
spinal cord injury mainly focuses on surgical spine stabilization, intensive
neurological rehabilitation, and prevention and treatment of acute and chronic
complications. No effective treatment for traumatic paraplegia or quadriplegia isavailable to date.

2/3About Mitsubishi Tanabe Pharma Corporation (MTPC)
Mitsubishi Tanabe Pharma Corporation (MTPC), founded in 1678, is one of the oldest
pharmaceutical companies in the world. Focused on ethical pharmaceuticals, MTPC
is headquartered in Doshomachi, Osaka, the birthplace of Japanʼs pharmaceuticalindustry. In light of the anticipated changes in healthcare needs in the future, MTPC
is advancing business activities under MISSION of “Creating hope for all facingillness,” and has set the corporate vision for 2030 (VISION 30) to “Be a healthcarecompany that delivers optimal therapy to each individual.” As part of the vision for2030, MTPC is prioritizing work on “precision medicine” to create effective therapies
and preventive methods by identifying patient populations with high potential for
efficacy and safety, focusing on the disease areas of central nervous system andimmuno-inflammation. In addition, MTPC is working to develop “around the pillsolutions” to address specific patient concerns based on therapeutic medicine,including prevention of diseases, pre-symptomatic disease care, prevention ofaggravation and prognosis. MTPC is a member of Mitsubishi Chemical HoldingsGroup.
For more information, please go to https://www.mt-pharma.co.jp/e/About Mitsubishi Tanabe Pharma Development America, Inc. (MTDA)
The U.S. headquarters of Mitsubishi Tanabe Pharma Development America, Inc.
(MTDA) is located in Jersey City, New Jersey. MTDA is a wholly-owned subsidiary of
Mitsubishi Tanabe Pharma Corporationʼs 100 percent-owned U.S. holding company,
Mitsubishi Tanabe Pharma Holdings America, Inc. MTDA is dedicated to the researchand development of innovative pharmaceutical products, satisfying unmet medicalneeds.
For more information, please go to https://mt-pharma-development-america.com/

About Osaka University
Originating from Kaitokudo and Tekijuku, centuries-old institutions establishedduring Japanʼs Edo period, Osaka University was founded in 1931 as the sixth
imperial university of Japan to satisfy the strong demand from the people, thebusinesses, and the government in Osaka. With 11 faculties, 16 graduate schools,
and 6 research institutes, Osaka University continues to develop as a leading
comprehensive research university. With its motto “Orchestration and co-creationof knowledge”, Osaka University conducts creative activities in cooperation withsociety, driving towards its goal of becoming a “World-Leading Innovative University
Contributing to Social Changes” by its 100th anniversary in 2031.
For more information, please go to https://www.osaka-u.ac.jp/en/

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