Development into a research-driven company with a strong global presence

「Through the development of an overseas development structure and proactive development activities, global Phase 3 enrollment for JR-141 was completed ahead of schedule. We are now working intensively toward applications for marketing approval worldwide.」

「As one of the few Japanese companies with biopharmaceutical and gene therapy technologies, our group will pursue stable business development, making the strengthening of our domestic business base over the medium to long term a priority while assessing policy and regulatory trends in Japan and overseas, so that our proprietary platform technologies and breakthrough medicines based on them can be brought from Japan to the world.」

「We entered into an exclusive co-development and licensing agreement with 武田薬品工業株式会社 for specific regions, planned launches overseas, and are conducting clinical trials and discussions with regulatory authorities. The agreement was terminated in June 2024. Our group will continue to conduct global Phase 3 clinical trials for the product in the United States, Central and South America, and Europe.」

「In May 2023, the company concluded an exclusive global development and commercialization agreement with Angelini Pharma for a novel biologic applying J-Brain Cargo® technology to epilepsy.」

「To realize full-scale global operations, we established JCR DO BRASIL in the Federative Republic of Brazil in 2020, JCR Europe in the Kingdom of the Netherlands in 2022, and JCR Luxembourg in the Grand Duchy of Luxembourg in 2022. Building on these achievements, we will continue making strategic investments to create new value and achieve full-scale globalization from the latter half of the 2020s onward.」

「During the second quarter of the consolidated fiscal year, JCR Luxembourg S.A. and JCR Europe B.V. were newly established and included in the scope of consolidation.」

「In fiscal 2021, the midpoint year of the medium-term management plan, we launched IZCARGO®, the world's first blood-brain barrier-crossing enzyme replacement therapy for Hunter syndrome, in Japan in May 2021, ahead of other countries, and began Phase 3 clinical trials in Europe, the United States and Brazil.」

「In September 2021, the company concluded an agreement with Takeda Pharmaceutical Company Limited for the joint development and commercialization of the next-generation Hunter syndrome treatment JR-141 in specified territories. This agreement enabled the Group to take another step forward as a global specialty pharmaceutical company.」

「As a global clinical trial, preparations are under way to initiate the trial in Brazil and Europe, following the United States.」

「The Company announced the three-year medium-term management plan “Henka” (Revolution into the Future), beginning in fiscal 2020 and leading up to its 50th anniversary in 2025.」

「In the Federative Republic of Brazil, we filed an application for marketing approval with the Brazilian Health Regulatory Agency (ANVISA) in December 2020. In other regions, the product received orphan drug designation from the U.S. Food and Drug Administration (FDA) in October 2018 and the European Medicines Agency (EMA) in February 2019. We are preparing for global expansion, including discussions with the FDA on initiating a global Phase 3 clinical trial.」

「During the second quarter of the consolidated fiscal year, JCR DO BRASIL was included in the scope of consolidation following the formal decision at a meeting of the Board of Directors held on 26 August 2020 to expand the business into the Federative Republic of Brazil.」

「For the blood-brain barrier-penetrating Hunter syndrome treatment JR-141 (development code: JR-141), the company began a Phase II clinical trial in Brazil in June 2018 and a Phase III clinical trial in Japan in August 2018. In other regions, the FDA granted orphan drug designation in October 2018 and the EMA granted orphan drug designation in February 2019, while the company continued preparing for global development.」

「Following the launch of Transform, the medium-term management plan beginning this fiscal year, global launches and clinical development of lysosomal disease therapies will enter full swing.」

「We will also promote the global rollout of these new drugs.」

「We will also promote the global rollout of these new drugs.」

「We will promote the global rollout of all these new drugs.」

「To realize our goal of becoming a company with a global presence, licensing products and technologies based on our technical capabilities and pursuing global expansion are extremely important challenges. In particular, the early overseas development of lysosomal disease products using J-Brain Cargo®, including JR-141, will be given the highest priority, making use of JCR USA, Inc., which was established in January last year.」

「Following this, development of the enzyme replacement therapy for Sanfilippo syndrome type A, a new drug to which J-Brain Cargo® has been applied, began in September last year under development code JR-441. Research and development of 15 lysosomal disease therapies, including the enzyme replacement therapy for Pompe disease (development code JR-162) and the enzyme replacement therapy for Hurler syndrome (development code JR-171), is proceeding in sequence, and we will promote the global rollout of all of them.」

「We are considering the global rollout of all these lysosomal disease enzyme replacement therapies. Clinical development in the United States will proceed through JCR USA, Inc., a joint venture established in the United States with メディパルホールディングス in January this year.」

「We are considering the global rollout of all lysosomal disease enzyme replacement therapies. Clinical development in the United States is scheduled to proceed through JCR USA, Inc., a joint venture established in the United States with メディパルホールディングス in January this year.」

「In January 2018, we established JCR USA, Inc., a U.S. joint venture with メディパルホールディングス, as a base for promoting the overseas rollout of products under development, including JR-141, and for commencing clinical development in the United States at an early stage.」

「To oversee the early-stage clinical development of JR-141 and JR-162 in the United States, we established the US joint venture JCR USA, Inc. with Medipal Holdings Co., Ltd. on 1 January this year. Going forward, we plan to consider the global expansion of our development portfolio from the US joint venture.」

「The Company has entered into development investment agreements with its largest shareholder, メディパルホールディングス, concerning multiple products under development. The parties have also agreed to conduct clinical development of various drug candidates through a joint venture to be established in the United States and to pursue broader business collaboration.」

「The breakthrough drug candidates created by the Company, including JR-141 using J-Brain Cargo®, are anticipated by patients worldwide. We are considering a specific framework for advancing global development, including corporate partnerships.」

「We will work to develop the Company into a research-driven company with a global presence, based on our management vision of using our proprietary biotechnology and cell therapy and regenerative medicine technologies.」