Commercialization, Joint Research and Licensing of J-Brain Cargo®

「In July 2025, we entered into a license option agreement with Acumen Pharmaceuticals for the out-licensing of J-Brain Cargo® technology, with the aim of developing a blood-brain-barrier-penetrating Alzheimer’s disease treatment.」

「As one result of this work, we created JUST-AAV, a new gene therapy technology using an adeno-associated viral vector. In addition to efficiently delivering vectors to the brain, it is expected to reduce adverse effects by reducing vector accumulation in the liver, and development is under way as a new platform technology. In December 2023, we began joint research with Modalis Co., Ltd. toward developing novel gene therapies using this technology. In January 2025, after achieving initial proof of the technology concept in this joint research, we entered into a new joint research agreement.」

「As one result of this work, we announced in May 2024 a new gene therapy technology using J-Brain Cargo® technology. By modifying an adeno-associated virus with J-Brain Cargo®, this technology reduces uptake by the liver and efficiently delivers the vector to the brain. Development is under way as a new platform technology.」

「In December 2023, we entered into a joint research, option and licensing agreement with Alexion AstraZeneca Rare Disease (Alexion) to create novel nucleic acid medicines using J-Brain Cargo® technology. In the joint research on a neurodegenerative disease treatment using J-Brain Cargo® technology under the agreement signed in March 2023, we achieved a research-stage milestone in March 2024.」

「In December 2023, the company concluded a joint research, option and license agreement with Alexion AstraZeneca Rare Disease (Alexion) for the discovery of novel nucleic acid medicines applying J-Brain Cargo® technology. This was the second partnership with Alexion, following the joint research, option and license agreement concerning a treatment for neurodegenerative diseases applying J-Brain Cargo® technology concluded in March 2023.」

「In September 2023, the company concluded a license agreement with Medipal Holdings Corporation for commercialization outside Japan, as well as a joint development and commercialization agreement in Japan.」

「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.」

「In addition to research expanding the potential application of JCR's proprietary blood-brain barrier-penetrating technology J-Brain Cargo® to various modalities, we are focusing on creating new foundational technologies beyond J-Brain Cargo®. In March 2023, we entered into a joint research, option and licensing agreement with Alexion AstraZeneca Rare Disease covering undisclosed drug candidates using J-Brain Cargo® technology for neurodegenerative diseases.」

「In the United States, the FDA granted Fast Track designation in February 2021 and Rare Pediatric Disease designation in December 2022. In the European Union, the EMA granted PRIME designation in October 2021.」

「In addition, in March 2022 we entered into a joint research and development agreement with Takeda Pharmaceutical Company Limited concerning gene therapies using J-Brain Cargo® technology for multiple lysosomal diseases. This represents a first step toward demonstrating that J-Brain Cargo® technology itself can be applied to a range of modalities.」

「For the blood-brain barrier-penetrating enzyme therapy for mucopolysaccharidosis type IIIA (development code: JR-441), the EC granted orphan drug designation in January 2022, making the product eligible for various incentives to accelerate development in the European Union. Preparations are currently under way to begin global clinical trials in the first half of 2023.」

「Preparations are currently under way to start the global Phase III clinical trial to be conducted in the United States, Brazil and Europe.」

「In the development of lysosomal disease treatments, which is currently a priority, we are developing new drugs using our proprietary blood-brain barrier-penetrating technology J-Brain Cargo®. We began selling the blood-brain barrier-penetrating Hunter syndrome treatment pabinafusp alfa (development code: JR-141; Izcargo® intravenous infusion 10 mg) in Japan in May 2021.」

「For the blood-brain barrier-penetrating mucopolysaccharidosis type I enzyme replacement therapy JR-171 (development code: JR-171), a clinical trial notification for a Phase I/II trial in Japan was accepted by the Pharmaceuticals and Medical Devices Agency (PMDA) in July 2020 as part of a global clinical trial, and the investigational drug was administered to the first subject in October 2020. ANVISA and the FDA have also approved the start of the clinical trial.」

「For the blood-brain barrier-penetrating mucopolysaccharidosis type I enzyme replacement therapy JR-171 (development code: JR-171), as our first global clinical trial, a clinical trial notification for a Phase I/II trial in Japan was accepted by the Pharmaceuticals and Medical Devices Agency (PMDA) in July 2020, and the investigational drug was administered to the first subject in October 2020. ANVISA and the FDA have also approved the start of the clinical trial.」

「For the blood-brain barrier-penetrating mucopolysaccharidosis type I enzyme replacement therapy JR-171 (development code: JR-171), a clinical trial notification for a Phase I/II trial was submitted to the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan in July this year and accepted, and to Brazil's National Health Surveillance Agency (ANVISA) in October this year and accepted.」

「In the development of lysosomal disease treatments, which is currently a priority, we are developing new drugs using our proprietary blood-brain barrier-penetrating technology J-Brain Cargo®.」

「After extensive long-term consideration of its strategy for developing lysosomal disease treatments for the global market, we determined that acquiring ArmaGen's technology assets, including its intellectual property rights for blood-brain barrier-penetrating technology, would secure our technological advantage.」

「In particular, the Phase II clinical trial in Brazil of the blood-brain barrier-penetrating Hunter syndrome enzyme replacement therapy JR-141 (development code: JR-141) began in June the year before last, and the Phase III clinical trial in Japan began in August the year before last. Both clinical trials progressed smoothly. In other regions, the product received orphan-drug designation from the US Food and Drug Administration (FDA) in October the year before last and from the European Medicines Agency (EMA) in February last year, while preparations for global expansion continue steadily.」

「In particular, the Phase II clinical trial in Brazil of the blood-brain barrier-penetrating Hunter syndrome enzyme replacement therapy JR-141 (development code: JR-141) began in June last year, and the Phase III clinical trial in Japan began in August last year. Both clinical trials progressed smoothly. In other regions, the product received orphan-drug designation from the US Food and Drug Administration (FDA) in October last year and from the European Medicines Agency (EMA) in February this year, while preparations for global expansion continue steadily.」

「In particular, the Phase II clinical trial in Brazil of the blood-brain barrier-penetrating Hunter syndrome enzyme replacement therapy JR-141 (development code: JR-141) began in June last year, and the Phase III clinical trial in Japan began in August last year. The product also received orphan-drug designation from the US Food and Drug Administration (FDA) in October last year and from the European Medicines Agency (EMA) in February this year, and development is progressing smoothly.」

「JR-141 is a groundbreaking Hunter syndrome enzyme replacement therapy using J-Brain Cargo®. As an innovative drug expected to be effective against central nervous system symptoms for which no treatment had previously existed, it received designation under Japan's SAKIGAKE designation system from the Ministry of Health, Labour and Welfare in March last year, orphan-drug designation from the US Food and Drug Administration (FDA) in October last year, and orphan-drug designation from the European Medicines Agency (EMA) in February this year.」

「JR-141's Phase II clinical trial in Brazil began in June last year, its Phase III clinical trial in Japan began in August last year and patient enrollment was completed in December. In addition, the US Food and Drug Administration (FDA) granted orphan drug designation last October, and development is progressing smoothly. Subsequently, the company began developing the enzyme therapy for type A Sanfilippo syndrome JR-441 last September as a new drug incorporating J-Brain Cargo®. It is sequentially conducting R&D on 15 lysosomal disease treatments, including the Pompe disease enzyme therapy JR-162 and the Hurler syndrome enzyme therapy JR-171, and plans to pursue global development for all of them.」

「Following this, we began development in September this year of JR-441 (development code: JR-441), an enzyme replacement therapy for Sanfilippo syndrome type A using J-Brain Cargo®. We will sequentially develop treatments for 15 lysosomal diseases, including the Pompe disease enzyme replacement therapy JR-162 (development code: JR-162) and the Hurler syndrome enzyme replacement therapy JR-171 (development code: JR-171).」

「In June this year, we began a Phase II clinical trial in Brazil of the blood-brain barrier-penetrating Hunter syndrome enzyme replacement therapy JR-141 (development code: JR-141), which uses our proprietary J-Brain Cargo® technology. A Phase III clinical trial in Japan is scheduled to begin in August. We will then advance research and development of the J-Brain Cargo®-based Pompe disease enzyme replacement therapy JR-162 (development code: JR-162) and the Hurler syndrome enzyme replacement therapy JR-171 (development code: JR-171), and aim to add these three products, together with treatments for a further 12 lysosomal diseases and their central nervous system complications, to our development pipeline as soon as possible following basic research.」

「Our proprietary J-Brain Cargo® technology may be broadly applicable to many drug candidates targeting central nervous system diseases, and we are pursuing technology out-licensing to companies in Japan and overseas.」

「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.」

「JR-171 (recombinant α-L-iduronidase using J-Brain Cargo®) — Preclinical — Enzyme replacement therapy for Hurler syndrome (lysosomal disease) — Uses J-Brain Cargo®」

「In research and development, the Phase III clinical trial of the biosimilar of the long-acting erythropoiesis-stimulating agent darbepoetin (development code: JR-131) and the Phase I/II clinical trial of the Hunter syndrome enzyme replacement therapy JR-141 (development code: JR-141), which uses the proprietary blood-brain barrier-penetrating technology J-Brain Cargo®, are progressing smoothly as planned. In preclinical development, research and development of the long-acting growth hormone JR-142 (development code: JR-142) and the Pompe disease enzyme replacement therapy JR-162 (development code: JR-162), which uses J-Brain Cargo®, are also progressing.」

「In March this year, we began clinical trials of the groundbreaking Hunter syndrome enzyme replacement therapy JR-141, a new drug using our proprietary blood-brain barrier-penetrating technology J-Brain Cargo®. In addition, we began developing JR-162, an enzyme replacement therapy for Pompe disease and the second new drug candidate to use J-Brain Cargo®.」

「The Hunter syndrome enzyme replacement therapy JR-141, a groundbreaking new drug using the proprietary blood-brain barrier-penetrating technology J-Brain Cargo®, has entered the final stage of preparations for starting clinical trials during the current fiscal year, and the long-awaited clinical trial will begin shortly.」

「In April this year, a new investigational drug manufacturing center and cell processing center were completed, accelerating the development of the groundbreaking Hunter syndrome enzyme replacement therapy JR-141, which uses the blood-brain barrier-penetrating technology J-Brain Cargo®, as well as new regenerative medicine products.」

「J-Brain Cargo® is a breakthrough technology that delivers intravenously administered drugs into the brain. The Hunter syndrome treatment currently under development using this technology (development code: JR-141) has already produced favorable results in animal studies, and a clinical trial is scheduled to begin during the current fiscal year.」