Expansion of International Partnering, Technology Licensing and API Supply

「同契約に基づき、当社は、2026年7月にアラブ首長国連邦で製造販売承認を取得し、このほどクウェート国での承認を取得しました。」

「In October 2025, the company entered into an agreement granting development and sales rights for agalsidase beta BS intravenous infusion "JCR" in 9 countries across the Middle East, Turkey and North Africa.」

「This was because, although domestic product sales amounted to ¥31,655 million, down ¥981 million year on year, research and development expenses totaled ¥15,431 million, up ¥4,196 million year on year, and the licensing agreements scheduled for execution during the fiscal year were not concluded, preventing recognition of contract upfront payments.」

「The company terminated certain licensing and other agreements relating to development products during the current interim consolidated accounting period, with the mutual agreement of the parties to the agreements.」

「For this product, the policy is to advance development through an out-licensing arrangement rather than in-house development, and negotiations are under way with prospective partners regarding the license.」

「Although the exclusive co-development and licensing agreement with Takeda Pharmaceutical Company Limited for the next-generation recombinant fusion protein JR-141 (INN: pabinafusp alfa) in specified territories ended in June 2024, the group will continue conducting a global Phase 3 clinical trial for the drug in the United States, Latin America and Europe.」

「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 March 2023, the company entered into a joint research, option and licensing agreement with Alexion, AstraZeneca Rare Disease for a proprietary therapeutic drug candidate applying J-Brain Cargo® technology and targeting neurodegenerative diseases.」

「For the treatment for fucosidosis (development code: JR-471), under the license agreement executed in October 2022, the company granted メディパルホールディングス an exclusive license, including the right to sublicense, to commercialize the product through research, development, manufacturing and sales worldwide excluding Japan.」

「To strengthen its relationship with Mycenax, a biopharmaceutical contract development and manufacturing organization with a track record of providing full-service CDMO operations, the company will use this subscription to increase the flexibility of its internal production capacity and accelerate production activities, thereby enabling multiple research and development programs to progress concurrently.」

「For Izcargo®, the company entered into an exclusive co-development and licensing agreement with Takeda Pharmaceutical Company Limited in specified territories (September 2021).」

「In September 2021, the company entered into an exclusive co-development and licensing agreement with Takeda Pharmaceutical Company Limited for JR-141 (INN: pabinafusp alfa), a next-generation recombinant fusion protein currently under development as a treatment for Hunter syndrome, in specified territories.」

「In addition, with respect to certain development products currently at the preclinical stage, the parties mutually agreed to terminate the previously executed right of first negotiation agreement in May this year. This resulted in a contract termination loss of ¥1,000 million, which was recognized as an extraordinary loss.」

「Contract revenue from the agreement concerning the out-licensing of the enzyme replacement therapy for lysosomal diseases has been pushed back to the consolidated accounting period for the fourth quarter.」

「Because agreement on the contract for the out-licensing of a lysosomal disease therapeutic enzyme product had not been reached by the end of September, partly because face-to-face negotiations had become difficult due to the spread of COVID-19, the contract payment income expected in the cumulative consolidated period through the second quarter was deferred to the third quarter and thereafter.」

「In addition, in terms of technology, the company established manufacturing technology for gene therapy products and will promote the development of new drugs and expansion of its licensing business together with its blood-brain barrier-penetrating technology, J-Brain Cargo®.」

「The out-licensing of products and technologies leveraging the company's technological capabilities, as well as global expansion, are extremely important issues.」

「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.」

「Regarding product out-licensing, beginning with JR-141 (Hunter syndrome), which uses J-Brain Cargo®, we will consider the optimal options for JR-162 (Pompe disease), JR-171 (Hurler syndrome) and subsequent lysosomal disease therapeutic enzymes, with a view to early overseas development in parallel with development in Japan and obtaining marketing approval and manufacturing authorization, and will also consider out-licensing where necessary.」

「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.」

「Conducting clinical development of various drug candidates through a joint venture to be established in the United States by the two companies, and the acquisition of the Company's shares by メディパルホールディングス.」

「Given the need to supply pharmaceuticals stably to countries worldwide, concentrating our bases in Japan is recognized as a business continuity risk in view of the country's geographic characteristics, including frequent earthquakes and localized disasters. To diversify these risks and establish a global pharmaceutical supply system, we are currently researching and considering the establishment of bases, including in Europe and the United States, with the use of the Swiss subsidiary among the options under consideration.」

「Based on the breakthrough technologies described above, we will actively pursue partnerships with overseas companies by leveraging the subsidiary established in Switzerland last year, seeking overseas expansion through both technology licensing and drug-substance supply.」