Creating New Drugs Through Development Pipelines and External Partnerships

「The domestic late Phase II clinical trial of Matsupexole (generic name; development code: KDT-3594), our internally discovered Parkinson's disease treatment, was unblinded in March 2026 and achieved its primary endpoint. In addition, following the start of the domestic Phase I clinical trial of KSP-0914 (development code), a treatment for Graves' disease, in August 2025, we began a domestic Phase I clinical trial of KSP-0576 (development code), a treatment for overactive bladder/interstitial cystitis and bladder pain syndrome, in February 2026, and a domestic Phase I clinical trial of KSP-0930 (development code), a treatment for narcolepsy, in March 2026.」

「As part of its R&D activities in the pharmaceutical business, in July 2025 the Company entered into an agreement with Viridian Therapeutics, Inc. (U.S.) to acquire exclusive development and commercialization rights in Japan for Veligrotug (generic name) and VRDN-003 (development code), treatments for thyroid eye disease. Following the commencement of domestic Phase I clinical trials of olutasidenib (generic name), a treatment for acute myeloid leukemia, in July 2025, the Company also commenced domestic Phase I clinical trials of KSP-0914 (development code), a Company-originated treatment for Graves’ disease, in August of the same year, continuing to advance its R&D projects through the development stages.」

「Our internally discovered linzagolix (generic name; development code: KLH-2109) was the subject of an application for approval in Japan for the indication of uterine fibroids in February 2025, and in March 2025 we began a domestic Phase III clinical trial toward obtaining an additional indication for endometriosis. We also began an additional domestic Phase III clinical trial of rovatirelin (generic name; development code: KPS-0373), a treatment for spinocerebellar degeneration, in March 2025. Development of KSP-0243 (development code), our internally discovered treatment for ulcerative colitis, was discontinued because it failed to achieve its primary endpoint in the Phase II clinical trial in the previous fiscal year.」

「In September 2024, we notified バイオジェニュイン社 (China) of the termination of the license agreement granting it development and commercialization rights in China and other territories.」

「As part of its R&D activities in the pharmaceutical business, the Company-originated product linzagolix (generic name; development code: KLH-2109) achieved its primary endpoints in 2 domestic Phase III clinical trials for uterine fibroids and began preparations for submission of a marketing approval application. In September 2024, the Company also entered into an agreement with Rigel Pharmaceuticals, Inc. (U.S.) to acquire exclusive development and commercialization rights in Japan, South Korea and Taiwan for olutasidenib (generic name), a treatment for acute myeloid leukemia.」

「CG0070 (development code), a treatment for muscle-invasive bladder cancer, is participating in an international joint Phase III clinical trial led by CG Oncology (United States), the source of the licensed technology, and clinical trials are under way in Japan. The sublicense agreement we entered into with Inmagene BioPharmaceuticals (China) to grant development and marketing rights in China for fostamatinib (generic name; development code: R788), a treatment for chronic immune thrombocytopenic purpura, ended in December 2023. The licensing agreement we entered into with AffaMed Therapeutics (China) to grant development and marketing rights in China and elsewhere for KDT-3594 (development code), our internally discovered Parkinson's disease treatment, also ended in May 2024.」

「The sublicense agreement we entered into with 印マジンバイオファーマシューティカルズ社 (China) granting development and commercialization rights for fostamatinib, a treatment for chronic immune thrombocytopenia, in China ended in December 2023.」

「Lovotirelin (generic name; development code: KPS-0373), a treatment for spinocerebellar degeneration, had its marketing approval application temporarily withdrawn in July of this year, and the Company is considering the feasibility of conducting additional clinical trials.」

「As part of its R&D activities in the pharmaceutical business, the Company obtained marketing approval in September of this year for difelikefalin (generic name; development code: MR13A9), a treatment for pruritus in dialysis patients for which 丸石製薬株式会社, its co-development partner, submitted an application for marketing approval in September last year. The Company is preparing to launch it after inclusion in the National Health Insurance drug price list under the product name Korsuva Injection Syringes for Dialysis 17.5 μg, 25.0 μg and 35.0 μg.」

「Regarding the spinal and cerebellar degeneration treatment rovatirelin (generic name; development code: KPS-0373), we temporarily withdrew the application for approval in July this year and are considering the feasibility of conducting additional clinical trials.」

「丸石製薬株式会社 submitted an application for marketing approval in September last year for difelikefalin (generic name; development code: MR13A9), a treatment for pruritus in dialysis patients that it is co-developing with the Company. In addition, the Company obtained marketing approval in December last year for fostamatinib (generic name; development code: R788), a treatment for chronic primary immune thrombocytopenia licensed in from Rigel Pharmaceuticals, Inc., for which it submitted an application for marketing approval in April last year. Toward its launch after inclusion in the National Health Insurance drug price list, the Company is conducting medical information activities under the product name Tavalisse Tablets 100 mg/150 mg, led by the Rare Disease Project, which specializes in marketing in rare disease areas.」

「Regarding R&D in the pharmaceutical business, the Company submitted an application for marketing approval in April of this year for fostamatinib (generic name; development code: R788), a treatment for chronic primary immune thrombocytopenia licensed in from Rigel Pharmaceuticals, Inc. In September of this year, 丸石製薬株式会社 submitted an application for marketing approval for difelikefalin (generic name; development code: MR13A9), a treatment for pruritus in dialysis patients that it is co-developing with the Company. The Company also commenced domestic Phase III clinical trials of its originated product linzagolix (generic name; development code: KLH-2109), a treatment for uterine fibroids and endometriosis, for uterine fibroids as the indication.」

「In April this year, we filed an application for approval of fostamatinib (generic name; development code: R788), a treatment for chronic immune thrombocytopenic purpura licensed in from Rigel Pharmaceuticals. We also began a domestic Phase III clinical trial for the indication of uterine fibroids of linzagolix (generic name; development code: KLH-2109), our internally discovered treatment for uterine fibroids and endometriosis.」

「The Company submitted an application for marketing approval in December last year for lovotirelin (generic name; development code: KPS-0373), a treatment for spinocerebellar degeneration licensed in from Shionogi & Co.,Ltd., and in April of this year submitted an application for marketing approval for fostamatinib (generic name; development code: R788), a treatment for chronic primary immune thrombocytopenia licensed in from Rigel Pharmaceuticals, Inc. In addition, difelikefalin (generic name; development code: MR13A9), a treatment for pruritus in dialysis patients co-developed with 丸石製薬株式会社, achieved its primary endpoint in domestic Phase III clinical trials.」

「Fostamatinib (generic name; development code: R788), a treatment for chronic immune thrombocytopenic purpura licensed in from Rigel Pharmaceuticals (United States), and difelikefalin (generic name; development code: MR13A9), a treatment for pruritus in dialysis patients co-developed with Maruishi Pharmaceutical Co., Ltd., each achieved their primary endpoints in domestic Phase III clinical trials. For CG0070 (development code), an oncolytic virus therapy licensed in from CG Oncology (United States), dosing of Japanese patients has begun in the international joint Phase III clinical trial for the indication of muscle-invasive bladder cancer.」

「Regarding research and development activities in the pharmaceutical business, we obtained marketing approval in September this year for avacopan (generic name; development code: CCX168), a treatment for microscopic polyangiitis and granulomatosis with polyangiitis licensed in from Vifor Fresenius Medical Care Renal Pharma (Switzerland), for which we filed an application for approval in February this year. We are preparing to launch it under the product name Tavneos Capsules 10 mg after its inclusion in the National Health Insurance drug price list. In May this year, EA Pharma Co., Ltd., with which we are co-developing carotegrast methyl (generic name; development code: AJM300), a treatment for ulcerative colitis, filed an application for approval. In June this year, we entered into a sublicense agreement with JW Pharmaceutical (South Korea) to grant development and marketing rights for fostamatinib (generic name; development code: R788) in South Korea, and in August this year entered into a sublicense agreement with Inmagene BioPharmaceuticals (China) to grant development and marketing rights in China, including Hong Kong and Macau.」

「In May this year, EA Pharma Co., Ltd., with which we are co-developing carotegrast methyl (generic name; development code: AJM300), a treatment for ulcerative colitis, filed an application for approval. In June this year, we entered into a sublicense agreement with JW Pharmaceutical (South Korea) to grant development and marketing rights in South Korea for fostamatinib (generic name; development code: R788), a treatment for chronic immune thrombocytopenic purpura that we licensed in from Rigel Pharmaceuticals (United States) and are currently studying in a domestic Phase III clinical trial.」

「CCX168 (development code; generic name: avacopan), a treatment for microscopic polyangiitis and granulomatosis with polyangiitis licensed in from Vifor Fresenius Medical Care Renal Pharma (Switzerland), has completed its Phase III clinical trial, and preparations are under way to file an application for approval. Linzagolix (generic name; domestic development code: KLH-2109; overseas development code: OBE2109), a treatment for uterine fibroids and endometriosis discovered by us and licensed out to Obseva (Switzerland), was the subject of an application for approval filed by the company with the European Medicines Agency (EMA) in November last year for the indication of uterine fibroids.」

「Regarding research and development activities in the pharmaceutical business, we began a domestic Phase III clinical trial of MR13A9 (development code; generic name: difelikefalin), a treatment for pruritus in dialysis patients that we are co-developing with Maruishi Pharmaceutical Co., Ltd., and continue to advance research and development across our programs, seeking to move them to higher development stages. In October this year, we entered into an agreement to grant AffaMed Therapeutics (Hong Kong) exclusive development and marketing rights and other rights for KDT-3594 (development code), a Parkinson's disease treatment discovered by us, in China, Taiwan, Hong Kong, Macau and 6 Southeast Asian countries: Singapore, Malaysia, Thailand, Indonesia, Vietnam and the Philippines.」

「Regarding R&D in the pharmaceutical business, the Company continues to advance R&D for each project and seeks to progress projects through the development stages. In addition, the Phase III clinical trial of KPS-0373, a treatment for spinocerebellar degeneration (development code; generic name: lovotirelin), has been completed. The Company is conducting detailed analyses of the results obtained in the trials, including subgroup analyses by severity, while continuing consultations with the regulatory authorities.」

「For R788 (development code; generic name: fostamatinib), a treatment for chronic immune thrombocytopenic purpura for which we entered into an agreement with Rigel Pharmaceuticals (United States) to acquire exclusive development and marketing rights in Japan, China, South Korea and Taiwan, we began the domestic Phase III clinical trial in September last year. In March this year, we entered into an agreement with CG Oncology (United States) to acquire exclusive development and marketing rights in Japan, South Korea, Taiwan and 20 Asian countries excluding China for CG0070 (development code), an oncolytic virus therapy for bladder cancer under development by the company. In addition, following a comprehensive review of the development strategy for YS110 (development code), a treatment for malignant mesothelioma that we had been developing after acquiring its domestic development and marketing rights from Wise A.C. Co., Ltd., and our research and development pipeline, we decided to discontinue development and returned the rights to the company.」

「Regarding research and development activities in the pharmaceutical business, we began the domestic Phase III clinical trial of R788 (development code; generic name: fostamatinib), a treatment for chronic immune thrombocytopenic purpura, in September last year under an agreement with Rigel Pharmaceuticals (United States) to acquire exclusive development and marketing rights in Japan, China, South Korea and Taiwan. The Phase III clinical trial of KPS-0373 (development code; generic name: rovatirelin), a treatment for spinocerebellar degeneration, has been completed, and we are conducting a detailed review of the results obtained to date, including subgroup analyses by disease severity, while continuing consultations with the regulatory authorities. The results of these pooled analyses, or post hoc analyses, have recently been published in a medical journal.」

「JR-131 (development code), a biosimilar of darbepoetin alfa (generic name) for the treatment of renal anemia, for which JCR Pharmaceuticals Co., Ltd., our co-development partner, filed an application for approval in September last year, received marketing approval in September this year. We are preparing to launch it under the product name Darbepoetin Alfa BS Injection JCR after its inclusion in the National Health Insurance drug price list. We have now begun the domestic Phase III clinical trial of R788 (development code; generic name: fostamatinib), a treatment for chronic immune thrombocytopenic purpura, for which we entered into an agreement with Rigel Pharmaceuticals (United States) to acquire exclusive development and marketing rights in Japan, China, South Korea and Taiwan. The Phase III clinical trial of KPS-0373 (development code; generic name: rovatirelin), a treatment for spinocerebellar degeneration, has been completed, and we are conducting a detailed review of the results obtained to date, including subgroup analyses by disease severity, while continuing consultations with the regulatory authorities.」

「JCR Pharmaceuticals Co., Ltd., our co-development partner, filed an application for approval of JR-131 (development code), a biosimilar of the long-acting erythropoiesis-stimulating agent darbepoetin alfa (generic name), in September last year, and we are responding to the review in preparation for approval. For R788 (development code; generic name: fostamatinib), a small-molecule tyrosine kinase inhibitor for which we entered into an agreement with Rigel Pharmaceuticals (United States) to acquire exclusive development and marketing rights in Japan, China, South Korea and Taiwan, we are preparing to start a domestic Phase III clinical trial. The Phase III clinical trial of KPS-0373 (development code; generic name: rovatirelin), a treatment for spinocerebellar degeneration, has been completed. We are conducting a detailed review of the results obtained to date, including subgroup analyses by disease severity, and have begun consultations with the regulatory authorities.」

「In September last year, JCR Pharmaceuticals Co., Ltd., our co-development partner, filed an application for approval of JR-131 (development code), a biosimilar of the long-acting erythropoiesis-stimulating agent darbepoetin alfa (generic name). In October last year, we entered into an agreement with Rigel Pharmaceuticals (United States) to acquire exclusive development and marketing rights in Japan, China, South Korea and Taiwan for R788 (development code; generic name: fostamatinib), a small-molecule tyrosine kinase inhibitor. We obtained marketing approval in February this year to add an oral disintegrating tablet formulation of the diabetes treatment Glubes combination tablets and plan to launch it after its inclusion in the National Health Insurance drug price list. The Phase III clinical trial of KPS-0373 (development code; generic name: rovatirelin), a treatment for spinocerebellar degeneration, did not show a statistically significant improvement over placebo in the change in the total SARA score, the primary endpoint used to assess ataxia. We are currently conducting a detailed review of the results obtained in the trials to date, including subgroup analyses by disease severity.」

「In October last year, we entered into an agreement with Rigel Pharmaceuticals (United States) to acquire exclusive development and marketing rights in Japan, China, South Korea and Taiwan for R788 (development code; generic name: fostamatinib), a small-molecule tyrosine kinase inhibitor. The Phase III clinical trial of KPS-0373 (development code; generic name: rovatirelin), a treatment for spinocerebellar degeneration, did not show a statistically significant improvement over placebo in the change in the total SARA score, the primary endpoint used to assess ataxia. We are currently conducting a detailed review of the results obtained in the trials to date, including subgroup analyses by disease severity.」

「In October this year, we entered into an agreement with Rigel Pharmaceuticals (United States) to acquire exclusive development and marketing rights for fostamatinib (generic name), a small-molecule tyrosine kinase inhibitor, in Japan, China, South Korea and Taiwan.」

「The Phase III clinical trial for JR-131, a biosimilar of the long-acting erythropoiesis-stimulating agent darbepoetin alfa (generic name; development code), which we are co-developing with JCR Pharmaceuticals Co., Ltd., has been completed, and we are preparing to file a marketing application. We are also continuing research and development across our programs and seeking to advance them through the development stages.」

「In September last year, we filed an application for approval to add a granule formulation of P-TOL chewable tablets. In the same month, KYORIN Pharmaceutical Co.,Ltd., our co-development partner, filed an application for approval of KRP-114V (development code; generic name: vibegron), a treatment for overactive bladder. CCX168 (development code; generic name: avacopan), a complement C5a receptor inhibitor for which we entered into an agreement with Vifor Fresenius Medical Care Renal Pharma (Switzerland) in June last year to acquire exclusive development and marketing rights in Japan, is participating in the Phase III international joint clinical trial being conducted by ChemoCentryx (United States). In the Phase III clinical trial of JR-131 (development code), a biosimilar of the long-acting erythropoiesis-stimulating agent darbepoetin alfa (generic name), which we are co-developing with JCR Pharmaceuticals Co., Ltd., equivalence to the reference biologic was verified.」

「Regarding research and development activities in the pharmaceutical business, in September last year we filed an application for approval to add a granule formulation of P-TOL chewable tablets. In the same month, KYORIN Pharmaceutical Co.,Ltd., our co-development partner, filed an application for approval of KRP-114V (development code; generic name: vibegron), a treatment for overactive bladder. CCX168 (development code; generic name: avacopan), a complement C5a receptor inhibitor for which we entered into an agreement with Vifor Fresenius Medical Care Renal Pharma (Switzerland) in June last year to acquire exclusive development and marketing rights in Japan, has entered Phase I clinical trials. In the Phase III clinical trial of JR-131 (development code), a biosimilar of the long-acting erythropoiesis-stimulating agent darbepoetin alfa (generic name), which we are co-developing with JCR Pharmaceuticals Co., Ltd., equivalence to darbepoetin alfa, the reference biologic, was verified. The application filed in July two years ago for approval to add an oral disintegrating tablet formulation of Glubes combination tablets was withdrawn in June last year, and additional trials are being conducted.」

「In R&D activities in the pharmaceutical business, we filed an application for approval in September this year for an additional formulation of Pitol chewable tablets, granules. In addition, because we obtained manufacturing and marketing approval in September this year for AJG511 (development code; generic name: budesonide), a treatment for ulcerative colitis for which our co-development partner EA Pharma Co., Ltd. filed an application for approval last October, we are preparing to launch the product under the name Rectable 2 mg rectal foam, 14 doses, after its inclusion in the National Health Insurance drug price list. Also in September this year, our co-development partner KYORIN Pharmaceutical Co.,Ltd. filed an application for approval of KRP-114V (development code; generic name: vibegron), a treatment for overactive bladder. In June this year, we entered into an agreement with Vifor Fresenius Medical Care Renal Pharma Ltd. (Switzerland) to acquire exclusive development and marketing rights in Japan for CCX168 (development code; generic name: avacopan), a complement C5a receptor inhibitor, and have initiated a Phase I clinical trial.」

「In March this year, we entered into an agreement with Maruishi Pharmaceutical Co., Ltd. to collaborate on the development and marketing in Japan of MR13A9 (development code), a κ-opioid receptor agonist for pruritus in dialysis patients. We have also begun Phase III clinical trials of JR-131 (development code), a biosimilar of the long-acting erythropoiesis-stimulating agent darbepoetin alfa (generic name), and an additional Phase III clinical trial of KPS-0373 (development code; generic name: rovatirelin), a treatment for spinocerebellar degeneration. In June this year, we entered into an agreement with Vifor Fresenius Medical Care Renal Pharma (Switzerland) to acquire exclusive development and marketing rights in Japan for avacopan (generic name), a complement C5a receptor inhibitor.」

「In addition to filing an application in July last year for approval to add an oral disintegrating tablet formulation of Glubes, EA Pharma Co., Ltd., our co-development partner, filed an application in October of the same year for approval of AJG511 (development code; generic name: budesonide), a treatment for ulcerative colitis. We have also begun Phase III clinical trials of JR-131 (development code), a biosimilar of the long-acting erythropoiesis-stimulating agent darbepoetin alfa (generic name), and an additional Phase III clinical trial of KPS-0373 (development code; generic name: rovatirelin), a treatment for spinocerebellar degeneration.」

「In July this year, we submitted an application for approval of an additional dosage form of Glubes, the orally disintegrating tablet. We also began a Phase III clinical trial of JR-131, a biosimilar of the long-acting erythropoiesis-stimulating agent darbepoetin alfa, and continue to advance R&D on each project and move projects to the next stage of development. In addition, during the cumulative consolidated period, we began an additional Phase III clinical trial of KPS-0373, a treatment for spinocerebellar degeneration, under development with the generic name lerotilelin.」

「For KPS-0373, a treatment for spinocerebellar degeneration (development code; generic name: troriluzole), we decided during the current consolidated cumulative period to conduct an additional Phase III clinical trial.」

「As we obtained manufacturing and marketing approval in February this year for an additional dosage form of Glufast, the orally disintegrating tablet for which we submitted an approval application in January last year, we plan to launch it after its inclusion in the National Health Insurance drug price list. In March this year, we also entered into an agreement with KYORIN Pharmaceutical Co.,Ltd. for the joint development and co-promotion in Japan of KRP-114V, a treatment for overactive bladder, under development with the generic name vibegron.」