Development of Drug Discovery Pipelines Other Than ALS

「In particular, KP2021 and KP2032 are currently being prepared for Phase I/II clinical trials.」

「For the development pipelines other than ALS, we extended the joint research agreement with Kitasato Institute on hearing loss through March 2027 and will further obtain nonclinical data and evaluate efficacy using various assessment systems toward the start of clinical trials.」

「We extended the joint research agreement with Kitasato Institute on hearing loss through March 2026 and are obtaining more specific data toward company-sponsored clinical trials and preparing for their smooth initiation. In addition, on 20 November 2025 we entered into a new joint research agreement with Jikei University for analysis of the mechanism of action of novel treatments for neurodegenerative diseases, with the aim of developing new pipelines in the iPS drug discovery business and systematically elucidating and accumulating the scientific evidence needed for future clinical application.」

「On 20 November 2025, the company also entered into a joint research agreement with 学校法人慈恵大学 concerning analysis of the mechanism of action of novel therapeutic agents for neurodegenerative diseases. The research is being conducted to identify and develop new development pipelines in the iPS drug discovery business and systematically elucidate and accumulate the scientific evidence required for future clinical applications.」

「For the development pipelines other than ALS, we extended the joint research agreement with Kitasato Institute on hearing loss through March 2026, obtained more specific data toward conducting company-sponsored clinical trials and prepared for their smooth initiation. We also presented research findings on FTD at the International Society for Stem Cell Research (ISSCR) 2025 annual meeting held from 11 to 14 June 2025.」

「For the development pipelines other than ALS, we have filed multiple patent applications, including the 2 February 2024 application for “Therapeutic Agent and Therapeutic Composition for Huntington's Disease” (Japanese Patent Application No. 2024-012936). We are also proceeding in line with our research and development plans, including presenting research findings on frontotemporal dementia (FTD; development code KP2021) and Huntington's disease (HD; development code KP2032) at Society for Neuroscience (SfN) 2024 in Chicago, Illinois, from 5 to 9 October 2024.」

「For the development pipelines other than ALS, we are proceeding in line with our research and development plans, including filing on 2 February 2024 a patent application (Japanese Patent Application No. 2024-012936) for “Therapeutic Agent and Therapeutic Composition for Huntington's Disease.”」

「For the development pipelines other than ALS, we are proceeding in line with our research and development plans, including filing on 2 February 2024 a patent application (Japanese Patent Application No. 2024-012936) for “Therapeutic Agent and Therapeutic Composition for Huntington's Disease.”」

「For the hearing-loss pipeline, we began joint research with Kitasato Institute in June 2023. For the frontotemporal dementia pipeline, we made progress including completing the selection of 1 final compound, obtaining the necessary data and filing a patent application on 2 November 2023. In the other iPS drug discovery pipelines, we are also advancing preparations for future clinical trials, including completing final compound selection for the Huntington's disease pipeline.」

「We are also researching the 5 development pipelines other than ALS using patient-derived iPS cells and are actively advancing development of treatments.」