Comprehensive support to accelerate R&D of biopharmaceuticals

Paving the way to the clinical stage as a "hub" connecting academia, industry, and government

The Research and Development Program for Innovative Biologics is a five-year initiative launched by the Japan Agency for Medical Research and Development (AMED) in fiscal year 2024. Its objective is to create innovative, high-functional biopharmaceuticals originating in Japan and to strengthen international competitiveness. To date, 19 promising research projects from across the country have been selected, each conducting research activities aimed at advancing their drug discovery "seeds" to the clinical stage.

One of the features of this project is the comprehensive support system provided by a support team consisting of approximately 20 experts. These members possess practical experience in universities, pharmaceutical companies, bio-ventures, and regulatory authorities, and are currently affiliated with Tohoku University, Keio University, the University of Osaka, and Hiroshima University.

The team covers a wide range of fields essential for drug discovery, including intellectual property, non-clinical research, CMC (Chemistry, Manufacturing, and Controls), regulatory affairs, and clinical development, ensuring a support structure geared toward clinical transition. Under the guidance of Program Supervisors (PS) and Program Officers (PO), the team provides "hands-on" support that works closely alongside researchers.

Since the start of the program in 2024, we have maintained a continuous understanding of research progress, fundraising, and corporate collaboration through on-site visits to principal investigators of all the research projects, regular participation in research meetings, and online consultations. This information is shared at joint meetings with PS, PO, and AMED held twice a month, where feedback from the participants is used to refine our support strategies. Currently, we are identifying key issues and clarifying support measures to increase the likelihood of achieving clinical stage-up, with an eye toward this fiscal year's interim evaluation.

In terms of operations, the support term established its own information management system. Both data aggregation and high security are ensured not only by the AMED information sharing infrastructure, but in addition by the support term's system. We also focused on creating an environment where researchers can share information with peace of mind through thorough training on the management of confidential information. We are also actively promoting interaction between researchers and related organizations, having held networking events in the first and second fiscal years of the program.

These gatherings of researchers, AMED, and the support team have sparked specific discussions on cross-project collaboration and concrete movements toward research advancement. Regarding commercialization support, we have begun formulating TPPs (Target Product Profiles) for all research projects. In addition to individual consultations regarding out-licensing and entrepreneurship, we are laying the foundation for social implementation of research results through study group meetings focused on startup creation.

For intellectual property, experts are involved in each project to advise on patent strategies and data preparation. Furthermore, for non-clinical trials, we provide meticulous support tailored to the needs of each project, such as preparing for PMDA consultations on data acquisition policies, conducting CDMO surveys for CMC, and selecting contractors.

Additionally, we have commissioned surveys by external specialist organizations targeting nucleic acid medicine, cancer therapeutics, early exploratory clinical trials, and conjugate medicines to organize the latest development trends and competitive information. The results of these surveys will be utilized in future clinical stage-up strategies.

We are also actively engaged in information dissemination through the launch of an official website, features in the New Year's Day 2025 issue of the Science News and this series, and business introductions at JBA (Japan Bioindustry Association) and JPMA (Japan Pharmaceutical Manufacturers Association).

Through these activities, we aim to share research results with society, achieve clinical stage-up, and facilitate corporate collaboration and out-licensing. The path to innovative drug discovery pursued in this program is by no means flat. As a hub connecting academia, industry, and government, the support team will continue to provide diligent assistance to deliver drug discovery seeds originating in Japan to patients as quickly as possible.