Drug Discovery Research

Technology enhancement to improve the capability for drug discovery and the probability of success in the clinical development stage is indispensable for the continual creation of excellent pharmaceutical products.

The Company is strengthening translational research to bridge the gap between non-clinical and clinical research. Specifically, the Company has established a comprehensive translational research platform capable of continuously generating clinical candidates. This unique platform consists of the combination of the proprietary drug discovery technologies for selecting potential candidates for clinical development, such as patient-derived iPS cell technology and neural circuit technology (electroencephalogram, brain imaging, etc.), original clinical data accumulated through the development of new drugs and clinical candidates in late-stage, unique drug targets, such as trace amine-associated receptor 1 (TAAR1), and new molecules that directly connected to pathophysiology, such as excitatory/inhibitory balance in brain disorders. Clinical samples obtained through collaboration with academia, particularly in oncology, are also utilized in translational research.
Furthermore, to increase the speed and success probability of drug discovery, the Company is promoting a data-driven drug discovery approach, based on its in-silico drug discovery platform, which utilizes data from drug discovery, clinical studies and real-world data from patients.
With its expertise in small molecule drug discovery, the Company is diversifying its approaches by pursuing chemical modalities (a unique platform for creating advanced medicines based on medicinal chemistry).

In addition to these activities in drug discovery research, under the Partnership to Realize Innovative Seeds and Medicines (PRISM) framework, which is an open innovation program launched in FY2015, the Company invites application of ideas that the Company's drug discovery research needs from research institutions and researchers in Japan.

iPSC-based drug discovery technology

iPS cells are a new type of stem cells first generated in 2006 by Dr. Shinya Yamanaka. These cells are established by introducing a set of specific factors into the cells derived from skin, blood, or other tissues. Human iPS cells have a potential to differentiate into various cell types that constitute human body, and are highly expected as a cell source in regenerative medicine and drug discovery.
By using iPS cells generated from patient's somatic cells, it is possible to recapitulate disease-related phenomena occurring in the patient body in a cell culture dish. Recently, a three-dimensional culture system such as organoid technology is making a great progress. This leads to the production of the organoids which more precisely incorporate features of human tissues and organs. The advance in iPS cell research will accelerate the identification of key disease mechanisms and the development of effective therapies.

The Company is conducting a number of drug discovery researches using patient-derived iPS cells with various diseases. For example, the Company succeeded in uncovering the novel mechanism of fibrodysplasia ossificans progressiva (FOP), a rare genetic disease in which heterotopic bones grow inside muscles, and discovered a new drug candidate by utilizing patient-derived iPS cells in a joint research project with the Center for iPS Cell Research and Application, Kyoto University.
The Company has also succeeded in identifying several clinical candidates using iPS cells in the area of Psychiatry & Neurology, one of its priority disease areas.

In-silico drug discovery technology

In-silico drug discovery technology becomes more and more essential not only to accelerate the conventional drug discovery, but also to provide innovative solutions for all drug discovery processes. Big and complex data in drug discovery are collected from biological, chemical, pharmacological and clinical domains, including phenotypical signals derived from cells, brains even patients. The Company engages in In-silico analysis of these data using state-of-the-art computational science and technologies. Especially, the Company focuses on three research aims: 1) studies that seek promising novel drug targets or mechanisms for specific diseases using informatics, including artificial intelligence method, 2) those that explore drug candidate compounds to target proteins using molecular simulation technology, and 3) those that develop better indicators of response to therapies and/or clinical conditions, called biomarkers, using biomedical informatics. The Company actively promotes rapid transformation from “conventional hypothesis-driven research” to “data-driven drug discovery research” based on comprehensive, multifaceted and objective data analysis and computational results.

PRISM, an open innovation program

Under the Partnership to Realize Innovative Seeds and Medicines (PRISM) framework, which is an open innovation program launched in FY2015, research institutions and researchers in Japan are invited to submit ideas that meet the Company's drug discovery research needs.