1) Diverse modality development (pharmaceuticals, medical devices, software as a medical device)
We hope to solve medical issues and create new medical treatments that enable people to enjoy lifelong health, both physically and mentally. In the pharmaceutical industry, modalities are diversifying, from development focused on small molecule drugs to biopharmaceuticals (antibody drugs, nucleic acid drugs, gene therapy, cell therapy). In addition, due to recent advances in engineering and information technology, the search for new medical treatment by integrating information and engineering technology is progressing, and major pharmaceutical companies in Europe, the United States and Japan have already expanded from single drug businesses to general medical solutions. Medical treatment options are also expanding, such as pharmaceuticals, medical devices, and even software as a medical device that utilizes artificial intelligence (AI). For this reason, in addition to the chemical and biological research that has been the focus of our company so far, we are expanding our horizons to include engineering and information research, creating a diverse and attractive research and business portfolio.
2) Addressing the medical issues of the declining birthrate and aging population
The World Health Organization (WHO) positions important diseases associated with aging and lifestyle (aging-related diseases) as “non-communicable diseases (NCDs)”, and targets cancer, diabetes, respiratory diseases, and cardiovascular diseases. These diseases count for 74% of all deaths worldwide in 2019 (WHO). Our development products target all of these four diseases, and we are developing important drugs not only in developed countries but also in emerging countries. The declining birthrate is also an important social issue, but there are not many pharmaceutical companies that are focusing on it. We also focus on medical issues for women (premenstrual dysphoric disorder, menopause, AI diagnosis of breast cancer) and children (rapid diagnosis of phenylketonuria).
3) Network with research institutes such as academia and medical institutions
The role of public research institutes such as academia and medical institutions in creating medical innovation is expanding. Unlike traditional small-molecule drugs, the technological base and seeds of biopharmaceuticals utilizing genetic engineering and other technologies reside in public research institutions. In addition, medical institutions, not private companies, possess the medical data necessary for the development of artificial intelligence (AI)-based software as a medical device (SaMD). Since we are conducting investigator-initiated clinical trials in many medical institutions and clinical departments for multiple medical fields, Renascience has many opportunities to hear from physicians about issues to be addressed in the medical field and is also working in an environment where it is easy to acquire big data in a relatively short period of time. Rather than sticking to our own resources and research environment, Renascience would rather focus on proactively utilizing external resources and the external environment to create a framework for efficient innovation. Renascience has the research and development base at Tohoku University and Hiroshima University is promoting the so-called ‘open innovation’.
4) Complete development from basic research to investigator-initiated clinical trials
Starting from basic research, Renascience conceives concepts and ideas for medical treatment, and manufactures pharmaceuticals, medical devices, etc. Renascience completes the necessary non-clinical studies with appropriate animals and cells, demonstrates the efficacy and safety of the medical treatment in clinical trials (clinical trials), and if possible, likes to develop the new treatment in-house to a stage close to applying for market approval. For example, the ultra-fine endoscope, a medical device that received approval in December 2022, has been jointly developed with several universities from product development to the implementation of non-clinical studies and the completion of clinical trials (investigator-initiated trials that physician/scientists involved in the research performs by themselves). It was filed for regulatory approval after out-licensing to a device company in the US. We are currently conducting a Phase III trial, the last clinical trial required for application for regulatory approval, for a therapeutic drug for chronic myeloid leukemia (CML), one of hematological cancers, and we would like to continue to conduct Phase III trials in-house in areas such as orphan diseases. The reason for this is that large pharmaceutical companies may find it difficult to focus on therapeutic drugs for orphan diseases when the development costs are high. By developing in-house to a point close to regulatory approval, it is possible to reliably implement important seeds in society. In addition, we believe that out-licensing at a later stage of development will result in greater compensation for Renascience, and we will be able to return it to our shareholders.
5) Use of investigator-initiated clinical trials
We have conducted 27 investigator-initiated clinical trials to date. The overwhelming advantages of investigator-initiated clinical trials are “quality” and “speed,” that are “efficiency”. In investigator-initiated clinical trials, physicians, who are at the forefront of research with access to the latest research results and who treat patients on a daily basis in the medical field, are able to draw up appropriate patient populations and study protocols. In addition, since physicians can conduct clinical trials themselves, it is a suitable clinical trial framework for early-stage trials of unapproved drugs (the stage in which the usefulness and safety of a drug are first confirmed, known as exploratory clinical trials). Furthermore, orphan diseases (rare diseases; The number of patients is small, and so sales cannot be expected to be large.) may have to be conducted from start to finish in investigator-initiated clinical trials, as pharmaceutical companies do not initiate drug development for these diseases due to low profitability. Almost the majority of R&D costs are spent in the clinical development phase, not the basic research phase. Investigator-initiated clinical trials can reduce development costs because the results of cutting-edge science and technology from universities and other medical institutions can be quickly utilized, and investigators can appropriately select patients to be treated. Unlike other companies, Renascience gives priority to this form of clinical trial, because, when appropriate coordinating investigators are found and the support of several large medical institutions such as universities can be obtained, investigator-initiated clinical trials have a significant advantage over corporate clinical trials, and large trials can be conducted in a short period of time.
The revision of the Pharmaceutical Affairs Law in 2003 opened the way for investigator-initiated clinical trials, in which physicians themselves conduct clinical trials. At that time of the law revision, there were many new drugs that were approved overseas and not approved in Japan (so-called ‘drug lag’), so domestic unapproved drugs and off-label drugs were the mainstream of investigator-initiated clinical trials. In terms of ease of conducting clinical trials (existing data such as manufacturing and safety tests can be used), many physicians at medical institutions such as universities proposed trials for new drugs that were approved overseas (not approved in Japan) and drugs for off-label use. In addition, in some cases, investigator-initiated clinical trials were conducted using existing drugs for rare diseases that pharmaceutical companies did not address. Against this background, there was a time when the impression that “investigator-initiated clinical trials are for expanded indications and orphan diseases for drugs approved abroad (domestically unapproved drugs) or existing drugs” was firmly established. However, all clinical trials conducted by Renascience aim for unapproved drugs (first-in-human) and are not clinical trials for overseas approved drugs (not approved in Japan) or for expanding indications of existing drugs.
In our drug development, non-clinical studies are conducted in compliance with Good Laboratory Practice (GLP) and the manufacturing of investigational drugs is conducted in compliance with Good Manufacturing Practice (GMP) for investigational drugs. In addition, investigator-initiated clinical trials are conducted in compliance with GCP (Good Clinical Practice), as are company-initiated clinical trials. Therefore, the results can be used in filing for applications and regulatory approval. In addition, since our drugs are unapproved drugs and we have secured the intellectual properties, we are able to commercialize them on an exclusive basis and generate sufficient revenues.
6) Formation of an ecosystem based on the open innovation
To date, many pharmaceutical companies and drug discovery ventures have focused on pipeline value chains, by building up all processes in-house and have focused on increasing business value. However, in fields such as pharmaceuticals that have extremely low success rates, long development periods, and large investments, R&D and business risks are high, so it is essential to form a portfolio that combines many pipelines and diversify risks. Backed by abundant funds, major pharmaceutical companies are often able to conduct development within the existing framework of forming their own pipeline value chains, but it is quite difficult for biotech companies that do not have ample funds. We have utilized the resources of external institutions (research institutions, medical institutions) and practiced development to increase development efficiency, including costs. We are considering building many value chains based on alliances with external institutions, and our strategies, research and development, and human resource management are different from those of existing ventures. We have been able to expand our pipeline and develop modalities with limited human resources and expenses, so we are beginning to see the outcomes. Rather than focusing only on our own resources and the internal environment, we would rather focus on proactive utilization of external resources and the external environment, and build a framework for efficiently creating the innovation. Renascience promotes open innovation and efficient development based on partnerships and collaborations with universities and other companies from various industries.
7) Promoting Comprehensive Collaboration with Multiple Universities by Establishing the Renascience Open Innovation Labs (RExes)
In January 2022, we opened the Tohoku University Renascience Open Innovation Labo (TREx) at the Tohoku University Graduate School of Medicine Medicinal Hub (2-1 Seiryo-cho, Aoba-ku, Sendai, Miyagi Prefecture, Faculty of Medicine Building 5). At the time of our founding, we had a research institute in the National Strategic Special Zone for Biotechnology in Kanagawa, which housed animal model breeding facilities for kidney disease. However, as the research target expanded from kidney disease to many disease areas, and the research stage progressed from basic research to clinical trials, the original research institute, which was mainly focused on animal models of kidney disease, was closed. However, we thought it was necessary to have “places” for utilizing cutting-edge scientific and technological achievements in many disease areas, “places” for face-to-face exchanges with doctors and researchers, and “places” for open innovation with governments and medical industry companies, so we established TREx. TREx is also the first base location project based on the “Agreement on Regional Economic Development between Sendai City and Tohoku University” concluded in April 2021. At TREx, 1) collaboration with researchers from the Tohoku University Graduate School of Medicine, doctors at Tohoku University Hospital, companies participating in the Medicinal Hub, governments, and other industries will be accelerated, and 2) research on existing development pipelines. 3) investigator-initiated clinical trials, acquisition of medical data, acquisition of public funds, formulation of licensing strategies, etc. can be handled efficiently and quickly, and 4) human resources are easily secured. We have been able to further accelerate the high efficiency of our research and development, which is one of our strengths.
IIn April 2023, we concluded a comprehensive collaboration agreement with Hiroshima University and established the Hiroshima University Renascience Open Innovation Lab (HiREx). HiREx will be utilized to conduct investigator-initiated clinical trials for pharmaceutical products such as non-small cell lung cancer and cutaneous hemangiosarcoma, and clinical performance tests of various software as a medical device (SaMD) including the support for diabetes treatment support AI and maintenance hemodialysis medical support AI.