Future Prospects With a One-of-a-Kind Drug Discovery Technology

Future Prospects With a One-of-a-Kind Drug Discovery Technology

The human body has the power to repair wounds on its own.
A drug discovery venture that focuses on the possibility of HGF (hepatocyte growth factor) discovered in Japan and challenges incurable diseases.
In collaboration with Keio University and cutting-edge robot therapy, we followed the trajectory and prospects of striving toward the realization of an unprecedented "medicine of hope."

Drug discovery bio-venture to confront "incurable disease"

Our company was established in Osaka in 2001 as a drug discovery bio-venture from Osaka University. Since then, we have continued research and development to commercialize drugs that mainly use HGF (hepatocyte growth factor) and help patients with intractable diseases around the world.
HGF is a protein in our body that was originally discovered by Dr. Toshikazu Nakamura (Professor and Chair Emeritus, Osaka University) about 30 years ago as a factor that promotes the proliferation of liver cells. The liver is known to be the organ with the highest regenerative capacity in the human body, and in the case of living-donor liver transplantation, even if one-third of the donor's liver is removed, that part will eventually regenerate. increase. As research progressed, it became clear that this protein is involved not only in the liver, but also in the regeneration and repair of various organs and tissues, and also has a very strong effect on the nervous system. Therefore, we are working on the development of fundamental therapeutic drugs that have not yet been established, targeting two intractable neurological diseases, Spinal cord injury and ALS (Amyotrophic Lateral Sclerosis).
By the way, "Kringle" in "Kringle Pharma" refers to the structure of the protein found in the molecule of HGF. As a company that develops pharmaceuticals for HGF and related substances, we named our company by combining two words: the kringle structure, which is a characteristic of HGF, and pharmaceuticals, which means pharmaceuticals.
While promoting the project through industry-academia collaboration, for Spinal cord injury, Professor and Chair Masaya Nakamura of Keio University School of Medicine, for ALS, Professor and Chair Masashi Aoki, Tohoku University School of Medicine, and for regenerative medicine, Professor and Chair Hideyuki Okano of Keio University School of Medicine. We are in charge of clinical development and aiming for commercialization. In contrast to Spinal cord injury, in which the central nervous system is damaged in an accident, etc., leaving the limbs and body incapable of movement, ALS is a disease of unknown cause in which nerve cells that control muscle movement are affected. Both of these diseases have a significant medical economic effect, as there is no fundamental treatment, and the patient's body becomes immobile, which places a heavy burden on caregivers. It has the effect of promoting survival and extending neurites, and dramatic effects are expected.

Encounter with a partner who supports a long way

For example, in an experiment conducted at Keio University, HGF was administered to monkeys who could not walk or grab something due to severe Spinal cord injury, and the limbs functioned in about 3 weeks. A remarkable recovery was observed. This is a turning point in the development of therapeutic agents for Spinal cord injury.
However, it takes a very long way to be approved as a drug. Drug development generally requires clearing three stages of clinical trials (Phase 1, 2, 3) in addition to non-clinical trials in a variety of animals. Regarding Spinal cord injury, we are conducting Phase 1/2 clinical trials in Japan that combine Phase 1 to confirm safety and Phase 2 to confirm the effect in a small number of patients. ALS, on the other hand, is in Phase 2. It can be said that we are facing the most important point in drug development in that if we do not produce results here, we will not be able to proceed to Phase 3 where we can increase the number of patients and verify the effects.
On the other hand, it must be said that the situation surrounding Japanese drug discovery ventures is extremely severe. Starting with non-clinical trials, manufacturing of investigational drugs, clinical trials, etc., each stage requires a cost of 100 million units and a period of many years, but during that time it is not possible to make a profit, so can we continue development? Please rely on external funding. If we do not find someone who can sympathize with the purpose and vision of the project and invest in future possibilities, not only the efforts we have made so far, but also the hopes of patients will disappear.
It was KII who raised his hand this time in such a situation. In addition to our unique technology in the world that can produce HGF as a recombinant protein, we have led the innovation with the achievements of jointly proceeding with the project with the teachers of Keio University and the research results of Keio University. I feel that it is the result of having you evaluate the possibility of going on. You can call it a benefactor of our project.

Opening up the future of drug discovery with the world's only technology

Actually, I was originally a microbial researcher, having been involved in research at an American bio-venture for about three and a half years, but through various experiences, I developed medicines that are useful for people suffering from illness. I returned to Japan with the desire to be involved in. I met this company when I wanted to be involved in a project to develop epoch-making medicines in Japan and contribute to the world, and joined the company in 2004. After working as General Manager of Research and Development Department and Director of Business Development Department, he was appointed President in December 2016, succeeding the current Chairman, Kunio Iwatani. It's been more than 10 years since I joined the company, and my motivation is to devote myself to the benefit of the world.
As for the current outlook, there is no doubt that we are the world leader in drug development of HGF recombinant proteins. HGF is produced as a recombinant protein by genetic recombination technology, but in order to administer it to humans, it is necessary to meet very strict standards. The most difficult part is how to establish a process that can mass-produce "genetically modified human HGF" with this "pharmaceutical grade", but it is the only one in the world that can be established and stably manufactured. That is our technology.
In addition, Cyberdyne Inc., which is attracting worldwide attention for its cyborg-type robot suit "HAL®" worn by humans, is also paying attention to the potential of HGF and is taking a stake in our company. A professor at Spinal cord injury Spinal cord injury, who is involved in regenerative medicine, introduced us to Professor and Chair Yoshiyuki Sankai of the University of Tsukuba, who is the president of Cyberdyne. Both parties share the vision of increasing the number of nerve cells and at the same time improving physical function through the regeneration of nerve function by medical HAL®, which is the medical treatment that should be in the future.
In the future, while expanding the scope of HGF to various diseases, we are thinking of establishing a relationship between research institutes such as universities and pharmaceutical companies and expanding the seeds of new biopharmacy. We would like to contribute to patients and their families around the world by realizing HGF-based pharmaceuticals and growing into a pharmaceutical company that specializes in "unmet medical needs" for which there is no cure yet. .. That is our goal and our wish.