The government decided to give reimbursement for CSL Behring’s hemophilia treatment, Abstilla, from June. SK Chemicals developed the original substance of the drug and transferred the technology to CSL Behring in 2009.

The key developers of Abstilla separated from SK Chemicals and established TiumBio, a domestic company specializing in drugs for rare diseases. TiumBio CEO Kim Hun-taek and his research team have continued to discover and develop new drug candidates for rare diseases since the establishment.

The company has recently won approval to conduct phase 1/2 clinical trials of cancer immunotherapy TU2218 from the U.S. Food and Drug Administration.

Korea Biomedical Review met with TiumBio CEO Kim to learn why the company developed new drug candidates for rare diseases and how far the studies have gone.

Question: Why did you leave SK Chemicals and established a new company?

Answer: As a new recruit at SK Chemicals, I have been developing new drugs. As the head of a department that develops new drugs, I thought fostering an environment for continuous development was significant. The stability of the research for new drugs may vary depending on the environment. I believed that was my role and built a company where people who have worked with me for a long time can see their dreams of developing new drugs.

Q: Among various diseases, why rare diseases?

A: We have chosen rare diseases because the market is quite attractive despite the public generally thinks that the market is small. We also have the strength and a history of developing new drugs. Global companies have already invested in the past with chronic diseases, such as diabetes and high blood pressure. They have enormous resources in new drug development.

So, we chose our first pipelines for hemophilia and fibrosis as they are diseases that we have been studying for over 20 years.

There are also clinically significant unmet needs in Korea. The governments of developed countries also are running many programs to support the development of rare disease treatments. The clinical trial of rare diseases has a high probability of making positive outcomes.

Q: What is the practical business model to go for an extended clinical trial?

A: Global societies view rare diseases somewhat differently from Korea. In Korea, rare diseases tend to be marginalized in terms of national sentiment. There was a lot of discrimination against those who were handicapped due to rare diseases in the past. However, the global market tries to take care of people with rare diseases.

When we established the company, the industry initially showed a somewhat negative response. Still, now, five years later, people seem to recognize how attractive the rare disease market is.

Q: What was most challenging in securing funds and finding a new drug research and development partner after starting the business?

A: We did not have to go through much trouble. We had researchers who had developed new drugs for so long, so they knew what they were doing. In addition, because of the excellent reputation of new drug developments, there was no difficulty in establishing cooperative relationships with partners and global pharmaceutical companies.

Q: What about the clinical studies of TU2218, treatment for idiopathic pulmonary fibrosis?

A: We submitted an investigational new drug (IND) application in Korea and the U.S. to conduct a phase 1/2 study of TU2218 on July 28. Once we get approval, we will carry out the trials in one site in the U.S. and two sites in Korea within this year. We plan to recruit about 50 to 60 people for the first phase of the study, using a combination therapy of TU2218 and immune checkpoint inhibitors of global drug developers to find the maximum tolerated dose.

Q: How does TU2218 work in treating idiopathic pulmonary fibrosis?

A: TU2218 blocks the angiogenic factor VEGFR2 pathway while inhibiting Transforming Growth Factor (TGF)-beta. The drug candidate helps immune cells to fight against cancer cells by inhibiting the two substances. So, we think the best partner drugs are immune checkpoint inhibitors for killing cancer cells by activating patients' immune cells.

Q: What about the ongoing hemophilia treatment (TU7710) pipeline?

A: We currently have no plan for running clinical trials in Korea. If our IND application for the global phase 1 clinical trial wins approval by December, we might be able to begin administering the drug candidate to patients during the first three months of 2022.

A global CDMO called Patheon in Australia is producing the drug materials. The number of people participating in the clinical trial will be around 20. We expect to see the test results in a short period.

Q: Does TiumBio have other major drug candidates?

A: Our treatments for hemophilia B and diabetes entered pre-clinical trials. However, we are focusing on research into fibrosis, hemophilia, and immuno-oncology.

The treatment for endometriosis has entered phase 2 clinical trial in Europe, and the administration will begin soon. In Korea, we have transferred technology to Daewon Pharmaceutical, and it is being developed as a treatment for uterine fibroids.

Q: There are gene therapies under development in the global market. Do they have the potential for becoming a new paradigm in treating hemophilia for now?

A: In the case of hemophilia-related gene therapy, there is a possibility that it might cause cancer as a side effect. Some patients produce typical clotting factors for a long time after receiving gene therapy. Still, others do not show a clear response to the treatment and require additional gene therapy.

The cost is also a problem as the drug usually requires about 1 billion won ($864,000) for a single administration. If a patient has to consider both the expense and risk of developing cancers, I think the gene therapies for hemophilia will settle in 20 to 30 years later.

Q: TiumBio recently invested 30 billion won in SK Plasma. What convinced you to make such a large investment?

A: We decided to invest 30 billion won in SK Plasma because we felt the need to invest in a business structure that has a stable and capable growth engine. SK Group also was looking for partners to build up SK Plasma as SK Bioscience or SK Biopharm. SK Plasma, specializing in blood products, approached us as we had a long experience developing new drugs in SK Chemicals.

Q: What is the top priority goal for TiumBio? Any plan for developing original drugs without partners?

A: We aim to develop drugs and transfer the technologies to global pharmaceutical companies. Regarding the development of new medicines on our own, I think we lack resources right now. Developing pipelines up to phase 2 clinical trials and transferring the technologies is the most efficient way to develop new drugs. We mainly focus on drug developments, not manufacturing or marketing medicines, so our business model to transfer technology and share risk factors and profits fits our companies’ long-term vision.

Q: What will TiumBio look like after 10 to 20 years?

A: Our short- and mid-term goal is to become a globally recognized new drug developer, not a pharmaceutical company that makes and sells drugs. There will be drugs under our research to be used as pharmaceutical products on the market.

It seems that the essential part of satisfying our stakeholders is the companies’ members. I believe our company will be where researchers can feel the sense of accomplishment for developing globally recognized drugs in 10 to 20 years.