[Biz Leader] TiumBio: "Building on experience developing Korea's first new drug, we aim to become a global drug developer"

Accelerating Development of Oral Immuno-Oncology Drug Tosposertib
Preparing for Phase 2b Based on Phase 2a Data
Advancing Global Commercialization in Collaboration with MSD

Go Hyeonsil, Chief Financial Officer (CFO) of TiumBio.

"Our goal is to become a company with globally successful new drugs, led by CEO Kim Huntaek, who has experience ranging from developing Korea's first new drug at SK Chemicals to licensing a global hemophilia treatment overseas."

Go Hyeonsil, Chief Financial Officer (CFO) of TiumBio, outlined the company's vision of growing beyond a biotech that merely owns drug candidates in clinical development and becoming a company that creates new drugs used to treat patients around the world. At the center of this vision is tosposertib (TU2218), an immuno-oncology drug candidate.

TiumBio develops new drugs primarily for rare and intractable diseases. While at SK Chemicals, CEO Kim Huntaek participated in the development of Sunpla, the first new drug approved in Korea, in 1999, and later worked on the development of the hemophilia treatment Afstyla. In 2009, Kim and his research team licensed Afstyla to global pharmaceutical company CSL. They then gained experience in global drug development that led to approvals from the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and other regulators.

CFO Go explained that Kim's experience has informed TiumBio's current research and development strategy. She said it is advantageous for a small or midsize biotech to focus on areas where the scientific mechanism is clear and specialized technology can provide a competitive edge, rather than entering fields such as hypertension, diabetes, and obesity, where it would have to compete with large pharmaceutical companies.

Tosposertib is a candidate at the heart of this strategy. Development initially began with a focus on treating fibrosis, but the company shifted its focus to cancer after recognizing that TGF-β contributes to reducing the effectiveness of immuno-oncology drugs in the tumor microenvironment. Tosposertib is a small-molecule drug candidate that simultaneously inhibits TGF-β signaling and vascular endothelial growth factor receptor 2 (VEGFR2). The key approach is to block both pathways, overcoming the limitations of existing TGF-β inhibitors and enhancing the anticancer activity of immune cells to boost the effectiveness of immuno-oncology drugs such as Keytruda.

Tosposertib is being developed as a first-line treatment for recurrent or metastatic head and neck cancer. The strategy is to add tosposertib to the current standard treatments—Keytruda alone or Keytruda combined with chemotherapy—to improve treatment outcomes.

Another advantage of tosposertib is that it is taken orally. Unlike some competing candidates that are antibody-based injectables, tosposertib is an oral small-molecule drug. The company sees its potential to improve convenience for patients while being used in combination with immuno-oncology drugs, as well as its suitability for combination with recently approved subcutaneous (SC) formulations of immuno-oncology drugs, as points of differentiation.

Collaboration with global pharmaceutical company MSD is another important part of tosposertib's development. TiumBio signed a joint research and development agreement with MSD in July 2022. MSD provides Keytruda for clinical trials free of charge, and the two companies continue to share clinical data. TiumBio places great importance on validating the drug's efficacy and clinical value alongside a global pharmaceutical giant, while also remaining open to the possibility of further collaboration.

The next key step is follow-up clinical trials. Based on data from its Phase 2a trial, TiumBio is preparing a Phase 2b trial that will include a comparison control group. The company said the Phase 2a trial exceeded its prespecified threshold for the primary endpoint, objective response rate. Because progression-free survival and overall survival require long-term follow-up, the company plans to collect additional data to strengthen the evidence supporting development.

"Based on the early clinical data for tosposertib, we are discussing licensing and co-development opportunities with global pharmaceutical companies," CFO Go said. "We see companies with oncology pipelines or experience developing and obtaining approval for targeted therapies as potential partners."

In addition to tosposertib, follow-up pipeline candidates include the women's health treatment merigolix (TU2670) and a hemophilia treatment (TU7710). In particular, in the hemophilia field, CEO Kim and key members of the research team are drawing on the experience they gained during the development of Afstyla.

TiumBio's ultimate vision is not simply to be a biotech with multiple candidates in clinical development. Its strategy is to build a virtuous cycle: increase the likelihood of success for new drugs through clinical data, work with suitable global partners to advance them to approval and commercialization, and reinvest funds secured through licensing and other means in the development of new drugs.

"Our ultimate goal is to have new drugs approved in global markets and to develop medicines that are used to treat patients," CFO Go said. "We plan to increase the likelihood of success for our current clinical-stage candidates while steadily expanding our early-stage drug discovery pipeline to ensure a continuing pipeline of follow-up candidates."

This content was produced with the assistance of AI translation services.