[Edaily Reporter KIM JI-WAN ] Vaxcell-Bio Therapeutics(323990)is pushing to enter the advanced regenerative medicine market with its autologous natural killer (NK) cell therapy “VCB-1102,” which demonstrated a tumor response in 11 out of 16 liver cancer patients.

According to the company on the 28th, Vaxcell-Bio Therapeutics is continuing its efforts to secure approval for “VCB-1102” as the second treatment under the Advanced Regenerative Medicine Act. Previously, on the 16th, a paper on a Phase 2a clinical trial of VCB-1102 in combination with hepatic artery infusion chemotherapy (HAIC) for locally advanced hepatocellular carcinoma was published in the international academic journal “Cancer Research and Treatment” (CRT). CRT is the official journal of the Korean Cancer Association, the Korean Society of Medical Oncology, and the Korean Lung Cancer Society.

With the publication of the Phase 2a clinical trial results for VCB-1102 in an international journal coinciding with the government’s relaxation of regulations on autologous immune cells, Vaxcell-Bio Therapeutics has been presented with an opportunity to translate its development achievements into actual patient treatment. Attention is now focused on whether this publication will pave the way for the drug to become the “No. 2 Advanced Regenerative Medicine Therapy.”
“Medium Risk → Low Risk”…

The Path to Patient Treatment Widens

First, an amendment to the Advanced Regenerative Medicine Act is imminent.

The government is pushing forward with a plan to lower the risk level of advanced regenerative medicine—which involves culturing and using a patient’s own immune cells—from the current “medium risk” to “low risk.” Previously, treatments required undergoing advanced regenerative medicine clinical trials, which took about 2–3 years prior to treatment; however, once the risk level is adjusted to “low risk,” providers will be able to submit clinical literature and other materials to apply for a review of the treatment plan. The Ministry of Health and Welfare reaffirmed this direction in its plan for rationalizing bioregulations released on the 23rd.

VCB-1102 is a therapy that utilizes the patient’s own NK cells. A representative from Vaxcell-Bio Therapeutics stated in an interview, “If NK cell therapies are reclassified from medium-risk to low-risk this month, we expect to benefit even more.”

Patient treatment using advanced regenerative medicine has already begun.

Yeouido St. Mary’s Hospital received the first domestic approval in April for a treatment plan for rare lymphoma using ViGenCell Inc.’s autologous immune cell therapy “VT-EBV-N,” and treatment began last September. Separate from the new drug approval process, this has opened the door for medical institutions to apply cell therapy technology to patients according to approved treatment plans. Vaxcell-Bio Therapeutics is preparing to enter the liver cancer treatment market through this pathway.

11 out of 16 Responded… Median Time to Tumor Progression: 16.82 Months

The key finding of this clinical trial is that 11 out of 16 evaluable patients showed tumor shrinkage or a response confirmed by imaging. The objective response rate (ORR) was 68.75%. This result is based on an analysis of 16 evaluable patients out of a total of 17 enrolled. Among them, 3 achieved complete remission (CR) and 8 achieved partial remission (PR).

In clinical trials of existing hepatocellular carcinoma treatments, the objective response rates reported were approximately 30% for the atezolizumab-bevacizumab combination therapy and approximately 11% for sorafenib.

The median time to progression (TTP) for VCB-1102, as assessed by the investigators, was 16.82 months. While the ORR indicates “how many patients experienced a reduction in tumor size,” TTP is a measure of “how long it took for the cancer to grow again or worsen.”

This treatment combines hepatic artery infusion chemotherapy (HAIC) with NK cells. HAIC is a treatment in which anticancer drugs are injected directly into the arteries leading to the liver, while NK cells are immune cells that seek out and attack cancer cells. The strategy aims to enhance the therapeutic effect by combining the power of immune cells with the attack of anticancer drugs.

The research team first administered HAIC twice; then, for patients whose cancer had not worsened, they administered additional HAIC while also administering NK cells. Treatment response was evaluated using the Modified Response Evaluation Criteria in Solid Tumors (mRECIST), which assesses not only the size of the liver tumor but also changes in the living tumor tissue.

Regarding the HAIC combination strategy, a representative from Vaxcell-Bio Therapeutics stated, “We researched combination therapy with VCB-1102 as part of our efforts to explore various treatment strategies that could improve treatment outcomes for liver cancer patients,” adding, “It is important to identify treatment strategies that can benefit patients and to evaluate their efficacy and safety through clinical trials.”

NK Cells Proliferated Up to 100,000-Fold… Simultaneously Advancing

Clinical

Development and Production Technology Alongside

clinical development, Vaxcell-Bio Therapeutics is also strengthening its NK cell production platform. Since cell therapies involve administering living cells to patients, production technology capable of securing sufficient quantities of cells while maintaining quality is key to commercialization.

Vaxcell-Bio Therapeutics has established a platform for large-scale expansion of NK cells from healthy donors. Under optimized culture conditions, the company achieved expansion of up to 100,000-fold and confirmed the high purity and cancer-killing ability of the expanded NK cells.

The company explains that it has verified not only the scale of culture but also the functionality of the cells after proliferation. The company is leveraging this platform research to advance the development and production technologies for NK cell therapies. It is expanding its research capabilities by concurrently conducting the VCB-1102 clinical trial for liver cancer patients and platform research using cells from healthy donors.

The market for liver cancer treatments is also expanding. Market research firm Global Market Insights forecasts that the global market for liver cancer treatments will grow from $4 billion (5.348 trillion won) last year to $16.7 billion (22 trillion won) by 2035. This represents a roughly 4.2-fold increase over the next 10 years, with an expected compound annual growth rate (CAGR) of 15.6% from 2026 to 2035. Vaxcell-Bio Therapeutics is pursuing entry into this expanding treatment market by building on its clinical results for liver cancer and its NK cell production technology.

A representative from Vaxcell-Bio Therapeutics stated, “Through the publication of this paper, we have shared the results of the VCB-1102 Phase 2a clinical trial with the international academic community,” adding, “We will continue the clinical development of VCB-1102 while working to secure approval for the treatment under the Advanced Biotechnology Act.”