[Edaily Reporter Kim Jinsoo ] Vaxcell-Bio Therapeutics(323990)is launching additional clinical trials, including a controlled study, to enter the market for the treatment of advanced liver cancer with its core pipeline product, the autologous natural killer (NK) cell therapy “VCB-1102.”

Although the government has decided to lower the risk classification for advanced regenerative medicine using autologous immune cells—making approval under the advanced regenerative medicine treatment plan possible—this requires, first and foremost, evidence to support the therapeutic efficacy of VCB-1102. Vaxcell-Bio Therapeutics plans to demonstrate the clinical efficacy of VCB-1102 independently of the regulatory easing expected next month following the risk classification adjustment, thereby reducing uncertainty in the commercialization process.

According to Vaxcell-Bio Therapeutics on the 22nd, the Committee for Advanced Regenerative Medicine and Advanced Biopharmaceuticals recently notified the company of its decision to reject the advanced regenerative treatment plan for advanced liver cancer combining VCB-1102 with hepatic artery infusion chemotherapy (HAIC).

This treatment plan was the proposal for the use of VCB-1102 that Vaxcell-Bio Therapeutics had submitted to apply for the regulatory sandbox. If approved as an advanced regenerative treatment plan, Vaxcell-Bio Therapeutics would be able to supply VCB-1102—which has not yet received formal approval—in exchange for a certain fee. This would have alleviated the financial burden associated with administering VCB-1102. However, with the review panel’s determination of ineligibility, plans for the commercial use of VCB-1102 have been somewhat delayed.

A Vaxcell-Bio Therapeutics official explained, “This review result does not mean that VCB-1102 is ineffective; rather, it indicates that additional research is needed to evaluate the existing clinical results in a more objective manner and to strengthen the evidence.”

Vaxcell-Bio Therapeutics immediately incorporated the review committee’s feedback and submitted a separate proposal for an advanced regenerative clinical trial that includes a control group. The company aims to use this to strengthen the evidence that will inform future treatment plan reviews and subsequent development.

A Vaxcell-Bio Therapeutics official stated, “According to previously analyzed results, the objective response rate in the HAIC and VCB-1102 combination group was 70.5%, which was higher than the 39.4% observed in the external control group that received HAIC alone. A statistically significant difference was confirmed even after adjusting for patient characteristics.”

Why Is the ‘Low-Risk’ NK Cell Therapy Moving Forward with Clinical Trials?

The government’s risk reassessment is a prerequisite for the commercial use of VCB-1102

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which has not yet received formal approval. On June 25, following a resolution by the Deliberation Committee, the Ministry of Health and Welfare decided to lower the risk level of advanced regenerative medicine clinical research and treatment using cultured autologous immune cells from “medium risk” to “low risk,” and announced plans to issue a formal notice by next month.

The key to this risk classification adjustment is the relaxation of the requirement to conduct preliminary clinical studies. Currently, high- and medium-risk treatments must be preceded by advanced regenerative medicine clinical studies with the same purpose and content, whereas low-risk treatments allow applicants to submit treatment plan reviews without separate clinical studies.

Vaxcell-Bio Therapeutics’ VCB-1102 is an autologous natural killer (NK) cell therapy, meaning the requirement for a prior clinical study will be eliminated next month due to the reclassification to low risk. However, this does not exempt the company from providing the safety and efficacy evidence required for treatment plan review. Consequently, Vaxcell-Bio Therapeutics has resubmitted an application for an advanced regenerative medicine clinical study despite the upcoming risk reclassification.

The main point raised during this review regarding VCB-1102 was that its efficacy when used in combination with HAIC had not been clearly demonstrated compared to HAIC monotherapy. The committee assessed that there were no safety concerns, taking into account factors such as the absence of reported serious adverse events in previous clinical trials. However, it determined that the therapeutic efficacy of VCB-1102 could not be objectively assessed based solely on the previously submitted data, prompting Vaxcell-Bio Therapeutics to initiate another clinical trial.

Vaxcell-Bio Therapeutics stated, “We understand that a prospective controlled study is necessary to more clearly confirm the efficacy of VCB-1102 when used in combination,” adding, “This follow-up study is focused precisely on verifying this aspect.”

In fact, the clinical trial recently proposed by Vaxcell-Bio Therapeutics compares a combination therapy group receiving VCB-1102 and HAIC to a control group receiving HAIC alone in patients with advanced liver cancer. This clinical design is intended to evaluate the results of both treatment groups within the same study to determine the extent of the benefit gained by adding VCB-1102 to HAIC.

While an investment of time and money is inevitable for the additional study, Vaxcell-Bio Therapeutics views this as significant because it can eliminate issues that might otherwise arise from the recurrence of the same problems during future treatment plan reviews. Furthermore, since the specific criteria and implementation procedures for risk adjustment have not yet been clearly defined, this move is seen as a proactive measure to minimize risks.

A representative from Vaxcell-Bio Therapeutics stated, “Once the detailed criteria and implementation procedures for risk adjustment are finalized, we plan to refine our clinical research and treatment strategy for VCB-1102 in accordance with the revised regulations, thereby enhancing patient access to treatment and improving commercialization prospects.”

The official added, “In addition to the HAIC combination therapy, we plan to submit an application soon for a clinical trial of advanced regenerative medicine that adds VCB-1102 to combination therapies involving atezolizumab and bevacizumab,” noting, “We will evaluate safety and efficacy in different treatment settings to broaden the evidence base for subsequent development.”