Shares of ViGenCell, long seen as a promising player in immune-oncology cell therapies, swung sharply on the release of phase 2 results. The candidate, touted as Korea's first NK-T cell therapy, failed to show a clear improvement in overall survival (OS), the period patients remained alive after treatment.
According to the final phase 2 results for the immune-oncology cell therapy "VT-EBV-N," disclosed on the 24th, the primary endpoint of 2-year disease-free survival (DFS) was 95% in the treatment arm, an improvement over the control group (77.58%). No deaths occurred in the treatment arm during the trial, while multiple deaths were reported in the control group.
However, the secondary endpoint of OS did not achieve statistical significance. While the mortality rate in the control group was 16% and there were no deaths in the treatment arm, the trial failed to prove a statistically meaningful difference. The company said, "The treatment group maintained a 100% survival probability, showing a numerical trend toward improvement."
The market reacted immediately. On the disclosure day, ViGenCell's share price plunged, finishing at 6,130 won, down 1,930 won (23.95%) from the previous day. Despite meeting significance on the primary endpoint, the roughly 17 percentage-point gap between the treatment and control groups and the failure to meet OS significance appear to have dampened investor sentiment.
VT-EBV-N targets NK-T cell lymphoma, a rare blood cancer. The rare disease occurs in about 1 per 100,000 people worldwide and is known to progress rapidly and be fatal. With no established standard of care and a relapse rate of about 80% within two years, the area is seen as having strong demand for new treatment options.
Currently, the only approved therapy for this indication is "Sugemalimab" developed by China's CStone. However, with evaluations that its efficacy is limited, competition to develop follow-on therapies continues. As major regulators in the United States, Europe, and Korea have designated this illness as a rare disease, the possibility of accelerated approval based on the phase 2 results is also being discussed.
ViGenCell is viewed as the most advanced in domestic development. VT-EBV-N received orphan drug designation from the Ministery of Food and Drug Safety in 2019 and from the European Medicines Agency in 2023. Based on these results, the company is pursuing conditional approval and aims to commercialize as early as next year. Subsidiaries and peers including Yuhan's ImmuneOncia Therapeutics, Eutilex, and GC Cell are also developing NK-T cell therapies.
The key issue is the earnings gap before commercialization. With Boryung, the second-largest shareholder, having sold about half of its holdings, concerns about an overhang (a potential large volume of shares for sale) have partially eased. Still, how to defend cash flow in the near term remains an open task.
ViGenCell recorded 258 million won in product revenue from wholesale medical equipment in 2024. It is also pursuing contract manufacturing (CMO) and contract development and manufacturing (CDMO) businesses for cell therapies, but there have been no meaningful order wins yet. The industry expects that whether the company can secure a tangible revenue base to endure until VT-EBV-N's commercial sales become visible next year will be the key variable for corporate value.
Meanwhile, the company said on the 25th it will terminate early the clinical trial of the acute myeloid leukemia candidate "VT-Tri(1)-A." During cohort 3 of phase 1, a grade 3 graft-versus-host disease (GVHD) adverse event occurred, and the company judged that pushing development too hard could burden corporate value. A company official said, "We suspect it stems from the product characteristic of using blood from a hematopoietic stem cell donor."
However, the market is interpreting it as a strategic decision to resolve uncertainties, and as of the 26th the share price has recovered to the 8,400-won range.