Clinical Trial of Genuine Biotech's Azvudine in Combination with PD-1 Monoclonal Antibody in the Treatment of Advanced Solid Tumors Approved by the National Medical Products Administration

On Feb. 13, 2026, Henan Genuine Biotech Co., Ltd. announced that the clinical trial of self-developed anti-tumor drug GEN-725 tablets (Azvudine) in combination with PD-1 monoclonal antibody in the treatment of advanced solid tumors was approved by the National Medical Products Administration.

In recent years, the anti-cancer research on immune checkpoint inhibitors based on programmed cell death protein-1 (PD-1) or programmed cell death ligand-1 (PD-L1) has developed rapidly, bringing long-term survival benefits to many cancer patients. However, there are still some patients with primary non-response or acquired drug resistance in clinical practice.

Taking colorectal cancer as an example, its incidence ranks third and second among all malignant tumors worldwide and in China, respectively. In 2023, there were approximately 2.03 million new cases worldwide. However, according to the National Comprehensive Cancer Network (NCCN) guidelines, immune checkpoint inhibitors are only recommended for patients with metastatic colorectal cancer with high microsatellite instability or mismatch repair deficiency. This group accounts for only about 5%, which means that there are more than 1.9 million new patients with microsatellite stable (MSS) (95% of colorectal cancer patients) worldwide each year, for which current PD-1/PD-L1 monotherapy is basically ineffective, and conventional regimens based on chemotherapy and targeted therapy have reached a bottleneck with significant toxic side effects. There is still a huge unmet clinical need for this patient population.

Last December, Genuine Biotech, in collaboration with the First Affiliated Hospital of Zhengzhou University and the Institute of Pharmaceutical Biotechnology, Chinese Academy of Medical Sciences, published a research paper on the combination therapy of Azvudine/PD-1 monoclonal antibody in Frontiers of Medicine. The results of animal experiments showed that Azvudine monotherapy significantly inhibited the growth of solid tumors and reshaped the tumor immune microenvironment; when combined with PD-1 monoclonal antibody, it produced a synergistic effect to induce complete and long-lasting tumor regression and establish a long-lasting immune memory.

Combined with previous studies, Azvudine, as a highly selective nucleoside drug with a unique dual mechanism, has been further validated for its potential in inhibiting tumor growth. On the one hand, it can directly inhibit the DNA synthesis of tumor cells, and inhibit the proliferation of cancer cells by terminating the prolongation of DNA chains and interfering with the functions of enzymes related to nucleic acid synthesis in cancer cells. On the other hand, as an immunomodulator remodeling the tumor microenvironment, Azvudine reduces the excessive accumulation of myeloid-derived suppressor cells (MDSCs) in the tumor microenvironment, while promoting the infiltration and proliferation of CD8+ T cells, CD4+ T cells, etc., thereby activating the body's own anti-tumor immune response and forming a synergistic effect with the PD-1 monoclonal antibody in relieving immunosuppression.

The study showed that in the CT26 colon cancer mouse model, all mice in the combination therapy group achieved complete tumor regression within 39 days, and none of them had recurrence by the end of the follow-up on day 88. Of particular interest was that in the subsequent "re-challenge" experiment, mice that had recovered from the combination treatment did not develop tumor growth after re-inoculation with the same tumor cells.

This combination regimen has also been further validated in human studies. In an investigator-initiated human trial, Azvudine in combination with PD-1 inhibitors and anti-VEGFR drugs in patients with MSS colorectal cancer who had received multiple lines of treatment in the past had a disease control rate of 100% in the higher dose group, an objective response rate of 40%, and a mean treatment duration of 33.9 weeks in the non-liver metastasis subgroup.

In the future, Genuine Biotech will accelerate the clinical study of Azvudine in combination with PD-1 monoclonal antibody in patients with advanced solid tumors, bringing new options to patients with solid tumors who are facing long-term treatment difficulties.