Genuine Biotech's Clinical Study of Azvudine for the Treatment of Advanced Solid Tumors Presented at ASCO Annual Meeting

From May 29 to Jun. 2, 2026, the American Society of Clinical Oncology (ASCO) Annual Meeting was held in Chicago, USA. As one of the most influential academic conferences globally in the field of neoplasms, the ASCO Annual Meeting highlights significant yearly advancements in clinical studies within oncology. 

At this ASCO Annual Meeting, Genuine Biotech, in combination with Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, presented the Phase 1 clinical study data of Azvudine (FNC) in patients with advanced solid tumors in the form of a poster, which preliminarily confirmed the potential of this domestic nucleoside drug in the therapeutic area of solid tumors, especially in patients with microsatellite stable (pMMR/MSS) colorectal cancer, showing encouraging efficacy and good safety. 

Previously, multiple preclinical studies of Azvudine for anti-tumor treatment completed by Genuine Biotech showed that the drug can significantly down-regulate the levels of myeloid-derived suppressor cells (MDSCs) in the tumor microenvironment and effectively increase the proportions of CD4⁺ T cells, CD8⁺ T cells, and NK cells. Based on this immune regulation effect, the combination of Azvudine and anti-PD-1 therapy can show superior anti-tumor efficacy.

This Phase 1 clinical study was initiated by the investigator to evaluate the preliminary efficacy and safety of FNC in combination with the investigator's choice of regimen in participants with advanced solid tumors after multiple lines of treatment. The study is currently in the dose escalation stage. As of Oct. 31, 2025, a total of 25 patients with advanced solid tumours were enrolled, including 22 patients with colorectal cancer (all pMMR/MSS) and 3 patients with lung cancer. All patients with MSS colorectal cancer received a triple therapy regimen combining FNC, penpulimab, and fruquintinib.

In terms of efficacy, among the 21 efficacy-evaluable patients with MSS colorectal cancer, the overall objective response rate (ORR) across all dose groups was 19.1%, and the disease control rate (DCR) was 90.5%. One patient experienced PR in the 5 mg group, 2 patients experienced PR in the 6 mg group, and 1 patient experienced PR in the 7 mg group. A total of 4 patients experienced PR. A total of 18 patients were enrolled in the 5/6/7/8 mg group, with an overall ORR of up to 22.2% and a DCR of 94.4%. By stratification according to the presence or absence of liver metastases, the ORR for patients without liver metastases reached 40%, and the DCR reached 100%. Most patients with MSS colorectal cancer after multiple lines of treatment can achieve stable tumor foci.

The median progression-free survival (mPFS) was 4.5 months and the median overall survival (mOS) was 7.6 months, effectively prolonging the survival cycle of patients with MSS colorectal cancer after multiple lines of treatment.

In terms of safety, no dose-limiting toxicity was observed, and the overall safety was manageable.

Colorectal cancer is the third most common cancer in the world. According to Frost & Sullivan data, there were nearly 2.05 million new patients with colorectal cancer worldwide in 2024. Although immune checkpoint inhibitors represented by PD-1/PD-L1 have revolutionized the treatment landscape of various cancers, their application in the field of colorectal cancer is relatively limited.

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, and such patients account for about 5% of all colorectal cancers. This means that more than 1.9 million new patients with microsatellite stable (pMMR/MSS) disease worldwide each year are basically ineffective for the current PD-1/PD-L1 monotherapy. These patients have long been facing the dilemma that traditional chemotherapy and targeted therapy have reached the bottleneck and have marked toxic side effects, and there is a huge unmet clinical need.

Azvudine is a Class 1.1 innovative drug independently developed by Genuine Biotech and has been conditionally approved for the treatment of HIV-1 and viral infections caused by SARS-CoV-2. Its potential in the anti-tumor field stems from its unique dual mechanism of action. On the one hand, as a nucleoside analogue, it can be incorporated into DNA or RNA molecules to interfere with cell replication, competitively inhibit DNA polymerase, etc., specifically interfere with nucleic acid metabolism, and prevent the division and proliferation of tumor cells. On the other hand, it acts as an immune modulator to significantly reduce the excessive accumulation of myeloid-derived suppressor cells in the tumor microenvironment. At the same time, it promotes the infiltration and proliferation of immune cells such as CD8⁺ T cells and CD4⁺ T cells, converting "cold" tumors to "hot" tumors. This mechanism is precisely in deep synergy with PD-1 inhibitors to relieve the immunosuppression of T cells.

The Phase 1 study data presented at this ASCO Annual Meeting provided validation of the clinical benefits of Azvudine in combination with PD-1 monoclonal antibody and anti-EGFR drugs in patients with pMMR/MSS colorectal cancer, offering a novel treatment regimen for this large patient population that has long lacked effective immunotherapy options.

In Feb. 2026, the application for the registration of Azvudine in combination with Innovent Biologics' sintilimab was approved by the National Medical Products Administration and entered formal clinical trials.

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, continuously exploring its therapeutic potential in more indications for solid tumors, which is expected to bring new options to patients with cancer who are facing long-term treatment difficulties.