81.8% CR rate! China's oncolytic virus YH01 shows promise in addressing unmet needs of BCG-unresponsive bladder cancer
VCBeat has learned that, YH01, a domestically developed novel oncolytic virus drug independently researched and developed by Beijing Yinmei Future Biomedical Technology Co., Ltd. (hereinafter referred to as "Yinmei Future"), has recently disclosed the phase Ⅱ clinical trial progress for high-risk Non-Muscle Invasive Bladder Cancer (NMIBC). Preliminary data show that among 11 evaluable patients, 9 achieved complete response (CR), with a CR rate of 81.8%, which provides a new biological treatment pathway exploration for the clinical challenge of bacillus calmette-guerin (BCG) treatment failure. In particular, among patients who received 6 doses, the 3-month CR rate reached 87.5% and the 6-month CR rate reached 80%.
According to Advances in Diagnosis and Treatment of Non-Muscle Invasive Bladder Cancer, bladder cancer ranks as the 10th most common malignant tumor worldwide. In 2020, there were approximately 573,000 new cases and 213,000 deaths globally; in 2022, China reported 92,000 new cases and 43,000 deaths. Among all patients, 75%–85% are initially diagnosed with Non-Muscle Invasive Bladder Cancer (NMIBC). Transurethral resection of bladder tumor (TURBT) followed by adjuvant intravesical bacillus Calmette-Guérin (BCG) instillation is the gold standard for the treatment of intermediate- and high-risk Non-Muscle Invasive Bladder Cancer. However, the existing regimens are associated with high recurrence rates and substantial progression risks, leaving significant unmet clinical needs.
In particular, for BCG-nonresponsive patients and those with recurrent disease or other treatment failures, radical cystectomy is the first-line treatment. While this procedure can eliminate primary tumor lesions, associated complications and postoperative urinary diversion can drastically impair patients' quality of life. Studies have shown that the incidence of perioperative complications can range from 28% to 64%, with a perioperative mortality rate of 2% to 3%. The causes of death include cardiovascular complications, sepsis, pulmonary embolism, hepatic failure and hemorrhage.
In the real world study, many patients are ineligible for or decline radical cystectomy due to underlying comorbidities and the significant decline in quality of life associated with the surgery. Therefore, it is of great importance to explore novel bladder-sparing treatment regimens that can achieve disease control or even cure without the need for surgery.
YH01 Adenovirus Injection is a recombinant oncolytic virus product developed independently by InnoFuture, which is engineered from human adenovirus type 5. It exerts a dual tumor-killing mechanism: it not only directly lyses tumor cells but also activates a systemic anti-tumor immune response. While enabling precise regulatory control, its local instillation administration route significantly reduces systemic adverse effects.
In January 2026, VCBeat conducted an exclusive interview with the principal investigators of the Phase Ⅱ clinical trial—Professor Niu Yuanjie, President of Tianjin Medical University General Hospital and Director of Tianjin Institute of Urology, and Director Hu Hailong from the Department of Urology, the Second Hospital of Tianjin Medical University—to conduct an in-depth interpretation of the study’s phased progress and clinical value.
Professor Niu Yuanjie
Chief Physician, PhD Supervisor
Postdoctoral Fellow, University of Rochester, USA
President, Tianjin Medical University General Hospital
Director, Tianjin Institute of Urology
Director, Tianjin Key Laboratory of Basic Urological Medicine
Professor Hu Hailong
Chief Physician, PhD Supervisor
Deputy Director, Department of Urology
The Second Hospital of Tianjin Medical University
1What are the significant values of conducting the clinical trial for the innovative drug YH01?
Prof. Niu Yuanjie: Healthy China 2030 Planning sets the important goal of increasing the overall 5-year cancer survival rate by 15% by 2030. Drug innovation, as a vital part of achieving this target, is an essential step. Bladder cancer is one of the most common malignant tumors of the urinary system. Globally, the cases of bladder cancer have reached nearly 600,000, with deaths standing at approximately 200,000. According to statistics in 2022, the annual number of bladder cancer cases in China is close to 90,000, with exceeding 40,000 deaths. Therefore, the treatment of bladder cancer remains a significant challenge for us. Approximately 80% of bladder cancer patients are diagnosed with Non-Muscle Invasive Bladder Cancer (NMIBC). For these patients, the standard treatment involves intravesical chemotherapy and bacillus Calmette-Guérin (BCG) instillation following transurethral resection of the bladder tumor (TURBT). However, the recurrence rate and progression risk with these treatments are quite high. YH01 is a newly developed drug that can be used for intravesical instillation therapy. Positive efficacy signals have been observed in clinical settings, making it a promising novel drug worthy of further research and exploration.
2What clinical pain points of BCG therapy is YH01 expected to address?
Prof. Niu Yuanjie: Currently, the main treatment method of Non-Muscle Invasive Bladder Cancer (NMIBC) is transurethral resection of bladder tumor (TURBT) followed by intravesical instillation of chemotherapy drugs or bacillus Calmette-Guerin(BCG). BCG is the most commonly adopted regimen. However, BCG instillation is associated with several notable issues: firstly, some patients are unresponsive to BCG, and the instillation fails to prevent tumor recurrence; some patients initially respond to BCG but lose responsiveness after a period of instillation, leading to tumor recurrence; additionally, a subset of patients are simply unable to tolerate BCG instillation due to adverse effects and other complications.The preliminary positive signals observed with YH01 include two key aspects: first, it can effectively reduce the tumor recurrence rate and deliver proven clinical efficacy; second, it has a more favorable safety profile with reduced adverse effects, and distinct improvements have been observed in patient tolerability and the incidence of adverse effects.
3What constitutes the innovative drug mechanism of YH01?
Prof. Niu Yuanjie: YH01 is an adenovirus instillation solution. First, it can distinguish between tumor cells and non-tumor cells, and express and replicate in large quantities specifically within tumor cells, leading to tumor cell death—in theory, it causes no damage to normal cells. Second, while killing tumor cells, the latter release a large number of antigens; and the adenovirus itself acts as an immune activator. This thus results in enhanced immunity post-killing and generates a robust anti-tumor effect.
4What do the interim efficacy data reveal about YH01?
Prof. Niu Yuanjie: In the Phase Ⅱ clinical trial, 9 out of 11 evaluable patients achieved complete response (CR) and 2 achieved stable disease (SD), with a CR rate of 81.8%. Among patients who received 6 doses, the 3-month CR rate reached 87.5% and the 6-month CR rate hit 80%. These data are extremely encouraging. In comparison with historical data (non-head-to-head), for the treatment of BCG-refractory patients, the commonly used intravesical chemotherapy with valrubicin yields a response rate of approximately 50% at 6 months, which drops to around 30% at 1 year and further to about 10% at 2 years post-treatment. When compared with the gemcitabine plus docetaxel chemotherapy regimen, the 1-year response rate is also only around 50%. It is clear from these comparisons that YH01 demonstrates greater efficacy potential for the retreatment of BCG-refractory bladder cancer.
5What does the "complete disappearance" of target tumor lesions mean for NMIBC patients?
Prof. Hu Hailong: The complete regression of tumors means patients may retain their bladders, namely gaining a window period for organ preservation and avoiding radical cystectomy. This is because radical cystectomy is associated with considerable surgical costs and a relatively high risk of mortality, given the extensive and prolonged nature of the procedure. Furthermore, urinary diversion is a necessary follow-up to radical cystectomy, which is accompanied by a multitude of complications. Meanwhile, cystectomy may cause certain nerve damage, leading to sexual dysfunction and thus taking a heavy toll on patients. More importantly, bladder preservation will significantly improve patients' quality of life. Physical integrity also reduces patients' social barriers.
6What safety characteristics has YH01 demonstrated?
Prof. Hu Hailong: To date, the overall tolerability of YH01 has been favorable in 15 patients. Adverse events were mainly mild, localized grade 1-2 bladder irritation symptoms (e.g., frequent micturition, urgent micturition, hematuria). But most of these symptoms were mild to moderate, and the majority of patients recover without intervention. Up to the current follow-up, no grade ≥3 adverse events or serious adverse events have been reported. In contrast to the well-recognized adverse events following BCG instillation—such as the most common flu-like symptoms, tuberculous cystitis, and even systemic disseminated infection—YH01’s local reactions are relatively manageable, and no obvious systemic reactions have been observed to date. Consequently, patients are able to complete the instillation cycle as scheduled without treatment discontinuation due to poor tolerability, which directly improves patients’ treatment compliance and is crucial for the sustainability of therapeutic efficacy.
7Given the preliminary data on YH01, how would you communicate about the clinical trial to patients?
Prof. Hu Hailong: Thanks to the preliminary data on YH01, our conversations with patients have become much easier. For elderly, high-risk patients with Non-Muscle Invasive Bladder Cancer who have failed BCG therapy, discussions were essentially limited to surgery in the past—talking through radical cystectomy and urinary diversion, all in a rather somber tone. Now, we are able to inform patients about ongoing clinical trials and novel treatment options that offer the potential for bladder preservation with markedly fewer treatment-related side effects. The focus of our conversations has shifted from "why bladder removal is imperative" to "what the advantages and uncertainties of this new regimen are". As physicians and researchers, we are committed to fully informing patients and their families that our research is still in early phase, with a limited sample size and a short follow-up duration. As such, the long-term efficacy remains to be further validated. Yet we can clearly sense a distinct shift in the demeanor of patients and their families—one brimming with renewed hope for bladder preservation.
8What is the significance of YH01 for bladder cancer treatment in China?
Prof. Hu Hailong: In the oncolytic virus field, the clinical data for YH01 truly underscores the potential of China's original innovative drugs. Moving forward, multi-center randomized controlled trials (RCTs) are required to rigorously validate its therapeutic efficacy. Historically, bladder cancer treatment strategies relied heavily on guidelines from Europe and the United States. Today, however, we have this domestically developed, original new drug with a relatively complete patent portfolio. In subsequent clinical trials, data generated in China will no longer serve as supplementary evidence but will instead emerge as a core component of the global evidence base. What's more, we will also have the opportunity to participate in international clinical trials and global clinical consensus-building efforts.
9What are your expectations for the subsequent clinical enrollment and efficacy data of YH01?
Prof. Hu Hailong: Currently, the Urology Department of the Second Hospital of Tianjin Medical University has enrolled nearly 20 patients. There are two key priorities we are focused on. Firstly, whether the complete response (CR) rate can be sustained above 80% following sample size expansion. Secondly, the outcomes of 1-year and even 2-year recurrence-free survival(RFS) data, particularly the progression-free survival (PFS) curve profile and PFS rate at a median follow-up of 12 months.
The Expert Consensus on Intravesical Instillation Therapy for Non-Muscle Invasive Bladder Cancer (2025 Edition) specifically points out that in attempting to conduct relevant clinical trials for BCG-nonresponsive patients, it has been found difficult to select an appropriate positive control—reselecting either BCG or chemotherapeutic drugs would violate ethical principles. For this reason, the FDA recommends not setting a control group in clinical trials at this clinical stage, while formulating corresponding regulations on the efficacy of single-arm studies.
Specifically, an efficacious treatment with clinical significance for BCG-nonresponsive patients with carcinoma in situ (CIS) shall meet the following criteria: a complete response (CR) rate of 50% at 6 months post-treatment, with CR maintained at ≥30% at 12 months and ≥25% at 18 months post-treatment. For BCG-nonresponsive patients with papillary tumors, the recurrence-free survival (RFS) rate shall be ≥30% at 12 months and ≥25% at 18 months post-treatment.
Worldwide, the recombinant oncolytic adenovirus CG0070 targets bladder cancer cells and expresses granulocyte-macrophage colony-stimulating factor (GM-CSF) intracellularly, and it has now entered Phase Ⅲ clinical trials. In the Phase Ⅱ BOND-002 study, BCG-nonresponsive patients with Non-Muscle Invasive Bladder Cancer (NMIBC) were treated with CG0070, achieving a complete response (CR) rate of 47% at 6 months. Treatment-related adverse events (AEs) were Grade Ⅰ-Ⅲ, mainly presenting as bladder spasm, hematuria and other symptoms.
To date, the preliminary data for YH01 has demonstrated remarkable advantages – its 80% complete response (CR) rate at 6 months has met the FDA’s benchmark requirements for single-arm studies and underscores its global competitive potential. Going forward, with an expanded sample size and ongoing follow-up, YH01’s progression-free survival (PFS) rate, recurrence-free survival (RFS) rate and CR maintenance rate at 12 and 18 months remain to be determined. The drug is poised to become a pivotal benchmark for oncolytic viruses and warrants continued attention.
Beijing Yinmei Future Biomedical Technology Co., Ltd., a high-tech enterprise based in Zhongguancun, focuses on the research and development and production of first-in-class Class I innovative drugs in the field of oncolytic viruses. It is now a wholly-owned subsidiary of Suzhou Yinghui Pharmaceutical Technology Co., Ltd. Our corporate vision is to deliver innovative and curative tumor therapies to cancer patients worldwide and become a global unicorn enterprise in the oncolytic virus sector.The founder of the company, Dr. Huang Yinghui, holds a PhD in Oncology from the University of Cambridge, UK. With over 20 years of study and work experience in the UK and the US, he has long been engaged in cancer-related research covering the molecular mechanisms of tumorigenesis, genetic diagnosis, chemotherapy, biotherapy and other related fields, and is a world-renowned expert in tumor biotherapy. He previously served as Dean of the College of Life Science and Bioengineering at Beijing University of Technology and a Tenured Professor at the Torrey Pines Institute for Molecular Studies in California, USA. He is also a recipient of the Young Scientist Award presented by the American Cancer Society and the California Department of Health.
Professor Huang Yinghui has created the OCDP™ Oncolytic Core Technology Design Platform. This platform is engineered to develop products by enhancing the replication and oncolytic activity of the virus itself. The oncolytic virus products designed on this platform specifically target cancer cells, with the design strategy bolstering their ability to infiltrate and destroy tumor tissues. These viruses lyse tumor cells in a direct manner and trigger a cascade amplification effect to eliminate cancer cells. Completed in vitro and in vivo experiments have demonstrated that these products exhibit a potent ability to inhibit the growth of malignant tumors, with minimal adverse effects and broad-spectrum activity against various types of tumors. They are poised to deliver a breakthrough therapeutic approach for the radical cure of cancer.
*This content is provided solely for informational purposes and does not constitute any medical advice.