Ractigen Therapeutics Kicks Off Multi-Center Phase II Clinical Trial of First-in-Class saRNA Therapeutic RAG-01 for Non-Muscle Invasive Bladder Cancer

Trial initiated at Fudan University Shanghai Cancer Center (FUSCC) under the leadership of Leading Principal Investigator Professor Ding-Wei Ye, uniting 11 premier urologic oncology centers across China
Phase II study incorporates a safety run-in phase with a subsequent multi-cohort design to evaluate intravesical RAG-01 both as a monotherapy and in combination with mitomycin C (MMC) in patients with intermediate- or high-risk NMIBC

SHANGHAI and NANTONG, China, Nov. 2, 2026 /PRNewswire/ — Ractigen Therapeutics, a leading clinical-stage biopharmaceutical company developing innovative small activating RNA (saRNA) therapeutics, today announced the official initiation of its multi-center Phase II clinical trial (Study RAG010201) of RAG-01 for the treatment of Non-Muscle Invasive Bladder Cancer (NMIBC) in China, marked by a clinical site initiation meeting held at Fudan University Shanghai Cancer Center (FUSCC).

Designed as a randomized, controlled, multi-center Phase II study, the RAG010201 trial evaluates the efficacy, safety, tolerability, and pharmacokinetics (PK) of intravesical RAG-01 as a monotherapy and in combination with the chemotherapeutic agent mitomycin C (MMC) in patients with intermediate- or high-risk NMIBC.

The multi-center Phase II trial is led by Leading Principal Investigator Professor Ding-Wei Ye, a renowned authority in urologic oncology at FUSCC and Chief Expert of the Urologic Oncology Multidisciplinary Team (MDT). A total of 11 premier medical institutions across China are participating in the study, including:

Fudan University Shanghai Cancer Center (Prof. Ding-Wei Ye)
Peking University First Hospital (Prof. Xue-Song Li)
Ruijin Hospital affiliated to Shanghai Jiao Tong University School of Medicine (Prof. Dan-Feng Xu)
The Second Hospital of Tianjin Medical University (Prof. Hai-Long Hu)
Hunan Cancer Hospital (Prof. Gong-Qian Zeng)
Nanjing Drum Tower Hospital, The Affiliated Hospital of Nanjing University Medical School (Prof. Hong-Qian Guo)
Union Hospital affiliated to Tongji Medical College of Huazhong University of Science and Technology (Prof. Xiao-Ping Zhang)
Qilu Hospital of Shandong University (Prof. Ben-Kang Shi)
Fujian Medical University Union Hospital (Prof. Shao-Xing Zhu)
The First Affiliated Hospital of Xiamen University (Prof. Jin-Chun Xing)
Sun Yat-sen Memorial Hospital of Sun Yat-sen University (Prof. Jian Huang)

To address the distinct clinical challenges across risk stratifications in intermediate- and high-risk NMIBC, the RAG010201 study incorporates two tailored cohorts:

Cohort A (High-Risk NMIBC): Patients with high-risk NMIBC face a substantial risk of tumor recurrence and progression to muscle-invasive disease, creating an urgent need for more potent bladder-sparing regimens. Cohort A will assess RAG-01 monotherapy versus RAG-01 in combination with MMC. Following a safety run-in phase to establish the recommended dosing regimen, the study will proceed to an open-label, 1:1 randomized controlled phase with the 24-month event-free survival (EFS) rate as the primary endpoint, evaluating both the standalone activity of RAG-01 and its synergistic potential with intravesical chemotherapy.
Cohort B (Intermediate-Risk NMIBC): For intermediate-risk patients, clinical management prioritizes preventing recurrence and extending recurrence-free survival while minimizing treatment-related toxicity. Cohort B is designed as a single-arm study evaluating intravesical RAG-01 monotherapy, with the 24-month recurrence-free survival (RFS) rate serving as the primary endpoint.

RAG-01 is a first-in-class saRNA therapeutic designed to selectively target and upregulate p21, a key tumor suppressor gene that regulates cell cycle arrest and cellular senescence, thereby inhibiting the abnormal proliferation of bladder cancer cells. Delivered locally via intravesical instillation using Ractigen’s proprietary LiCO™ delivery system, RAG-01 has already demonstrated a favorable safety profile, clear dose-dependent target engagement, and encouraging durable clinical responses in its Phase I clinical trial in Australia. Preclinical study results demonstrated that RAG-01 in combination with MMC exerted significant synergistic inhibitory effects on bladder cancer cell proliferation.

Professor Ding-Wei Ye, Leading Principal Investigator of the study at Fudan University Shanghai Cancer Center, commented: “Following transurethral resection of bladder tumor (TURBT), patients with NMIBC continue to face daunting rates of tumor recurrence and progression. In clinical practice, persistent Bacillus Calmette-Guérin (BCG) shortages, BCG intolerance or failure, and the suboptimal durability of conventional intravesical chemotherapies highlight a critical unmet need for therapies with novel mechanisms of action. By leveraging cutting-edge RNA activation (RNAa) technology to selectively upregulate the p21 tumor suppressor gene, RAG-01 introduces an entirely new therapeutic paradigm for localized bladder cancer treatment. We look forward to collaborating with top urology centers nationwide to advance this rigorously designed Phase II study and evaluate the potential of RAG-01, both alone and in combination, to reduce recurrence, delay disease progression, and preserve patients’ bladders.”

“Kicking off our Phase II trial at Fudan University Shanghai Cancer Center alongside Professor Ding-Wei Ye and 10 other top-tier clinical centers across China is a major milestone for the RAG-01 program,” said Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics. “As a former urologist myself, addressing the unmet needs of bladder cancer patients has been a driving force behind translating RNA activation technology from bench to bedside. Building on the encouraging durability and dose-response signals observed in our Phase I study, we are confident that the world-class surgical quality and rigorous clinical execution of our participating Chinese centers will enable us to fully unlock the therapeutic potential of RAG-01.”

About RAG-01

RAG-01 is a pioneering saRNA candidate engineered to target and activate the tumor suppressor gene p21 via the mechanism of RNA activation (RNAa). Traditionally considered “undruggable” by conventional small molecules or antibodies, p21 presents a unique opportunity for saRNA-based targeted gene activation. Delivered through intravesical instillation using Ractigen’s proprietary LiCO™ delivery technology, RAG-01 has demonstrated encouraging preliminary efficacy, a favorable safety profile, and clear target engagement in its Phase I clinical trial conducted in Australia. RAG-01 previously received IND clearance from the U.S. Food and Drug Administration (FDA), was granted Fast Track Designation (FTD) by the FDA in 2024, and received Phase II IND approval from China’s NMPA Center for Drug Evaluation (CDE).

About NMIBC

Non-muscle invasive bladder cancer (NMIBC) is a common malignancy confined to the mucosal and submucosal lining of the bladder, accounting for approximately 75% of all newly diagnosed bladder cancer cases. The standard first-line treatment for intermediate- and high-risk NMIBC is transurethral resection of the bladder tumor (TURBT) followed by intravesical Bacillus Calmette-Guérin (BCG) immunotherapy or intravesical chemotherapy. However, a significant proportion of patients experience frequent recurrence, disease progression, or become unresponsive to BCG, often leaving radical cystectomy (complete removal of the bladder) as the only remaining option and highlighting the critical need for novel bladder-sparing therapies.

About RNAa

RNA activation (RNAa) is a clinically validated platform technology discovered and developed by Dr. Long-Cheng Li and his team. It utilizes small activating RNAs (saRNAs) to target gene regulatory domains at the transcriptional level, activating endogenous gene expression and restoring therapeutic protein levels. This transformative technology holds vast potential for developing first-in-class therapeutics across oncology, genetic disorders, and chronic diseases, particularly for targets where traditional pharmacological approaches fall short.

About Fudan University Shanghai Cancer Center (FUSCC)

Founded in 1931, Fudan University Shanghai Cancer Center (FUSCC) is China’s earliest established Grade-A tertiary oncology hospital directly affiliated with Fudan University and managed by the National Health Commission. Operating across its Xuhui and Pudong campuses with homogeneous multi-site development, FUSCC consistently earns the highest national performance rating (Grade A) among specialized cancer centers in China. It features national key clinical specialties, Shanghai key laboratories and innovation platforms for precision oncology, cell and gene therapy. In 2025, it recorded 2.5 million outpatient visits, with an overall 5-year cancer survival rate of 72%, matching leading international benchmarks. FUSCC advances clinical research, delivers findings that revise international clinical guidelines, facilitates research translation, and conducts medical support programs and global collaborations as a leading, internationally recognized cancer center.

About Ractigen Therapeutics

Ractigen Therapeutics is a clinical-stage biopharmaceutical company dedicated to discovering and developing transformative small activating RNA (saRNA) and siRNA therapeutics for diseases intractable to conventional modalities. Powered by its proprietary smart-TCC™ saRNA discovery engine and extrahepatic delivery platforms (LiCO™ and SCAD™), Ractigen is advancing a differentiated clinical and preclinical pipeline targeting neuromuscular, metabolic, cardiovascular, oncology, and rare genetic disorders, including clinical-stage programs RAG-01 (NMIBC), RAG-18 (DMD), and RAG-17 (SOD1-ALS). For more information, please visit www.ractigen.com, and follow us on X and LinkedIn.

Ractigen Therapeutics Kicks Off Multi-Center Phase II Clinical Trial of First-in-Class saRNA Therapeutic RAG-01 for Non-Muscle Invasive Bladder Cancer

ข่าวที่เกี่ยวข้อง

krapalm