Ractigen Therapeutics Announces Positive Phase I Clinical Data for saRNA Therapy RAG-01 in NMIBC at EAU 2025

Mar 27, 2025 09:23 CST Updated 09:23

Ractigen

Innovative Drug R&D Developer

｜Edited by the Content Team of Zhong肽 Biochemical

March 202524Day,Ractigen Therapeutics, a clinical-stage biopharmaceutical company focused on small activating RNA (saRNA) therapies, announced the positive preliminary data from the Phase I clinical trial of RAG-01 for the treatment of non-muscle invasive bladder cancer (NMIBC) at the late-breaking abstract session of the 40th Annual European Association of Urology Congress (EAU 2025) held in Madrid, Spain. The Royal Melbourne Hospital, AustraliaPaul AndersonThe doctoral presentation showcased these research findings. The data indicates that, among patients for whom BCG treatment has failed, the lowest two dosage groups showed...Carcinoma in situ(CIS) patients achieved a complete response rate (CR) of 66.7%, with excellent safety performance.

RAG-01, as an innovative saRNA therapy, aims to upregulate the expression of the p21 tumor suppressor gene. The p21 gene plays a crucial regulatory role in cell cycle progression; however, traditional therapies struggle to effectively target it. Leveraging Ractigen Therapeutics' proprietary LiCO™ delivery technology, RAG-01 is administered via intravesical instillation, offering a novel treatment option for NMIBC patients who have failed BCG therapy. In light of its demonstrated potential in addressing unmet clinical needs, RAG-01 has successfully received Fast Track Designation and IND approval from the U.S. FDA.

AboutPhase I Clinical Trial of RAG-01

The ongoing Phase I clinical trial (NCT06351904) is an open-label, multi-center study utilizing a standard “3+3” dose-escalation design. The primary objectives are to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of RAG-01, as well as to explore preliminary efficacy. As of December 15, 2024, a total of 9 patients have participated in the trial, receiving treatment at three dose levels: 30 mg, 100 mg, and 300 mg. The treatment regimen includes a 6-week induction phase with once-weekly dosing, followed by maintenance therapy at weeks 12, 24, 36, 48, and 72.

Key Research Results Presented at EAU 2025

Excellent Safety:No dose-limiting toxicity (DLT) was observed in any of the dose groups. Treatment-related adverse events (AEs) were reported in 88.9% (8/9) of patients, but all adverse events were grade 2 or lower in severity. The most common adverse reactions (each with an incidence rate of 11.1%) included urinary urgency, urinary frequency, and urinary tract infection.

Precise Drug Delivery and Target Activation:Pharmacokinetic analysis showed that the systemic exposure of RAG-01 in the blood was extremely low, confirming the effectiveness of intravesical administration and the LiCO™ delivery system. Urinary concentrations of RAG-01 increased in a dose-dependent manner, ranging from 83.3 to 1820 µg/ml at 2 hours post-administration. Meanwhile, the proportion of p21-positive cells in the bladder urothelium also increased in a dose-dependent manner, confirming target engagement of the drug.

Significant Early Efficacy:In patients with carcinoma in situ, the complete response rate in the two lowest dose groups (30mg and 100mg) reached 66.7% (2/3). Additionally, 66.7% (2/3) of patients with papillary tumors showed no disease recurrence at the 3-month follow-up. These early efficacy data strongly indicate that even at lower doses, RAG-01 still demonstrated remarkably significant therapeutic effects.

AboutNon-Muscle Invasive Bladder Cancer (NMIBC)

Non-Muscle-Invasive Bladder Cancer (NMIBC) is a common malignant tumor, with lesions confined to the inner wall of the bladder. The standard first-line treatment is Transurethral Resection of Bladder Tumor (TURBT), followed by intravesical BCG immunotherapy. However, a significant proportion of patients experience recurrence or show no response to BCG treatment, highlighting an urgent need for the development of novel and effective therapeutic approaches.

AboutRNA Activation (RNAa)

RNA Activation (RNAa) is a clinically validated technology platform pioneered by Dr. Li Longcheng and his team. RNAa selectively activates gene expression by targeting specific gene regulatory regions with small activating RNA (saRNA), thereby restoring the production of therapeutic proteins. This innovative technology holds broad therapeutic potential across various disease areas, particularly in fields such as genetic disorders and cancers where traditional treatments have shown limited efficacy.

AboutRactigen

Ractigen Therapeutics is a clinical-stage biopharmaceutical company dedicated to developing breakthrough small nucleic acid drugs and disease treatment methods. Ractigen Therapeutics is one of the few global small nucleic acid drug companies that simultaneously possess both intrahepatic and extrahepatic delivery capabilities, having developed multiple internationally leading small nucleic acid drug delivery platform technologies with independent intellectual property rights, including SCAD™, LiCO™, and GLORY™. Based on RNA activation technology and its self-developed Smart-TTC saRNA drug development platform, the company has established a highly differentiated small nucleic acid drug pipeline, with indications covering neurodegenerative diseases, neuromuscular diseases, cancer, metabolic diseases, and hematological disorders, providing innovative therapeutic solutions for undruggable targets and currently incurable diseases across various fields.

Source:

1.Ractigen