RAG-01 / Ractigen Therap - LARVOL DELTA

RAG-01-01: A Study of RAG-01 in Patients With Non-muscle-invasive Bladder Cancer (NMIBC) Who Have Failed Bacillus Calmette Guérin (BCG) Therapy (clinicaltrials.gov) - P1 | N=72 | Recruiting | Sponsor: Ractigen Therapeutics. | N=15 ➔ 72 | Trial completion date: Aug 2025 ➔ Aug 2028 | Trial primary completion date: Jun 2025 ➔ Aug 2028

Enrollment change • Trial completion date • Trial primary completion date • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

RNAa: Mechanisms, therapeutic potential, and clinical progress. (PubMed, Mol Ther Nucleic Acids) - "Notably, MTL-CEBPA, the first saRNA drug candidate, shows promise in hepatocellular carcinoma treatment, while RAG-01 is being explored for non-muscle-invasive bladder cancer, highlighting clinical advancements in RNAa. This review synthesizes our current understanding of the mechanisms of RNAa and highlights recent advancements in the study of mi-RNAa and the therapeutic development of saRNAs."

Journal • Review • Alzheimer's Disease • Bladder Cancer • CNS Disorders • Genito-urinary Cancer • Hepatocellular Cancer • Metabolic Disorders • Oncology • Otorhinolaryngology • Solid Tumor • CEBPA

Ractigen's RAG-01 Shows Promising Early Complete Responses in Phase I NMIBC Trial, Data Presented at EAU 2025 (PRNewswire) - P1 | N=15 | NCT06351904 | Sponsor: Ractigen Therapeutics. | "Ractigen Therapeutics...announced the presentation of positive preliminary data from its ongoing Phase I clinical trial of RAG-01, a first-in-class saRNA therapy for the treatment of non-muscle invasive bladder cancer (NMIBC)....Targeted Drug Delivery and Activity: PK analysis revealed minimal systemic exposure of RAG-01, confirming the effectiveness of the intravesical administration and LiCO technology. Dose-dependent increases in RAG-01 urine concentrations were observed, ranging from 83.3 to 1,820 µg/ml at 2 hours post-instillation....Remarkable Early Efficacy: In patients with carcinoma in situ (CIS), the complete response (CR) rate was 66.7% (2/3 patients). Furthermore, 66.7% (2/3) of patients with papillary tumors remained disease-free at the 3-month assessment."

P1 data • PK/PD data • Bladder Cancer

Ractigen Therapeutics Secures FDA Fast Track Designation for RAG-01, a First-in-Class saRNA Therapy (PRNewswire) - "Ractigen Therapeutics...proudly announces that its flagship program, RAG-01, has been granted Fast Track Designation (FTD) by the U.S. Food and Drug Administration (FDA)....The milestone establishes RAG-01 as the first saRNA drug worldwide to achieve FTD."

Fast track • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

Ractigen Therapeutics Announces FDA Approval for RAG-01, a First-in-Class saRNA Therapy for BCG-Unresponsive NMIBC (PRNewswire) - "Ractigen Therapeutics...announced today that the U.S. Food and Drug Administration (FDA) has approved the company's Investigational New Drug (IND) application for RAG-01, a groundbreaking saRNA therapy targeting non-muscle invasive bladder cancer (NMIBC). This approval facilitates the launch of U.S. clinical trials, following the successful initiation of a Phase I trial in Australia...RAG-01 is a pioneering therapy in bladder cancer treatment, representing the first of its kind to specifically target and activate the p21 tumor suppressor gene....Currently, the Phase I clinical trial of RAG-01 in Australia has successfully enrolled and dosed the first three patients."

IND • Trial status • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

A Study of RAG-01 in Patients With Non-muscle-invasive Bladder Cancer (NMIBC) Who Have Failed Bacillus Calmette Guérin (BCG) Therapy (clinicaltrials.gov) - P1 | N=15 | Recruiting | Sponsor: Ractigen Therapeutics. | Not yet recruiting ➔ Recruiting

Enrollment open • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

A Study of RAG-01 in Patients With Non-muscle-invasive Bladder Cancer (NMIBC) Who Have Failed Bacillus Calmette Guérin (BCG) Therapy (clinicaltrials.gov) - P1 | N=15 | Not yet recruiting | Sponsor: Ractigen Therapeutics.

New P1 trial • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

Ractigen Announces First Patient Dosed in the Phase I Clinical Trial of RAG-01 for NMIBC (PRNewswire) - "Ractigen Therapeutics...achieved a major milestone today with the first patient dosed in its First-in-human phase I clinical trial for RAG-01 conducted in collaboration with GenesisCare, Australia's leading provider of cancer care services. This marks a significant advancement in the fight against non-muscle invasive bladder cancer (NMIBC)."

Trial status • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

Ractigen Therapeutics Advances into Clinical Stage with Groundbreaking saRNA Drug, RAG-01 (GlobeNewswire) - "Ractigen Therapeutics...is excited to announce the submission of a clinical trial application in Australia. This application paves the way for a phase Ⅰ, open-label, multi-center study, aiming to evaluate the safety, tolerability, pharmacokinetics, and preliminary efficacy of RAG-01, a groundbreaking saRNA drug candidate, in patients with non-muscle-invasive bladder cancer (NMIBC) who have not responded to Bacillus Calmette-Guérin (BCG) therapy."

New P1 trial • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

Preclinical development of RAG1-40-31L: A novel small activating RNA-lipid conjugate targeting tumor suppressor gene p21 for treatment of non-muscle invasive bladder cancer. (ASCO 2023) - "These data constitute a preclinical proof-of-concept for saRNA as a novel p21-targeting modality in the treatment of cancer. The C5X5 lipid conjugate appears to be a viable approach to enhance oligonucleotide delivery and activity in bladder cancer. Preclinical safety assessment studies to enable clinical trials of RAG01-40-31L are currently underway."

Preclinical • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer • CDKN1A • RAG1