Ractigen Therapeutics Submits Phase I Clinical Trial Application in Australia for First-in-Class saRNA Drug RAG-01

RAG-01 Successfully Submits for Clinical Trials, Becoming the Second saRNA Drug Globally to Enter the Clinical Stage and the First and Only saRNA Drug in China to Achieve This Milestone.

Dr. Longcheng Li, founder and CEO of Ractigen, stated:"Today marks not only an important moment for Ractigen Therapeutics to enter the clinical stage, but also a significant achievement in the field of global innovative drugs. Based on the original platform technology of RNA activation (RNAa), the preclinical data of RAG-01 has demonstrated excellent tumor suppression effects and safety. We look forward to the clinical performance of RAG-01 and hope that it will become an innovative targeted cancer treatment drug with global impact. We will actively promote more promising saRNA products into the clinical stage, providing innovative therapies for many currently difficult-to-treat diseases."

RAG-01 is a first-in-class double-stranded saRNA drug that specifically targets and activates the tumor suppressor gene p21. It enhances the expression of the p21 gene through the RNAa mechanism, thereby inhibiting tumor cell proliferation and inducing apoptosis and senescence. Preclinical efficacy studies have shown that, delivered via Ractigen Therapeutics' self-developed extracellular small nucleic acid delivery system LiCO™, RAG-01 treatment significantly inhibits the growth of bladder cancer tumors in animal models while demonstrating robust safety.

RAG-01 utilizes Ractigen Therapeutics' self-developed LiCO™ delivery system, which employs a unique lipid chemical approach to support multiple administration routes such as subcutaneous, intravenous, intravesical, and intravitreal injections. The versatility and efficacy of LiCO™ have been validated in numerous preclinical projects, enabling efficient and sustained delivery to tissues including the heart, muscle, fat, and eyes, completely revolutionizing the delivery method for oligonucleotides.

Bladder Cancer is the most common cancer of the urinary system, with NMIBC accounting for 50~80%. The standard treatment for NMIBC is primarily transurethral resection of bladder tumor (TURBT), but the postoperative recurrence rate is high, reaching up to 50%~70% within five years. Therefore, there is an urgent need to develop drugs that effectively prevent bladder cancer recurrence and treat advanced bladder cancer.

RNA Activation (RNAa) is a platform technology pioneered internationally by Dr. Long-Cheng Li, the founder of Ractigen, and his team, which has already been clinically validated. This technology uses double-stranded RNA targeting the promoter region of genes to activate gene expression, restoring the levels of therapeutic proteins. RNA activation technology is a highly scarce platform technology in the pharmaceutical field, with broad application prospects in developing therapeutic drugs for cancer, genetic diseases, chronic diseases, metabolic diseases, cardiovascular and cerebrovascular diseases, and more.

Ractigen Therapeutics is a platform-based new drug research and development company based in China and targeting the global market, committed to developing breakthrough small nucleic acid drugs and disease treatment methods. Ractigen Therapeutics is one of the very few small nucleic acid pharmaceutical companies globally that simultaneously masters both intrahepatic and extrahepatic delivery technologies, having developed multiple proprietary small nucleic acid drug delivery platform technologies with international leading standards, including SCAD™ and LiCO™. 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 and neuromuscular diseases, cancer, metabolic and hematological disorders, providing innovative therapeutic solutions for undruggable targets and currently incurable diseases across various disease areas. For more details, please visit the official website at www.ractigen.com.

JIANGSU, China, Dec. 06, 2023 — Ractigen Therapeutics, a leader in the field of small activating RNA (saRNA) 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. This milestone marks Ractigen's transition into clinical development, with RAG-01 targeting NMIBC.

RAG-01 is recognized as the second saRNA drug worldwide to enter clinical trials and the first of its kind in China to achieve this critical milestone. This development signifies a remarkable advancement in RNA-based therapeutic applications, especially in the context of challenging cancer treatments.

Reflecting on this significant development, Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics, shared his confident outlook: "This milestone represents more than a procedural step for us; it marks a crucial advancement in the application of RNAa technology in clinical settings. Our proprietary RNAa platform, as evidenced by the development of RAG-01, has shown promising results in preclinical tumor suppression and a solid safety profile. We hold a strong belief in the potential of RAG-01 in advancing bladder cancer treatment and remain dedicated to pushing the boundaries with more innovative saRNA candidates in clinical trials, targeting diseases that have historically been difficult to treat."

About RAG-01: RAG-01 stands as a pioneering saRNA candidate from Ractigen Therapeutics, engineered to target and activate the tumor suppressor gene p21. The drug, delivered intravesically using Ractigen’s advanced LiCO™ technology, has shown significant tumor suppression in mouse orthotopic bladder cancer models. Its development marks a significant stride in RNA-based therapies, addressing the unmet needs of NMIBC patients.

About LiCO™: LiCO™, Ractigen's proprietary delivery system, enables the delivery of duplex RNA into various tissues and organs. It supports multiple administration routes, including subcutaneous, intravenous, intravesical, and intravitreal, providing profound and durable activity. Its versatility and effectiveness have been validated in many preclinical studies, marking it as a cornerstone in Ractigen's therapeutic arsenal.

About NMIBC: NMIBC represents 50-80% of all bladder cancer cases. Despite standard treatments like transurethral resection of bladder tumor (TURBT) followed by intravesical BCG or chemotherapy, recurrence rates remain high, estimated at 50-70% within the first five years. RAG-01's development is a significant step towards addressing this substantial unmet need in bladder cancer therapy.

About RNAa: Pioneered by Dr. Long-Cheng Li and his team, RNAa is a clinically validated platform technology. It employs saRNA to target gene regulatory domains, activating gene expression and restoring therapeutic protein levels. This technology has vast potential for developing therapeutic drugs across various diseases, especially where traditional methods fall short, including cancer, genetic disorders, chronic diseases, and metabolic and cerebrovascular disorders.

About Ractigen Therapeutics: Ractigen Therapeutics is at the forefront of developing saRNA drugs, utilizing RNA activation (RNAa) to up-regulate gene expression endogenously. This innovative approach involves saRNA targeting specific genes to enhance transcription, thereby restoring normal protein functions. Ractigen's cutting-edge technology is pivotal in treating diseases unaddressable by conventional methods, such as those resulting from epigenetic silencing or gene downregulation.