Ractigen Therapeutics, a pioneering clinical-stage biotechnology company developing innovative small activating RNA (saRNA) therapeutics, announced that China's National Medical Products Administration (NMPA) Center for Drug Evaluation (CDE) has approved its Investigational New Drug (IND) application to initiate a Phase II clinical trial of RAG-01 for the treatment of Non-Muscle Invasive Bladder Cancer (NMIBC).

This significant milestone makes RAG-01 the second saRNA therapeutic to receive IND approval in China, both of which were developed by Ractigen. This approval not only solidifies Ractigen's global leadership in saRNA technology but also serves as a critical clinical validator for the company's broader RNA activation (RNAa) platform and its LiCO^TM delivery technology.

RAG-01 is a novel saRNA therapeutic designed to upregulate p21, a key regulator of cell cycle arrest and cellular senescence, to inhibit the abnormal proliferation of bladder cancer cells. This innovative saRNA therapeutic aims to treat NMIBC by increasing p21 mRNA and protein levels.

The progression of RAG-01 into Phase II clinical development follows highly encouraging Phase I data from Australia, which demonstrated clear target engagement (p21 protein upregulation), a favorable safety profile, and promising complete response (CR) signals.

"We are very pleased to receive IND approval from the China NMPA CDE for the Phase II trial of RAG-01," said Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics. "This approval is an important validation of both the RAG-01 program and the broader potential of saRNA therapeutics. RAG-01 reflects our innovation strategy of activating endogenous disease-relevant genes that have long been considered difficult to drug. We believe this differentiated mechanism, together with local bladder delivery, may offer a meaningful new treatment option for patients with NMIBC. We look forward to working closely with investigators and regulatory authorities to advance this program."

The Phase II study is a randomized, controlled, multi-center trial designed to evaluate the efficacy and safety of RAG-01 as monotherapy and in combination with chemotherapy in patients with intermediate- and high-risk NMIBC. The China study builds on encouraging preliminary data from the ongoing Phase I clinical trial in Australia, where RAG-01 has demonstrated favorable safety, clear target engagement, and anti-tumor activity.