▎WuXi AppTec Content Team Report

On May 29, Ribo Life Science announced its independently developedAnti-thrombotic siRNA Drug Targeting FXIRBD4059 was recently granted Phase 2 clinical trial authorization by the European Medicines Agency (EMA).It is a randomized, double-blind, placebo-controlled Phase 2 clinical study,Aimed to evaluate RBD4059 inPatients with stable coronary artery diseaseSafety, efficacy, and pharmacokinetics in China. According to the introduction in the news release by Ribo Life Science,RBD4059With the potential for ultra-long-acting antithrombotic effects lasting six months per injection.

Anticoagulant drugs are fundamental medications for the prevention and treatment of thrombosis, with a wide range of indications including coronary artery disease, peripheral artery disease, end-stage renal disease, atrial fibrillation, venous thromboembolism, and postoperative care in orthopedic surgeries. Currently used anticoagulants in clinical practice, such as direct oral anticoagulants, VKAs, and heparin, all carry a certain risk of bleeding. Therefore, there remains an unmet clinical need for novel anticoagulants that are potent, long-acting, and associated with a low risk of bleeding.

RBD4059 is a GalNac-conjugated siRNA drug developed by Ribo Life Science based on its liver-targeting technology platform.By inhibitingCoagulation Factor XI（FXI）By blocking the activation of the endogenous coagulation pathway, thereby achieving its anticoagulant/antithrombotic effect.FXI is a key molecule in the intrinsic coagulation pathway. An increasing number of studies have shown that inhibiting FXI can block the intrinsic coagulation pathway, providing a new and effective antithrombotic method with a low risk of bleeding for clinical patients.According to the press release from Ribo Life Science, inhibiting FXI has the potential to become a more effective anti-thrombotic treatment compared to current therapeutic approaches, andBleeding risk is relativelySmall。

In Phase 1 clinical trials, RBD4059 demonstrated good safety and efficacy. According to Ribo Life Science earlier in 2023,European Society of Cardiology (ESC）Data presented at the conference,Preclinical study data show that the product has a highly efficient and sustained inhibitory effect on FXI expression and activity, and is expected to have long-lasting efficacy in clinical practice., improve patient compliance.

Dr. Zicai Liang, Chairman and CEO of Ribo Life Science, stated that RBD4059With the potential for ultra-long-acting antithrombotic effects lasting six months per injection, which is expected to bring benefits to patients. We are very pleased that the drug has been approved for Phase 2 clinical trials by the EMA. Based on Ribo Life Science's differentiated development strategy, they will also launch more innovative small nucleic acid drugs successively, bringing substantial clinical benefits to patients as soon as possible.

[1] Ribo Life Science's Self-Developed World's First FXI-Targeting Small Nucleic Acid Drug Approved for Phase II Clinical Trial in the EU. Retrieved May 29, 2024, from https://mp.weixin.qq.com/s/Hf9cmRJvI4tZAlR5d_xCCA

[2] Ribo Life Science announced the research data of three small nucleic acid drugs under development for the first time at the 2023 European Society of Cardiology Annual Meeting.. Retrieved Sep 4, 2023, from https://mp.weixin.qq.com/s/7_SE4uTeNvzeYr_SMtyCuA