RiboBio's siRNA Drug RBD4059 Receives EMA Approval for Phase II Clinical Trial in Thrombotic Disorders

On May 29, 2024, Suzhou Ribo Life Science Co., Ltd. ("Ribo Life Science") announced that its self-developed RBD4059, the world’s first anti-thrombotic siRNA drug targeting FXI, had recently received Phase II clinical trial approval from the European Medicines Agency (EMA). This trial is a randomized, double-blind, placebo-controlled Phase II clinical study aimed at evaluating the safety, efficacy, and pharmacokinetics of RBD4059 in patients with stable coronary artery disease. The trial has also been approved to be conducted at Ribo Life Science’s European R&D center in its own Phase II clinical facility, which meets European and American regulatory standards.

Anticoagulant drugs are fundamental for the prevention and treatment of thrombosis, with a wide range of indications including coronary artery disease, peripheral artery disease, end-stage renal disease (ESRD), atrial fibrillation (AF), venous thromboembolism (VTE), and post-operative care in orthopedic surgery, among others. Currently used anticoagulants in clinical practice, such as direct oral anticoagulants (DOACs), VKAs, and heparin, all carry a certain risk of bleeding. Therefore, there is a significant clinical demand for novel anticoagulants that are potent, long-acting, and have a low risk of bleeding.

RBD4059 is a GalNac-conjugated siRNA drug independently developed by Ribo Life Science based on its RIBO-GalSTARTM liver-targeting technology platform, for which it holds global rights. It achieves its anticoagulant/antithrombotic effects by inhibiting FXI and blocking the activation of the intrinsic coagulation pathway. Compared with current treatment methods, inhibiting FXI is expected to become a more effective antithrombotic treatment with minimal bleeding risk. In Phase I clinical trials, RBD4059 demonstrated good safety and efficacy, and relevant data will be presented at this year’s European Society of Cardiology (ESC Congress) meeting to be held in London.

Dr. Zicai Liang, Chairman and CEO of Ribo Life Science, stated: "RBD4059 is the world's first siRNA-based small nucleic acid drug targeting FXI, and another significant achievement from our RIBO-GalSTARTM liver-targeting platform. RBD4059 has the potential for ultra-long-lasting antithrombotic effects with a single injection every six months, which could bring benefits to patients. We are very pleased that this drug has received Phase II clinical approval from the EMA. Based on Ribo Life Science’s Best-in-Class/First-in-Class differentiated development strategy, we will continue to introduce more innovative small nucleic acid drugs, aiming to bring substantial clinical benefits to patients as soon as possible."

About Ribo Life Science

Ribo Life Science is an innovative R&D company based on small nucleic acid (RNAi) technology and dedicated to the development of small nucleic acid-based drugs. With its innovative R&D capabilities and a fully integrated technology platform, Ribo Life Science has built a robust product pipeline. The company has established an international R&D center in Europe (Ribo International), which focuses on the global development of oligonucleotide therapies, leveraging extensive experience in translational medicine and clinical development to address unmet medical needs for patients with major diseases in the liver, cardiovascular system, kidneys, and oncology, as well as rare diseases. Ribo International's proprietary Phase II clinical center, which complies with European and American regulatory standards, has passed qualification audits by multinational pharmaceutical companies. This recent approval by the EMA will further support Ribo Life Science in advancing high-quality clinical research efficiently.

Editor: Baiji

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