RiboBio's siRNA Drug RBD5044 Receives EU Approval to Initiate Phase II Clinical Trial in Patients with Mixed Dyslipidemia

October 29, 2024

Ribo Life Science and its subsidiary Ribocure Pharmaceuticals today announced that their siRNA drug RBD5044 has been approved by the EU to commence Phase II clinical trials.

RBD5044 is a product based on RIBO-GalSTAR^TMRBD5044, an siRNA drug targeting ApoC3 developed on a liver-targeting technology platform. This Phase II clinical trial will be a randomized, double-blind, placebo-controlled multicenter study aimed at evaluating the efficacy and safety of RBD5044 in patients with mixed hyperlipidemia.

RBD5044, as a GalNAc-conjugated siRNA drug, achieves potent, precise, and long-lasting reduction of ApoC3 protein levels in plasma through siRNA-mediated ApoC3 gene silencing. ApoC3 is a key component of triglyceride-rich atherogenic lipoprotein particles; studies have shown that plasma ApoC3 levels can independently predict coronary events beyond LDL-c. Genetic research has also found that loss-of-function mutations in ApoC3 and reduced plasma triglyceride levels are associated with a lower risk of cardiovascular disease, indicating that RBD5044 may reduce the risk of developing cardiovascular disease. In patients with severe hypertriglyceridemia, RBD5044 may also reduce the recurrence of acute pancreatitis by inhibiting ApoC3 and lowering plasma triglyceride levels.

RBD5044 is the world's second siRNA drug targeting ApoC3 to enter the clinical stage. Based on the safety and efficacy data from Phase I clinical trials, Ribo Life Science is highly confident in the potential of RBD5044 for the highly effective treatment of mixed hyperlipidemia and hypertriglyceridemia.

Dr. Gan Liming, Global R&D President of Ribo Life Science, stated: "Hypertriglyceridemia, whether in a fasting or postprandial state, is closely associated with residual cardiovascular risk, and there is currently a lack of effective treatment options. We are fully committed to accelerating the development of RBD5044 to address this urgent clinical need. This marks the fifth clinical trial approval obtained by the company in 2024, signifying that our siRNA drug development has entered a fast track. I am immensely proud of our team, who have not only achieved remarkable success in developing highly effective and safe siRNA drugs but have also demonstrated agile and precise execution in their communication and collaboration with regulatory authorities."