Suzhou Ribo Life Science and subsidiary Ribocure Pharmaceuticals, collectively known as Ribo, has reported initial results from its Phase IIa clinical trial of vortosiran (RBD4059) in patients with chronic coronary artery disease (CCAD) in Europe.
Data were presented at the China Pharmaceutical Innovation Conference (CPIC) in Shanghai.
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Vortosiran is an investigational small interfering ribonucleic acid (siRNA) therapy designed to target coagulation Factor XI (FXI).
The randomised, double-blind, placebo-controlled trial assessed vortosiran in patients with CCAD who had previously experienced a myocardial infarction and were taking aspirin as standard of care.
Patients who completed the high-dose cohort (maintenance dose of 400mg) achieved on average a 92% reduction in FXI activity, a level of suppression sustained for several months.
The results suggest that dosing every three to six months may be feasible across a range of indications.
No treatment-related serious adverse events, major bleeding events, or clinically relevant non-major bleeding events were recorded in the study.
Ribo states that the extent of FXI inhibition achieved with vortosiran exceeds that reported for ongoing small molecule programmes in Phase III development, which typically require dosing once or twice daily.
Ribo co-CEO and R&D global president Dr Li-Ming Gan said: “These Phase IIa data, generated in the target patient population on top of standard of care, reinforce our belief that vortosiran is a safe and highly differentiated FXI-inhibition approach for thromboembolic diseases.
“Building on these findings, we have initiated the ORBIT-XI programme (Optimizing RNA-Based Inhibition of Thrombosis), including several Phase IIb trials designed to support rapid progression into Phase III development across multiple indications.”
Vortosiran is a GalNAc-conjugated siRNA candidate from the RiboGalSTAR liver-targeting platform. By selectively suppressing FXI synthesis, the therapy inhibits the intrinsic coagulation pathway.
