Ribo has reported initial Phase IIa results for vortosiran (RBD4059) in chronic coronary artery disease patients, showing an average 92% reduction in Factor XI activity in the high-dose (400 mg maintenance) cohort. The FXI suppression was sustained for several months, suggesting dosing every three to six months may be feasible. No treatment-related serious adverse events or major bleeding events were observed. Ribo has launched the ORBIT-XI programme to advance Phase IIb and Phase III development.
Ribo's Vortosiran Achieves Sustained 92% FXI Suppression in Phase IIa CCAD Trial
The trial assessed vortosiran in chronic coronary artery disease patients with prior myocardial infarction taking aspirin as standard. Credit: Jo Panuwat D / Shutterstock.com.
Suzhou Ribo Life Science and its subsidiary Ribocure Pharmaceuticals, collectively known as Ribo, have released initial Phase IIa data for vortosiran (RBD4059) in patients with chronic coronary artery disease (CCAD) treated in Europe. The data were presented at the China Pharmaceutical Innovation Conference (CPIC) in Shanghai.
Trial Design and Key Findings
The randomised, double-blind, placebo-controlled study evaluated vortosiran in CCAD patients who previously experienced a myocardial infarction and were receiving aspirin as standard of care. Participants in the high-dose cohort, which used a 400 mg maintenance dose, showed an average 92% reduction in Factor XI (FXI) activity. That level of FXI suppression was sustained for several months.
Ribo reports these results suggest dosing intervals of every three to six months may be feasible across multiple indications, potentially reducing dosing frequency compared with oral small-molecule programmes that typically require once- or twice-daily administration.
Safety
No treatment-related serious adverse events were reported in the study. There were also no major bleeding events or clinically relevant non-major bleeding events recorded among participants in the reported cohorts.
"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," said Ribo co-CEO and R&D Global President Dr Li-Ming Gan.
Mechanism and Development Path
Vortosiran is a GalNAc-conjugated small interfering RNA (siRNA) from Ribo's RiboGalSTAR liver-targeting platform. By selectively suppressing hepatic synthesis of FXI, the therapy targets the intrinsic coagulation pathway to reduce thrombotic risk while aiming to preserve haemostatic balance.
Building on the Phase IIa findings, Ribo has launched the ORBIT-XI programme (Optimizing RNA-Based Inhibition of Thrombosis), which includes several Phase IIb trials intended to support rapid progression into Phase III across multiple indications.
Note: These are initial results presented by Ribo; full datasets and peer-reviewed publication details were not provided in the announcement. The original article was published by Clinical Trials Arena, a GlobalData brand.
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